Rare Disorders Organization Stresses Testing, Transparency on Biosimilars

By Amanda Conschafter, blog editor

Maximizing biologic medicine’s potential for rare disease patients means observing needed safety precautions. So explained the National Organization for Rare Disorders (NORD) in its recent webinar, “Understanding Biologics & Biosimilars.” Citing molecular distinctions between biologics and their biosimilar follow-ons, NORD called for specific precautions – distinct naming, physician notification, comprehensive testing and transparency – to ensure patient safety.

Distinct naming. Distinct naming, NORD argued, can help the FDA and care providers better track adverse events related to biosimilars. Tracking such events, and accurately monitoring new biosimilars’ effect on patients with rare diseases, helps safeguard patient wellness.

Physician notification. Likewise, physicians should know when their patients’ prescriptions for a biologic are filled with a biosimilar substitute, NORD explained. Just as distinct names encourage clarity in monitoring patient use en masse, physician notification allows physicians to monitor individual patient’s response to medications with accurate data.

Comprehensive testing. NORD also advocated for testing of each separate condition for which biosimilars receive FDA approval. More patient advocacy organizations are calling for comprehensive testing as the issue of indication extrapolation gains public awareness in the United States. While biologics undergo clinical testing for each use for which they’re approved, extrapolation allows biosimilars to receive approval for indications for which they haven’t been tested. The approval extrapolates the biosimilar’s testing data for one condition and applies it to additional conditions based upon the biologic reference product’s approved uses. The practice raises significant safety concerns.

Transparency. Finally, the FDA should aid patient safety, NORD contended, by making its approval process transparent – and by establishing clear criteria for interchangeability among biosimilars and their biologic reference products. NORD isn’t alone in requesting clear information from regulators. In recent weeks, Members of Congress have also called upon the FDA for clarity as the agency considers its first two applications for biosimilar approval.

As NORD notes, in only 30 years biologics have expanded to aid more than 350 million patients worldwide. Biosimilars could play a pivotal role in further expanding access for patients with rare disorders. But NORD suggested that providers, patient advocates and regulators must exercise caution moving forward, balancing the desire to expand access with the imperative to protect patient health.


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