In my first post, I published the cover letter of the winning appeal. Here is the rest of the letter.
Why My
Condition Requires Proton Beam Therapy (PBT)
Proton radiotherapy is the preferred
treatment modality for Ms. Call’s life-threatening malignant paraganglioma of
the left carotid body. The targets will include the left neck to the base of
the skull. Protons will minimize dose to the surrounding critical structures
including the spinal cord and brainstem… Proton beam irradiation therapy will
be particularly beneficial for Ms. Call given her young age of 28 years old and
long life expectancy. (Dr. Adam Garden, M.D. Anderson Radiation Oncologist,
Appeal letter to Anthem, May 11, 2012)
Within days of my diagnosis with this rare cancer, I met
radiation oncologist Dr. Allen Chen at UC Davis Medical Center. As lead author
of nine recent papers on head and neck cancers—two studies comparing different
radiation treatment types, a study on radiation beam location and implications
for tissue damage, and a study on IMRT outcomes for unknown cancers—he is among
the top physicians in his field and uniquely qualified for work on rare tumors
such as mine. Given the exceptional nature of this case, however, he quickly
reached out to other colleagues in the radiation oncology community. It was
under Dr. Chen’s encouragement that I sought a second opinion at M.D. Anderson.
There I met Dr. Adam Garden, who first introduced me to
Proton Beam Therapy (PBR). Also specializing in head and neck radiation
oncology, Dr. Garden has published eight articles on the detrimental side
effects of radiation therapy. As I learned from Dr. Garden, PBR should minimize
the risk of both acute and chronic side effects, including secondary cancers
and damage to important structures in my neck, head, and jaw. With this
information, the decision to pursue PBR was easy.
Anthem’s recommendation to the University of California to deny
coverage for PBR is based on the claim that the medical reviewers have not seen
any medical studies that show that PBT is as good as other treatments for my
condition. Since immersing myself in the literature, I can only conclude that
Anthem, acting on behalf of the University of California, simply has not
looked. The evidence for PBR’s effectiveness for head and neck cancer—indeed
its superiority—is overwhelming. I
believe that on a more thorough review of the literature, Anthem would overturn
its previous recommendation to the University.
For example, Yoon, et al., (2010) compared IMRT and proton
therapy for patients with head and neck tumors, prostate cancer, and brain
tumors. They found that for head and neck treatment there was a lower relative
secondary cancer rate for all organs, even for the thyroid gland, with PBT.
Purdy (2008) explains that relative to IMRT, PBT reduces the radiation dose to
healthy tissue and the risk of leakage and scattered radiation. In a study of
conventional radiotherapy, IMRT, and PBT, Palm and Johansson (2007) find that
“the data consistently show that proton beam therapy substantially decreases
the OAR [organs at risk] average dose compared to the other two techniques.”
Metz (2002) reaches a similar
conclusion: “Side effects, both acute and long-term, typically seen with
X-ray therapy can thus be markedly reduced with proton beams due to the sparing
normal tissues that are situated around the tumor. These considerations are
directly related to the physical characteristics of the proton beam, and
require no demonstration or study. However, data are available from clinical
series that support them.”
In addition to the physical benefits I expect from receiving
PBT instead of IMRT, there is strong evidence that it will reduce my long-term
healthcare costs as well. Lundkvist, et al., (2005) conduct a cost-benefit
analysis and estimate that a patient of 65 years would spend over $5000 less in
dental bills over her lifetime (in 2012 dollars) by choosing PBR over IMRT. This
number would be substantial higher for me, since at 28 I have a longer life
expectancy, and it does not include any valuation of quality of life or
non-dental health-care costs. The long-term cost-savings and quality-of-life
benefits of proton therapy are discussed by Pijls-Johannesma, et al., (2008),
who write that “It is expected that in the long run, due to a decrease in the
absorbed integral dose, fewer secondary cancers will be induced. Also, due to
the expected decrease in side effects and increase in tumor control probability
(TCP), it is expected that long-term costs will decrease as a result of less
hospitalization, less palliative care and improved quality of life.”
Another head and neck cancer patient, Steve Harris, recently
sought proton treatment at M.D. Anderson. Although his application for
insurance coverage was initially denied, his appeal was upheld. According to the radiation oncologist who
treated Harris, “It's important to minimize any other side effects that can
occur… With head and neck cancer, you’re close to the centers that affect taste
and smell, the swallowing function, and day-to-day speech” (Work, 2012).
M.D. Anderson is not alone in supporting patients seeking
PBR. In fact, “to most radiation oncologists and physicists, the benefit of
proton therapy in many clinical cases is without question; protons allow
sparing of normal tissue, which is unarguably beneficial to patients. Indeed,
to a radiation oncologist experienced in delivery of proton therapy, the
decision of whether or not to employ it in a given case is analogous to a
surgeon’s decision regarding the choice of a particular scalpel or cautery
device”’ (Hill-Kayser, et. al, 2011).
The Term "Investigational" Has No Meaning When Applied to a Rare Cancer
Ms. Call should not be denied the
options available for this treatment of her malignant paraganglioma of the left
neck. Proton therapy is the best and only treatment option and medically
necessary for Ms. Call considering her age of 28 years and her prior
surgical/treatment history. (Dr. Garden, Appeal letter to Anthem, May 11, 2012)
For my case, PBT is no more “investigational” a procedure
than the IMRT therapy that Anthem is recommending the University of California offer.
An investigational procedure, according to my Benefit Booklet, is one that is
“not widely accepted as proven and effective within the organized medical
community.” Anthem’s dismissal of my request for PBR on these grounds is
problematic in several respects. First, as is detailed throughout this
document, the organized radiation oncology community strongly supports the use
of PBR for cancer treatment. Second, the rarity of my cancer and my young age
relative to most radiation patients preclude the identification of a “proven
and effective” treatment for my condition. Finally, advancement of the state of
the art in radiation therapy has never relied solely on clinical trials. IMRT,
for instance, was “widely adopted without comparative trials based on the same
type of surrogate dose distribution modeling that supports the case for proton
therapy” (Robert L. Foote, MD, Professor of Radiation Oncology, Mayo Clinic, as
quoted in Morgan, 2009). Proton therapy for prostate has also been considered
investigational (Suit, et al., 2008), but patient Paul A. Morgan was approved
for treatment by Independence Blue Cross despite the lack of Randomized Phase III Clinical Trials comparing proton
therapy to photon beam therapy.
Awaiting results on clinical trials in a case like mine raises
ethical issues. In a particularly relevant recent article in the International Journal of Clinical Oncology,
a leading journal for peer-reviewed papers on newer treatments, Ogino (2012)
challenges the ethics of demanding clinical trials as criteria for coverage of
care, especially for “rare malignancies … where the limited number of patients
may not enable randomized clinical trials to be conducted. Therefore, a
clinical decision regarding the optimal treatment should be taken even though
the highest level of evidence may not be available.” As in the cases discussed
by Ogino, my tumor’s life cycle is unknown and its rarity makes clinical trials
infeasible. Anthem argues that “medical studies we have seen do not show that
it is as good as other treatments for your condition.” Unfortunately for both
me and Anthem, there are no studies of my condition. However, Chan (2006) studies
another rare head and neck tumor and finds that it was more responsive to PBT
than IMRT.
These ethical considerations are discussed in other top
journals in the field as well. In the Journal
of Clinical Oncology, Goiten and Cox (2008) make the following strong
argument: “Although we can understand (though
not necessarily agree with) the desire to rely on RCTs to establish the
advantage of a superior therapy, we find it totally unacceptable to insist on
what we judge to be unethical RCTs purely to establish the financial cost-effectiveness
of an admittedly better technology—nor would patients, if fully informed,
consent to participate in such studies.”
The most persuasive voice in my case, however, is that of my
own radiation oncologist at M.D. Anderson. As he wrote in his appeal to Anthem,
“proton therapy is FDA-approved and is neither investigational nor
experimental. It has the potential to expedite patient recovery after
treatment and reduces the potential morbidities that may result from
irradiating surrounding organs with more conventional techniques such as x-ray
therapy. This approach minimizes toxicity to the patient and results in a
more rapid recovery resulting in less cost to her (as the patient) and to you
(as her insurer). Specifically, proton therapy will be an outpatient
treatment, and will not require hospitalizations due to disease or
treatment-related side effects.”
Reducing
My Radiation-Related Side Effects Is Medically Necessary
PBT is medically necessary for the treatment of the rare
malignant tumor in my left neck because this treatment would deliver less
radiation dose to the surrounding healthy structures than any other treatment
option. The effects of radiation are
detrimental—this is precisely why radiation will be used to target my
tumor. But if Anthem and the University
of California refuse to approve the recommended PBT treatment, I will be forced
to undergo IMRT, a less suitable form of radiation that will unnecessarily
expose my submandibular (salivary) glands, oral cavity, spinal cord, and brain
stem to radiation.
As explained in greater detail below, leading experts in the
field have determined that PBT is a less risky form of treatment for me, and is
medically necessary for my treatment.
PBT likely will cause far fewer serious side effects than IMRT,
resulting in significant improvement in quality of life, and it carries less
than half the risk of secondary malignancy.
For all of these reasons, I request that Anthem and the University of
California reverse the decision to override the recommendation of the
experienced head and neck cancer specialists, and immediately authorize my treatment
with PBT.
Leading
experts in the field of head and neck oncology determined that PBT is medically
necessary because it is the safest treatment option for the malignant
paraganglioma in my neck, based on factors specific to my tumor.
I was fortunate enough to be evaluated by leading experts in
the field at M.D. Anderson, one of the nation’s foremost comprehensive cancer
treatment centers. Dr. Garden is a graduate of Johns Hopkins Medical School and
Board Certified Radiologist with 24 years of medical experience and practice in
Radiation Oncology. He specializes in
the treatment of cancers affecting the head and neck and has published at least
30 peer-reviewed original research articles addressing the treatment of these
cancers. Based upon my specific case
factors and the risks of radiation-related side effects, Dr. Garden determined
that PBT is medically necessary for me:
Proton beam radiation therapy has shown to
deliver less radiation dose to other critical structures; in Ms. Call’s case
this would include the surrounding organ/tissue
structures of the left neck, spinal cord and brainstem, where pared to
x-ray treatment, including intensity modulated radiation therapy (IMRT). We strongly feel that it is medically
necessary that Ms. Call receive proton therapy for treatment of her
life-threatening malignant paraganglioma of the left neck. (Appeal letter to Anthem, May 11, 2012)
The medical opinion of Dr. Garden should not be disregarded
simply because of a lack of documentation in the medical literature on the
treatment of my specific condition. The
medical evidence concerning the substantial risk of harm arising from treatment
of the head and neck with IMRT are clear.
It is well-documented that PBT eliminates many of these risks. For this reason alone, Anthem should follow
the recommendation of the medical experts, and authorize my treatment with PBT.
PBT
will cause fewer serious side effects than IMRT, resulting in significant
improvement in my quality of life.
“Treatment-related toxic effects of radiation therapy for
head and neck malignancies can be severe” (Slater, 2006). The radiation exposure to the areas of my
head and neck surrounding the tumor will likely cause both short- and long-term
side effects. As a 28-year old soon-to-be cancer survivor, the detrimental side
effects of IMRT could negatively impact my quality of life and my ability to be
fully productive in my fieldwork for decades to come.
The decreased exposure to radiation through treatment with
PBT is significant in cases involving malignancies of the head and neck. “With PBT, unnecessary dose to the adjacent
normal head and neck structures can be limited, resulting in significant improvement
in quality of life during and after treatment” (Slater, 2006). PBT’s unique ability to target the tumor
provides radiation oncologists with the means to deliver higher radiation doses
to the target, improving chances of eliminating tumor cells, while
significantly reducing radiation doses to the surrounding tissues, resulting in
fewer radiation-related side effects.
IMRT causes more serious damage to critical organs than
PBT. In my case, submandibular
(salivary) glands, oral cavity, spinal cord, and brain stem are located in the
area surrounding the tumor bed, and would receive excessive radiation should
Anthem limit my treatment options to IMRT.
A 2007 medical study compared dose
distributions and impacts between PBT, conventional radiotherapy, and IMRT
(Palm and Johannson). This study found
that proton therapy substantially decreases radiation to nearby organs compared
to conventional radiotherapy and IMRT, while providing improved dose
distribution in non-target tissues. The
reduced radiation can preserve organ function, significantly reducing the risk
of normal tissue complications and secondary malignancies.
Two specific side effects arising from irradiation of the
head and neck region are acute mucositis (painful inflammation and ulceration of
the mucous membranes lining the digestive tract) and acute and chronic
xerostomia. Radiation treatment that is
focused near the salivary glands may inflict temporary or permanent damage.
Xerostomia occurs when the salivary glands do not make enough saliva, producing
“serious negative effects on the patient’s quality of life, causing
difficulties in tasting, chewing, swallowing, and speaking” and can lead to
dental problems including causing mouth infections, sores, cavities, tooth
loss, and gum disease (Lundkvist, et al., 2005).
The side effects of the course of treatment I am provided
affect the efficacy of the treatment.
This is not solely a matter of eliminating my tumor. The preservation of critical bodily functions
also is important.
PBT
carries less than half the risk of secondary malignancy caused by the radiation
treatment.
The decreased exposure to radiation through treatment with
PBT has a substantial effect on the risk of secondary malignancy. A comparative analysis of secondary malignancy
risk in 503 patients treated with proton therapy between 1974 and 2001 versus
1591 matched treated with conventional photon therapy, revealed a significantly
lower risk of second malignancy after proton radiotherapy when compared with
photon radiation therapy (Chung, et al, 2008).
Moreover, after adjustment for clinical and demographic variables,
traditional photon radiation therapy was associated with almost a three-fold
greater risk of second malignancy.
The reduction in secondary cancers has been specifically
documented in the treatment of head and neck cancers (Yoon, et al., 2010).
In the medical literature, compared with IMRT, treatment with PBT for head
and neck tumors carries a lower relative secondary cancer rate for all organs.
It is unethical to disregard the documented
tissue-sparing effect of PBT—resulting from the inherent characteristics of the
proton beam—simply because there is a lack of medical literature
documenting its application to rare malignant carotid body tumors such as
mine.
PBT’s sparing of normal tissue is well documented in the
medical literature, including in the treatment of head and neck cancers. For
rare malignancies like mine, where the limited number of patients does not
enable randomized clinical trials to be conducted, it is unethical to deny
treatment as investigative simply because the highest level of evidence is not
be available. The well-documented tissue-sparing effects of PBT result
directly from the physical characteristics of the proton beam (Metz, 2002). Anthem
and the University of California should not require proof of the impossible:
that the documented tissue-sparing effect applies equally to
malignant carotid body tumors. The tissue-sparing effect
is unambiguous. PBT would spare the tissues surrounding the
tumor in my neck from unnecessary damaging radiation. The
recommendation of the head and neck specialists who evaluated my tumor have
cited this tissue-sparing effect as supporting their determination that PBT is
the optimal treatment for my condition. This recommendation should
not be disregarded. It is unethical for Anthem to authorize only a
treatment that carries a documented significantly greater risk of harm to my
submandibular glands, spinal cord, and brainstem when the less risky treatment
option of PBT could be made available to me.
Anthem
has justified authorizing PBT for other cancers based on these factors. The
same authorization is justified here.
Anthem’s own policy, justifying the use of PBT:
Anthem considers Proton beam radiation therapy “medically
necessary” for the initial monotherapy radiation treatment of
localized prostate cancer. … This is based on evidence that demonstrates that
PBT is able to deliver higher doses of radiation to a targeted area while
decreasing exposure to adjacent healthy tissue. (Policy #: RAD.00015)
Anthem’s policy justifying PBT coverage for prostate cancer
uses the same reasoning that should be used in my case. It appears that the
only reason that prostate cancer would be covered when my cancer is not has to
do with my cancer’s rarity. Indeed, PBT
is also offered for CNS lesions whose pathologies similar to mine: a) close to
delicate, nonhealing and crucially functioning parts of the anatomy and b) not
a whole body metastasis which would make such an expensive and focused
treatment futile.
In sum, the increased radiation-related side effects
resulting from treatment with IMRT are clearly established. These consequences are not insignificant, but
have the potential to affect the quality of my life for the next seventy-plus
years. Because PBT will leave me with
fewer serious side effects than IMRT, PBT is medically necessary for me.
Anthem
and the University Have Caused Dangerous Delay
When first considering proton therapy in late April, I
called Anthem customer service to verify that it would be covered. The
representative told me that it would be, so long as the procedure was
FDA-approved. M.D. Anderson’s proton
therapy facility has been operating with FDA-approval since May 2008 (Matsuda,
et al., 2009). I reached out to friends to set up lodging for me in Houston and
prepared to leave home for the six to eight weeks of treatment. Since then, I
have been waiting for confirmation daily.
Receiving Anthem’s denial was unexpected, and navigating the
appeals process under this time pressure has been extremely confusing and
anxiety producing. My Benefit Booklet specifies that “Only
the Peer Clinical Reviewer may determine that the proposed services are not
medically necessary and appropriate.” He or she must be “licensed in California
with the same license category as the requesting provider.” If my request for
treatment is denied, the denial letter should include the name and telephone
number of the Peer Clinical Reviewer who made the decision. It did not.
The letter includes the name of the medical reviewer who made the determination
not to approve PBT (Steven Rosenblum) as well as the Medical Director (Gil
Solomon), but neither are board certified in radiation oncology or experienced
with diseases of the head and neck.
Dr. Garden appealed Anthem’s denial on May 11. Both he and
Dr. Chen recommended that my therapy start four to eight weeks after surgery,
i.e. no later than May 16. I was not able to obtain documentation of Anthem’s
decision to deny Dr. Garden’s appeal until May 23. Again, the outside doctor is
unnamed. I called Anthem to request his or her name and written opinion, but
was told I would have to wait up to thirty days. If I had to wait thirty more days, I would
receive the relevant information on June 22, more than five weeks after the
8-week maximum post-surgical wait prescribed by the experts who treated me. Anthem and the University of California are
asking me to choose between submitting to more harmful and less effective
treatment without making available their reasoning for doing so, and putting my
life in danger by further delaying treatment.
Whereas the doctors recommending PBT for my treatment are
highly qualified practicing radiation oncologists, those denying my right to
the treatment have unclear qualifications. Furthermore, by restricting access to
information about the outside reviewers, Anthem has violated its own policies.
Meanwhile, each additional day of delay in approving my treatment reduces my
chances of beating this cancer. I urge Anthem and UC officials to immediately
overturn the denial and grant me the care I need to survive.
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