Marcia Boyle, President and Founder of IDF, was one of three people representing patient organizations to speak at a 2-day Food and Drug Administration (FDA) public hearing to receive input regarding how the FDA should implement a recent law establishing a shortened process for certain biologic products to get to the marketplace. Basically, the discussion centered on, “What is the best way to introduce generic biologics into the marketplace.”
Of course, a shortened approval pathway for biologics raised red flags for IDF. IDF is committed to act and advocate on behalf of the PIDD community.
You probably know that in traditional pharmaceutical chemical based manufacturing there must be years of research, development and testing for safety before reaching the marketplace - an expensive process. Generic manufacturers take the chemical formula and manufacture an “equivalent” drug without the expense of the research. Hence generic drugs are less expensive than brand drugs.
Until now, biologics (drugs that are derived from animal products or other biological sources) have had to follow strict research, development and clinical testing guidelines before receiving FDA approval. However, Congress recently approved and authorized the FDA to develop a shortened approval pathway for the generic equivalent to biologics – “biosimilars.” This is vital to the PIDD community because immunoglobulin is a biologic. Link to Congressional Research Services background report on Biosimilars
Marcia’s testimony (link to read testimony) expressed various concerns to the FDA panel about the potential safety hazards to patients with primary immunodeficiency diseases (PIDD) of creating shortcuts to the manufacturing of products like immunoglobulin. She urged the FDA to follow the lead of the European Medicines Agency (EMA) and exclude immunoglobulins from the biosimilars pathway altogether.
She urged the panel to require human clinical studies (not just animal studies) of all biologics to demonstrate that a generic biologic (biosimilar) is safe. For patients with PIDD, the notion of being forced to be infused with a drug that has never been tested on a large cohort of people is toxic. She stressed that even with clinical trials there needs to be a strict surveillance system in case there are problems down the road.
As a case in point, Marcia noted that even after the FDA approved a manufacturing change, a major immunoglobulin manufacturer, who knows the business well, had to voluntarily withdraw its immunoglobulin product worldwide because of a significant increase in thromboembolic events – just because of a manufacturing change which produced structural differences in the immunoglobulin product. It is hard to imagine what would happen with a generic immunoglobulin manufacturing company with no experience in the plasma product world and no clinical studies to test whether the product is safe for patients with PIDD.
This hearing was the first step in the FDA process of defining rules and regulations regarding a pathway for biosimilars into the marketplace. Rest assured that IDF will be very active in the process to assure that patients with PIDD are protected.