A new provision under the health care reform law could help people with inflammatory arthritis afford biologics – a class of highly expensive disease-modifying drugs – by enabling the Food and Drug Administration to create a pathway for similar drugs to come to market at a lower cost.

In a two-day hearing in Washington, D.C. this week, the FDA heard from drug companies, physicians, patients and their advocates about the importance of so-called biosimilars and what the FDA should consider when establishing regulations for them.

“A little over 10 years ago, a diagnosis of RA or [juvenile arthritis] would have meant I could look forward to a future of gnarled joints, chronic pain, pulmonary and cardiac damage, and increased risk for early death, loss of function, limited mobility and often complete functional disability,” Arthritis Foundation board member Jan Wyatt, who has rheumatoid arthritis, told the FDA panel. Biologics, she added, have been “life-transforming.”   

Biologics typically used to treat people with arthritis, generally by injection or infusion, are Enbrel, Rituxan, Actema, Remicade, Symponi, Orencia, Humira, Cimzia and Kineret. They can cost up to tens of thousands of dollars a year without health insurance coverage, and even with health insurance can cost hundreds of dollars a month.

Access to biologics “is an unmet public health need that lower-cost biosimilars will help to fulfill,” said Mark McCamish, MD, PhD, of Novartis, whose wife has ankylosing spondylitis. “Vigorous biosimilar competition will encourage innovation,” he added.

Similar, but not the Same

Health care reform’s Biologics Price Competition and Innovation Act, or BPCI, would allow a fast-track testing process to get the biosimilars to market more quickly and cheaply than the original biologics.

At issue is how much testing and tracking of these new drugs is needed to ensure they’re safe and effective, because biosimilars are very different from other types of generic drugs.

Biosimilars, like biologics, are developed not with chemicals like other drugs, but from proteins in living cells, including viruses and blood components. Because they are made from biological components, biosimilars are similar to the original biologic drug, but not exactly the same. Generic drugs, by contrast, are synthetic, and chemically identical in every way to their name-brand originals, which have a known structure.

Biosimilars, as a result. are not as easy as chemical drugs to fit into a one-size-fits-all approval plan.

BPCI allows for less testing for biosimilars to come to market because the safety and efficacy already have been shown for their original drugs. The question the FDA is grappling with is how much clinical testing is appropriate and how much would be considered unnecessary.  

Redundant Tests, High Costs

Some pharmaceutical industry representatives argued that biosimilars would be so similar as to be interchangeable with the originals, so they would require little or no additional clinical testing.

Scott Reid, speaking for CVS Caremark Corp., suggested that even the name of the original be used for the biosimilar. Lot numbers could be used to track side effects, he said.

Others speaking for the pharmaceutical industry also urged more latitude in testing requirements.

“Solid clinical evidence” is needed before the FDA approves a drug as biosimilar, but “unnecessary clinical trials” would increase the economic burden, said Rivka Riven-Kreitman, a representative of Teva North America, which has been focusing on biosimilars production.

Jay Siegel, MD, head of regulatory affairs for Johnson & Johnson, told the panel, “Clinical outcomes measurements may not always be necessary.” In some cases, existing studies could be used to extrapolate a new drug’s effects, he said, including research of the 20 or more biosimilars that are already approved for use in Europe.  

“No Substitute for Testing”

But Gregory Schimizzi, MD, a rheumatologist speaking for the Coalition of State Rheumatology Organizations, said biosimilars may act differently in the body than the biologics upon which they are based, causing different side effects. The FDA should require clinical trials for all new products, regardless of their similarity to already-approved drugs, he argued. “[A] minor difference can cause a biosimilar protein to have vastly different effects. There simply is no substitute for testing these products given their complexity.”

The Foundation’s Wyatt added, “There will be subtle product variations that can have severe consequences.”

In addition, a surveillance system is critical for tracking adverse reactions to biosimilars once they come to market, said Wyatt. Patients have to be able to report side effects accurately to help ensure public safety.

Making these drugs affordable so more people can benefit from them is critical, but not at the risk of their health and safety, Wyatt argued.

“People will not trade a less favorable risk-benefit profile for a cheaper drug,” she said.  “That is simply unacceptable.”

The FDA will accept written comments through the end of the year at www.regulations.gov. Include the docket number: FDA–2010–N–0477.
Reporting by Larry Lindner