As Europe revises substantially its pharmaceutical legislative framework for the first time in 20 years, it remains critical that the region has the right environment to bring the next generation of treatments to patients faster. Where innovation happens matters, and the updates to the General Pharmaceutical Legislation (GPL) present a once-in-a-generation opportunity to improve access to medicines and ensure the EU remains an attractive place to invest in pharmaceutical research and manufacturing.ⁱ As all stakeholders engage with the European Union Institutions on their proposals, we must reflect on what could be at stake for patients, the region and scientific progress for decades to come.

A Healthier Future Depends on Innovation

There is a growing recognition that investing in health and manufacturing delivers long-term benefits for patients, societies and economies. It is positive to see the European Commission propose ideas for streamlining the European Medicines Agency’s procedures, aiming to enhance predictability in regulatory processes for applicants. However, we recognize there is still more work to do on other aspects of the GPL that could have a negative impact.

For example, a recent study from Dolon, commissioned by EFPIA, shows that proposals to reduce regulatory data protection reduces the incentive for companies to invest in breakthrough medicines by 55% in Europe over the next 15 years.ⁱⁱ Moreover, it could further accelerate Europe’s decline in innovation compared to the USA, China and Japan. This could result inⁱⁱ:

• A one-third reduction in Europe’s share of global R&D investment by 2040, dropping from 32% to 21%.
• Loss of one in five medicines in development (22%) which would no longer be economically viable in Europe.
• A loss of 16 million years of life lost (YLL) due to increased mortality and premature death across the EU.

Unless changes are made, Europe will become reliant on other regions' medical innovation and supplies and patients will wait longer for the latest advances in treatments. Investing in health leads to people living healthier, longer lives and promotes overall economic prosperity. Therefore, doing so should be viewed as an investment to EU public health and EU citizens and not just a cost.

Rare Disease Therapies at Risk: Threats to a Proven Success Story

A clear example of a successful EU regulation is the Orphan Medicines Product Regulation from the early 2000s, which introduced regulatory and economic incentives targeting rare diseases. This has increased scientific activity and led to new innovation of treatments in the rare diseases space, including boosting the number of EU-approved orphan medicines from single digits to more than 230.ⁱⁱⁱ Estimates indicate that up to 74% of orphan medicines authorized in Europe between 2000-2017 were developed as a result of the regulation.^iv

However, the European Commission has proposed a provisional set of criteria to define high unmet medical need within rare diseases, in which some criteria are ambiguous, creating uncertainty for companies, like Amgen, when investing in innovative research in rare disease. It is estimated there would be a negative impact on the development of 45 rare disease products which may not be carried out elsewhere, corresponding to a 12% decrease in innovation. This could deprive around 1.5 million people living with a rare disease of a novel treatment option.^iv

Long-term European Strategies Should Encourage Investment

Small and medium enterprises (i.e., Europe’s biotech sector) would be the most impacted, due to the unpredictability and less favourable conditions for R&D created by the GPL proposals.ⁱⁱ Amgen’s history is an example of why a predictable and favorable framework is important. Initially beginning as a small start-up with a team of three and a good idea in 1980, Amgen has grown to be one of the world’s leading independent biotechnology companies and is developing a pipeline of medicines with breakaway potential. Amgen’s medicines have reached millions of patients around the world, and those currently available in Europe treat some of the region’s most serious diseases. The company’s current and future solutions are a result of an environment that favoured and supported innovation and investment in healthcare.

As Europe aims to deliver on the core purpose of the GPL, we encourage Members of the Parliament and Council to not undermine competitiveness and slow down research, development, manufacturing and delivery of new treatments for patients. Amgen remains committed to working with all stakeholders to champion pro-patient and pro-innovation policies that enables the EU to enhance its role as a key driver of medical innovation.

References

1. EFPIA (January 2022). Where innovation happens, matters. https://www.efpia.eu/news-events/the-efpia-view/blog-articles/where-innovation-happens-matters/. (Accessed November 2023)
2. Dolon (2023). Revision of the General Pharmaceutical Legislation: Impact Assessment of European Commission and EFPIA proposals. https://www.efpia.eu/media/msadqxbf/revision-of-the-general-pharmaceutical-legislation-gpl-impact-assessment.pdf. (Accessed November 2023)
3. European Medicines Agency. Orphan Medicines in the EU https://www.ema.europa.eu/en/documents/leaflet/leaflet-orphan-medicines-eu_en.pdf.(Accessed November 2023)
4. Dolon (2020). Estimated Impact of EU Orphan Regulations on Incentives for Innovation. Available at: https://dolon.com/dolon/wp-content/uploads/2020/10/Estimated-impact-of-EU-Orphan-Regulation-on-incentives-for-innovation.pdf.(Accessed November 2023)