There is a superficial attraction to common names. They have contributed to extraordinary usage of generic drugs, now over 80% of all prescriptions filled in the U.S. However, the steady, rapid uptake of generics ultimately rests on the level of proof required by the FDA. A generic drug must be identical and bio-equivalent to its reference product.
Biologics are different. They are complex molecules that cannot be duplicated in a precise manner with current technology. Congress recognized this when it passed the Biologics Price Competition and Innovation Act. A biosimilar product can be approved for marketing only if data show that, among other things, the product is “highly similar” to an already-approved biological product.
The naming of biologics has special significance for the 30 million Americans with rare diseases, whom my organization represents. Any disease affecting fewer than 200,000 Americans is considered rare, and there are approximately 7,000 such diseases.
Rare disease patients welcome the potential for cost-savings and improved access that biosimilars may bring. Yet, we also are concerned. Without distinguishable names for all biologic products, there is a significant risk to our community that prescribers and payers will gloss over the critical difference between “identical” (generic drugs) and “highly similar” (biosimilar biologics).
Rare-disease patients are often among those most sensitive to even small differences among products. “Highly similar” may be fine for some patients for some indications in some medical circumstances. It will not always be good enough to ensure safety and efficacy for rare-disease patients.
To protect rare disease patients, distinguishable names are needed so that every patient, prescriber, payer and pharmacist can be certain exactly what product was dispensed. If there are problems—particularly patterns of adverse events or therapeutic failure—we will then be able to educate payers and prescribers as to what product will work best for specific rare disease populations and sub-populations.
Specifically, distinguishable names for biosimilars support the medical community's vital post-approval learning curve regarding which medicines are best for rare-disease patients. Further, distinguishable names enable 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. Finally, distinguishable names reinforce the critical distinction in the BPCI Act that biosimilars are highly similar, not identical to the reference product.
The FDA will make the key decisions on biosimilars; patients, providers and payers will have to live with the consequences. For rare-disease patients, distinguishable names for biologics are the fundamental core of maximizing the benefits and minimizing any potential harm from biosimilars.
Peter L. Saltonstall is president and CEO of the National Organization for Rare Disorders.