RARE Daily

European Pharma Organization Warns New Legislation Slowing Rare Disease and Other Clinical Trials

March 20, 2023

Rare Daily Staff

New European legislation designed to protect patients is delaying clinical trials for thousands of people with cancer and rare diseases, according the European Federation of Pharmaceutical Industries and Associations.

The industry organization said that the legislation will affect hundreds of clinical trials and as many as 42,000 patients over the next three years based on responses to an EFPIA survey.

At question is the In Vitro Diagnostic Regulation (IVDR), which took effect May 2022. In vitro diagnostics (IVDs) are tests done on samples such as blood or tissue taken from the human body. They can detect disease or other conditions, and they can be used to monitor a person’s overall health to help cure, treat or prevent diseases. The regulation aims to ensure patients’ safety, provide a more transparent framework for IVDs, and deliver access to innovative medical technologies, aims that EFPIA fully supports.

The organization said the implementation of the new regulation has been challenged by a lack of infrastructure, guidance, and coordination, triggering a series of unintended consequences. While EFPIA said it was intended to improve public health and protect patients, it is causing delays to clinical trials and blocking access to new treatments for conditions like cancer and rare diseases.

The EFPIA is urging all of its partners to enter talks to find solutions and mitigate the negative impacts of the legislation.

An EFPIA survey of its membership found as many as 160 trials are currently being delayed in Europe, up to 420 trials could be delayed during the next three years. The launch of as many as 89 therapies could be delayed as a result.

Up to 400 trials are expected to enroll fewer patients, meaning some people missing out on innovative new treatments. These include trials for cancer, rare disease, neuroscience, inflammation, cell and gene therapies, pediatrics, and cardiovascular diseases, the organization said.

Compliance with the IVDR means IVDs used in clinical trials go through an assessment process when the diagnostic test result influences patient medical management. However, this process is currently complex and uncoordinated. It results in patients waiting longer to participate in clinical trials, or even not participating at all.

EFPIA is currently working on complementary policy recommendations that would alleviate some of the barriers created by the implementation of the IVDR and improve access to clinical trials for European patients. These recommendations are looking at delaying the application of the IVDR for clinical trials using an IVD, organizing voluntary coordination processes at Member States level to improve the assessment procedure, and developing new guidance clarifying the assessment process as well as other steps.

“These figures are extremely worrying for patients with rare and life-threatening conditions,” said Nathalie Moll, director general of EFPIA “These patients should be at the front of the queue, getting fast access to care, not having to wait due to complicated bureaucracy and a lack of coordination across Europe.”

Photo: Nathalie Moll, director general of EFPIA 

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