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June 2007
MEMORANDUM IN SUPPORT OF RARE DISEASE TREATMENT BILL A8882
New Yorkers for Accessible Health Coverage (NYFAHC) strongly supports A. 8882,
the Rare Disease Treatment Bill. The bill would amend New York's External Review
Law (Insurance Law Article 49, Public Health Law Article 49) to provide more
realistic access to promising treatments for people with life threatening or seriously
disabling rare diseases. This proposed legislation also has the support of, among
others, the American Cancer Society and the Center for Medical Consumers.
While the existing external review law works extremely well to expand access to
care for most seriously ill patients by providing coverage for medically appropriate
clinical trials, it has become apparent that the law fails to provide access to
medically appropriate treatment for a small sub-class of seriously ill people it was
surely designed to benefit – persons with very rare conditions. Some conditions are
so rare that it is not economically feasible to test innovative or investigational
treatments for them through the clinical trial system. Terminally ill people with
such conditions are highly unlikely to find clinical trials specific to their disease.
Consequently, the External Review Law is unlikely to provide those with rare
diseases access to innovative treatment because such treatment – even if deemed
medically appropriate by medical experts in the particular disease – is frequently
administered outside of clinical trials, and because peer reviewed medical literature
is unlikely to directly establish that it is more likely to benefit the person than
treatments currently accepted as “standard” for their particular condition. For
these people, a more flexible standard is required.
The Rare Disease Treatment bill would expand the External Review Law to give
people with rare diseases the same opportunity to obtain promising life saving
treatment as is already provided to their neighbors with more common diseases. A
rare disease patient could establish the “likely benefit” of an innovative treatment
by relying on evidence accumulated in the context of diseases of higher incidence
but with comparable features. The patient would still have to satisfy the Law’s
additional requirements: (i) certification by a board certified treating physician that
the treatment is likely to benefit the patient, and (ii) endorsement of that treatment
choice by board certified peer reviewers appointed under the External Review Law.
In such circumstances, the effect will be the same as the Law’s mandate of
coverage for clinical trial treatments for more common conditions like breast cancer
or colon cancer: to provide access to investigational treatments for which there is a
medical consensus, supported by reliable evidence – even if that evidence is not
yet conclusive or (in the case of rare diseases) is derived from analogous conditions
– that the treatment is likely to benefit the insured’s condition.
The urgent need for this statutory improvement was recently demonstrated in the
case of Suzanne Mauer, a New Yorker facing an extremely rare and deadly form of
cancer who has been denied access to a treatment likely to benefit her because of
her purported inability to satisfy the current External Review Law's requirements.
As described in Ms. Mauer’s statement, she has been diagnosed with metastatic
NYFAHC is an independent project of the Center for Independence of the Disabled in NY (CIDNY)
hemangiopericytoma (“HPC”), a disease which simply does not respond to standard
chemotherapy. Her in-network and out of network doctors, both highly respected
oncologists at Beth Israel Medical Center and Memorial Sloan Kettering Cancer
Center, recommended that she receive a drug regimen including a drug called
Avastin, which was administered to 6 patients with her condition at the University
of Texas MD Anderson Cancer Center in Houston, Texas, and to which all 6
evidenced good response, a remarkable response rate in HPC. Based on certain
disease characteristics which make HPC comparable in its disease process to colon
cancer, for which Avastin is approved, the doctors at MD Anderson correctly
anticipated that the drug could be useful, and Ms. Mauer’s New York doctors
concurred.
Ms. Mauer’s health plan denied coverage as experimental. She appealed and lost.
She then sought external review, and three peer reviewers rendered opinions. Two
of the three reviewers found the treatment to be reasonable or the best option, but
the plan’s determination was not overturned because the reviewer who found the
treatment choice "not unreasonable" nonetheless could not say it met the current
statutory standard, based on published medical literature, of being “more likely” to
benefit her than standard treatment. Without access to the treatment, her disease
has been progressing. Her own health plan ultimately, on June 6, determined to
provide coverage for the treatment and to adjust its internal guidelines regarding
rare disease, but numerous other health plans would continue to deny coverage to
individuals in similar circumstances.
On the following pages, Ms. Mauer tells her story in her own words. She
recognizes that even a new statutory standard would not guarantee access to the
new treatment, just as there is no guarantee that the treatment will definitely
benefit her. But she believes that she, and the few other New Yorkers who face
similarly rare diseases, should have coverage – equivalent to New Yorkers with
more common diseases – for reputable, promising and potentially life-saving
treatments.
_____________________________________________________________
Suzanne Mauer's Story
May 2007
I am 50 years old and live with my husband in Brooklyn, NY. Until February, 2007,
when my illness necessitated going on long-term disability, I was a Senior
Relationship Manager overseeing accounts for national and multi-national
corporations, having raised over $10 million dollars of investment capital for
internet start-up companies. My professional life also includes having been a
Corporate Banking Officer for a US, top-15 bank. While pursuing my career, I have
also continued my education and will be ordained as an interfaith minister in June
2007.
In 1999, I was diagnosed with a large brain tumor and received a successful
craniotomy. The pathology was Hemangiopericytoma (HPC). HPC is considered a
type of soft tissue sarcoma (soft tissue sarcomas, as a class, constitute less than
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2% of all cancer diagnoses). Only about 250 people each year in the U.S. are
diagnosed with HPC. For most of them, the tumor is not malignant and is treated
successfully with surgery. In my case, my tumor pathology was reported as a 4 on
a scale in which 0 is benign and 10 is malignant, yet my neurosurgeon told me that
I did not have cancer. (Had they recognized the cancer and referred me to an
oncologist for management, I might not have the disease I have today.)
In January 2004, however, I was diagnosed with a tumor on my thoracic spine and
underwent a vertebrectomy and radiation. In May 2004, in addition to my network
specialist, Dr. Ronald Blum at Beth Israel, I began seeing sarcoma specialist Dr.
Robert Maki at Sloan Kettering. I also began a monthly non-toxic treatment of
Zometa, which helps to prevent cancers from growing.
Unfortunately, other tumors were found in numerous locations throughout my
body. It has since been determined that I have metastatic HPC. Metastatic cancer
is diagnosed in less than 50 cases a year. In January, 2007, my doctors found a
new regimen through a colleague at MD Anderson in Texas where all 6 metastatic
HPC patients treated with a 2 drug protocol (Temodar/Avastin) have shown
improvement or stabilization of the progression of the disease. This is remarkably
better than the partial response reported for only one of seven HPC patients
receiving standard chemotherapy at Memorial Sloan Kettering. The treatment at
MD Anderson is not, however, in a clinical trial, and my doctors and I could find no
appropriate clinical trial for HPC treatment for which I could qualify.
Oxford, my insurer, would not approve Avastin since it contended other drug
therapies were available. My doctors told me that other drug therapies show no
benefit to HPC patients and have very high morbidity rates. I lost an internal
appeal and an external review, even though two of the three reviewers found the
treatment choice to be at least reasonable. As a result, I have not been receiving
any treatment since I am unable to personally pay for the high cost of Avastin
($6,000 every two weeks). My efforts to obtain the drug at no cost from Avastin’s
manufacturer, Genentech, have not yet borne fruit.
On a personal level, the effects of having this disease have been extremely taxing
for me and my family. Before I was affected by this disease I socialized quite a bit
and was very involved with many, many activities professionally, personally, and
physically. The first symptoms of the brain tumor made me easily stressed and
short-tempered, causing my husband and me to separate for 10 months preceding
the craniotomy. In spite of utilizing numerous stress reduction techniques, I found
my job as a Corporate Banking and Trust Officer became too stressful in my last 2
years there and I left my job to avoid making an error that could have ruined my
career and professional reputation. My family relationships and friendships were
also quite strained and I reduced them in number and became increasing lonely
and alienated.
My neurologist let me know that I would fully recover by participating as fully in life
as possible. But after the craniotomy, I required a much less intellectually
demanding job and had to relearn other key social skills. For instance, I had
“forgotten” the importance of supporting my superior’s position when it was
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different from my own; and therefore lost a job that I had in the post-production
business.
While I thankfully no longer feel impeded mentally, physically my condition has
steadily deteriorated, especially over the last 2 years. My life has become
considerably smaller. I require at least 10-14 hours of sleep nightly and am still
frequently exhausted. I have to deal with constant pain at 11 different sites in my
body. Recently, my doctors discovered that a tumor had broken my right rib twice
and now a tumor on my left rib is doing the same. The pain often wakes me up
screaming in the night. I can only walk approximately 3 short city blocks daily in
total or the pain becomes excruciating even with pain killers and I will be laid up for
at least 2 days. The amount of pain killers I need daily has doubled from April 1 to
May 10. A tumor on my liver is causing me to perspire excessively 8 days out of 10
regardless of temperature. I often wake up chilled in a wet bed. All social plans
need to be given the proviso “if I am up to it.”
Fighting for the chance to get a drug that could improve my condition has been
almost impossible for me given my constant pain and exhaustion. I have relied
heavily on my husband and on the efforts of a lawyer who is now donating his
services. I am willing to jump hurdles to arrest the progress of my disease. But I
cannot jump over hurdles that are impossibly high. I hope that the Legislature and
Governor will see the wisdom of giving even people with the very rarest diseases,
those for whom it’s not economically worthwhile to create clinical trials, a chance at
the most promising treatments reasonably recommended by their specialists.
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