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Public Policy Issue Brief
Appropriations for Medical Research and Drug Approval:
Ensuring Support for Brain Tumor Research and Drug Development
Background
• There have been very few new brain tumor therapies approved in the last 30 years, BUT
there is hope.
• Critical research at the National Institutes of Health (NIH) is laying the groundwork for
breakthroughs. The NIH is the largest funder of brain tumor research in the United States.
• NIH’s work impacts the brain tumor community through many institutes including: the
National Cancer Institute (NCI), National Institute of Neurological Disorders and Stroke
(NINDS), National Human Genome Research Institute (NHGRI), and National Center for
Advancing Translational Sciences (NCATS).
• NCI’s clinical trials cooperative groups are conducting state-of-the-art research on adult
glioblastoma therapy.
• NCI’s Pediatric Preclinical Testing Platform (PPTP) is an essential component for evaluating
the safety and efficacy of new drugs before testing directly in children.
• NCI’s Neuro-oncology branch is a critical place for patients throughout the United States to
seek cutting edge treatment advice.
• The U.S. Food and Drug Administration (FDA) is more committed than ever to jumpstarting
brain tumor drug development by improving clinical trials.
The Problem and Opportunity
• Brain tumors are one of the deadliest forms of cancer, are also a neurologic disease, and
have among the fewest therapeutic options – thus research funding is absolutely critical to
finding new effective treatments.
• Private philanthropy cannot fill in for the key leadership role that NIH plays in funding,
coordinating, and advancing research, and no amount of private funding can replace the
FDA’s regulatory role in evaluating and approving new treatments.
• The era of precision medicine is here. The opportunity to leverage previous Congressional
investment in the NIH’s genomic research should be seized through new research.
Become an Advocate
Congress should renew its historic, bipartisan commitment to the fight against cancer,
including brain tumors, by increasing appropriations for NIH in FY 2016 to $33 billion and
a commensurate increase for NCI to $5.44 billion. We also urge Congress to provide an
appropriation of $2.8 billion for the FDA.
www.braintumor.org
4/2015
Public Policy Issue Brief
How Congress Can Help Research on Childhood Brain Tumors:
The Leading Cause of Cancer Death in Children Under 14
We Must Do More To Fight Childhood Brain Tumors
• Pediatric brain tumors are the leading cause of cancer death in children under 14.
• In 2015, an estimated 3,420 new cases of pediatric brain tumors will be diagnosed.
• While some pediatric brain tumor types have long survival rates, for gliomas – the most
prevalent pediatric brain tumor type – there has not been progress to extend survival rates
in decades.
• Children with high-grade gliomas only have a 28% chance of living five years past diagnosis.
• Even if a child with a pediatric brain tumor survives their disease, current standard treatment
approaches can leave a child with developmental delays and a lifetime of neurological problems.
• Industry is not a significant source of funding for pediatric brain tumor research.
Past Congresses Advanced Research Through DOD CDMRP
Thanks to funding from the Peer Review Cancer Research Program, a program of the Department of
Defense’s Congressionally Directed Medical Research Programs, pediatric brain tumor research is
experiencing advances, which are benefiting military families and all Americans. Grants have:
• Enabled development of mouse models to identify drug targets in Diffuse Intrinsic Pontine
Glioma (Duke and Baylor University studies).
• Enabled studies to identify potential approved drugs for adults, which may be capable of use in
pediatric brain tumor clinical trials (St. Jude Children’s Research Hospital).
• Identified possible drivers of medulloblastoma metastasis – which are the more frequent cause
of death in patients that were first diagnosed with a primary medullablastoma – that represent
novel therapeutic targets for future studies to capitalize on (Emory University).
The 114th Congress Can Help
Congress should include pediatric brain tumors among the eligible topics in the Peer Review
Cancer Research Program of CDMRP for FY 2016.
Pediatric brain tumors have been one of the eligible topics for research funding under the Peer
Review Cancer Research Program since it’s beginning in 2009. Inclusion of pediatric brain tumors
as a topic in FY 2016 is a revenue neutral issue – the words “pediatric brain tumors” just need to
be added to the appropriations report language for the Program.
Research is Critical to Discovering Treatments that Extend Survival and Protect a Child’s
Normal Development.
For more information contact: David F. Arons, National Brain Tumor Society, darons@braintumor.org.
www.braintumor.org
4/2015
Public Policy Issue Brief
Oral Chemotherapy Parity:
Ensuring Affordability of Oral Chemotherapy for Brain Tumor Patients
Background
There are very few therapies available for brain tumors patients. One type of chemotherapy, temozolomide
(Temodar), is a widely used treatment, and often the standard of care, for many patients with malignant
brain tumors. Temozolomide is almost exclusively prescribed orally.
The Problem
•M
any private health insurance plans cover temozolomide as a pharmacy benefit and not as a medical
benefit, as traditional IV chemotherapy is covered. The result can be high co-pays or co-insurance in the
hundreds or even thousands of dollars per month.
•A
n increasing number of brain tumor patients are reporting that they cannot afford the out-of-pocket
costs per month to access temozolomide, yet they must access this medicine as part of their oncologistprescribed brain tumor treatment.
•F
or many brain tumor patients there is not an IV chemotherapy substitute. Thus, health insurance costsharing can create real economic hardships and present a barrier to the affordability of a medically
necessary chemotherapy regimen.
•A
dditionally, research has found that more than 25% of all anticancer agents currently in development
are planned as oral drugs. Many of these new oral drugs have shown significant clinical advantages
over traditional IV/injected forms of cancer treatment in early trials. As new treatments come into the
marketplace, the affordability of oral and other patient administered anticancer medications will become an
even larger problem, both for patients and the doctors that must prescribe them as part of standard care.
The Solution
Congress should support oral chemotherapy parity legislation.
The Cancer Drug Coverage Parity Act, supported by Congressmen Higgins and a bi-partisan group of
House co-sponsors, and The Cancer Treatment Parity Act, supported by Senators Kirk and Franken, are
both expected to be reintroduced in the Spring of 2015, and would require health plans to cover oral
chemotherapy on an equal basis as chemotherapy given through hospital administered IV or injection.
Because it will only apply to health plans that already cover chemotherapy, this is not a mandate.
Not only is access to oral chemotherapy critical to delivering the standard of care, it can be beneficial to the
patient’s quality of life because he or she can undergo treatment at home instead of traveling to a hospital
Health insurance should facilitate brain tumor treatment, and not create a financial barrier to it.
About National Brain Tumor Society
National Brain Tumor Society is the largest nonprofit organization dedicated to the brain tumor community
in the United States. We are fiercely committed to finding better treatments, and ultimately a cure, for people
living with a brain tumor today and those who will be diagnosed tomorrow. This means aggressively driving
strategic research and advocating for public policies, which meet the critical needs of this community. It’s time
to build on progress and transform tomorrow, today. To learn more visit www.braintumor.org.
www.braintumor.org
4/2015
Battling Brain Tumors
Help Americans – And Your Constituents:
3 Ways Congress Can Help the Brain
Tumor Community in 2015
A Cause for All of Us
Indeed, the fight against brain tumors should be everyone’s cause.
Anyone can get a brain tumor including persons of any age, race, gender
or ethnic origin. There is currently no standard prevention or early
detection measures, and very few effective treatments and no cure exist.
Adults and Children Impacted
Tragically, malignant brain tumors are among the deadliest cancers
with just a 34% five-year relative survival rate. And brain tumors are
now the leading cause of cancer death in children under the age of 14.
Even those with a benign brain tumor face a potentially life-threatening
disease and may suffer from chronic neurological problems.
Elizabeth and Lauren
are among the
many we honor and
remember for their
battle, and Owen is
one of the people
currently living with
a brain tumor that we
fight for everyday.
The Need to Leverage “Precision Medicine”
Should you be discouraged? No! We hope you’ll be inspired and
determined to help our cause. Thanks to previous investment by
Congress the era of precision medicine is here, now it is time to
leverage it.
National Brain Tumor Society “asks” Congress to fight brain tumors by:
1.Support an increase in FY 2016 Appropriations for the National
Institutes of Health to $33 billion and the National Cancer Institute
to $5.4 billion.
2.Support continued inclusion of pediatric brain tumors as an eligible
topic for research in the Peer Review Cancer Research Program
within the Defense Department’s Congressionally Directed Medical
Research Programs.
3.Support legislation to help make anti-cancer medications more
affordable such as the Cancer Treatment Parity Act (Sens. Kirk/
Franken) and the Cancer Drug Coverage Parity Act (Rep. Higgins)
expected to be introduced shortly.
For more information contact: David F. Arons, National Brain Tumor
Society, darons@braintumor.org.
www.braintumor.org
4/2015