Patient Groups, Industry Seek Changes to Rare Disease Drug Guidance

Regulatory NewsRegulatory News | 04 April 2019 |  By 

Patient groups, drugmakers and other organizations are calling for changes to the US Food and Drug Administration’s (FDA) recently revised draft guidance on developing drugs to treat rare diseases.
The 24-page draft guidance was revised last February after first being published in 2015. The revision included updates to the agency’s approach to natural history studies, a discussion of issues for evaluating biomarkers for consideration as surrogate endpoints and a new section on evaluating safety.
In comments submitted to the public docket for the guidance, the National Organization for Rare Disorders (NORD) says it supports the revised draft guidance and “particularly appreciates the additions and clarifications made to the natural history studies section.”
NORD also says it would like to see FDA revise its disease-specific drug development guidances to align with the draft guidance, especially regarding its recommendations on natural history data registries, flexible clinical trial designs, inclusion criteria and expanded access protocols (EAPs).
However, NORD says it is still concerned that the guidance could lead to “redundant registry efforts and restricted data ownership” and recommends that FDA take firmer steps to encourage data sharing.
Additionally, NORD calls on FDA to promote the use of existing sources of real-world evidence (RWE), such as NORD’s own IAMRARE registry program, in the design and conduct of natural history studies.
In separate comments, contract research organization IQVIA and drugmakers Takeda and Biomarin call for FDA to address the use of real-world data (RWD) and real-world evidence (RWE) as potential external controls for natural history studies.
IQVIA also requests that FDA add a section discussing patient registries, which are not explicitly mentioned in the guidance.
“A registry may be used to recruit patients for clinical trials, monitor patient care and outcomes, advance research hypotheses, observe patient behavior patterns, provide external comparators, establish disease specific standards of care, and support reimbursement discussions. As such, registries often play a vital role in the design of natural history studies,” IQVIA writes.
Biomarin also requests that FDA provide more clarification on how sponsors can modify existing biomarkers and other assessment measures in cases where developing novel measures would be too time consuming as suggested in the guidance.
While supportive of the draft guidance, the non-profit Critical Path Institute (C-Path) says the guidance could be improved by recommending the use of Clinical Data Interchange Standards Consortium (CDISC) standards, common data elements and global identifiers in natural history studies.
“In rare diseases, where many data collections are small, the value of standardization of data collection cannot be understated. In order to combine or compare data from different sources, such standardization is key,” C-Path writes.
Public Docket


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