Are Solutions for Rare Diseases in Jeopardy?

This blog was originally published in French, in Les Echos newspaper.

How can we align the progress of medicine with the patients’ needs? How can we ensure that new life-saving medicines continue to be produced? How can Europe keep its status of a world-leading hub for medical research and innovation? How can we improve prevention? Should governments prioritise public health spending?   

These are the questions feeding the scientific debate at the EU level for the moment.

The European Commission is indeed engaged in reflections on whether to update the existing model for IP protection in pharmaceutical development, or to strengthen it, and safeguard Europe as a flagship for global medical research.

Such an important debate is in the interest of all of society. We need to see what works – and how we can protect it.

An age of medical progress, thriving like never before

Since the early 2000s we have witnessed extraordinary advances in medical research, especially in the field of rare diseases.

While there are still sadly plenty of conditions without treatments, there have been incredible progress in healthcare, leading to increases in life expectancy, quality of life and reduction of morbidity in almost all corners of the globe.

A large number of our member companies invest in medical research for rare diseases. At the EU level, it is estimated that 30 million persons suffer from a rare disease, and because most rare diseases have a genetic origin, 75% of them affect children.

It’s in this corner of medical research where the development made in the past decades is truly remarkable. Haemophilia is a telling example: children born today with this condition can expect to live long and healthy lives.

EUCOPE members focus on rare diseases

Medical research into rare diseases has always been slow and difficult. From fundraising to regulations, many hurdles were hampering the development of research in rare diseases and discouraging investors in supporting research entities.

And this was a fundamental problem. Before 2000, there were only 8 medicines authorised in Europe to treat rare diseases. A dreadfully low figure for about 5,000-8,000 rare diseases prevalent in Europe.

What made the difference – From 8 to 165

Fortunately, policy-makers around the globe acknowledged the need for change, and in 2000, the EU Orphan Regulation entered into force. A game-changer for medical research in Europe.

Thanks to a combination of scientific discoveries and the changes introduced by the Orphan Regulation, medical researchers in universities and hospitals have been able to set up companies in ways that weren’t been possible before. They finally had a system that allowed them to test their ideas and look for more solutions for patients while having financial backing for the first time.

Eighteen years after the Orphan Regulation, we can see the results. We finally have medicines for patients living with rare diseases. There were 165 medicines authorised in the EU by March 2019. Patients are now reaping the benefits of this regulation.

When an idea becomes a product

The affordability of and the access to the most innovative medicines remain a challenge for healthcare systems in Europe. We all know it. Member States’ budgets are under great pressure, with implications for healthcare budgets.

Medicines are only a part of the healthcare costs borne by governments, of which orphan medicines represent an extremely small fraction.[1]

Changing the incentives system will not solve the issue of affordability. We need to find new and creative solutions to ensure our healthcare systems are sustainable.[2]

Intellectual property protection allows for an idea to be first protected and then transformed into a pharmaceutical discovery. The time between obtaining intellectual property protection and the medicine’s approval and pricing is usually over a decade apart. If that part of the chain is weakened, the whole life cycle of medical research will be endangered. As a result, less research will be done and fewer medicines will be discovered.

Policy-making doesn’t always work the first time around, just like research and development.

In this case, it has worked; the European Orphan Regulation has produced amazing results for the health of people with a rare disease.

We need to remember that the incentives system has been the backbone of this booming medical progress. We need to think twice about the long-term consequences that reforming or even slightly changing the system might have on patients’ access to new medical solutions for rare diseases.

[1] In a 2011 budget impact study, orphan drugs accounted for only 3.3% of the share of EU’s total pharmaceutical budget in 2010. [Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2010-2020, 2011]

[2] An option could be to look for alternative payment models.

March 12, 2019

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