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Progressive Familial Intrahepatic Cholestasis

Regulatory Approval and Commercialization: Developing Treatments for Rare Diseases

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During a presentation at the 2023 PFIC Family & Scientific Conference, Lara Longpre, Chief Development Officer at Mirum Pharmaceuticals provided an overview of the therapeutic development process from the initial concept through completion of clinical trials.

In a continuation of the development process, Lauren Wilkinson, who works in patient advocacy at Albireo Pharmaceuticals, highlighted the tail end of this process, including FDA filing, review, and commercialization.

To start, pharmaceutical companies organize all of the data from the completed clinical trials and submit all of that evidence along with a proposed product label (or prescribing information) to the FDA with the new drug application (or NDA).

“That is where we’re trying to prove to them that the drug is safe and effective. It outlines the dose, what the benefits of treating symptoms are if there are any associated risks or side effects,” Ms. Wilkinson said. The FDA also checks to make sure the label is proper and clear on what some of the risks may be and that the standards for manufacturing control have been met.

Next, the FDA appoints a team to review all the data and the label, providing written evaluations, detailing their recommendations. Ms. Wilkinson noted that the FDA never actually tests the drug or food product themselves—something that many people don’t realize.

In some cases, the FDA will announce a PDUFA (Prescription Drug User Fee Act) date. “This is basically a fee paid by a drug company to the FDA that helps them establish clear timelines on the review process on their way to approval. There are 2 benefits to this: One, it gives the FDA a clear timeline, and two, it funds some of their efforts to help them meet those timelines,” Ms. Wilkinson explained.

Prior to the announcement of a PDUFA date, drug companies are not allowed to communicate much to the public except for an NDA being submitted. Once the PDUFA date is announced, companies can begin to prepare for an announcement, focusing on parents, caregivers, and advocacy partners, to build momentum around a potential approval.

During this time, the company is usually preparing sales materials and training the sales team, as well as working with insurance and access organizations.

Once the FDA has given approval, the drug is available for patients. “All the preparation that [these companies] were doing while they were waiting, now comes to fruition,” Ms. Wilkinson said. “There’s lots of news releases and outreach to media so that we can get that news to the community, the patients, and the caregivers. As well as working with access and reimbursement organizations to make sure that the drug can be covered.”