FDA Planning Five Guidance Documents to Help Children With Rare Diseases

Posted 10 July 2014 | By

FDA Planning Five Guidance Documents to Help Children With Rare Diseases

The US Food and Drug Administration (FDA) has released a new report indicating four core ways it plans to accelerate the development of treatments for pediatric rare diseases (PRD).

The report, Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases, was required under Section 510 of the Food and Drug Administration Safety and Innovation Act (FDASIA) and based off a three-day public meeting held last year by FDA.

The subsequent report, released on 8 July 2014, established a strategic plan meant to make it easier to develop new products to treat rare diseases affecting pediatric populations.

That plan focuses on four primary objectives:

  1. enhance foundational and translational science for pediatric rare diseases
  2. strengthen communication, collaboration, and partnering for pediatric rare diseases within and outside FDA
  3. advance the use of regulatory science to aid clinical trial design and performance for pediatric rare diseases
  4. enhance FDA’s review process for pediatric rare disease products.

On the last point, in particular, FDA said it's looking to reconsider its existing standards for safety and efficacy in favor of a patient-centered approach.

"We also plan to further develop and implement a structured approach to benefit-risk assessment in the drug review process and establish a patient engagement panel as part of the medical device advisory committee process," Jill Hartzler Warner, associate commissioner for special medical programs at FDA, wrote in an accompanying blog post.

FDA also indicated that four guidance documents are under development for PRDs and are intended to "aid in efficiently executed product development for PRD." Those guidance documents are intended to "contain pediatric-specific information," FDA said.

Also in the works is FDA's Rare Pediatric Disease Priority Review Voucher Program, which distributed its first voucher in February 2014 to BioMarin after its rare disease drug Vimzin was approved. The point of the voucher is to incentivize the development of rare disease drugs for pediatric patients. In return, companies are given a priority review voucher which can either be used for other drugs or sold to another company.

A guidance document specifically on the program will be forthcoming, FDA said.

 

Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases

FDA Voice Blog


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