New legislation designed to improve public health and protect patients has unforeseen implementation effects that are delaying clinical trials and blocking access to new treatments for conditions like cancer and rare diseases. The number of clinical trials delayed is expected to run into the hundreds, affecting up to 42,200 patients over the next three years, according to a survey run by EFPIA. The legislation is also likely to result in delays to therapies reaching patients in Europe. EFPIA is urging all partners to enter talks to find solutions and mitigate the negative unintended impact of the legislation.

The In Vitro Diagnostic Regulation (IVDR) came into effect in 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. However, the implementation of the regulation has been challenged by a lack of infrastructure, guidance, and coordination, triggering a series of unintended consequences.  

After hearing concerns from numerous companies, EFPIA surveyed its membership to collect data about the impact. The survey showed: 

• Between 82 and 160 trials* are currently being delayed in Europe, with an expected 238 to 420 trials* to be delayed over the next 3 years. 
• These delays mean that between 33,815 to 42,200 patients* in Europe are expected to have delayed access to clinical trials over the next 3 years, around half of them (up to 27,400) being cancer patients. 
• The launch of 89 therapies could be delayed because of this legislation, in innovative therapeutic areas such as oncology and rare diseases.  
• 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. 
• 43 percent of companies surveyed said they expect delays of 6 to 12 months to current clinical trials, with 48 percent expecting 6 to 12 month delays over the next three years. 
• 67 percent of companies would consider reducing the number of EU trial sites if IVDR requirements remain the same, noting these trials would move to the US, Canada, UK, and Asia, among other locations.

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. 

The launch of these novel therapies to the European market is also likely to be delayed, many of which are for patients with life-threatening diseases and those with unmet medical needs, where no treatment is available.

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,
• organising voluntary coordination processes at Member States level to improve the assessment procedure, 
• developing new guidance clarifying the assessment process, 
• considering a risk-based approach to avoid assessing IVDs that are low risk for patients, 
• accepting on a case-by case basis, and with agreement of Member States involved, to not conform with IVDR requirements if certain conditions are fulfilled, 
• clarifying the scope of in-house testing to broaden it.

Speaking about the concerning situation, Nathalie Moll, Director General of EFPIA, said: 

“These figures are extremely worrying for patients with rare and life-threatening conditions. 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. 

As the European Commission reviews the pharmaceutical legislation, this is real time example of how a lack of forward thinking in Europe could penalise patients and drive research out of the region. There is a serious risk that a number of companies could have to omit European sites from trials and divert the research to the US and Asia while these challenges are being resolved – meaning much later access to treatments for Europeans. Our Members are telling us that this is already happening. We have to act now to keep clinical trials in Europe and the benefits that they bring to European patients.”

Ends

* Range of numerical responses provided by respondents