The introduction to an FDA draft guidance for industry published in June 2015, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment,” makes an unusual statement:
Development of this guidance was preceded by the submission to FDA of a proposed draft guidance independently prepared by a consortium of stakeholders including patients, parents and caregivers, clinicians, scientific experts, and industry representatives. The proposed draft guidance submitted by the consortium was made available through a Federal Register notice seeking public comment. Both the independently prepared proposed draft guidance and the public comments received in response to the Federal Register notice were considered in writing this guidance.
Stakeholder-authored draft guidances are a promising way to accelerate availability of clear FDA guidance for industry in indications that lack such guidance. It is beyond the capabilities of any regulatory agency to issue development guidances for every indication identified in ICD-10. Nevertheless, the absence of such guidances inevitably increases the burden that clinical development places on both drug developers and regulators. More importantly, lack of specific guidances delays the availability of new therapeutics for patients with unmet medical needs in serious and life-threatening diseases.
An Invitation to Help Fill Gaps in FDA Guidance
We commend FDA for agreeing in December 2013 to allow Parent Project Muscular Dystrophy (PPMD) and other interested parties in the DMD community to submit a draft for FDA’s consideration. The stakeholders deserve high praise for submitting their draft to FDA six months later, in June 2014. The FDA’s own draft guidance appeared one year later.
With the example of the DMD community to show the way, identification of a missing volume in the FDA guidance library should be an invitation to generate a document that helps FDA fill the gap. Stakeholder initiative and incorporation of earlier public review cycles should at a minimum shorten the review cycle following publication of FDA’s own draft guidance. Furthermore, the participation of advocacy organizations in the authorship of a guidance document is an important step toward patient-centric development. While there is a degree of collaboration now, perhaps the next step in accelerating development of FDA guidance documents will be a more collaborative and parallel process that further reduces time required to review FDA’s draft guidances and issue final versions.
Uncertainty about the path forward compounds already great risks in clinical development. Here’s to stakeholder authorship of more pre-FDA draft guidances to assist FDA in articulating a clear path forward wherever possible as soon as possible.