Orphan diseases and pediatric studies FDA

fda-logo1The Food and Drug Administration (FDA) recently released its FDASIA-mandated report and strategic plan on ways to encourage and accelerate the development of therapies for pediatric rare diseases. This plan builds on the FDA public meeting held in January, which CCCA participated in and followed closely.

“FDA believes the strategies in this document will help to accelerate the development of new therapies to treat [pediatric rare diseases] by increasing stakeholders’ awareness of the many FDA programs and initiatives to facilitate the development process.” [FDA Report]

The report summarizes the issues discussed at the January public meeting and the strategic plan outlines what the FDA believes it can reasonably achieve within its existing areas of jurisdiction. Four key objectives were identified in the report and outlined in an accompanying FDA blog post:

  • Enhance foundational and translational science for pediatric rare diseases through strategies that fill information gaps using natural history studies and the identification of unmet pediatric needs in medical device development.
  • Strengthen communication, collaboration, and partnering for pediatric rare diseases within and outside FDA, in recognition of the fact that single entities cannot overcome the rare disease product development challenges on their own.
  • Advance the use of regulatory science to aid clinical trial design and performance for pediatric rare diseases: To further this objective, four separate guidance documents are under development relating to pediatric-specific information.
  • Enhance FDA’s review process for pediatric rare disease products: This objective includes addressing the complexities and challenges surrounding benefit-risk assessments, which are important issues to the pediatric rare disease community. FDA has been developing a structured approach to benefit-risk assessments and exploring ways to evaluate and communicate benefit-risk uncertainty.

In the industry publication FDA Week, CCCA’s founder Susan Weiner, PhD, commended the fairness of the report and its strategies but urged the agency to go further and become more innovative. Dr. Weiner stressed the importance of advisory panels of families and advocacy group representatives for disease-specific interests. Dr. Weiner also noted that the report doesn’t address the upcoming guidance on laboratory-developed tests, a key component of pediatric cancer clinical research.

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Popular Q&A

What does the FDA do? a topic, disease, or research study that the FDA is involved with and describe it?

what does the FDA do? a topic, disease, or research study that the FDA is involved with and describe it’s involvement?

The FDA takes possible cures to diseases and slows down the process of making it legal to buy and use to fix your ailments. They do this because of all the donated money to help finding a cure (for example cancer, or aids) If someone were to find a cure for a disease then they would stop getting the donation money. Theres more money to be made in finding cures rather then creating a cure. FACT

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