Orphan Regulation: Time for a rethink?

The upcoming review is an opportunity to not only accelerate the development of rare disease treatments, but also to provide equitable access for patients across Europe, says MEP Nicolás González Casares, S&D group shadow rapporteur on the EU’s Beating Cancer Plan report.
Source: European Parliament audiovisual

By Nicolás González Casares

Nicolás González Casares (ES, S&D) is a member of Parliament’s Industry, Research and Energy Committee

19 May 2022

Two decades ago, Europe was leading pharmaceutical innovation worldwide. However, between 2014 and 2018, just 22% of new drugs were of European origin, while 48% were developed in the United States.

Research and innovation into health and wellbeing are essential for society, but it is a complex sector with great risk for investment. Development of a new drug usually takes 10 years and €2.5bn of investment, with less than 10% of the molecules finally reaching the market.

On the other side, thousands of patients await the approval of new therapies for yet untreatable diseases that, in many cases, do not have the same accessibility in all countries or regions after their marketing. This scenario requires a regulation that successfully finds the balance between accessibility to medicines, financial sustainability, and innovation.

In this sense, this is a decisive moment. After 20 years, the European Union is going to review its pharmaceutical legislation and, following the lessons learned during the pandemic, re-evaluate key aspects such as incentives for the pharmaceutical industry, efficiency of the regulatory systems or the inclusion of new technologies in the evaluation processes.

“The time has come to assess the suitability of selective rapid approval pathways, which eliminate unnecessary bureaucracy and give regulatory agencies a proactive role in guiding new products into clinical practice”

Special attention should be given to orphan medicines, where specific legislation governing their research, development and marketing will also be reviewed. The previous legislation, which has been in place for two decades, was effective in many respects.

Since its approval, 142 orphan drugs have reached the market, with an estimated 18-24 drugs directly derived from this legislation, and the process of research and development has been significantly accelerated. Strategic incentives made it possible to redirect investment to neglected areas, but it has also entailed a great cost for the EU, through reimbursement and public investment, and it did not manage to reach certain pathologies in which the development of new drugs is especially necessary.

A recent analysis by the European Commission highlighted some aspects that could be improved in order to develop a more efficient and beneficial system of incentives for all parties.

However, the pandemic showed us to what extent the evaluation of the safety and efficacy of medicines can be accelerated, without compromising their quality. Therefore the time has come to assess the suitability of selective rapid approval pathways, which eliminate unnecessary bureaucracy and give regulatory agencies a proactive role in guiding new products into clinical practice. The inclusion of new technologies, such as the use of real-world data or artificial intelligence, will be key to optimising these processes.

In the coming months, all the stakeholders involved, namely the pharmaceutical industry, regulators, clinicians, researchers and, of course, patients need to sit down at the table and discuss all these aspects to find, ultimately, the best way to drive innovation in Europe and ensure a quick and equitable access to medicines.


This content was commissioned by AbbVie and produced by Dods Impact

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