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

The debate over the issue is expected to heat up in the coming months especially in light of the new context of the pandemic and the industry’s efforts to come up with a vaccine against COVID-19 in record time. [Shutterstock/unoL]

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

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 Orphan Regulation was introduced in 2000 and its main objective was to address the challenge of treating patients with rare diseases. The number of people who suffer from such diseases in the EU is around 246,000.

“Most patients suffer from even rarer diseases affecting one person in 100,000 or more,” the executive says.

The pharma industry has been reluctant to invest in medicines and treatments for patients with rare diseases because of the low commercial interest due to a comparatively small size of the market.

The Orphan Regulation introduced some incentives for the pharma industry to invest in these areas, which has brought so far, some positive results. The number of orphan medicines went from only eight products to more than 160 today while clinical trials increased by 88% between 2006-2016.

But now the Commission wants to re-open the legislation and make “corrections”, which critics suggest may prove to be risky for both the pharma industry and patients.

The equation is tough to solve. Patients with rare diseases obviously want greater access to drugs, the pharma industry pushes for more incentives to invest in areas of unmet medical needs while governments want to save as much money as possible.

Sources have told EURACTIV that a legislative proposal to amend these regulations is due in late 2021 or early 2022. “The impact assessment of the legislative proposal has already started in the Commission,” the sources said.

Opening Pandora’s Box?

An EU official told EURACTIV that the executive acknowledges a positive impact on the development of drugs for patients with rare diseases, including through financial incentives.

But the official pointed out there is still room for improvement, especially when it comes to the incentives, to enhance research on the 95% of rare diseases which still have no treatment options.

The official explained that the market situation cannot compare to 20 years ago, when the regulation took effect, but said that in some cases the ten-year market exclusivity may not be the most appropriate way to stimulate the development of new treatments for rare diseases.

Critics suggest, though, that shortening the timeframe of market exclusivity period has low added value in practice.

The reason is that the generics industry does not step in to produce generic orphan drugs because of their small market share and low profitability prospect.

The official also estimated the healthcare cost related to orphan medicines to have risen by €20-25 billion since 2000. But at the same time, this has resulted in significantly better health outcomes, meaning a better quality of life for patients.

Industry: the regulation has brought results

For the pharma industry, the regulation has so far brought tangible results for patients and any attempt to change the incentives’ regulatory framework could produce unwanted counter-effects.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) says all healthcare stakeholders should work closely to ensure patients’ access to treatments.

“In addition, we need to further incentivise the research and development of new treatments for patients where, currently, no treatment options exist,” EFPIA said.

“EFPIA calls on European institutions to ensure a stable and predictable regulatory and incentives’ framework. This will support further research and development into much needed new treatments for rare diseases.”

According to the industry, 24 European Reference Networks have been established by 2018, while 23 European countries had developed national rare disease plans.

Another critical argument is that the orphan regulation has helped create 220 small- and medium-sized enterprises, which are responsible for the development of 51% of orphan medicines in Europe.

The debate over the issue is expected to heat up in the coming months, especially in light of the coronavirus pandemic and the industry’s efforts to come up with a vaccine against COVID-19 in record time.

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