The European Commission on Tuesday published the results of a yearslong evaluation of the EU’s orphan and pediatric medicines regulations it says will be used to guide future legislative changes and shape the EU pharmaceutical strategy.
The more than 100-page evaluation reviews the positive impacts and shortcomings of the orphan regulation, Regulation (EC) No 141/2000, and the pediatric regulation, Regulation (EC) No 1901/2006, based on external studies and various consultations with stakeholders.
“The evaluation found that both regulations have fostered the development and availability of medicines for patients with rare diseases and for children,” the Commission writes. “Nevertheless, both regulations have not adequately managed to support the development in areas where the need for medicines is greatest. Products tend to be developed in certain more profitable therapeutic areas for which the number of available treatments is increasing.”
According to the report, 142 orphan medicines have been authorized in the EU since 2000, 131 of which are still on the market. While the Commission notes that the orphan regulation alone did not spur the development of most of those products, it estimates that 18-24 orphan medicines were developed as a result of the regulation and credits it with speeding access to patients by nine months on average.
For the most part, the Commission concludes that the orphan regulation has had a positive impact by adding 210,000-440,000 quality-adjusted life years for patients in the EU despite increasing costs by €23 billion from 2000-2017.
Nearly three quarters (73%) of orphan medicines had annual sales of less than €50 million in the European Economic Area, while only 14% had annual sales over €100 million. The report found that orphan medicines on average received an additional 3.4 years of market exclusivity, which equates to about 30% of sales revenue for those products.
While some sponsors may have been “overcompensated,” the Commission says that the additional market exclusivity “has helped to increase profitability, without giving the sponsor an unbalanced compensation” in most cases.
The evaluation also looks at whether the current threshold of affecting fewer than 5 in 10,000 patients in the EU “is the right tool” for defining rare diseases.
“The evaluation results raise the issue of whether the current prevalence criterion (on its own) is still an appropriate way to define a rare disease, whether a different method for calculating prevalence is needed, or whether a different criterion should be applied,” the Commission writes. “Advances in science, such as personalised medicine and the use of biomarkers could add another layer of complexity to the current regulatory framework. While such developments may hold great potential for optimal tailoring of treatments to diseases, they should not lead to unnecessary multiplications of rare diseases out of common diseases, either proliferation of exclusivity periods.”
The report also raises the failure of one of the pathways for obtaining orphan designation to gain traction. While orphan medicines are typically thought of as those intended to treat rare diseases, under the regulation sponsors can also obtain orphan designation for medicines for life-threatening or serious conditions that are unlikely to make a sufficient return on investment.
However, the Commission notes that, “This possibility was difficult to implement and has never been used.”
As for the pediatric regulation, the Commission finds that it increased the amount of clinical trials that included children by nearly 50% and resulted in more than 1,000 pediatric investigation plans (PIPs) but has not addressed “the greatest therapeutic needs of children” as the regulation does not have any provisions to direct development.
Likewise, the Commission found that supplementary protection certificates (SPCs) awarded for the completion of pediatric studies according to a PIP, “[have] not shown to be effective in stimulating the development of medicines whose development for adults is not attractive.”
The Commission also acknowledges that both regulations have led to increased costs for healthcare systems, but that the benefits to children and patients with rare diseases, “Outweigh the costs imposed on both industry and society.”
Additionally, the Commission found inefficiencies in processes related to both orphan and pediatric medicines. “There may be room for simplification and streamlining of internal processes including different scientific committees within the European Medicines Agency to avoid the risk of inconsistencies and delays,” the Commission writes, noting that some administrative burdens, such as annual reports on orphan designations, should be reconsidered.
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Assessment Report