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2023 Wrap-Up: Regulatory Updates in Specialty Pharma

The world of regulation surrounding specialty pharma is ever-changing. As 2023 ends, we take a look at some of the regulatory news updates that affect the industry and provide pharma market insights.

Regulatory Actions and Guidance

Recent regulatory action and guidance from the U.S. Food and Drug Administration (FDA) and other state, federal, and international agencies is changing the playing field for remote monitoring devices and other rare disease treatments.

FDA Revises Final Guidance on Remote Patient Monitoring Devices

In October 2023, the FDA released updated final guidance on its enforcement policy for remote patient monitoring devices. The updates affect guidance released in 2020 on using non-invasive monitoring devices during the pandemic. The guidance seeks to reduce the number of 510(k) submissions related to modifications of devices for remote monitoring.

MDCG Guidance Seeks to Regulate Medical Device Software

The European Commission’s Medical Device Coordination Group (MDCG) recently released guidance on how to ensure that medical device software meets EU regulations. It clarifies when hardware that uses medical device software would count as a “medical device” and how these items should be regulated. The guidance is expected to affect a number of popular devices, including smart watches that track pulse, respiration and similar health-related indicators.

FDA Releases New Guidance Documents on Various Topics

In the second half of October 2023, the FDA released several new guidance documents covering various topics including specialty pharmacy regulations. These included guidance on remote patient monitoring devices, discussed above. The guidance documents also include:

  • Benefit-Risk Assessment for New Drug and Biological Products (pdf).
  • Communications from Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products: Questions and Answers (pdf).
  • Development and Licensure of Vaccines to Prevent COVID-19: Guidance for Industry (pdf).
  • Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies: Guidance for Industry (pdf).

The FDA further released guidance on the development of new tobacco products and compliance policies for blood donor screenings.

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Drug Approvals and Market Moves Provide Pharma Market Insights

Several new drugs have received approval or are making their way through the FDA’s approval process or received other regulatory nods as 2023 draws to a close. Top moves in this area include:

  • FDA approval of PENBRAYA, Pfizer’s meningococcal disease vaccine. PENBRAYA combines components from two existing vaccines to create the first pentavalent vaccine against meningococcal disease, says Annaliesa Anderson, senior vice president and head of vaccine research and development at Pfizer.
  • The EU approves AstraZeneca and Daiichi Sankyo’s Enhertu for treatment of advanced non-small cell lung cancer. The European Union gave the go-ahead to AstraZeneca and Daiichi Sankyo for use of Enhertu, a treatment for certain types of lung cancer, following a positive opinion from the Committee for Medicinal Products for Human Use. Enhertu opens up more treatment options for these patients, says Dave Fredrickson, executive vice president of the oncology business unit at AstraZeneca.
  • CDER grants novel drug approvals to several new products. In October 2023, CDER added three new treatments to its novel drug approvals list. They include Bimzelx (bimekizumab) to treat plaque psoriasis, Zilbrysq (zilucoplan) to treat generalized myasthenia gravis, and Velsipity (etrasimod) to treat ulcerative colitis.

The regulatory landscape changes quickly. Staying on top of the pharma market insights and specialty pharmacy news coming out of the FDA and related organizations is essential. Pharmaceutical organizations that stay in tune with the news and changing specialty pharmacy regulations can organize their information effectively. They are thus better positioned to spot opportunities and address oncoming challenges.

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