EU Orphan Law A 'Remarkable Success' But Approval Speed and Patient Access Still Poor
This article was originally published in SRA
While the 15-year old EU orphan drugs legislation has "significantly helped to accelerate the translation of science into therapies for patients with rare diseases", orphan drugs are still not being approved - or reaching the patients who need them- fast enough.
Almost 120 orphan drugs have been approved under Orphan Drug Regulation No 141/2000 compared with a mere eight before the regulation came into force, notes Jean-Louis Roux, public affairs director of rare disease organization EURORDIS.
But "despite so much progress in so few years, advances in medical research still fail today to be translated with sufficient speed into approved therapies that effectively reach, at the end of the line, the patients who need them most urgently," Roux told Scrip Regulatory Affairs.
The EU should do more to complement and strengthen the existing legislation with regard to improving patient access, he says.
Roux was speaking in response to a recent report issued by the European Commission on the "state of play" of the orphan drugs regulation1. The report revealed that that between 2000 and September 2015, 117 orphan drugs were approved under the regulation and orphan designations were granted to 1,544 products.
Roux says the regulation "was a historic breakthrough at the time of its adoption and remains, more than 15 years later, a remarkable health policy success"
But EURORDIS still has concerns over the eight to 14 years of research and studies that are generally needed before a positive or negative decision on authorization and/or reimbursement of an orphan medicinal product and the problems of patient access.
"Even after authorisation and reimbursement are granted, about 1/3 of European patients living with a rare disease still have no access whatsoever to the orphan medicinal products they require for their condition," Roux told SRA. "Another 1/3 may obtain access but only after further substantial delays of several more years (much later than foreseen by the EU Transparency Directive)as a given product may end up being introduced first in major EU member states and only later, years after authorisation, in other, smaller EU member states."
"Most recently," he says, "the availability of orphan medicinal products of major importance has been restricted by EU member states solely due to cost and budget considerations."
"The reality is that the aspirations and potentialities of the EU orphan drugs legislation "remain far from being fully achieved, particularly when it comes to patient access to approved orphan medicinal products," Roux told SRA.
"While we recognise that this aspect extends well beyond the scope of the EU Orphan Drug Regulation, EURORDIS believes that the issue of patient access should nevertheless be subject to greater attention and more resolute action on the part of the EU to complement and strengthen the existing legislation," he said.
According to the commission's report, most of the orphan drug designations awarded between 2000 and September 2015, were granted on the basis of "prevalence criteria," ie the condition affected no more than 5 in 10,000 people in the EU. In one case only, the designation was granted on the grounds that marketing the product would result in insufficient return on investment.
Designated products benefit from incentives, such as protocol assistance and fee waivers, and can also more easily attract public or private funding. Since the regulation came into force, 951 protocol assistance procedures (of which 264 involved SMEs) have been completed and fee reductions totaling €78.4 million have been granted, the report says.
Of the 117 orphan drugs that received approval between 2000 and 2015, most pertained to treatments for pulmonary arterial hypertension, acute myeloid leukemia, cystic fibrosis, multiple myeloma and acute or chronic lymphoblastic leukemia. Sponsors of authorized orphan drugs benefit from a 10-year market exclusivity period, which may be extended for another two years for medicines that also have complied with an agreed pediatric investigation plan (PIP).
The commission's report also contains an inventory of incentives being offered by EU member states at the national level to support research and development of orphan drugs.
Roux says the Orphan Drug Regulation is "a success of the EU action in support of entrepreneurship and industry, as the legislation has encouraged the unprecedented development of innovative pharmaceutical and biotechnology undertakings, as well as the creation of start-ups and jobs." He says the regulation "continues to represent today a strong and distinct competitive advantage of the EU on the global marketplace, in support of the EU’s current strategic focus on growth, innovation and jobs."
References
1. Commission Staff Working Document: Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan mecidinal products — state of play 2015, Posted online February 2016, http://ec.europa.eu/health/files/orphanmp/doc/orphan_inv_cwd_20160126.pdf