The safety of biosimilars and FDA regulation

by John Horton

When the FDA was created in 1906, not even Nostradamus could have predicted the medical advances that would come to extend and improve our lives: childhood vaccines, insulin, and the use of lasers in surgery were all unimaginable a century ago.

While many future advances are beyond our imaginations’ reach, it seems certain that the advances in biologics — medicines made from living organisms — will stand out as some of the most important health care developments of our time. (Unlike biologics, “traditional” pharmaceuticals are made from non-living chemicals.)

A new debate is happening in Washington that will likely impact the pipeline of hope for those suffering from some of the most serious diseases and conditions. The FDA now has the authority from the new health care law to implement a biosimilars pathway.

There is a lot at stake. At issue are the safety standards to which “biosimilars” — copycat version of the original biologics — will be held by the FDA. When biologic drugs, like Ixiaro (for encephalitis) and Cervarix (to prevent cervical cancer), are first approved, they have to be certified by the FDA as safe and effective, just like any other drug. But unlike chemical drugs, like Lipitor and Zoloft, exact “generic” versions cannot be made of biologic drugs (which is why they are called “biosimilars” and not “generic biologics”.)

There’s the rub: for “biosimilars”, how similar is similar enough for the FDA to approve it as safe and effective? The FDA gave drug companies and other interested parties until the end of 2010 to submit proposed answers to this question — input that the FDA will consider as it develops safety standards for biosimilars this year. Perhaps predictably, some copycat drug companies called for the safety standards to be lowered, which will reduce their barrier to market entry.

The biosimilars companies’ argument is: if we are held to the same burdens of proof for safety and efficacy as the original innovator companies, we will be unable to significantly reduce costs, which is an important patient benefit for generic or biosimilar drugs. This argument contains a valid point: new drugs, including biologics, can be expensive.

But, there is the danger that if the FDA lowers its rigorous safety standards for biosimilars, patients will be put at risk. Indeed, some biosimilar companies have publicly argued that the benefits of reducing costs for biosimilars mean that there is a level of “acceptable risk” for patients. This will be cold comfort for a cancer patient provided a biosimilar for a drug like Abraxane if, for her, the drug does not work, or causes unforeseen side effects worse than the cure.

There is an easy answer: even if a biosimilar can never be physically identical to the original biologic, the FDA should hold it to the same high safety standards that protect patients from harm. Whether the agency insists that biosimilars be held to the same safety standards as other drugs will determine if, 100 years from now, biosimilars are seen as a historic medical advance or as a dangerous aberration in the forward march of medicine and health.

John Horton is the President and founder of LegitScript, a service that verifies and monitors the online sale of pharmaceutical products. From 2002 until 2007, Mr. Horton served as a policy advisor in the drug policy office of The White House.

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