Recent Developments in Medical Devices and Biopharma You Should Know

At a Glance

• The Centers for Medicare & Medicaid Services has issued a final procedural notice on the Transitional Coverage for Emerging Technologies pathway.
• The Center for Devices and Radiological Health has issued final guidance on clinical considerations for studies of devices intended to treat opioid use disorder.
• The Office of Foreign Assets Control has released guidance on the extension of the statute of limitations to 10 years for violations of the International Emergency Economic Powers Act (IEEPA) or the Trading With the Enemy Act (TWEA).
• The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research has released guidance on using real-world evidence to support regulatory decisions for medicines.
• CDER has published new clinical pharmacology guidance for investigational drugs.

With a rapidly evolving regulatory landscape, staying on top of the latest updates is crucial for companies in the biopharma and medical devices sectors. In our inaugural biopharma and medical devices briefing, we highlight a selection of recent regulatory and legislative developments that should be top of mind for companies operating in these sectors.

Regulatory and Legislative Developments

CMS Issues Final Notice on Transitional Coverage for Emerging Technologies

On August 7, 2024, the Centers for Medicare & Medicaid Services (CMS) issued a final procedural notice on the Transitional Coverage for Emerging Technologies (TCET) pathway. First proposed in June 2023, the TCET pathway uses existing national coverage determination (NCD) and coverage with evidence development (CED) processes to expedite Medicare coverage of certain breakthrough devices.

Manufacturers of FDA-designated breakthrough devices that fall within a Medicare benefit category may submit a nonbinding letter of intent to self-nominate for TCET participation 18 to 24 months before an anticipated FDA market authorization date. CMS will review nominations on a quarterly basis and will approve up to five candidates per year.

CMS plans to make a coverage decision within six months from market authorization for breakthrough devices accepted to the TCET pathway, providing upwards of five years of transitional coverage while evidence is generated to support a long-term NCD.

Guidance for Developers of Medical Devices for Treatment of Opioid Use Disorder

In 2018, U.S. Food & Drug Administration’s Center for Devices and Radiological Health (CDRH) issued an “innovation challenge” to promote development of medical devices aimed at curbing opioid use disorder. Hundreds of companies applied for the challenge. Over the next few years, CDRH provided further help to all developers by articulating the agency’s expectations for clinical trials needed to obtain marketing authorizations for their medical device products.

In July 2024, a final guidance was issued, with the title Clinical Considerations for Studies of Devices Intended to Treat Opioid Use Disorder. Clinical studies in this target population are particularly challenging. The guidance lays out helpful recommendations for study design, including the assignment of subjects to the treatment versus control groups, participant retention, length of the study, prevention of missing data, and appropriate ways to measure treatment’s success.

Increase in Statute of Limitations and Recordkeeping for OFAC Violations

In April 2024, President Biden signed legislation that extends the statute of limitations (SOL) for civil and criminal violations of the International Emergency Economic Powers Act (IEEPA) or the Trading With the Enemy Act (TWEA) from five years to 10 years. This primarily affects regulations implemented by the Department of Treasury’s Office of Foreign Assets Control (OFAC) relating to sanctions and embargo programs.

On July 22, 2024, OFAC released guidance concerning this SOL increase, clarifying it intended to apply the new 10-year SOL to any violations committed after April 24, 2019. OFAC also stated that it anticipates releasing new recordkeeping regulations consistent with this new 10-year period later this year. Read more in our recent article.

Using Real-World Evidence to Support Regulatory Decisions for Medicines

In clinical practice, rich information is routinely collected about the health status, medications used and changes in each patient’s condition over time. With modern-day electronic systems for storing, searching and sharing that information, new efficiencies could be realized for gaining additional knowledge about the safety and risk-benefit profile of medicines. Industry and regulators both in the U.S. and Europe have been developing ways to tap that resource. However, using real-world data responsibly requires tackling real-world challenges such as different naming conventions and reporting practices, missing data, duplicate reporting, and others. For this reason, the latest guidance from the FDA Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research represents an important step forward.

That guidance, titled Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products, discusses such topics as selection and vetting of data sources, defining study design elements (e.g., “exposure,” “outcome” and “covariates”), dealing with missing data, assuring data quality during data acquisition, curation, and integration into the final dataset for analysis. This guidance complements the previously issued Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data Sets.

FDA Publishes New Clinical Pharmacology Guidance for Investigational Drugs

To understand how an investigational new drug gets processed by the body, radiolabeled versions of the drug are sometimes given in clinical trials. The amount of the drug absorbed, distributed throughout tissues and organs, metabolized, and ultimately eliminated could then be readily measured, giving researchers insights into the fate of the new molecule.

The recently finalized guidance from the FDA’s Center for Drug Evaluation and Research, Clinical Pharmacology Considerations for Human Radiolabeled Mass Balance Studies, includes recommendations for drug developers regarding study design, participants, product formulation and administration, and bioanalysis, as well as on the appropriate timing of these studies and reporting requirements. The guidance reflects the agency’s experience and current thinking, based on the recently reviewed new drug applications.