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Congress, FDA Must Put Patient Safety First in Biosimilar Approval Process | Commentary

Breakthrough medicines known as biologics are already benefitting millions of people in the United States and around the world. With the prospect of an emerging category of biologic drugs known as biosimilars, however, concerns about patient safety and the efficacy of biosimilars have been raised. Congressional and Food and Drug Administration oversight is critical to make sure patients will not be at risk.

Made from living organisms, biologics are far more complex and much more difficult to develop and manufacture than traditional chemical drugs. The advent of sophisticated biologic medicines such as immunoglobulin, clotting factor and monoclonal antibodies has saved and transformed the lives of Americans who have been diagnosed with many life-threatening diseases.

People living with primary immunodeficiency diseases, for example, rely on lifelong use of biologics for long-term management of their conditions. Without biologics, many primary immunodeficient individuals are unable to fight off even minor infections and viruses, including the common cold.

When Congress passed the Affordable Care Act, it gave the FDA regulatory authority to review and approve a new class of biologic drugs known as biosimilars. 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.

Now is a critical moment for the future of biosimilars and patient health.

Although there are not yet any approved biosimilars in the U.S., the FDA has accepted the first three applications for approval — the first biosimilars may be approved within a year.

Despite the potentially imminent arrival of biosimilars to the U.S. marketplace, the FDA is still developing the biosimilar approval process. It has yet to offer for public review and comment critical details about standards for biosimilar approval and about what steps it will take to safeguard public health.

Congress has yet to hold even a single hearing on biosimilars to ensure the FDA is properly implementing this key provision of the Affordable Care Act. Congress needs to exercise its critical oversight role.

The FDA has held more than 55 meetings with the pharmaceutical industry manufacturers of biosimilar drugs, but has had few meetings with representatives of the patient advocacy community.

To help ensure the voices of patients are at the center of this important health care debate, and to ensure that patients ultimately have access to safe and effective biosimilars, 22 leading patient advocacy organizations have come together to form Patients for Biologics Safety and Access, the only coalition on biosimilars made up entirely of organizations that represent the interests of patients.

As biosimilars are developed and approved in the United States, we urge the FDA to take steps that uphold patient safety above all else. Federal regulators should consider essential factors when approving biosimilars. The FDA should recognize that there are tremendous variations in the complexity of biologics and there are substantial differences in therapeutic responses to biologics from patient to patient. Even a minor difference between a biosimilar drug and the original biologic product, known as its innovator biologic, could have a significant adverse health impact for a patient. Consequently, a one-size-fits-all approach to the approval of biosimilars could threaten patient safety.

The FDA should 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.

Physicians and patients must have all the information necessary to make a fully informed choice about whether to use an innovative biologic or biosimilar. Moreover, pharmacists should not be allowed to automatically substitute biosimilars for biologics without first notifying the patient and their doctor.

Steps must also be taken to assure appropriate tracking of adverse events for all biologics, including biosimilars, so that safety problems are promptly and accurately identified and corrected. All products, including biosimilars, should carry distinguishable, nonproprietary names, as well as brand and lot information to quickly trace a product to an adverse event. As new biologic products are made available, additional post-marketing surveillance will also be needed.

As both the president of a patient organization and a mother of a son who relies on a biologic medicine, I recognize the great potential of biosimilars to improve access to affordable treatments for many patients living with significant and life-threatening conditions. However, we should not jeopardize patient health by rushing at the expense of safety.

Marcia Boyle is the president and founder of the Immune Deficiency Foundation, a national non-profit patient organization dedicated to improving the lives of patients with primary immunodeficiency diseases.

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