FDA releases industry guidelines for epidermolysis bullosa therapies
The FDA has released new guidelines aiming to assist industry production of drug therapies for the prevention and treatment of epidermolysis bullosa (EB) cutaneous manifestations.
The term epidermolysis bullosa (EB) refers to a varied collection — ranging from mild in severity to fatal — of inherited skin conditions that result in mechanical skin fragility and blistering. This can cause recurrent wounding and resultant infections. Currently, therapies to manage and/or treat EB represent an urgent, unmet clinical need. The recent release of new industry guidelines by the FDA — focusing on EB drug product development, clinical trial design and endpoints — signifies a step towards achieving successful EB treatment development.
In the guidelines document, the FDA state: “The purpose of this guidance is to assist sponsors with the development of drugs for treatment or prevention of the serious cutaneous manifestations of the heterogeneous group of disorders collectively known as epidermolysis bullosa (EB). The paucity of effective treatment options for EB represents an important unmet medical need.”
Further in the document, the FDA note: “This guidance focuses on drug development and trial design issues specific to the treatment of EB, including FDA’s current thinking on trial endpoints. There is not yet sufficient clinical trial experience to establish definitive endpoints.”
The newly released guidelines build on previous FDA guidance concerning the development of targeted cell and gene therapies and wound healing products, set out over the course of 2006–2015.
Learn more about cell therapy for epidermolysis bullosa:
The guidelines cover three categories of considerations that impact on clinical trial design:
- Factors relating to the trial population
Recommendations in this section include guidance on defining patient cohorts for clinical trials such that patients recruited for the testing of products aimed at either palliative disease treatment or disease modification are only done so if, for example, they represent the phenotypic spectrum of disease interest under investigation. Patients should not have to undergo certain genetic testing if this is not relevant to the claims of the therapy being investigated.
- Endpoints from which to assess efficacy of trialed therapies
This guidance pertains to practical study design; recommendations concerning the random assignment of participants to treatment-receiving groups, blinding and appropriate control group measures are made. Further, it is advised that subjective endpoints and the timescales with which to assess these are discussed prior to trial design. Additionally, the importance of assessing the validity of methods used to evaluate these endpoints is highlighted, such as the current lack of adequate patient-reported outcomes from which endpoints can be evaluated.
- Additional special considerations
The advice in this section concerns measures that can be taken to maximize patient comfort during trials, such as electronic-informed consent, measures that reduce the number of patients’ visits to trial sites amongst others.