Biologics & Biosimilars

Biologics are medicines made from living organisms and are far more complex and much more difficult to develop and manufacture than traditional chemical drugs. Millions of people are benefiting from these breakthrough medicines in the treatment of many conditions including rare diseases. And with many more biologics under development, millions more will benefit in the coming years.

Biosimilars are medicines that are highly similar, but not identical to biological medicines. Because of the uniqueness and complexity of biologics, biosimilars are NOT generic copies of biologic medicines. While there are a small number of biosimilars approved in other countries, there are no approved biosimilars in the U.S. Some, however, are under development. The U.S. Food and Drug Administration (FDA) accepted its first two applications for approval of a biosimilar in late summer 2014.

The Law

As part of the Affordable Care Act (ACA), Congress enacted the Biologics Price Competition and Innovation Act (BPCIA) establishing authority for FDA to review and approve biosimilars.

BPCIA requires that a biosimilar be “highly similar” to an already-approved innovative biologic product and that “there are no clinically meaningful differences” between it and the reference product “in terms of the safety, purity, and potency of the product.”

The law also gives FDA authority to deem a biosimilar to be “interchangeable.” Interchangeability requires a significantly higher standard to confirm that an interchangeable biosimilar will achieve the same clinical result in any given patient. 

FDA

FDA has been developing processes and standards to review and approve biosimilars.

Key decisions, such as the kinds of studies that must be done and standards for biosimilars, have not yet been made. There are currently two draft guidance documents out for comment and more are expected.

This means we are at a critical time. FDA is expected to put forward biosimilar approval standards and consider approval of the first U.S. biosimilar against those standards possibly in 2015.

Congress

Congress approved the BPCIA without extensive hearings as a part of the much larger ACA. No hearings were devoted to the safety of biosimilars prior to the approval of the legislation. The law left key details related to biosimilar review and approval to the discretion of the FDA.

While key Congressional leaders promised to hold a hearing on patient safety, Congress has yet to hold any oversight hearings regarding the BPCIA and biosimilar safety.

The Concerns

The complexity and uniqueness of each biologic medicine make it difficult to produce a biosimilar. A slight variation in the manufacturing can result in serious, life-threatening adverse events in our patients. It is imperative that the FDA ensure all biosimilars are thoroughly tested and meet the highest safety standards the same as all biologics are today. And it is crucial that the FDA get it right from the beginning.  As we know, it is far easier to fix something before it takes effect than it is after the fact.

Given the significance of the decisions that will be made on a number of very important patient safety and access issues by FDA, we need a strong and united patient voice to be heard by policymakers at this critical time. We are concerned that without such an effort, a regulatory structure could be established and products approved and administered to patients without adequate patient protections in place. By putting forward a clear and strong patient voice, collectively we can make an important difference for our members and millions of Americans now and into the future. 

PBSA proposes to the FDA the following actions as it finalizes its standards for oversight and approval of biosimilar medicines:

  • The FDA must put patient safety first and require biosimilars to prove they are just as safe and effective as reference innovative biologic products.
  • FDA must recognize that there are tremendous variations in the complexity of biologics ­and the substantial differences in therapeutic responses to biologics from patient to patient. FDA standards for the review and approval of biosimilars should not provide for a “one-size-fits-all” approach that could threaten patient safety.
  • FDA must require that manufacturers of both innovator biologics and biosimilars conduct rigorous clinical testing to prove that their product works safely and effectively in each and every condition and distinct patient population for which it is approved for use. Failure to perform adequate testing in each group of patients and disease states puts patients at risk.
  • FDA must require strict standards, including clinical trials prior to FDA approval, for determining whether a biosimilar is “interchangeable” and can be expected to produce the same clinical result as the reference product in any given patient. Without clear, understandable, and strict standards for interchangeability patient safety could be at risk.
  • There must be no automatic substitution of biosimilars for biologics. The choice of products should not be determined by a pharmacist, regulator, or insurer but by a physician in consultation with his or her patient; over a biosimilar. 
  • Given the vast differences with biologics and biosimilars, each biologic including biosimilars must have a distinguishable non-proprietary name. Distinguishable names facilitate a process of determining the cause of an adverse event in an expeditious way. Distinguishable naming is a critical component of transparency in healthcare.
  • Steps must be taken to assure appropriate tracking of adverse events for all biologics, including biosimilars, so that safety problems are promptly and accurately identified.

For more information, contact Larry LaMotte, Vice President of Public Policy at llamotte@primaryimmune.org or 443-632-2552.