FDA gets treatment guidance from patient advocates

Share this article:
PPMD President Pat Furlong
PPMD President Pat Furlong

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.

Share this article:
You must be a registered member of MMM to post a comment.

Email Newsletters

MM&M EBOOK: PATIENT ACCESS

Patient access to pharmaceuticals is a tale of two worlds—affordability has improved for the majority, while the minority is hampered by cost, distribution and red tape. To provide marketers with a well-rounded perspective, MM&M presents this e-book chock full of key insights. Click here to access it.

More in Channel

Five things for pharma marketers to know: Monday, September 15

Five things for pharma marketers to know: ...

Pharma has sought 76 meetings with FDA over biosimilars; Gilead licenses Sovaldi to India generic drugmakers; Pfizer and Ranbaxy Lipitor lawsuit dismissed.

Liraglutide, aiming for new indication, gets new name

Liraglutide, aiming for new indication, gets new name

Why Novo Nordisk is choosing not to leverage Victoza's brand equity as it seeks a weight-loss indication for liraglutide.

Five things for pharma marketers to know: Friday, September 12

Five things for pharma marketers to know: Friday, ...

An FDA panel voted in favor of liraglutide for weight loss; Allergan investors backing an attempted takeover of the firm crossed a critical threshold; and 100 million health wearables are ...