I am a firm believer in celebrating Europe’s successes. By pausing to acknowledge examples of policymakers getting it right, we can do more of what works in the future.

Over the past two decades, an excellent example of how European policy and legislation can positively impact people’s health can be found in the field of rare diseases. The EU Orphan Medicinal Products (OMP) Regulation, passed in the year 2000, has changed the lives of people with a number of rare diseases by stimulating the development of new treatments for previously untreatable conditions. 

However, there are still many patients hoping that future innovation will deliver new therapies for their diseases.

Take a moment to recall just how challenging the rare disease landscape was 25 years ago. For the 6,000 rare diseases that have been identified, a total of eight medicines were available before the year 2000.

The prospects for developing a new product to address unmet need in rare diseases were not promising, due to scientific hurdles and market failures. After engaging with stakeholders to understand the major bottlenecks to progress in medicines development, the OMP Regulation was introduced. This marked a turning point in the history of medicines development in Europe.

Orphan designation offers several routes to support new medicines targeting a rare disease. These include scientific advice, reduced regulatory fees, incentives for SMEs, and 10 years of market exclusivity which can be extended by two years under certain circumstances.

It worked. Between the years 2000 and 2018, the Regulation resulted in more than 2,700 new orphan designations, which in turn led to more than 230 authorised new treatments for around 90 rare diseases.

Pharma package jeopardises progress

However, there is still an enormous unmet need in the rare diseases space. We need to accelerate progress in this area by learning from the success of the OMP Regulation. Unfortunately, there is a real risk of reversing our collective progress.

In April 2023, in the context of the revision of the entire legal framework for pharmaceuticals, the European Commission proposed a revision of the incentives framework for orphan medicines. To inform the debate on this complex topic, EFPIA commissioned a report by Dolon. The study[1] found that, in its current draft, the proposed changes would have a negative impact on the development of 45 products in Europe between 2020 and 2035. This would represent a 12% decrease in innovation, potentially depriving around 1.5 million European rare disease patients of a novel treatment option. It would also translate into a decrease of €4.5 billion in R&D spending in Europe. 

Incentivising innovation, improving access 

Looking back at the positive impact of the OMP Regulation, one thing is clear: policy makes a difference. The policy environment shapes future trends in innovation, with dramatic real-world impacts for millions of patients. Today, it is within the power of EU policymakers to build a revised legislative framework that will support a world class ecosystem in which innovative medicines are developed.  

We also know that industry cannot stand still. In partnership with seven stakeholders from across the rare disease communities, we are working to overcome some of the main stumbling blocks to discovering new therapies for rare diseases by addressing gaps in scientific knowledge to accelerate the development of new therapies via the Rare Disease Moonshot.

Of course, the impact of innovation depends on patients having access to that innovation. Data from EFPIA’s annual Patients W.A.I.T Indicator show that market authorisation and patient access to orphan medicines can vary from three months to 2.5 years, across the EU. EFPIA shares the EU Pharmaceutical Strategy’s ambition to address delays and disparities in the time it takes for new therapies to reach patients.  

Our aspiration is to achieve equal access on day one for all Europeans, as of when a new medicine gets the green light from the EMA.

So where do we start? An EFPIA Root Causes Analysis identified 10 factors that cause delays. These include slow value assessment processes, a lack of alignment on evidence requirements as well as health system constraints and resource allocation.  

There is no one-size-fits-all-solution and no one stakeholder that can fix these problems alone, but industry is already taking action. EFPIA member companies are committed to filing pricing and reimbursement applications in all EU countries within two years of EU market authorisation, and we have launched a portal to track that commitment. Companies are also willing to collaborate with Member States on novel payment and pricing models to support health systems to introduce innovative technologies. In addition, our industry is also proposing an approach for Europe where the countries that can afford less, pay less for medicines. This type of agreement would require solidarity among Member States to embed these ‘fair principles’ and prevent them from being undermined, for example, through international reference pricing or supply diversion – where a medicine sold more cheaply in one country is immediately exported to a country with higher prices. 

These proposals require new ways of thinking and political will. If we are serious about making a step-change for patients on medicines’ access, these conversations are the place to start. 

Building on success, together

The OMP Regulation is a success story. It delivered on its promise by recognising the specific challenges facing medical research in the areas of rare disease and devising concrete solutions that worked for stakeholders. We have come too far to jeopardise this success.

Now is the time to imagine if everyone living with a rare disease had a treatment within reach. It is time to strengthen not weaken the legislation. It is time to come together and get this right.

[1] https://www.efpia.eu/media/tigiq5g5/revision-of-the-orphan-regulation-estimated-impact-on-incentives-for-innovation-of-changes-proposed-by-the-european-commission.pdf