FORCE advocates for families facing hereditary breast and ovarian cancer in areas such as access to care, research funding, insurance, and privacy.
Read the written testimony drafted by FORCE and advocacy groups for other hereditary cancer syndromes.
The FDA's Prescription Drug User Fee Act (PDUFA) authorizes the FDA to collect fees from companies that produce certain human drug and biological products. These fees are used to expedite the drug approval process. The law was first enacted in 1992 and renewed most recently in 2012 (PDUFA V).
A key provision of PDUFA-V is to improve drug development and review by taking a more systematic approach to benefit-risk assessment for new drugs; one that considers disease severity and unmet medical needs. The Patient-Focused Drug Development Initiative directs the FDA to choose 20 different disease areas for concentration. During this time, the FDA will conduct quarterly public meetings for each of the chosen diseases. Topics discussed will include:
These meetings will include participation of FDA review divisions, the relevant patient advocacy community, and other interested stakeholders.
FORCE, in partnership with advocacy groups representing other hereditary cancer syndromes, presented oral and written testimony to the FDA for the inclusion of hereditary cancer syndromes in the list of disease states which would receive special consideration. The FDA chose breast cancer but not ovarian cancer or hereditary cancer as one of the disease areas on which it will focus. The full list of diseases to be addressed in FY 2013-2015 can be found on the FDA's Patient Focused Drug Development webpage. A date for the Breast Cancer meeting has yet to be set. A slide-show on Patient-Focused Drug Development can be viewed here.
The FDA will initiate a second public process to determine the list of disease areas for FY 2016-2017, at which time FORCE will petition again for the inclusion of hereditary cancer syndromes.
Although the FDA did not select hereditary cancers as an area of focus for 2013-2015, we will stay involved in the discussions about breast cancer to be certain that the unmet research needs of the HBOC community are addressed. Further, we will continue to urge the FDA to consider hereditary cancers for the next cycle of patient-focused drug development.
We urged the FDA to add hereditary cancer syndromes to the list of disease priorities in the patient-focused drug development initiative. The hereditary cancer community bears a heavy cancer burden. It's time to remedy this disparity while optimizing the promise of personalized medicine. Below is a discussion of reasons why we believe that hereditary cancer syndromes should be included as a disease focus by the FDA:
Hereditary cancer treatment and pharmacoprevention offer unique opportunities for exploiting the
known gene defects and associated genes to develop population-specific treatments and preventions.
However, they also offer distinct challenges including the fact that hereditary cancers are rarer and
consist of a smaller subset of the larger cancer cohort. As such:
Nevertheless, it is important to develop new preventive and therapeutic agents for those with inherited cancer because of their unique challenges:
Although the FDA did not select hereditary cancers as an area of focus, we will stay involved in the discussions about focus on breast cancer to be certain that the specific unmet research needs of the HBOC community are addressed. Further, we will continue to urge the FDA to consider hereditary cancers for the next cycle of patient-focused drug development.