New European Regulations Could Hinder Rare Disease Treatments, Pharma Industry Raises Concerns Over Stricter Clinical Trial Rules

Pharmaceutical companies, researchers, and patient groups are raising concerns about new European Union regulations that could hinder the development of treatments for rare diseases.

The new rules, set to take effect in January, impose stricter guidelines on medical trials, particularly limiting the use of single-arm trials, which are often essential for rare disease research.

Nearly 40 organizations, including Cancer Patients Europe, the European Association of Urology, and the World Federation of Hemophilia, have urged Brussels to revise these regulations.

The Financial Times highlights that single-arm trials, in which all patients receive the experimental treatment without a control group, are vital for developing therapies for rare diseases, which typically affect few patients.

According to Paolo Morgese, Europe Vice-President of Public Affairs at the Alliance for Regenerative Medicine (ARM), randomizing control trials is often too expensive and unethical for rare diseases.

Morgese emphasized that the new guidelines could hinder the EU’s ability to provide transformative therapies to patients quickly and may prevent access to these treatments in some member states.

The introduction of these guidelines comes amid growing interest in innovative cell and gene therapies, which have seen significant global growth.

The FT report, citing data from IQVIA, notes that by the end of 2023, 76 cell and gene therapies had been launched globally, double the number from a decade earlier.

ARM’s analysis revealed that about 80% of advanced therapies approved and available in the EU relied on single-arm studies, highlighting the importance of this trial method in the field.

While the EU Commission argues that the guidelines will enable quicker and more coordinated authorization of medicines across member states, critics believe the restrictions on single-arm trials could stifle innovation in rare disease treatment.

The Regulation on Health Technology Assessment, which applies to cancer and advanced therapeutics from January 12 and to all medicines from 2030, does not prohibit single-arm trials, but the industry is concerned about the stringent implementation guidelines.

Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.

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