Biomedical R&D advances in the aftermath of the pandemic: the pharmaceutical industry has 8,000 drugs under investigation, 10% of them for rare diseases

Oncology, infectious diseases, neurology, haematology and endocrinology are the areas with the highest number of clinical trials with new treatments underway.

Seven out of ten molecules in clinical trials worldwide are first-in-class, i.e. break new ground.

The European Federation of the Pharmaceutical Industry warns that Europe’s share of biomedical R&D is declining and already lags behind the US and Asia.


Biomedical innovation and the commitment of pharmaceutical companies to research into new medicines continues after the historic effort made in the fight against the Covid-19 pandemic. This is shown in the latest EFPIA Pipeline Innovation Review, conducted by the consultancy Iqvia for the European Federation of Pharmaceutical Industries (Efpia). The study shows that there are currently more than 8,000 drugs under investigation, 10% of which are specific to rare or rare diseases. Of these molecules that pharmaceutical companies have in clinical development around the world, almost 70% are first-in-class, i.e. they break new ground and are the best proof of this sector’s commitment to innovation and people’s health.

Specifically, the report indicates that a total of 6,835 clinical trials were launched worldwide in 2021, a historic figure that is almost 20% more than the number of trials in 2020 (5,537) and more than 50% higher than the clinical trials of just four years earlier, in 2017, when 4,457 trials were initiated worldwide. This year, 2022, the number of clinical trials continues to rise, with a total of 3,520 trials having been initiated as of August.

“These figures are a clear indicator of a pharmaceutical industry at the cutting edge of science, an industry that continues to invest heavily in finding new treatments for hundreds of medical conditions. Behind every piece of data in the report is a story of hope for patients, for their families and for medical professionals,” says Nathalie Moll, Efpia’s CEO.

In the last five years – from 2017 to 2022 – five therapeutic areas have been the focus of much of the research into new medicines. These are oncology (24% of all trials); infectious diseases (12% of the total), neurology (10%), haematology (8%) and endocrinology (6%). In addition, the report highlights the strong commitment that the pharmaceutical industry has been making in recent years to research into new drugs for rare diseases. Thus, over the last decade (see graph), there has been a steady increase in the number of clinical trials for these pathologies and they now represent between 10% and 12% of the total.

Europe is losing pace in biomedical R&D

The Iqvia report also focuses on the place of origin of research. This confirms the decline in biomedical R&D in Europe compared to the United States and the new emerging countries. The latest data show that, of the total number of clinical trials initiated in 2021, up to 30% were launched in Asian countries, while the United States was the second region with 27% and Europe is already in third place with 23% of the total number of trials. The long-term trend observed is the increase in the proportion of clinical trials conducted in Asia (mainly China and South Korea), which has grown by more than 10% in the last decade, the report says.

It should be recalled that in the early 1990s Europe was the leading region – ahead of the US and Japan – in R&D investment in new medicines. “While our industry invests around €41.5 billion a year in research and development in Europe, with a higher percentage of revenues devoted to R&D than any other research-based sector, global investment in biomedical research has increasingly shifted elsewhere and Europe is facing growing competition from emerging countries, such as Brazil, China, Korea and India, which are increasing their investment in biomedical research,” says Moll, who points to the future European Pharmaceutical Strategy as an opportunity to change course.

Some important steps have already been taken. Thus, with the aim of strengthening the European framework for clinical trials, while maintaining a high level of participant protection, data robustness and transparency, the ACT EU, or Accelerating Clinical Trials in the EU, initiative was launched in January 2022. This project, led by the European Commission, the European Medicines Agency (EMA) and the Network of Heads of Medicines Agencies (HMA), includes the active participation of the Spanish Agency for Medicines and Health Products (AEMPS) in its work plan to transform the way clinical trials are initiated, designed and developed.

However, as Efpia’s director general points out, “what matters now are the rules and policies that encourage investment, such as a robust and predictable IPR ecosystem and a state-of-the-art regulatory framework,” she argues. If we are not able to implement them in Europe, the gap between us and the rest of the world will continue to widen and Europe will miss its chance to become a leader in health and life sciences.

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