Now that the Affordable Care Act (ACA) was mostly upheld by the United States Supreme Court, one of the major provisions—the Biosimilar Price Competition and Innovation Act or BCPA—will likely be moving to the forefront of priorities for the Food and Drug Administration (FDA) as well as pharmaceutical and biotechnology companies.
The BCPA created an abbreviated licensure pathway for biological products shown to be biosimilar to or interchangeable with an FDA-licensed biological reference product [section 351(k) of the Public Health Service Act]. This process is similar to the 1984 Hatch-Waxman legislation FDA uses to approve generic drugs. Biologic products are therapies used to treat diseases and health conditions. They include a wide variety of products including vaccines, blood and blood components, gene therapies, tissues, and proteins. Unlike most prescription drugs made through chemical processes, biological products generally are made from human and/or animal materials.
Biologic drugs are highly complex drug therapies for patients diagnosed with some of life’s most challenging diseases like cancer, rheumatoid arthritis, and Multiple Sclerosis.
The FDA defines a biosimilar as “a biological product that is highly similar to an already approved biological product, notwithstanding minor differences in clinically inactive components, and for which there are no clinically meaningful differences between the biosimilar and the approved biological product in terms of the safety, purity, and potency.”
According to a recent article from MedCity News, biologic products area a growing share of the U.S. drug market: they accounted for seven of the top 20 medicines sold in the United States in 2012. Despite their widespread use, there are no copycat or ‘biosimilar’ versions in the United States; unlike generic versions of traditional chemical drugs, which have been widely available for the past three decades.
Consequently, the author noted that “a major challenge for scientists and regulators remains ensuring patient safety. Since biologics can never have identical copies, seemingly insignificant changes in the composition of these medicines can have unexpected or even harmful effects in patients.”
To address these concerns, FDA released three Draft Guidance documents back in February of this year. Janet Woodcock, M.D., director of FDA’s Center for Drug Evaluation and Research (CDER), noted that these “draft documents are designed to help industry develop biosimilar versions of currently approved biological products, which can enhance competition and may lead to better patient access and lower cost to consumers.” Some of the key points in the FDA guidance are:
- Biosimilars will be reviewed as a package, with the FDA considering the totality of the evidence
- FDA is taking a stepwise approach
- Biosimilar does not mean identical
- Biosimilarity and interchangeability are sequential decisions. They can be done in the same 10-month approval period if that is requested initially. If interchangeability is requested later in the approval process, it extends the approval period.
- Interchangeability will require a switching study.
- The reference product has to be FDA approved; European approval is not sufficient.
As FDA gets closer to establishing the Biosimilar pathway, the article noted that the Agency “must consider several factors to safeguard patient safety.”
First, the article noted that “clinical trials will be needed to ensure a biosimilar product is as safe and effective as the innovator product.”
Second, “because a biosimilar will never be the same product as the original biologic, a patient should be made aware if he or she is receiving the original biologic drug or its biosimilar. It is also important that the patient, working with his or her primary physician, fully understands the differences between the two products being offered.”
Finally, the author recommended that FDA “give unique names to each biosimilar that comes on the market.” The article noted that this will help avoid any confusion between doctors, pharmacists, and patients over what medication the patient is taking. In addition, unique names will help trace any biosimilar drug on the market that begins to cause unexpected side effects in patients, which will allow patients to easily determine whether their drug is affected ’ and can learn what steps to take to prevent or mitigate the side effects.
As FDA continues to evaluate comments from the public, including industry, it will be important to keep in mind patient safety, innovation and transparency in creating this new approval pathway. More importantly, however, the companies that begin to develop biosimilars will need to provide educational grants to continuing medical education (CME) providers to educate physicians and health care providers about the diferences in these new products. As noted above, biosimilars and biologics are complex products and there will be a tremendous need for education. While there may not be any biosimilars on the market presently or in the near future, a framework for CME educational grants and programs will need to be established.