In a letter submitted today to the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD) urged Commissioner Margaret Hamburg to give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars.
With over 7,000 rare diseases identified and 30 million Americans affected, the patient population represented by NORD is extraordinarily heterogeneous. Without thoughtful and consistent naming protocols for biologics, there is the potential for significant confusion among treatment options and increased adverse events, both of which could jeopardize patient safety. Distinguishable naming of all biologics is imperative to recognize for health care professional to deliver the degree of customized care that is routinely required for patients with complicated, uncommon and less well-studied diseases.
"Every patient deserves the care best suited for their medical situation and most likely to give them the best outcomes," said NORD President and CEO Peter L. Saltonstall. "Biologics are often the most advanced and effective treatments for patients we represent and everyone in the treatment continuum should be able to readily identify the specific drug product a patient was given."
The letter raises a number of concerns specific to rare disease patients. Rare disease patients usually have unique treatment courses and while similar medications might work equally well, such a judgment can only be made through tracking of outcomes made possible through distinguishable names.
According to the letter, distinguishable names for biologics would:
- Support the medical community's vital post-approval learning curve regarding which medicines are best for their rare disease patients;
- Support surveillance and tracking of adverse events given that rare disease patients often do not respond to medications in the same way as other individuals might; and
- Reinforce a critical distinction in the biosimilars law between biosimilars (similar not identical) and interchangeable biosimilars (similar but demonstrated to have comparable clinical results).
National Organization for Rare Disorders (NORD)