EU to tighten up on pharmaceutical research

The new rules would put less emphasis on so-called single-arm trials, where all participants receive the same treatment. In contrast, randomised control trials, which are the most rigorous method of clinical research, compare new treatments with the current standard treatment or placebo, and participants do not know which one they are receiving.

The new EU guidelines do not rule out single-arm tests, leaving it up to each Member State to decide. The Health Technology Assessment Regulation will apply to cancer medicines and advanced therapy products from 12 January and to all medicines from 2030.

According to objectors, this will make it more difficult to get rare disease drugs approved, as expensive mass trials will be prioritised.

According to the Alliance for Regenerative Medicine (ARM), an advocacy organisation for the cell and gene therapy industry, randomised control trials, which often require a group of patients to receive a sham therapy, are too expensive and unethical because they give a person with such a serious disease an ineffective placebo.

The guidelines were introduced in response to the growing interest in innovative but expensive new cell and gene therapies for cancer and rare diseases. According to market researcher IQVIA, 76 cell and gene therapies will have been launched worldwide by the end of 2023, double the number a decade earlier. According to ARM's analysis, around 80% of the advanced therapies licensed and available in the EU use single-arm trials that do not include a control group.

The researchers discussed the difficulty of randomized control trials for rare diseases, citing Wiskott-Aldrich syndrome as an example, which affects an average of only four out of 1 million men, and feared that if single-arm trials risked being "inconclusive", countries would no longer send patients for testing.

The researchers add that the direction of regulatory change is positive, but the implementing directives are too restrictive. Many pharmaceutical companies have stopped developing treatments for rare diseases because they are too expensive and the payback is difficult for a small number of patients.

Cover photo (for illustration purposes only): Portfolio