This article is part of our special report Pharma innovation.
Organisations of patients with rare diseases have warned EU policymakers to “think carefully” before reviewing the incentives in the orphan drugs regulation, claiming that the pharma industry should not be discouraged from investing in new therapies.
With national health budgets under pressure by austerity, health ministries are seeking ways to cut drug spending by focusing on industry incentives.
Pharmaceutical companies, for their part, cite OECD figures pointing that drug expenditure has remained stable at 15% over the past years. They also claim that cutting spending could pose a threat to innovation and that there are other ways of cutting costs in the healthcare sector.
In June 2016, EU health ministers called on the European Commission to perform an overview of the current EU legislative tools and incentives that aim to facilitate investment in the development of medicinal products.
The 2000 orphan drug regulation could be part of that review. The regulation includes special incentives (market exclusivity, regulatory data protection) for the industry and orphan drugs, which are used for rare diseases and are marketed at high prices.
The European Commission should consider a “revision of the regulatory framework on orphan medicinal products without discouraging the development of medicinal products needed for the treatment of rare diseases,” the EU ministers said in their 2016 conclusions.
However, the fine line between the need for pharma innovation, affordability and patients’ safety in practice proves hard to identify.
‘Exception becoming the rule’
Yannis Natsis, Policy Manager at the European Public Health Alliance (EPHA), said the problem with orphan drugs is the growing number of medicines obtaining the orphan drug designation.
“So, the exception seems to be becoming the rule,” Natsis told EURACTIV.com at the European Health Forum Gastein last week (4-6 October).
Another issue is that orphan drugs often come to the market with very limited and incomplete data. “They are supposed to be for very limited sub patients’ groups but they reach the blockbuster status very quickly,” Natsis said.
“This is why the companies see a business opportunity – every drug wants to be an orphan drug.”
Natsis admitted that the 2000 orphan drug regulation has encouraged many good drugs to be placed on the market. But he believes that incentives have been used more for profit maximisation rather than genuine innovation.
“We need to have an extensive review of the system. There is nothing wrong with looking back and learning from our mistakes.”
The European Medicines Agency (EMA) said policymakers should fully exploit the legal possibilities to reduce protection periods for orphan medicines that do not meet the criteria.
Orphan drugs enjoy a 10-year market exclusivity but according to the regulation, this period could be reduced to six years when there is sufficient evidence that the product is profitable at the end of the fifth year.
“The EMA and member states must send a clear signal to the market that ‘rare’ does not and should not mean ‘orphaned’. We need to uphold the spirit of the orphan legislation but not encourage the abuse of the incentives,” Natsis emphasised.
Orphan drug regulation is a success
Patients with rare diseases would be directly impacted by a review of the orphan drug regulation incentives considering that they are already faced with accessibility issues, delays, or absence of medicines.
Simone Boselli, Public Affairs Director of Rare Diseases Europe (EURORDIS), which represents patients with rare diseases, agrees with EMA’s proposal to eventually reduce protection periods for orphan medicines that do not meet the criteria over time.
But he insisted that, contrary to what many believe, the orphan status of a product is not easy to obtain in Europe, and is not meant to last forever.
From all orphan designation applications submitted to the EMA, only 72% receive a positive opinion for designation, he pointed out. And 27% more will be losing their orphan status at the time of receiving their marketing authorisation.
“This shows that the evaluation of the orphan status is strict and only for therapies addressing unmet medical needs or non-satisfactory treatments,” Boselli said.
However, EURORDIS is fully against a review of incentives in the orphan drugs regulation, a move supported by the European Public Health Alliance (EPHA), a grouping of health NGOs.
The patient organisation claims that the orphan regulation has succeeded in fulfilling its primary goal of attracting investment in the development of therapies for life-threatening or debilitating diseases for millions of people who have today either no treatment at all or no satisfactory treatment. This success should be attributed to the incentives granted by the regulation, they claim.
“To date, 143 products have been approved under the EU Orphan Drug Regulation since its adoption, 97 of which are still currently holding orphan status after their period of market exclusivity, hence open to head-to-head competition. This is a genuine success, and one that should be celebrated proudly by all,” Boselli told EURACTIV.
As far as the high prices argument is concerned, Boselli highlighted that amongst 70+ orphan medicines approved by EMA up to April 2014, 24% of them had an annual cost below €11,000, and only 18% of them an annual cost higher than €111,000.
“It is also fair to point out that most orphan drugs are to treat cancers and prices of orphan medicines in cancer are not more expensive on average than other new cancer therapies. Simply put, some innovations are expensive, but it is not just orphan products,” Boselli stressed.
Turning investors elsewhere
EURORDIS also believes that without the current incentives, investments that have been encouraged by the legislation will most likely be channelled to other segments of the economy offering higher returns, rather than health and pharmaceutical research for rare diseases.
“We should think carefully about the long-term consequences of criticising current incentives, which could very quickly create sufficient concern or unpredictability to deter investors, pharmaceutical and biotech companies from investing their resources into the research and discovery of new rare disease medicines,” Boselli said.
“In order to link innovative science with the economy and the real therapeutic added value with value for money, we need to not challenge the EU Orphan Drug Regulation,” Boselli concluded.
Nathalie Moll, the Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA), commented, "The orphan drug regulation is very clear on what can be designated as an orphan drug. There are very clear criteria based on prevalence. The rapidly advancing science is creating the capacity to more carefully target medicines to a cohort of patients where they are most effective."
"That’s a good thing for patients, clinicians and healthcare systems. Maybe it’s because I am a scientist but I am excited by the potential of more and more carefully targeted, even personalised medicines," she added.