FDA Advisory Committee Preparation: Stakeholder Engagement
A specialty biopharmaceutical company was preparing for an advisory committee meeting and FDA approval of their self-administered therapy for a rare and potentially life-threatening genetic condition that had no self-administered treatment available at time of review. In addition to the majority of Advisory Committee members having no direct experience treating the condition, the company’s data supporting self-administration was limited.
To demonstrate the need for this self-administered therapy, 3D partnered with the leading patient organization in the category, as well as thought leaders in the U.S. and internationally, where the product was currently available. We coordinated a robust open public hearing and a letter writing campaign to educate advisory committee members on: the clinical and geographic limitations of current therapies; the day-to-day anxiety of patients who feared not reaching a hospital in time for treatment; and the ability of patients to recognize the symptoms of an oncoming attack and self-administer treatment.
The FDA advisory committee voted 12-1 to support approval of the product. They then went a step further and asked the FDA to add a special question on self-administration. When the agency agreed, the advisory committee members voted 11-1 recommending self-administration of the drug by patients. The FDA approved the product as the first self-administered subcutaneous treatment for this condition.