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FDA's Patient-Focused Drug Development Program

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Read the written testimony drafted by FORCE and advocacy groups for other hereditary cancer syndromes.

Background on PDUFA and Patient-Focused Drug Development Program

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:

  • the impact of the disease on patients
  • the spectrum of severity for those who have the disease
  • the measures of benefit that matter most to patients, and
  • the adequacy of the existing treatment options for patients

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.

FORCE's recommendations

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:

  • There are fewer financial incentives for pharmaceutical companies to target these cancers vs. the larger disease population.
  • Patients with hereditary cancer are motivated to participate in clinical trials and registries.
  • However, they often enroll in studies that focus on sporadic cancers because of the number and
    availability of these larger, less targeted studies. This leads to a vicious cycle where fewer
    pharmaceutical companies are willing to develop drugs due to recruitment challenges.
  • Tests to identify and define members of hereditary cancer populations create their own challenges to
    therapeutic development because they are often expensive, lack FDA approval or simply aren't utilized.

Nevertheless, it is important to develop new preventive and therapeutic agents for those with inherited cancer because of their unique challenges:

  • The lifetime risk for familial cancer is significantly higher, and cancers often strike younger and
    are more aggressive than sporadic cancers. This may lead to a different risk-benefit ratio for
    prevention and treatment than for sporadic cancers.
  • The genes and pathways associated with these cancers are frequently known, lending the opportunity to target vulnerabilities that may not exist for sporadic cancer.
  • Currently, risk-management for hereditary cancer syndromes focuses on cancer screening and surgical interventions. Many inherited cancers, including ovarian and pancreatic cancer, have no good early detection and a high mortality rate. Development of drugs explicitly to prevent cancers in individuals with hereditary cancer syndromes is achievable and could save thousands of lives and dollars per year.
  • Hereditary gene mutations are sometimes associated with different risks and benefits for therapeutic agents compared with published standard-of-care for sporadic cancers. Much more needs to be learned about 'standard therapy? in these specific populations and how to develop protocols that don't expose patients to increased harm. For example:
    • BRCA mutation carriers may be more sensitive to the heart damaging effects of Adriamycin, a standard breast cancer chemotherapy agent;
    • 5-FU, a mainstay of standard chemotherapy for colon cancer, works only for some patients with Lynch Syndrome;
    • People with Li Fraumeni may be more sensitive to radiation therapy; and
    • Individuals with Ataxia-Telangiectasia cannot receive standard radiation or chemotherapy because they are extremely sensitive to the cytotoxic effects associated with these therapies.

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.

Page updated 04/01/14

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