National Brain Tumor Society Public Policy and Advocacy Update

Vol. 5, No. 4

April 2015

Head to the Hill is Here! (Follows Race for Hope,
Increasing Funding for Brain Tumor Research
Oral Chemotherapy Parity Update
Advancing Development of New Treatments
Advocacy Stories of Impact

Head to the Hill is Here! May 4 & 5 – Washington DC
In just a couple of days, more than 200 advocates from 38 states will use their voice to advocate for public policy issues critical to the brain tumor community. Head to the Hill, our annual Washington, DC–based advocacy event, is set to have record attendance in its fourth year.

Can’t attend Head to the Hill in person? No problem! You can still be a part of Head to the Hill by calling the offices of your federal representatives. Please be on the lookout for an email on the morning of May 5 that will provide you the link to the Head to the Hill call-in page.

We are grateful to everyone who will participate in this year’s advocacy day in person or on the phone.

Finally, its not too late to also attend Race for Hope DC! Join over 11,000 brain tumor community members as they walk and run to raise resources and awareness to find a cure.

Increasing Funding for Brain Tumor Research
Congressional hearings for FY 2016 Appropriations are on-going. National Brain Tumor Society, as part of the One Voice Against Cancer Coalition, is asking Congress to increase funding for  the National Institutes of Health (NIH) by 9% to $33 billion and to include a proportionate increase for the National Cancer Institute (NCI) to $5.4 billion. 

In addition, we are asking Congress to continue to include pediatric brain tumors among the eligible topics for funding in the Peer Review Cancer Research Program of the Department of Defense’s Congressionally Directed Medical Research Programs. Given that there is almost no industry funding for pediatric brain tumors, this is a critical area in need of federal funding. Many of you have written letters and made phone calls to your Members of Congress on this funding issue – Thank you!

Oral Chemotherapy Parity Update
We are working with Senators Franken (MN) and Kirk’s (IL) offices as well as the office of Representative Brian Higgins (NY) on the strategy for re-introduction of federal legislation that would make sure that brain tumor patients prescribed oral chemotherapy such as temozolomide (brand name: Temodar) do not have to pay more out of pocket in co-pays than they would if they were able to take it intravenously.  Following bill introduction in a matter of weeks, we will be asking advocates to encourage their Members of Congress to co-sponsor the bills.

Advancing Development of New Treatments
There has been an extraordinarily high number of new pieces of legislation produced in this Congress related to the development of new treatments. In particular, the Energy and Commerce Committee led by Representatives Fred Upton
(MI) and Diana DeGette (CO) has developed draft legislation that would provide greater authority to the FDA to advance review and approval of drugs and devices and would call for new funding for precision medicine research initiatives. National Brain Tumor Society has weighed in with the Committee and is calling for the establishment of standards for the qualification of surrogate endpoints (clinical trial measures) as a high priority in order to bring consistency and clarity to industry-led clinical trials. We will keep you informed and engaged so we can work as a team.

Advocacy Stories of Impact
The public policy process can be slow, it can be long, and it can be frustrating. It can take years to see ultimate success. Thus we must celebrate and take note of the process victories along the way and keep fighting together. Below please find short stories that remind us of why advocacy is so important and impactful:

** Breakthrough Therapies
In 2012, the National Brain Tumor Society joined with other groups in the rare disease community to support legislation called Breakthrough Therapies. Breakthrough Therapy Designation gives the FDA authority to create an ‘all hands on deck’ review of drugs that show potential benefit at an earlier stage in the clinical trials process. An investigational drug may get breakthrough therapy designation if the preliminary clinical evidence demonstrates the drug may have substantial improvement on at least one clinically significant endpoint (such as survival) over available therapy. Drugs receiving breakthrough therapy designation are in a greater position to be evaluated more quickly and intensively by the FDA. 

Thanks to the efforts of thousands of the National Brain Tumor Society's volunteer advocates across the country, and the many more with other organizations, the legislation passed Congress and was signed by the President in 2012.  As a result, we are now pleased to report that the first breakthrough therapy designation for a brain tumor drug seeking FDA approval for both newly diagnosed and recurrent glioblastoma patients was made in February of this year. Celldex Therapeutics’ Rindopepimut is an investigational immunotherapeutic vaccine that targets EGFRvIII (epidermal growth factor variant 3) - a molecule that is expressed by the tumor cells of an estimated 30% of glioblastoma patients - received breakthrough therapy status. Their clinical trials are currently in phase II and phase III studies, respectively. While we don’t know what the ultimate outcome of the clinical trials will be, or if an approval will be made, we do know that the advocacy of National Brain Tumor Society volunteers helped create a more favorable and expeditious process for brain tumor drugs to be approved if they are found to be safe and effective.

** Creating Hope Act
In 2012, the National Brain Tumor Society supported the enactment of legislation spearheaded by Kids v Cancer, St. Baldrick’s, and others called the Creating Hope Act.  The Creating Hope Act bill has since become a law, which created a new economic incentive for pediatric rare disease drug development through the use of a voucher for priority review by the FDA. Companies that have an eligible new drug and receive a voucher obtain faster FDA review. In March, the FDA announced approval of the first pediatric cancer drug (for neuroblastoma) that had received a priority review voucher. The drug is called Unituxin and the sponsor is United Therapeutics. Our hope is that the same voucher program will someday be used for a pediatric brain tumor.

Both of these stories illustrate the power of your advocacy to identify problems, propose solutions, and advance research and treatment.

Thanks again for all your support and advocacy. Together we are making a difference!

David F. Arons, JD

Chief Public Policy
and Advocacy Officer

Lainey Titus Samant

Associate Director

Tyler Gately

Manager of Advocacy
and Volunteer Leadership

National Brain Tumor Society is 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 effecting change in the system at all levels. It's time to build on progress and transform tomorrow today.

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