FDA Must Forge Ahead With Biosimilar Drug Review

Over the past several decades, medical innovation has unlocked the power of biologics — scientifically complex therapies used to treat diseases such as rheumatoid arthritis, ulcerative colitis, psoriasis, and several forms of cancer.

Unfortunately, many of these life-changing therapies remain prohibitively expensive, and even patients with health insurance coverage struggle to afford the astronomical co-pays. That’s why it’s more important than ever that the Food and Drug Administration, which has a dual mandate to promote patient access while ensuring consumer safety, strike a delicate balance between the two. This is especially true in the area of biosimilar drug review, where the safety stakes are high, and where additional FDA-approved therapies can have real value and impact for the American healthcare consumer.

Rheumatic diseases alone affect more than 52 million Americans, and FDA-approved biologic therapies and those in the development pipeline are critical lifelines that can dramatically improve long-term outcomes. These drugs are highly effective, helping my patients to manage pain, avoid long-term disability, and lead healthy, full lives.

For this reason, clinicians and patients have welcomed the advent of biosimilars — comparable versions of biologic therapies that have the potential to increase competition and eventually lower the cost of these life-changing treatments. In late 2016, the first biosimilar to treat rheumatic diseases hit the U.S. market and a handful of other biosimilar therapies intended for the treatment of rheumatic diseases have been approved. Dozens of these therapies are currently undergoing FDA review.

The addition of new and potentially lower-cost therapies is good news for patients who struggle to access and afford the biologic therapies they need to manage their conditions, as well as for the efficiency of the overall U.S. healthcare system.

But the safety stakes are also high. Biosimilars are incredibly complex, and unlike generic or small molecule drugs, even small changes in the manufacturing process can lead to ineffective or dangerous interactions with the immune system. For this reason, it is imperative that there be full transparency in biosimilar naming, labeling and post-market surveillance practices, as well as a robust FDA workforce to support the biosimilars review and regulatory process.

As prescribing doctors, we need to know that new biosimilars are not only affordable, but also entirely safe for our patients — and this is only possible with a sufficient workforce at the FDA.

Congressional leaders can support these efforts by ensuring adequate funding to enhance and expedite the FDA’s review of biosimilar therapies and to issue industry guidances. To achieve this goal, Congress should create a dedicated program for biosimilar review with its own source of funding, similar to what exists for prescription drugs, generic drugs, and medical devices.

Lawmakers should also quickly pass the Biosimilar User Fee Act. Under BsUFA, the FDA is permitted to collect user fees on biosimilar products in order to fund the review process. The law expires in September 2017. If Congress is interested in making sure patients have access to these life-saving therapies, it should reauthorize the BsUFA as soon as possible.

Within the FDA, action is also required.  Ensuring patient safety requires full transparency between biosimilar manufacturers, regulators, doctors, and patients so that physicians know precisely what they’re prescribing and patients know what they’re taking.  To achieve this, biosimilars need to be adequately studied, have distinct names, clear information on drug labels, and consumer-oriented pharmacy dispensing practices.  The agency has made some good progress on this front and must now issue and implement final industry guidances, including the excellent plan for interchangeable biosimilars.

Moreover, the incoming administration should enact hiring reforms.  At present, bringing on highly qualified employees is far too difficult.  However, the 21st Century Cure Act (section 3072) authorized decision-makers to make a change.  There are many directions that the Trump Administration could go on this front.  For instance, federal agencies could use market surveys of salaries in related competing industries which FDA candidates might work in.  And using such data, they could better align remuneration rates to attract the very best talent with the expertise to study these complex drugs.  Put simply, innovative and efficient onboarding of experts in the complex field of biologic therapies should be a top priority in the years ahead.

In the end, biosimilars give us the opportunity to overcome the trade-off between cost and innovation. We can have the best of both worlds, providing patients with state-of-the-art treatments and manageable prices. It is imperative that our policymakers dedicate more time and resources towards biosimilar policy at the FDA to realize that goal.


Angus Worthing, M.D., is a practicing rheumatologist in Washington, D.C., and chair of the American College of Rheumatology’s Government Affairs Committee.

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