FDA Releases Report on Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases

By Alexander J. Varond —

FDA recently released its report entitled “Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases Including Strategic Plan: Accelerating the Development of Therapies for Pediatric Rare Diseases.”  The report fulfills the requirements set out in PDUFA V and in Section 510 of FDASIA, which require FDA to hold a public meeting to discuss ways to encourage and accelerate the development of new therapies for pediatric rare diseases (“PRDs”), and to issue a report that includes a strategic plan for encouraging and accelerating such therapies See our previous posts here and here).

FDA’s 86-page report summarizes the difficulties facing sponsors of drugs for PRDs, outlines legislation and approaches to expedite and accelerate rare disease medical product development, provides an overview of the three-day workshop held on January 6-8, and sets forth FDA’s strategic plan.  The first two days of the workshop focused on “complex issues in developing drug and biological products for rare diseases,” while the third day focused on “complex issues in developing medical devices for pediatric patients affected by rare diseases.”

FDA noted that its strategic plan is not a legislative wish list, but is instead an outline of what FDA can reasonably achieve under its existing authority to encourage and accelerate the development of new therapies for PRD.  The report includes a helpful summary of FDA’s strategic plan that we include here:

Objective 1:  Enhance foundational and translation science for PRD

• Drugs/biologics strategies
• Facilitate the conduct of natural history studies for PRD
• Publish draft guidance on common issues in rare disease drug development
• Device strategies
  • Identify unmet PRD needs in medical device development
  • Refine and expand the use of computational modeling
  • Explore the use of registry data for use in both premarket and postmarket evaluation of medical devices intended for pediatric populations

Objective 2:  Strengthen communication, collaboration and partnering for PRD within and outside FDA

• Drugs/biologics and devices strategies
• Continue to foster interagency (public-public) and public-private partnerships
• Continue to foster international collaborations
• Continue to foster Intra-Agency collaborations

Objective 3:  Advance the use of regulatory science to aid clinical trial design and performance for PRD

• Drugs/biologics strategies
• Develop additional FDA guidance relevant to PRD
• Increase engagement of the Study Endpoints and Labeling Development (SEALD) Staff early in instrument development to navigate COA process
• Facilitate increasing the knowledge of biomarkers and COAs useful for PRD, including engaging with investigators and organizations in biomarker and clinical outcome qualification programs to advise during their development
• Develop training programs for pediatric clinical investigators
• Explore modeling and simulation approaches  (e.g., physiologically-based pharmacokinetic [PBPK] models) to provide preliminary data for drugs used in PRD to inform the design and conduct of PK/PD studies and other clinical trials for investigational drugs in PRD population
• Device strategies
• Develop expedited approval pathway for certain devices intended to treat unmet medical needs
• Evaluate the results of an analysis of approved medical devices to explore the feasibility of shifting some premarket data requirements to the postmarket setting for future medical devices
• Support the development of Medical Device Development Tools to improve clinical trial performance
• Develop curriculum for undergraduate/graduate studies to increase understanding of regulatory approval process for device development
• General strategies
• Use FDA web-based resources to update and expand awareness of PRD product development issues
• Increase awareness in pediatric rare disease researchers, product developers, and patient community of funding opportunities through OPD grant program

Objective 4:  Enhance FDA’s review process for PRO products

• Drugs/biologics strategies
• Foster FDA’s efforts to obtain patients’ and caregivers’ perspectives for incorporation in drug development
• Further develop and implement a structured approach to benefit-risk assessment in the drug review process
• Issue Rare Pediatric Disease PRV draft guidance document
• Continue reviewer training for rare and PRD
• Explore potential for innovation in data analysis
• Device strategies
• Further develop methods to implement the incorporation of patient preferences into assessments of premarket approval and de novo classifications of devices
• Establish a patient engagement panel as part of CDRH’s Medical Advisory Committee
• Analyze the HDE process for medical devices that diagnosis and treat PRD
• Set standards for whole genome sequencing that can he used as a comparator
• General strategies
• Continue to enhance FDA’s expertise to review innovative products