MEP: A common EU tool to incentivise production and availability of orphan drugs

MEP Stelios Kympouropoulos: "“168 innovative drugs have been made available to patients with a rare disease improving the health and therefore the quality of life of thousands of people." [European Parliament]

This article is part of our special report Reviewing orphan drugs law: A tough equation.

When it comes to rare diseases and ‘orphan drugs’, technology is there to support an “objective” EU tool that could provide the pharma industry with the necessary incentives to research and produce orphan drugs and simultaneously avoid exhausting ailing health systems, MEP Stelios Kympouropoulos told EURACTIV in an interview.

This tool, according to Kympouropoulos, will not allow unnecessary price fluctuations and will ensure the best price for each member state separately in order for orphan drugs to be granted to patients at the right price for national health systems.

“This will maintain the incentive to continue production and research for pharmaceutical companies and on the other hand, will help health systems afford orphan drugs,” he said.

The Orphan Regulation was introduced in 2000 and its main objective was to address the challenge of regulating medicines that treat patients with rare diseases, i.e. those that affect fewer than five in 10,000 people.

All relevant stakeholders acknowledge the added value of the regulation when it comes to the production of new orphan drugs, but the European Commission now wants to re-visit parts of the legislation.

For the pharma industry, touching the incentives could harm innovation and limit much-needed access to therapies for patients.

EU member states, on the other hand, prioritise the sustainability of their healthcare systems and therefore seek the lowest price possible.

Commission’s ‘risky’ move to re-visit orphan drugs regulation

The European Commission is expected to present by the end of July an evaluation study on the pros and cons of the application of orphan and paediatric regulations, which deal with a niche segment of rare diseases that affect fewer than five in 10,000 people.

The regulation has worked

An evaluation study on the pros and cons regulation is due at the end of July.

An EU official recently told EURACTIV that orphan drugs have cost EU health systems an additional €20-25 billion between 2000-2017 but at the same time, between 210,000-440,000 ‘quality-adjusted life years’ were gained to the benefit of patients.

The term quality-adjusted life years expresses the additional number of years which a person lives as a result of receiving a treatment, taking into account the quality of life in those years.

Kympouropoulos said the regulation has brought remarkable results so far.

“168 innovative drugs have been made available to patients with a rare disease, improving the health and therefore the quality of life of thousands of people,” the European People’s Party (EPP) MEP said.

“Businesses, on the other hand, seek legal security and stability to make financial investments, and this Regulation offers this, bringing many drugs to our domestic market. And that’s the bottom line, existing treatments to be available to everyone.”

However, he said, improvements are needed, especially in terms of European funding and opportunities given to start-ups, small and established companies to build their know-how on the development and distribution of new drugs.

“In order to develop innovation and maintain it within the EU, economic incentives should be given to researchers, but also at the European Commission level, research should be a priority for future goals,” the MEP said.

“At universities, working with research centres, civil society and businesses can help shape a curriculum that is up-to-date and future-oriented,” he emphasised.

EU orphan drugs law and the emergence of SMEs

The legal framework provided by the current EU orphan drugs regulation has encouraged a number of small and medium sized companies (SMEs) to develop innovative technologies in the fight against rare diseases in Europe, stakeholders told EURACTIV.com.

Lessons learnt from COVID-19

Referring to the lessons learnt from the ongoing COVID-19 pandemic, the Greek MEP said there is enormous room for improving European coordination.

“Europe’s main goal is to preserve health for all. As an MEP and doctor, I am proud to serve this purpose,” he said, adding that investment in research and a unified way of distributing medicines to all member states is an area that the EU needs to target even more.

He emphasised that policies, parties and different working committees intersect at some point.

“The European Union has civil society at its core and to maintain this heritage, it must offer prosperity. One way to do this is through medication for all, even with Orphan Medicines,” he concluded.

[Edited by Zoran Radosavljevic]

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