Regulatory uncertainty mires breakthrough drugs

Food labels now cater to consumers who are as hungry for data as for, say, potato chips.   A quick glance at your snack’s packaging can tell you whether it’s organic, vegan, GMO- or gluten-free, or if it was packaged in a factory that also processes peanut, tree nut or wheat products. Consumers want transparency about what they eat.  It should come as no surprise that physicians and patients want the same with the medications they prescribe. 

Yet the issue of transparency in prescription drug labeling, testing and naming has given rise to debate in recent years, specifically related to biological and biosimilar drugs. Biological drugs are the breakthrough medications developed from living cells or tissue that change lives for rheumatoid arthritis, multiple sclerosis, cancer, migraine headache patients and others.  Biosimilars are follow-on biologic medications, which many hope will offer similar results at lower prices.

{mosads}But there’s a snag.  The United States and its regulatory system are accustomed to small molecule drugs and their follow-on generics.  These can be duplicated by following a series of chemical reactions. Yet biological medicines are inherently unique because of their living source. Thus, all biosimilars vary in small but potentially important ways from the original biological medicine. They are simply not generic copies. 

So at each step of the regulatory process, the Food and Drug Administration must consider how to treat biologics and biosimilars.  The agency must develop a new approach that acknowledges biosimilars’ distinctions—and the effect these distinctions can have on patients.

Take the issue of naming.  Biologics and biosimilars are similar but not identical.  How similar, then, should their non-brand names be?  Even as the Food and Drug Administration approved the country’s first biosimilar earlier this year, the agency could not make up its mind about this fundamental question. So it approved the drug, designed to boost white blood cells in cancer patients, with a “placeholder” name—the nonproprietary name followed by a four-letter suffix indicating the drug’s manufacturer.

Distinct names make prescription drugs traceable in the case of side effects or adverse events.  They minimize chances for confusion or accidental substitution of one drug for another. They help keep patients’ records accurate.  But the tentativeness of FDA’s solution stopped physicians short of a collective sigh of relief, leaving them to wonder instead if a distinct naming system is here to stay.

Prescribing information presents another challenge.  Physicians trust the paperwork that accompanies a prescription medication as the authoritative information source on dosing levels, conditions the drug is approved by the FDA to treat, clinical trials data and details on safety.  Yet the FDA allowed its first approved biosimilar to share prescribing information with the original biological drug. 

If this decisions sets a precedent for future biosimilars, physicians will lack important data about the actual biosimilar, even basic details such as whether the medication is in fact a biosimilar.   The information gap presents patient safety concerns. Biological medicines sometimes affect patients’ immune systems, and nuances in biosimilars may trigger these side effects differently or with different patient groups.  Physicians need accurate and fully transparent data to make prescribing decisions that protect patients’ safety.

Yet on these basic issues, the FDA’s long-term regulatory stance remains uncertain.  Moving forward without first establishing basic regulatory standards is concerning.

Biosimilars could bring lower costs and expanded options for patients, but a lack of clarity may undermine the physician confidence needed for widespread adoption. This much is clear: confusion serves no one; least of all the patients that biosimilars are designed to treat.

Conschafter is blog editor for the Institute for Patient Access. Charles is a neurologist from Nashville, Tenn. and chairman of the Alliance for Patient Access.


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