Patient and Industry Groups Warn New EU Rules Will Reduce New Rare Disease Treatments

Rare Daily Staff

New guidelines set to take effect in January in the European Union threaten to reduce the number of new treatments for rare diseases, according to industry and patient groups that oppose the measures.

A report in the Financial Times said nearly 40 patient and industry organizations are seeking revisions to the new rules, which place less weight on single arm trials that do not use a placebo-control. They argue these new guidelines would make it harder to win approval for new therapies to treat rare diseases with small patient populations.

The Alliance for Regenerative Medicine, a cell and gene therapy trade group, said randomized control trials were too expensive and unethical because giving an ineffective placebo to a person with a severe illness would cause unnecessary suffering, the FT report said.

“In rare diseases, single-arm trials are often necessary for ethical, practical, and scientific reasons,” said Paolo Morgese, ARM’s Europe vice-president of public affairs, told FT. “The joint clinical assessment guidelines largely ignore this reality and miss a huge opportunity to build a modern EU system. Without changes, the current approach will undermine the EU’s goal to get transformative therapies to patients faster and could prevent access in many EU member states.”

The new guidelines come as a growing number of cell and gene therapies are moving through the pipeline. Though randomized, placebo-controlled studies are considered the gold standard for clinical trials, a study by the Alliance for Regenerative Medicine found that 80 percent of approved advanced therapies in the European Union won approval on the basis of a single-arm study.