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Medicare Ponders Poorly Conceived Anti-proton Regulation

Citizen whistleblower (me) speaks truth to power

Medicare is proposing a regulation that would pay the same for all types of radiation, including proton therapy. Such a draconian cut in reimbursement would drive some proton centers into bankruptcy. This is my longest blog, detailing the origins of this proposal, including using ancient, out-of-date reports dating to the earliest years of proton therapy, double-dealing by the American Society for Radiation Oncologists (ASTRO), and general governmental politics, influencing, and incompetence.

If you want to help proton therapy, please read at least the summary in my first few pages and contact your congress person and senators demanding this proposal be stopped. If you want to do that right now, go here: Alliance for Proton Therapy Access: https://www.votervoice.net/PROTON/1/campaigns/68657/respond

In 2018 the Medicare Payment Advisory Committee (MedPAC) issued a report that detailed three scenarios in which they felt Medicare was being taken for a ride by low-value services:
1) Early initiation of dialysis,
2) proton beam therapy, and
3) H.P. Acthar Gel.
The latter is a hormone used for treating multiple sclerosis and kidney damage. It costs $40,612.75 for one injection. That certainly looks excessive. MedPAC feels the same way about proton therapy.

It's decision is based on the faulty premise that proton therapy and x-rays are equally effective treatments. Therefore, since proton therapy costs more, it must be of low value. I thought low value just meant overpriced. Wrong.

Low value is defined as a technology that provides little or no benefit, in which the potential harm outweighs the potential benefit. That’s how MedPAC rates proton therapy: little benefit and might harm people. Anyone who knows anything about proton therapy can see such an assessment is outrageous and inaccurate.

Yet, based on this erroneous assumption, the Center for Medicare and Medicaid Services (CMS) has proposed a policy (CMS Rule No. CMS-5527-P) that would pay the same for all types of radiation, thereby reimbursing x-rays and proton therapy at the same rate. Such a policy would strike a blow against the advancement of proton therapy, possibly throwing some centers into bankruptcy and deny this life-saving technology to thousands of people.

THIS IS A VERY CRITICAL MOMENT FOR PROTON THERAPY. WE MUST ALL WORK TO REVERSE THIS TERRIBLE DECISION.

The American Society of Clinical Oncology (ASCO) states in a recent presentation investigating cost of treatments, “. . . the steady increase in the number of patients being treated with PBT (proton beam therapy) for cancers with additional requirements for health insurance coverage is primarily in those with Medicare coverage.” Yes, many Americans depend on Medicare, especially older men like me who have prostate cancer. After spending weeks reading hundreds of pages of reports and regulations I am speaking TRUTH TO POWER to reveal what lies behind of this draconian proposal.

For the convenience of those just perusing this essay, here are the main points I will be making at greater length and with substantial proof.

1. The proposed rule represents an ATTACK ON PROTON THERAPY by proponents of x-rays, hijacking CMS procedures for their own purposes. Is this an exaggeration? I will show specific examples.

2. CMS based its policy on OUTDATED STUDIES AND SOURCES that do not accurately reflect the true picture and performance of proton therapy. Computer programers say, “Garbage in, garbage out.” Such is the case with the proposed rule. Would you judge a new software, for example, by a review written fifteen years ago for an earlier version? Hardly. But that’s what CMS is doing in its rating of proton therapy.

3. The amount of PROOF DEMANDED OF PROTON THERAPY IS UNREASONABLE, near impossible, and unethical. The insistence on waiting for level I studies ignores a plethora of other current data that supports proton therapy. Such a strict standard was never imposed upon current x-ray technologies because their benefit was considered obvious. The benefits of proton therapy are equally obvious.

4. The proposed rule would IMPEDE THE DEVELOPMENT of proton therapy, which promises to be the cancer treatment of the future. Frankly, if it weren't for the lack of insurance coverage, many more cancer patients would avail themselves of proton therapy.

5. The physics and technologies of X-RAYS AND PROTON THERAPY ARE SUBSTANTIALLY DIFFERENT, producing differing results. Proton therapy is more complex and rightfully higher priced. To suggest an equivalency of results, value and payment is unsupportable.

6. The CMS policy is based on studies and input from people who HAVE NO DIRECT EXPERIENCE with proton therapy. Their understanding is theoretical and their actions uncaring.

7. Thwarting proton therapy will DENY THE GENERAL PUBLIC of an effective choice in fighting cancer, thereby reducing the quality of available care. The goal of the proposed CMS rule is to increase efficiency, lowers costs, and maintain the quality of care. Limiting the availability to proton therapy would have the opposite effect. Moreover, considering cost over results violates Medicare’s own regulations.

8. The rule NEEDS MORE TIME to be considered properly, without a hasty rush to judgment. Much of the general public, me included, learned about the situation when the comment period had already expired. The implementation of this rule should be delayed.

A legitimate problem, a flawed response

Because proton therapy is quite expensive, it has become an easy target for reducing costs. For example, health economists Katherine Baicker and Amitabh Chandra have identified NEW TECHNOLOGY as being one source of high spending. (What do they suggest, not having new technology? Placing crippling penalties against it?)

Another area of expenditure they identify is INFRASTRUCTURE COST AND CAPITAL EXPANSION. Proton therapy falls under this category, also. Hence, Amitabh Chandra has raged against proton therapy. From his prestigious Harvard position, he has called proton therapy “useless” and a “death star.” He has said that when looking for wasteful spending, proton therapy is an obvious place to start. From his ivory tower, Amitabh Chandra hasn’t a clue about the efficacy and promise of proton therapy.

Some bureaucrat at CMS considers an overpriced drug and proton therapy to be examples of the same problem, namely, unjustified cost. So he judges proton therapy to be low-value and decides to cut the price. For someone uninformed about proton therapy, such logic seems to make sense. But, CMS IS WRONG on all counts, as I will show.

That negative assessment of proton therapy by PubMED didn’t come out of the blue. Shortly, I will look deeper into the origins of their faulty conclusion, especially a bogus report by ICER and double-dealing by ASTRO. Reducing government waste is admirable, but penalizing proton therapy is not the way to do it. Consider this metaphor to help make my point.

Suppose the government needs to purchase a vehicle for transportation. Car A is cheaper than car B; so, car A should be the choice, right? Studies conducted by car A show that it can get across town just as well as car B. But what if there are other considerations. The roads are bumpy. Car A has poor shocks, causing kidney damage to the driver (with resulting claims and medical costs) whereas car B has a smooth ride. Car A is poorly designed, whereas car B is ergonomically proportioned. The driver of car A arrives exhausted and needs a chiropractor, but the driver of car B arrives rested and ready to work. Car A offers no guidance whereas car B has a great GPS system. Car A is plain Jane, whereas car B has features that improve safety and reduce accidents.

Clearly, price alone should not be the only consideration. But suppose car A has strong lobbying, a large organization, and political pull. It proposes a regulation that the government should pay the same for all cars, knowing full well that such an action will put car B out of the running. Now, car B is at a disadvantage, rated low-value, and the government's savings are imaginary. You probably have seen through my metaphor. Car A is x-rays, car B is proton therapy.

MedPAC has a tough job in controlling expenditures. Recently they admitted that even efficient hospitals can’t cover costs with Medicare reimbursements. They are too low. So MedPAC has recommended a two percent increase for 2020. Yet at the same time they propose cutting reimbursement for proton therapy to below its cost. They have the right mission, but the wrong solution.

The misrepresentation of proton therapy

Some would see my mission as Quixotic. Clearly this is a David and Goliath situation. I am fully aware that others of much higher standing have tried and failed. Here is an example.
In May of 2018, representatives of the National Association for Proton Therapy (NAPT) took aim at the mischaracterization of proton therapy by the Medicare Payment Advisory Commission (MedPAC) as being of low-value. Here are some excerpts from their letter to James Mathews, Ph.D. Executive Director of MedPAC.

Dear Dr. Mathews:

On behalf of the National Association for Proton Therapy (“NAPT”), thank you to you and your colleagues for taking the time to meet with us earlier this month. We appreciated the engaging discussion regarding the value of proton beam therapy to Medicare beneficiaries and to the program overall. We look forward to continuing the dialogue with you regarding this important service. We are writing in regards to the upcoming June MedPAC report to Congress which, as you noted at the end of our meeting, will include a discussion of proton beam therapy as a case study for potentially low-value services.


The NAPT representatives offered better data and information, in person, to enable a more appropriate designation for proton therapy. Here is what they said.

We request that additional consideration for analysis and conclusions by the Commission be given based on the data we presented. The Commission defined a "low-value service" as one where the service provides little to no clinical benefit and the risk of harm outweighs any potential benefit that may exist. We respectfully disagree with the characterization of proton beam therapy as a low-value service for the following reasons.

Oh, how polite those NAPT folks were in their letter. They “respectfully disagreed?” They should have grabbed the guy by the throat and said, “LOOK AT THE FACTS.” Here are the points they made.

--Proton Beam has a long clinical history

--Proton therapy was cleared by the Food and Drug Administration (FDA) thirty years ago.

--A critical clinical benefit of proton beam therapy is the elimination of excess radiation to healthy tissues and organs, minimizing costly side effects and secondary tumors.

--It is the standard of care treatment for multiple diagnoses and clinical evidence has demonstrated its value in other common cancers.


How did it work out? The report went to congress as scheduled, unchanged. (See: http://medpac.gov/docs/default-source/reports/jun18_ch10_medpacreport_sec.pdf?sfvrsn=0) The unfair rating of proton therapy as low-value continues to this day and was part of the basis for the current CMS proposal. Was that just an oversight? Did they really find no reason to switch their old and outdated information for the new more recent data presented by NAPT? Were they just incompetent, or did they get some help from the critics of proton therapy?

.

1. CMS fell prey to a deliberate and orchestrated attack against proton therapy.

For almost three decades now there has been a competition between x-rays and proton therapy. X-ray practitioners come to the battle with an overwhelming arsenal of tools. Their numbers, organizations, financial and political clout present formidable barriers which put proton therapy at a disadvantage.

Radiologists must coexist, sometimes under the same roof, the same practice, or the same organization. And so, the elephant in the room that no one seems to be mentioning is that x-ray radiologists have deliberately thrown obstacles in the way of proton therapy. Whether their actions have been deliberate is up for debate. They have circled the wagons and taken the following strategies.

-- Continuing to quote statistics and results from the earliest studies with passively scattered protons instead of more recent ones with pencil beam scanning.

-- Condemning with faint praise: “Oh, yes, proton therapy has some potential, but it is still unproven. Here, let me show you my shiny new x-ray equipment.”

-- Demanding peer-reviewed double-blind comparative studies before recognizing the efficacy of proton therapy, even though the current popular x-ray technologies such as IMRT never underwent similar examination and such studies may be unethical.

-- Publishing “official” standards for insurance coverage and public information suggesting limited approval of proton therapy.

-- Failing to give proton therapy reasonable coverage in their publications (with one major exception).

-- Failing to refer patients for proton therapy even when it has been shown to be more suitable (and even when proton therapy is available in their own health facility).

-- Lobbying for official government policy that thwarts the development of proton therapy (i.e., the proposed CMS rule).

Am I being hysterical? Overcome by conspiracy theories? Let’s look. The proposed CMS regulation that would penalize proton therapy was suggested by the American Society of Radiation Oncologists (ASTRO), which must have known perfectly well what it was doing and the damage its policy would cause to proton therapy. In a letter to CMS, ASTRO points out, “We recognize that CMS has sought to align some of the proposal with the Radiation Oncology Alternative Payment Model that ASTRO submitted in April 2017.”

Further, it says, “ASTRO has worked for many years to craft a viable payment model that would stabilize payments, drive adherence to nationally recognized clinical guidelines, and improve patient care. We appreciate the administration's focus and commitment to ensuring radiation oncologists' ability to participate in an advanced payment model.”

The anti-proton therapy CMS regulation was SUGGESTED by ASTRO!

“The proposed payment model is a step forward in allowing the nation's 4,500 radiation oncologists to participate in the transition to value-based care that improves outcomes for cancer patients,” said Paul Harari, MD, Chair of the board of directors of the American Society for Radiation Oncology (ASTRO).

“Value-based care” means not paying higher rates for proton therapy.

ASTRO would be the first to point out that some of its members are proton therapy radiologists, and its journals publish articles about proton therapy. True. In 2016, they were kind enough to send me the special edition of the Red Journal that is dedicated to proton therapy. Despite this, they have been no friend to proton therapy. After attending one of their conventions in San Antonio, my review about the presence of proton therapy was entitled “Drop in the Bucket.” (See www.proton-beam-therapy.com/blog.DropintheBucket.html). The event and the organization were overwhelmingly focused on x-rays.

While there, I encountered a stand with a selection of ASTRO publications intended for the general public about the treatment of cancer. Proton therapy was poorly represented, if at all. For example, in the treatment of brain tumors, one area in which there is almost universal appreciation of the superiority of proton therapy, the sole paragraph said this:

Proton beam therapy delivers radiation therapy using particles instead of an x-ray beam. The benefit of proton therapy is that there is little or no radiation dose beyond the treatment area. This means that your doctor may be able to decrease dose to the surrounding healthy brain, which could lead to fewer side effects during or after completion of radiotherapy. Proton therapy may also allow delivery of radiation a second time or a high dose of radiation for certain tumors involving the base of the skull. This treatment is not yet widely available throughout the United States. Ask your doctor if proton therapy might be beneficial for you.

Notice the qualifying “may.” It should say "does." Chances are “your doctor” is unfamiliar with proton therapy. They didn’t suggest getting a second opinion from a proton therapy center, where they could get a realistic assessment of their situation, or learning more about proton therapy.

One ASTRO booklet describes radiation therapy as “the careful use of high-energy x-rays or particles to safely and effectively treat brain tumors.” How many people would even understand the reference to “particles?” It doesn’t even say the words “proton therapy.” (Yes, there are also carbon particles, but I hardly think the intent was to be inclusive.)

What about prostate cancer? The brochure says:

Intensity modulated radiation therapy (IMRT) and image guided radiation therapy (IGRT) are treatment approaches that allow the radiation beams to treat the cancer and lessen the risks of side effects.

Did we forget something here? Something about how proton therapy has fewer side effects than x-rays? Oh wait, look in the next paragraph:

In most cases, external radiation is in the form of high-energy photons, or x-rays. In a few clinics around the country, proton beam therapy is used to treat prostate cancer. Proton therapy is a form of external beam radiation therapy that uses protons rather than photons to treat cancer cells.

That’s it. A few clinics (thirty-three, actually). Nothing more. A whole page is dedicated to a list of potential side effects which may be controlled through medications or changes of diet. The brochure implies that these side effects are typical of all radiation treatment, without pointing out that they are considerably diminished with proton therapy.

Surely they must give proton therapy more credit when it comes to breast cancer, as it has been shown to reduce or eliminate serious damage to the lungs or heart, right? The ASTRO brochure says this: “Typically, radiation therapy is done with high-energy x-rays or photons.”

That’s it. There is NO MENTION of proton therapy in the ENTIRE BROCHURE. None! It goes so far as to state that heart injury for left-side breast cancer is a ”very rare potential side effect.” As I was writing this paragraph (early November) the evening news reported a new study (Harvard?) that showed how frequently x-rays for breast cancer have led to fatal heart conditions down the road. Fatal! Not so rare.

Maybe the author didn’t read ASTRO’s own Red Journal in 2016 showing the benefits of proton therapy for breast cancer. For example:
Bradley JA, Dagan R, Ho MW, Rutenberg M, Morris CG, Li Z, Mendenhall NP. Initial Report of a Prospective Dosimetric and Clinical Feasibility Trial Demonstrates the Potential of Protons to Increase the Therapeutic Ratio in Breast Cancer Compared With Photons. Int J Radiat Oncol Biol Phys. 2016 May; Vol. 95(1):411-21

One more example. The ASTRO brochure on external beam radiation states, “The treatment team uses a machine outside the body to direct radiation beams such as high-energy x-rays at the cancer.” At some length, there follow descriptions of 3-D CRT, IMRT, IGRT, and stereotactic radiation, all forms of x-rays. Finally, they mention “neutron beam therapy” that is “available at only a few centers in the country.” Neutron beam therapy? Really? There is no further explanation there, although elsewhere ASTRO offers this description: “Neutron beam therapy (NBT) is a less widely available form of EBRT that utilizes neutrons. Its clinical use is very limited due to difficulties in the delivery of this treatment modality.”

They mention neutrons in their booklet but not proton beam therapy! Yeah, those neutron centers are hard to find. Better choose x-rays instead.

Not only does the ASTRO literature underplay the benefits of proton therapy, it overstates those of x-rays.

Long-term side effects of radiation therapy may occur in any normal tissues in the irradiated area. With careful radiation therapy planning using improved technologies, serious long-term side effects are uncommon.

Uncommon? Really?

Nancy P. Mendenhall reports in The International Journal of Radiation, Biology, Physics (Vol. 88, Issue 3, pp. 596-602) that IMRT (x-rays) increases the risk of potential recurrence for prostate cancer by fifteen times! MD Anderson Clinic in Houston calculated for esophageal cancer X-rays have a whopping 96% additional cancer risk. How uncommon is that!

Here’s another example.

Secondary cancer is a significant cause of death of patients cured for Hodgkin's lymphoma by radiation therapy. IMPT (proton therapy) is expected to reduce cardiac toxicity compared to VMAT by reducing dose to the heart and substructures. Patients with both pre-existing heart disease and tumour and nodal spread overlapping with or inferior to the T7 vertebrae are likely to benefit most from proton over photon therapy.
(From “Proton vs photon: A model-based approach to patient selection for reduction of cardiac toxicity in locally advanced lung cancer.” Radiother Oncol. 2019 Aug 17. pii: S0167-8140(19)32973-1. doi: 10.1016/j.radonc.2019.06.032.)


Note this is a 2019 reference. I wouldn’t call death a side effect nor would I call any treatment a cure that leads to death.

A study in the United Kingdom reported that 523 (52%) out of every 1,000 new cancer patients are prescribed X-rays as part of their treatment. Out of these, 120 patients (23%) will require re-treatment. X-rays fail in almost one out of four treatments.

Another ASTRO roadblock to proton therapy has been their recommendations for insurance coverage. Initially, insurance companies were covering proton therapy. Then, in 2009, ASTRO suggested that proton therapy is unproven and gives no better result than x-rays. They acknowledged proton therapy was suitable for a small number of cancers which they included in Group I. The rest, in group II, should be reimbursed by insurance companies only if they are in a trial or a registry. Using this document as their rationale, most insurance companies then cancelled their coverage for proton therapy. That in turn forced many patients to choose x-rays, which were covered.

Here is the kind of language ASTRO uses in describing proton beam therapy (PBT).

An assessment of patient suitability for PBT is an important step in the process of care. Changes in the density and composition of tissues in the path of the beam have much greater impact on the delivered dose for protons than photons. Tissue interfaces, especially those with large differences in electron density, can lead to larger or unacceptable dosimetric uncertainties in PBT for certain patients. . . While PBT plans may be more conformal than X-ray therapy plans, they may also be more susceptible to uncertainties in patient positioning or proton range in the patient.

This is an example of condemnation by faint praise. Yes, proton therapy is more conformal (in other words, accurate) but watch out for those uncertainties. It’s true that there are uncertainties caused by changes in tissue density and available electrons. Proton therapy is unique in having some uncertainty with the distal edge at which the protons stop and release their energy The question is one of precision. MD Anderson describes proton therapy as a “196-ton, cancer-killing machine with sub-millimeter precision.” Similarly, the proton center at Miami Cancer Institute promises, “Proton therapy delivers radiation with pinpoint accuracy, with little to no dose to tissues beyond the tumor, making treatment very effective and much gentler.”

On the other hand, X-rays have no distal uncertainty. They just keep going right on through with great certainty. Protons have been shown to be more accurate, have fewer side effects and spare most healthy tissue. That’s what counts. Oh, ASTRO forgot to mention that.

I have spent quite a few pages outlining why ASTRO is not a friend to proton therapy. Their proposals to CMS regarding insurance coverage would cripple the proton therapy industry, which they both know and want.


2. Faulty data underlies the poor policy proposal.


The proposed CMS rule is based on FLAWED DATA. What is even more aggravating is that they are either unaware of this fact, or are deliberately refusing to update their information or change their assessment. When considering negative studies about proton therapy, one or more of the following is usually true.

--The study is seriously out-of-date and no longer applicable.

--The study is biased in favor of a specific result.

--Recent upgrades of proton therapy are not taken into consideration.

Suppose a piece of software was invented in 1990 and consistently updated and improved. Would you base your purchasing decision in 2019 on an evaluation that was written in 2008 for an earlier version with many fewer features? Hopefully not. Yet, that’s exactly what’s happening with Medicare’s evaluation of proton therapy. As I will show, fallacious and outdated sources continue to be used as the basis for current policies.

For the last six years, the capability of proton therapy has been enormously enhanced by the introduction of pencil beam scanning (PBS). Instead of using a shaped continuous beam (passive double scattering), PBS lays down a series of overlapping spots in successive rows and layers, “painting” the target. This improvement vastly increases the viability of proton therapy from 20% of all cancer types to 80%. (See my book, Proton Therapy: Revolutionary Treatment for 80% of ALL Cancers.)

Picture a jar filled with a random mixture of red and white marbles. PBS could target one color marble while sparing the other. The targets don’t need to be contiguous. Neither double scattering nor x-rays can do that. PBS is a game changer.

In my books and blog entries, I advise prospective patients to seek out a proton center that has PBS. Three of the earliest proton centers (MD Anderson, Mass General, and the University of Florida) are expanding their facilities so as to have PBS capability. Eventually, all centers will have it.

Almost all the studies that say proton therapy is no better than x-rays are comparing early double scattering procedures to the best and most accurate version of x-rays. Even studies published as recently as 2018 still use comparisons with double scattering. A study using data prior to 2014 is out-of-date, and the results should be regarded as historical, not current. You must be careful in that some recent articles still quote old data. Check the footnote and see the date of the reference. Decision makers at CMS clearly don't even know about this critical distinction between double scattering and pencil beam scanning. To them, radiation is radiation.

A study that has greatly influenced CMS policies is a stellar example of being out-of-date. The MedPAC analysis that judges proton therapy to be of low value specifically quotes the outdated 2014 study by the Institute for Clinical and Economic Review (ICER).

ICER is fast becoming very influential on the US healthcare scene. ICER is a Boston-based independent nonprofit organization that seeks to improve healthcare value by providing comprehensive clinical and cost-effectiveness analyses of treatments, tests, and procedures.

On March 28, 2014, ICER published an impressive 256-page Health Technology Assessment for the state of Washington that was far from scientifically vigorous, despite its length. While it states that the general time frame for the data in the report was January 1990 to January, 2014, the vast majority of the studies date to the first fledgling years while proton therapy was still establishing its protocols more than a decade ago. For a particular cancer, the report decried the fact that only a single study was available regarding proton therapy. Really? In 2014?

Using their proprietary evaluation system, ICER rated the proof for proton therapy as scarce and of low quality, thereby concluding proton therapy itself to be of low value. Not only did their analysis lead to denial of proton therapy coverage in Washington State, this unfair and self-serving conclusion continues to do damage to proton therapy even today, influencing the currently proposed policy of CMS.

So, let’s take a closer look. After much verbiage, frequent repetition, and a full 116 pages of graphs, the ICER report stated the following conclusion:"

“...we judged PBT to have superior net health benefit for ocular tumors, and incremental net health benefit for adult brain/spinal tumors and pediatric cancers. We felt PBT to be comparable to alternative treatment options for patients with liver, lung, and prostate cancer as well as one noncancerous condition (hemangiomas). Importantly, however, the strength of evidence was low or moderate for all of these conditions. We determined the evidence base for all other condition types to be insufficient to determine net health benefit, including two of the four most prevalent cancers in the U.S.: breast and gastrointestinal (lung and prostate are the other two). Current authoritative guideline statements and coverage policies relevant to Washington State reflect these uncertainties through coverage restrictions or limitations on recommendations for use.”

Am I reading this wrong? Did it first say PBT is comparable to x-rays when it comes to lung and prostate cancers, and then immediately say those cancers are among the four cancers for which there is insufficient evidence to determine net health benefit? “Importantly, however, the strength of evidence was low or moderate for all of these conditions.” Despite their own admission of insufficient evidence, they made a drastic conclusion that exists to this day.

I found that many conclusions in the 2014 Washington report were taken from a 2008 report done by ICER six years earlier comparing proton therapy with brachytherapy. At that time, there were only four proton therapy centers in the United States. Four! (Today there are thirty-three.) That’s ancient history.

The ICER report led to limiting the availability of proton therapy in the state of Washington. So egregious was their under-evaluation of proton therapy that the National Association for Proton Therapy published a rebuttal. Yet, this flawed ICER report is mentioned in the MedPAC study calling proton therapy low-value, to wit:

Unlike Medicare’s relatively broad coverage of proton beam therapy, Washington State has more limited coverage of this treatment for state government health insurance programs. The state covers proton beam therapy for ocular cancers, pediatric cancers, and central nervous system tumors, but covers it for other nonmetastatic cancers only at the state agency’s discretion and only if the patient has had prior radiation in the expected treatment field with contraindication to all other forms of therapy (Washington State Health Care Authority 2014). Washington State has a unique health technology assessment program to determine which services will be covered for state employees, FFS Medicaid beneficiaries, and workers-compensation claimants. An independent clinical committee of health care practitioners—the Health Technology Clinical Committee (HTCC)—reviews evidence-based reports about whether certain medical devices, procedures, and tests are safe and effective to determine whether the state should pay for the technology. The HTCC bases its decisions on the safety, effectiveness, and cost-effectiveness of the technology. The state used this process to determine coverage for proton beam therapy.

Sounds like the HTCC did its homework, right? MedPAC was impressed by their unique health technology assessment program. Note this article.

Boston, Mass., July 9, 2014 – The Institute for Clinical and Economic Review has produced a final evidence report on the comparative clinical effectiveness and comparative value of proton beam therapy (PBT) relative to conventional radiation treatments for 16 types of cancer and 3 benign tumors. The report, which highlighted the lack of evidence for most uses of PBT, was prepared for the Washington State Health Technology Assessment program’s Health Technology Clinical Committee and was an integral part of the ultimate coverage decision on the use of PBT for individuals covered in the state’s Medicaid, public employee and workers’ compensation programs.

We see that the high-sounding Health Technology Clinical Committee got its information from the discredited ICER report. This is part of the evidence CMS refused to update when approached in 2018 by the National Association of Proton Therapy. In their letter, the NAPT clearly notes that they presented newer data and information, likely about pencil beam scanning and more recent favorable studies. It was ignored.

Think about the motivation for a private company that sells itself as a watchdog for excessive spending. Suppose you are Washington State and have paid six figures for a report that concludes, “No problem. Proton therapy is a wonderful technology. Spend away.” Did you get your money’s worth? Would you hire ICER in the future? But, what if the report says, “We conclude that you should watch out for this new technology. Avoid paying that high price.” Then, your expenditure for the study seems justified, and ICER gets more business.

Or does it? ICER has gone on to become involved in the analysis of drugs, for which it has been criticized for being biased by a Medicare agency that stated:

Other stakeholders assert that ICER’s evaluations of the affordability of drugs favor insurance companies. In addition, representatives of pharmaceutical and medical device manufacturers and other health care organizations have raised many concerns about ICER. For example, these stakeholders have (1) asserted that ICER’s models used to assess a therapy’s value are not sufficiently transparent to the public; (2) taken issue with the methods used to assess value (e.g., the over-reliance on data from randomized clinical trials and the use of QALYs to assess cost-effectiveness); (3) asserted that patients, patient groups, family caregivers, and others have not been sufficiently engaged in the analytical process.

Considering this criticism that ICER gives the customer what they want to hear, compare again their conclusion about proton therapy in light of what we know about proton therapy today.

There is only sparse evidence comparing PBT to other forms of radiation as well as alternative treatments. To date, there has been only one randomized controlled trial comparing PBT to an alternative treatment, and few high-quality observational studies are available. . . . There was insufficient evidence to determine whether PBT offers any improvement over alternative treatments for many other conditions, including bone, breast, gastrointestinal, head and neck cancers, lymphomas, and benign arteriovenous malformations. . . In other areas, however, including common cancers such as breast and prostate, the poor evidence base and residual uncertainty around the effects of PBT is highly problematic.

I read all 265 of those ICER pages and made about 60 pages of rebuttal notes. Here are just a few more examples of how inadequate the ICER report is, which in turn discredits the MedPAC report. Regarding treatment for lung cancer, ICER made this conclusion.

Nearly all outcome and toxicity measures were reported for the entire cohort only. However, the rate of pneumonitis was found to be significantly higher among PBT patients (33% vs. 15% for IMRT/3D-CRT, p=0.04).

Now let’s go forward five years to an article in Applied Radiation Oncology by Cynthia E. Keen entitled “Safer RT treatments for esophageal cancer with proton therapy.” Here is what she reported about a study at MD Anderson in Houston.

Average doses to the heart and lung were 6.5 Gy and 11.6 Gy for PBT compared with 10.0 Gy and 19.9 Gy for IMRT.  The PBT group was also associated with a significantly lower distant recurrence rate (45.0%) compared to the IMRT group (33.3%). Five years following treatment, the PBT group also had significantly higher overall survival (41.6% vs 31.6%), progression-free survival (34.9% vs 20.4%) and distant metastasis-free survival (64.9% vs 49.6%).

I think a 30% superior survival rate trumps a comparison of pneumonitis. However, let’s continue with that thread, as there is another oft-quoted study that says proton therapy is no better than x-rays when it comes to pneumonitis when treating lung cancer lymph nodes. So misleading were the conclusions in that study, that I dedicated an entire blog post to its refutal. (See www.proton-beam-therapy.com/blog.Flawed.html).

The lung study is widely quoted by critics as demonstrating that protons are no better than X-rays. This article in MedPage Today heralded the results of the trial with the headline: “No Reduced Toxicity for Proton Tx vs IMRT in NSCLC.” Proton Tx stands for proton treatment. IMRT is Intensity-Moderated Radiation Therapy, a type of X-ray treatment. NSCLC stands for Non-Small Cell Lung Cancer.

The purpose of the study was not to determine the effectiveness in killing the cancer. Both x-rays and proton therapy do that. The study concedes that proton therapy exposes less heart tissue to radiation than x-rays, but it was measuring pneumonitis.

The study was done in 2012 but published in 2018. That alone is suspicious. So is the fact that the proton procedures were poorly done for the first half of the study. Both x-rays and protons were beamed from one direction to treat all the lymph nodes, resulting in considerable pneumonitis by the protons. Then, halfway through the study, the proton therapy technique was changed to beam from both sides, which is possible because of the stopping power of protons. Instead of treating protons as if they were x-rays, they used proton therapy to its best advantage.

From that point onward, x-rays continued to produce pneumonitis at the same rate as before, but proton therapy produced none. None! Zero! And this wasn’t even pencil beam scanning. Yet, when the statistics were totaled for the entire length of the study, rather than the second half, proton therapy produced slightly more pneumonitis. So the headlines proclaimed that proton therapy had a worse outcome than x-rays when, once each modality was properly utilized, the results overwhelmingly favored proton therapy.

How data are handled and studies designed are relevant to the fair representation of proton therapy. Some of the most widely repeated negative statements about proton therapy are biased, outdated, and unrepresentative.

3. The level of proof demanded of proton therapy is unreasonable and probably unethical.


Level I prospective comparative clinical trials have long been considered the strongest type of proof. This is the standard frequently promulgated by organizations both within the government and in the public sphere. It is not surprising, then, that such demands are being made of proton therapy. For example, this from ASTRO: “There is a need for more well-designed registries and studies with sizable comparator cohorts to help accelerate data collection.” Over and over critics of proton therapy repeat, “unproven, needs more data, must have double-blind comparative studies.” Critics of proton therapy feel justified in their demands, even when they don’t apply that same stringent requirement to x-ray technology.

The insistence on level I trials is flawed, unlikely, and unethical. Lack of such studies should not be the basis for proposing a rule that would set back the availability of such a promising technology. As well-meaning as they may be, demands requiring level I studies before endorsing proton therapy are wrong for these reasons:

-- Such a requirement is not universally recognized as necessary.

--There are many types of studies and proof besides level I.

--Opponents make their demands for reasons other than scientific purity.

--Random clinical trials in many cases are difficult to the point of being impossible.

--There are serious ethical considerations that obviate such studies.

--This level of proof has not been demanded of current x-ray technologies.

--Lack of insurance coverage makes such studies difficult.

There are hundreds of studies about proton therapy. Just go to PubMed and look. Every proton therapy center traces its results, conducts trials, has registries, and keeps quality of life data. Yet, claim the critics, this isn’t enough. In an opinion piece in the Red Journal in 2016, Harvard radiation oncologists Drs. Braunstein and Warren contend the following:

. . . although cohort studies can demonstrate the feasibility of protons, they cannot alone be used to shift the standard of care. Leaders in the field have cited profound resistance to the initiation of randomized controlled trials, owing to the widely accepted physical benefits of protons.

Note “demonstrate the feasibility of protons” and “widely accepted physical benefits.” Still, they feel more level I studies are necessary. To achieve this, they suggest proper financing to make such trials more likely. That’s a better idea than taking away support for proton therapy as suggested by CMS. As a patient advocate, my position is that we don’t need to wait for more trials. Let’s get on with proton therapy.

The Harvard doctors go on to suggest that lacking such studies, proton therapy should be reimbursed at the same rate as x-rays. They admit, “This reference pricing approach will undoubtably limit the viability of certain centers that were financially dependent on the price premium of protons.” They don’t seem to be bothered by the closing of some centers. I am, and so should Medicare.

Comparative trials are not only expensive they are hard to fill. Without sufficient participation, the results are not as reliable. The National Cancer Institute (NCI) and the Patient-Centered Outcomes Research Institute (PCORI) have funded seven phase III randomized trials comparing proton therapy and photon therapy. After two years, the breast cancer trial enrolled only 317 patients of 1,716 needed and in five years, the prostate cancer trial enrolled only 254 out of 400.

Reportedly, cancer patients refuse comparative trials because they want to be assured they will be in the proton therapy arm. This was true for my wife’s cousin who declined to be in a trial for that very reason. He proceeded to be treated with proton therapy (covered by his government insurance).

A very important trial occurring right now is the ongoing COMPPARE trial (NCT03561220) documenting bowel, urinary, and sexual dysfunction in prostate cancer treatments. The University of Florida proton center has organized this PCORI-funded trial in which “almost all of the proton facilities throughout the country are cooperating, as well as a large number of academic centers that don't have protons. The trial will accumulate 3000 patients in a fairly short period of time, and will allow us to really compare those results.” How ambitious is that! There is no shortage of trials for lack of trying.

Besides enrollment issues, there are important ethical considerations that constrain random trials. If one were testing the best design for parachutes, would it be proper to give one arm of the study inferior models to see whether they open? Can you ethically cause suffering or death to prove a point? If you sincerely believe proton therapy is better, can you subject people to x-rays instead?

The clamor for proof fails to acknowledge historical precedent. When 3D x-ray technology replaced 2D technology, the benefits were so clear that acceptance became universal within a couple of years. Similarly, IMRT and other x-ray modalities have been embraced because the benefits were obvious. They were not required to provide years of level I trials. The benefits of proton therapy are equally obvious.

To complicate things even further, lack of insurance coverage reduces the availability of proton therapy participants. Insurance companies have been encouraged by model policies suggested by ASTRO to deny coverage for proton therapy. This resistance has reached such an unsupportable level that several states have now passed legislation forbidding insurance companies to hold proton therapy to a higher standard than it demands for other modalities.

The insistence on level I prospective comparative clinical trials should not be the sole standard by which proton therapy is best judged. There is a preponderance of favorable evidence consisting of clinical results, prospective studies, client-reported results, quality of life, and other input available right now. Let’s move forward.

4. Obstacles are being thrown in the way of proton therapy.


The proposed rule would throw up serious obstacles to the development of a promising technology. Actually, “promising” isn’t the right word, as it implies a future state. Proton therapy is available now. Still, by medical standards, it may take decades to arrive at its ultimate capacity. After thirty years and 200,000 people treated around the world, is proton therapy still in its infancy? Hopefully. Just think what it will be doing when it reaches adulthood.

Even if Medicare (wrongly) feels protons and x-rays are equivalent, it would still be worth supporting the extra cost of proton therapy due to its superior characteristics. It’s common for developing technologies to be expensive at first, and then become more modest as they progress. This pattern is true for proton therapy. Promising developments such as fractionation (fewer sessions) and flash (a single treatment session) foretell the lowering of costs in the future.
ASTRO has taken a position regarding new x-ray technologies, as follows.

Innovation in radiation oncology has contributed greatly to increased cure rates and reduced side effects from treatment. Yet, the RO Model does not adequately account for the next generation of advances in the delivery of radiation oncology. Practices should be able to continue to invest in innovations that provide clinical benefit for patients. ASTRO recommendation: CMS should pay for new technology at fee-for-service rates and adopt a rate review mechanism for new service lines and upgrades.

They want to invest in innovations that provide clinical benefit for patients. However, they are referring to new x-ray technologies, not proton therapy. Nor, you can be sure, will those new x-ray advances be first studied for ten or twenty years before they can be used. At the same time, ASTRO declares that proton therapy should be consigned to the CMS equal pay model.

The Commission supports the development of alternative approaches to paying for RT services that aim to reduce spending while preserving or improving quality and outcomes. In general, we agree that the proposed RO Model would encourage providers to be more efficient in delivering RT services by holding them accountable for the cost of an episode of care. We encourage CMS to proceed with testing an episode payment model for RT services. However, we have concerns about how CMS would calculate the episode payment rates in this model and how CMS would measure quality. In addition, this model is not consistent with the Commission’s principles for A–APMs. Finally, we support CMS’s proposal to include PBT in the model.

Ah, there you have it in black and white. This is no oversight. It is a deliberate attack on proton therapy. They want their own advances in delivery to be paid at a higher rate, but not proton therapy. Another quotation from ASTRO:

We support including PBT in a radiation oncology model because Medicare’s payment rates for PBT are substantially higher than for other types of external beam radiation therapy. In addition, the use of PBT has expanded in recent years from pediatric and rare adult cancers to include more common types of cancer, such as prostate and lung cancer, despite a lack of evidence that it offers a clinical advantage over alternative treatments for these types of cancer. Therefore, including PBT in the episode payment would create an incentive to use lower-cost, comparable modalities.

The patient would choose a lower-cost, comparable modality; i.e., x-rays. With this kind of double-dealing, I don't see why any proton therapy radiologist would want to be a member of ASTRO. It is working against their interests.

Should Medicare support new and developing technology? An April, 2018, article on the Future Medicine website (see https://www.futuremedicine.com/doi/full/10.2217/bmt-2018-0001) by Kimberly S. Corbin and Robert W. Mutter entitled “Proton therapy for breast cancer: progress and pitfalls” is quite comprehensive. This is the conclusion.

The burgeoning field of breast cancer PBT is in its infancy. The improved target coverage and normal tissue avoidance over conventional photon techniques are well established. Presently, promising PBT applications for both early and advanced stage breast cancer patients are emerging, with potential to improve the therapeutic ratio. . . There are exciting opportunities to improve treatment delivery, optimize schedules, refine patient selection and understand the unique radiobiology of PBT that will impact patient care. This work and robust comparative effectiveness research will establish the future role for PBT for breast cancer in the years ahead.

A joint study by the American College of Radiology (ACR) and ASTRO said this:

Proton radiotherapy may permit improved therapeutic ratios with lower doses to sensitive normal structures and greater dose to target tumor tissues [4]. . . Increasingly, there are now clinical data documenting the outcomes of proton radiotherapy across disease sites with many experiences supportive of a role for proton therapy [7-11]. Proton radiotherapy may be seen as a technological option for the delivery of radiation treatment.

Here’s another example that came up just hours ago as I was writing this. A medical news site I visit has a new article about a trial for liver cancer comparing x-rays and photons.

Treating 133 patients, researchers saw a median survival rate of 31 months in the 49 treated with proton therapy, compared to 14 months in those who received photon therapy. They also observed a decrease in incidences of non-classic radiation induced liver disease (RILD), the onset of which is associated with worse overall survival rates and has been traced back to high doses of radiation. The authors say overall survival in proton patients may have been higher due to lower occurrence of post-treatment liver decompensation, and claim this is the first study to compare both forms of radiation for the treatment of HCC patients.

Here we see a very real difference with a median survival rate of 31 months for proton therapy and 14 months for photons. The study showed proton therapy to be twice as effective as x-rays. Here is the response to that study by Laura Dawson, president-elect of ASTRO:

Generation of high-quality evidence in support of protons to better understand the patient populations most likely to benefit from protons should motivate for more affordable and improved global accessibility of protons.The best type of evidence is from randomized trials. The present studies motivate for randomized studies to be completed, so indirectly they hopefully will help proton therapy to become more affordable in the future.

Instead of saying proton therapy did very well in this study and should be considered for liver cancer, the emphasis is on more studies and lower prices in the future. Nice try, but, the future is now.

5. X-rays and protons are not comparable and should not be treated or reimbursed as such.


Equal reimbursement rates imply equivalency in the results of x-rays and proton therapy. This is the foundation of the proposed regulation for CMS. IT IS WRONG.

Everyone acknowledges that proton therapy is more complex and more expensive to build and run than x-rays. The key is whether it is better than x-rays. Critics say no, but those who work with proton therapy say yes. This difference is the crux of the issue driving the CMS regulation.

In “Three Ways to Make Proton Therapy Affordable,” authors Thomas R. Bortfeld and Jay S. Loeffler state:

If cost was not an issue, proton therapy would be the treatment of choice for most patients with localized tumours. Protons can be targeted more precisely than X-rays, so the tissues around the tumour receive two to three times less radiation. This lowers the chance of causing secondary tumours or impairing white blood cells and the immune system. High doses of protons can be delivered safely to hard-to-treat tumours: for instance, those at the base of the skull or in the liver. Such accuracy is crucial when treating cancers in children.

“If cost was not an issue . . .” protons would be the treatment of choice. So, what can critics of proton therapy do? Make cost a big issue. The correct model for cancer treatment is not free market survival-of-the-fittest. A better plan would be to encourage and enhance proton therapy with sufficient reimbursement, studies, and funding.

I’ve heard many stories in which x-ray radiologists promoted their modality to patients while either undervaluing proton therapy or not mentioning it at all. This is such a prevalent practice that even within health systems that have both x-rays and proton therapy, x-ray practitioners fail to refer their patients for proton therapy. They continue to resist the inevitable.

I have a friend who was referred to an x-ray radiologist at MD Anderson by his family practitioner who was likely unfamiliar with proton therapy. So my friend was treated for a brain tumor with traditional x-rays, which caused considerable cognitive damage. After attending one of my lectures about proton therapy, he asked me why he hadn’t been treated with protons at MD Anderson to prevent the damage that he had experienced. Good question. His x-ray radiologist certainly knew the superiority of proton therapy for brain tumors.

How different are x-rays and protons? Electrons used to create x-rays have negligible mass or electrical charge whereas protons have substantial mass and a positive charge. If I were a hydrogen electron (I weigh 175 pounds), my corresponding proton would be sixty tons. Tons! They aren’t equivalent. Their physical characteristics are hugely different. The large mass of protons reduces beam broadening and scatter seen with x-rays.

The widely accepted relative biological effect (RBE) for x-rays is 1.0 and for protons 1.1. That means protons have the same effect with ten percent lower dose, or with the same dose, they are ten percent more effective.

I previously mentioned flash technology in which a single blast of very high dose protons obliterates the cancer but spares the healthy tissue. Think of how that will revolutionize cancer treatment. The cost will plummet.

The proton center in Beaumont, Michigan, is developing SpaARC, in which protons are beamed from multiple angles as the gantry turns. Proton therapy can adopt techniques from x-rays, but x-rays will never be able to deliver spots or layers or stop at the target. Proton therapy was approved by the FDA in 1988 as being safe and effective. The cogent question is whether it is worth the extra cost. The answer is that it has lower toxicity and side effects. These conditions make a big difference to us patients, but not much to my insurance company which doesn’t care if I wear diapers the rest of my life or lose sexual function as long as it can save a few dollars and make a higher profit.

I remember back during the passage of the Affordable Care Act (Obamacare) talk of “death panels” that would decide who is or isn’t covered for specific treatments. Insurance coverage by Medicare that forces patients to choose x-rays over proton therapy can lead to greater pain and suffering and more deaths.

X-rays and protons are different in the short run as well. One case in which greater toxicities happen is in combining chemotherapy and radiation. A new study presented June 1, 2019, at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago analyzed data collected on 391 adults who received proton therapy and 1,092 adults who received standard photon therapy within the University of Pennsylvania Health System in Philadelphia. Patients had eight different cancer types, with lung cancer and head and neck cancers being the most common. The patients all received chemotherapy alongside their radiation therapy. Over a 90-day period, 11.5% of the proton therapy patients and 27.6% of the x-ray patients experienced side effects requiring hospitalization.
With proton therapy, paying more up front can save greater expense and more complications in the long run. Medicare is short-sighted not to take this into account.

ASTRO does mention proton therapy in its letter to CMS, as follows.

CMS invites comment on its proposal to include proton beam therapy in the RO Model. The Agency sites reports from Institute for Clinical and Economic Review (ICER) and the Medicare Patient Advisory Commission (MedPAC) in which both groups purport that proton beam therapy is of lower value when compared with other forms of radiation therapy.

ASTRO wishes to point out that this blanket statement is not applicable to all disease sites. While ASTRO agrees with and actively works to address inappropriate utilization of services that do not benefit patient care, PBT can serve as an effective, evidence-based treatment for a specific group of clinical indications, such as ocular tumors, chordomas, chondrosarcomas, primary or metastatic tumors of the spine, hepatocellular cancer, and certain malignant and benign primary CNS tumors, just to name a few. The Agency proposes to exclude proton beam therapy from the included modalities in instances where a beneficiary is participating in a federally funded, multi-institutional, randomized control clinical trial for proton beam therapy so that further clinical evidence assessing its health benefit comparable to other modalities can be gathered.

Excluding proton therapy from the equal payment model would indeed help. While this statement initially appears to be supportive and concedes the benefit of proton therapy for some cancers, it also suggests Medicare pay for most proton therapy only if the beneficiary is in a study or trial, and not just an ordinary trial but a federally funded, multi-institutional, randomized control clinical trial. How likely is that?

This requirement effectively limits coverage. Further, by saying that protons are good for some cancers and suggesting CMS pay for more studies to see whether it is good for other cancers, the implication is that proton therapy is still unproven for those other cancers and therefore should not be covered. ASTRO's list of approved cancers is predictably short.

If ASTRO is a friend to proton therapy it’s hard to see. The beginning of their comment says that the CMS blanket statement regarding lower value for proton therapy is not applicable to ALL disease sites implies that it may still apply to some of them. In addition, the ASTRO statement limits the approval of proton therapy to a much smaller selection of cancers than is currently the practice. So this supposed support for proton therapy is really a thinly disguised attempt to further limit reimbursement.

Let’s give some examples that show x-rays and protons are not equivalent. At the 2017 PTCOG-NA conference in Chicago, the following studies were presented.

Two presentations addressed the treatment of prostate cancer with proton therapy. In a follow up to previous studies utilizing SERR (Surveillance & End Results Registry) and Medicare claims data to compare outcomes between proton therapy and IMRT, physicians from Northwestern Medicine Chicago Proton Center and the University of Florida Health Proton Therapy Institute analyzed a more contemporary and larger population of patients treated with proton therapy and IMRT. The study included patients treated by multiple proton therapy providers from across the country. Compared with the IMRT cohort, the proton patients had a significantly higher 5-year survival rate (94% vs 87%), fewer bladder complications and fewer secondary cancers.

In a direct comparison of clinical outcomes between cohorts of prostate cancer patients treated with IMRT and proton therapy, investigators from the University of Florida Health Proton Therapy Institute and the Mayo Clinic in Arizona found similar complication rates between IMRT and proton therapy, but significantly higher disease control rates with proton therapy for low and intermediate risk prostate cancer patients. Disease control rates for high risk prostate cancer patients were similar between the proton and IMRT cohorts, but hormone therapy was not used as often or for as long a duration in patients who received proton therapy. (See website: www.ptcog.ch)

Commenting on the studies, Dr. William Hartsell, Medical Director for the Northwestern Medicine Chicago Proton Therapy Center stated, "These studies show the potential for proton therapy to improve outcomes for patients, especially in reducing the side effects from treatment, which may also allow for more effective treatment and better disease control."

A review of the above study states:

In particular, this work shows that for a clinically relevant parameter set, proton therapy is superior to photon therapy for a number of reasons. That is, regardless of the schedule of administration, it leads to a superior TCP because of its greater RBE, and because of its greater ability to be targeted to tissue, it has a minimal modelled risk for secondary malignancy. 

I decided to give the critics their voice in this essay. I looked for studies comparing pencil beam scanning proton therapy and photons (x-rays). I went to PubMed and other sites to find any such studies published within the past three or four years. Sorry photons, I just couldn’t find a study saying you were superior to pencil beam scanning. Here are some typical results from studies incorporating pencil beam scanning. These are the type of studies CMS should be considering.

"Proton beam therapy for cancer in the era of precision medicine" (December, 2018)
The dosimetric advantage of protons results in a finite range with little or no exit dose and a smaller volume of normal tissue to be irradiated. It is worth noting that the precision is becoming increasingly more important to take advantage of PBT for patients. The technical advances allow that the precision PBT will become widely available, and it may be the lead application in the treatment of cancer in the future.

“Comparison of Pencil Beam Scanning Proton- and Photon-Based Techniques for Carcinoma of the Parotid” (Spring, 2016)
Pencil beam scanning allows for superior normal tissue sparing while still maintaining excellent target coverage in patients with resected parotid gland cancers. These findings suggest that PBS may allow for an improved therapeutic index for these patients.

“Pencil beam scanning proton therapy vs rotational arc radiation therapy: A treatment planning comparison for postoperative oropharyngeal cancer” (November, 2016)
The significant dosimetric sparing seen with proton therapy may lead to reduced side effects such as pain, weight loss, taste changes, and dry mouth. Prospective comparisons of protons vs photons for disease control, toxicity, and patient-reported outcomes are therefore warranted and currently being pursued.

“Evaluating the benefit of PBS vs. VMAT dose distributions in terms of dosimetric sparing and robustness against inter-fraction anatomical changes for pediatric abdominal tumors” (July, 2019)
For pediatric patients with abdominal tumors, improved dosimetric sparing was obtained with PBS compared to VMAT. In addition, PBS delivered by posterior-oblique irradiation fields demonstrated to be robust against anatomical inter-fraction changes. Compared to PBS, daily anatomical changes proved to affect the target coverage of VMAT dose distributions to a higher extent.

“A Comparison of Grade 4 Lymphopenia With Proton Versus Photon Radiation Therapy for Esophageal Cancer” (2019)
XRT was associated with a significantly higher risk of G4L in comparison with PRT. 

The next study is the “worst” result I could find for proton therapy so I quote it in more detail.

"Temporal lobe sparing radiotherapy with photons or protons for cognitive function preservation in paediatric craniopharyngioma" (Aug., 2019)
RESULTS:
The exposed volumes of the temporal lobes and their substructures were consistently reduced with PBS compared to DSPT and VMAT, e.g. the left hippocampus V10Gy from 100% (VMAT) or 41% (DSPT) to 5% with PBS (p = 0.002). Some of the ventricular substructures were better spared with VMAT compared to both proton modalities. The reduced doses to the temporal lobes achieved with PBS translated into improved predicted memory outcomes, but not for the estimated IQ.
CONCLUSION:
The irradiated volumes of temporal lobe BSCs were consistently the lowest with PBS, whereas the model-based estimates of cognitive outcomes were less consistent.

That’s it. A model-based estimate was inconsistent. That's the best I could do for photons.If ICER and MedPAC had included more recent data, their conclusions would have been much different. Or would they? Is it useless to argue about getting the facts right? Is this really a post-truth world? Is the CMS policy based on politics rather than science? As a concerned observer, it's hard for me not to become cynical.

6. Researchers and decision makers lack direct experience with proton therapy.


Criticism of proton therapy comes almost exclusively from those who have little or no direct experience with the technology. Either they are protecting their own turf (usually x-rays, but also surgery and other modalities) or they are dealing with proton therapy as a theoretical consideration, such as doing research and making recommendations about cost. Some of the studies used by CMS repeat fallacies about proton therapy that an knowledgeable person would know is wrong.

Whoever makes decisions at CMS clearly has little or no direct experience with proton therapy. Their knowledge is secondhand, dependent on the quality and accuracy of the studies that they consult. As we have shown in this paper, the studies that have most influenced the CMS proposed regulation are dated and flawed and misleading, resulting in bad policy. But, they don’t know enough about proton therapy to recognize that.


7. Considering quality of care and cost effectiveness.


The CMS regulation proposes to create more efficiency and lower prices while maintaining the quality of care to patients. By penalizing proton therapy this rule would reduce the quality of care available to patients by limiting access to proton therapy, thereby violating its own purpose.

Proton therapy is indeed expensive. The building, the equipment, the larger staff, the high degree of expertise, all these factors make proton therapy more costly than x-rays. Or is it? Several studies that took into consideration the long-term costs, including the treatment of side effects and cancer recurrence, found that in some cases proton therapy is of equivalent or better value. MD Anderson has demonstrated that partial irradiation for breast cancer is less expensive with protons than x-rays.

But there is another consideration. Considering only cost violates some of Medicare’s own rules and has failed in the past. On two occasions, Medicare tried to interpret Section 1862 of the Social Security Act that requires Medicare to pay only for services that are reasonable and necessary. In 1989, the agency issued a proposed regulation that explicitly considered the cost-effectiveness of services in the coverage process. The proposed rule was never finalized, with stakeholders arguing that the agency could not use criteria for coverage that extended beyond clinical evidence and that the statute did not permit the agency to deny coverage based on cost effectiveness.

May I say that again? Medicare may not deny coverage based on cost effectiveness. Yet that was the whole emphasis of the flawed ICER report and the implication of the “low-value” MedPAC report.
In 2000, CMS released a notice of intent (NOI) on new criteria for considering cost in the coverage process only for services that provided equivalent clinical benefits compared with an existing covered service but that were more costly. This is what they are currently trying to do, again. As with the 1989 proposed rule, the cost criteria were not finalized.

Stakeholders, including medical providers and the medical device industry, argued that
(1) cost had no role in the coverage process,
(2) CMS could not use criteria for coverage that extended beyond what medical experts thought was reasonable and necessary for an individual’s medical need, and
(3) the statute did not permit the agency to deny coverage based on whether a service was or was not cost-effective.
Some stakeholders feared the use of cost effectiveness was a move toward rationing health care.
So, history repeats itself. Or so I hope, meaning the proposed rule will not be implemented.

The two objectives of CMS are somewhat contradictory, namely reduce costs and encourage new technology. But new technology is expensive. At the World Medical Innovation Forum in Boston, CMS Administrator Seema Verma argued that government can often foil innovation. 

I think we are at a turning point in healthcare. We are at an amazing opportunity or crossroads where we have all of this information that is now readily available and we have the tools to move forward. And I think what we need to do is get government out of the way and make sure we are supporting innovators to do what they do best.I think we all have to recognize the important role that the U.S. plays in bringing innovation to the entire world, and recognize it is the private system that we have in place that is spurring innovation, and be very careful about those initiatives that would put more government in control and would thwart innovation going forward. . . The reality is government has often been a barrier to innovation.

Unfortunately, she is talking about administrative types of innovation, artificial intelligence, and software, not treatment modalities such as proton therapy. However, the point is relevant that the government should not stand in the way of innovation or instigate excessive regulations.

The United States leads the world in number of proton therapy centers and research. If the CMS rule deprived centers of adequate income, besides closing their doors, they will cut back on research, trials and registries. As a result, we will cede leadership in proton therapy to other countries, leading many Americans to go overseas for treatment, to England, the Czech Republic, Switzerland, Korea, India, China. Other countries are encouraging the development of proton therapy; we should not discourage it here. On the other hand, if everyone went abroad for proton therapy, ASTRO and the x-ray lobby would be overjoyed. My hope is that the government will side with cancer patients and not with ASTRO.

Here are some pertinent comments by Andrew K. Lee, medical director at Texas Center for Proton Therapy as published online in OncLive.

It is time to look beyond the immediate costs and short-term results when determining what this therapy brings to healthcare. Proton therapy should be viewed for what it is: an investment in improving outcomes and long-term quality of life for patients. At some point, we need to differentiate costs versus value when it comes to this technology. We all agree cost control is important. At the same time, if an incremental cost provides value to a patient, the value needs to be measured over the lifetime of the patient and not just during active treatment or for a few short months into survivorship. The reality of cancer care is that no matter what we do, sometimes the ramifications of treatment stay with the patient for months or years, impacting quality of life and increasing costs for managing toxicities.

A recent study of cancer survivors found that 8%, or 1 in 12 of adults with the most common cancers, eventually develop a second malignancy unrelated to the first. The study also determined second cancers were often lethal, as 55% died of their second cancer and only 13% died from their first malignancy. The implications of these findings are important, because there is a limit to the amount of treatment patients can tolerate during their lifetime. When focusing on a patient’s first cancer, physicians may not always be considering the lifelong therapeutic tolerance of that patient, a factor that becomes critical if a second cancer develops. Proton therapy provides a way to not only control the initial cancer, but also minimize the toxicities of primary treatment, providing an opportunity to more effectively treat a second cancer if necessary. This is a good example of the value added by proton therapy that is difficult to measure and cannot be calculated in the short term.

8. The decision process is a rush to judgment


For changes this drastic, the period for response, review, and revision was inadequate (expiring September 16). After working on my response for several weeks, I discovered the deadline for response had already passed. So I turned this essay into a post on my proton therapy blog (while hoping for wider dissemination). In its letter, ASTRO pointed out that it is inconceivable that this regulation could go into effect on January 1, 2020.

Additionally, it is impossible to imagine that more than 1,100 practices will be notified in early November of their required participation and then start on January 1, 2020. As will be discussed further in our comments, the time, cost and effort burden of implementation on practices, particularly smaller practices, is substantial and CMS estimates of implementation burden are insufficient. Likewise, requirements for EHR vendors and systems needed to operate the model, will take time to develop, test, and implement. Given these factors, ASTRO urges CMS to delay implementation until no sooner than July 1, 2020 to ensure participating practices have had adequate time to prepare for model implementation.

ASTRO believes that the proposed model is likely to decrease quality of care. They point out this very relevant definition. Agency action is considered arbitrary and capricious if the Agency:
1) Has relied on factors which Congress had not intended it to consider,
2) Entirely failed to consider an important aspect of the problem,
3) Offered an explanation for its decision that runs counter to the evidence before the agency,
4) Is so implausible that it could not be ascribed to a difference in view or the product of agency expertise.

It seems clear that by their own definition, the proposed action by CMS is clearly arbitrary and capricious. In the end, I don’t actually want a delay, I want the rule changed to remove the equal pay for x-rays and protons. That is bad policy for the reasons I have been discussing in this paper.


Conclusion


I’m sure the proton industry will respond to this threat, hopefully hiring some effective heavy-hitting lobbyists. As an individual proton therapy advocate, I think we need to have a groundswell of input from citizens, both those who already know about proton therapy because they received it, and those who may someday find themselves with a cancer diagnosis.

I’ve written about this matter to my representative and senators who are all Republicans. That worries me. The Republican Party historically represents the establishment, which means corporations and wealthy people. That could put them on the side of traditional radiologists at the expense of proton therapy. On the other hand, cancer treatment is a concern of all constituents. To fail to stop this damaging proposed regulation as currently written is to restrict public access to a remarkable cancer treatment.

If you agree that proton therapy should be given a better deal, contact Medicare and your congress people and ask them to revise CMS Rule No. CMS-5527-P to allow higher payments for proton therapy. Here’s a link to make it easier: Alliance for Proton Therapy Access: https://www.votervoice.net/PROTON/1/campaigns/68657/respond

Many thanks.

Robert Ferre
www.proton-beam-therapy.com
robert@ProtonTherapyBook.com
November 11, 2019