It’s usually FDA that issues guidance documents to its stakeholders. Now, for the first time, a group of stakeholders has reversed that and sent the agency a guidance document.
Parent Project Muscular Dystrophy (PPMD) and allied stakeholders in June gave FDA the first patient advocacy-initiated guidance for a rare disease to help accelerate development and review of potential therapies for Duchenne muscular dystrophy.
PPMD says that more than 80 representatives of the Duchenne community, including parents and patients, medical experts, academics and biopharmaceutical industry representatives, participated in seven working groups that met over the past six months to draft the guidance.
“By working closely with FDA to provide industry and other clinical trial sponsors with clearer guidance from the patient perspective, we will increase the likelihood that clinical trials will be designed to better match the unique needs of Duchenne patients,” said PPMD president Pat Furlong.
The group says that clinical trials for rare diseases like Duchenne are difficult to design and implement due to challenges such as small study populations, incomplete and evolving understanding of rare diseases, and the need for effective ways to measure clinical impact of therapies being studied. The cornerstone of the guidance encourages FDA and trial sponsors to engage patients and their families at all stages of trial development and to take into account what they consider to be acceptable clinical trial risks.
“As FDA evaluates new drug applications for Duchenne therapies, it is imperative to take into consideration the value that parent decision-makers place on even moderate benefits to function and mobility, and their tolerance for considerable risk and uncertainty,” Furlong said. “Parents can make these decisions thoughtfully and FDA must recognize that. They cannot protect us from what we think is important in the drugs we need now.”
The guidance’s sections include published or in-press peer-reviewed articles focusing on six areas aimed at overcoming the challenges in trial design and implementation—benefit/risk assessment, diagnosis, natural history, clinical trial designs, muscle biopsy-based biomarkers and non-muscle biopsy-based biomarkers.