BRUSSELS, Belgium - In a bid to catch up with the U.S., Japan and Australia, the European Union (EU) will look again at new rules to promote orphan drugs - medicines neglected because of limited commercial potential.
“Most applications for the designation of orphan medicinal products are filed by small firms specializing in biotechnology and genetic engineering, since the vast majority of rare diseases are developmental disorders of a genetic or other kind,“ said the European Commission (EC) in a preliminary draft, which draws heavily on U.S. experience.
The head of the EC's pharmaceuticals unit, Patrick Deboyser, said the new legislation will help authorize human-use agents for the diagnosis, prevention and treatment of conditions affecting five in 10,000 members of the population or less, or that are life-threatening or seriously debilitating and unlikely to generate a commercial return.
Decisions on whether applications for help would qualify could fall to the new Committee on Orphan Medicinal Products, made up of representatives of the member states, patient groups and the Committee for Proprietary Medicinal Products - the EU's senior drug science committee.
The aim would be a speedy and flexible system in which the European Medicines Evaluation Agency (EMEA), in London, would validate applications, and a decision would emerge in 90 days.
The incentives for companies to develop orphan drugs in this latest draft (the seventh version the EC has developed internally over the last three years) would include access to the centralized registration procedure at the EMEA - including assistance in developing a protocol and in carrying out or following up clinical trials - and a waiver of its $100,000 authorization fees.
The proposal anticipates five such applications in 2000, eight in 2001, 12 in 2002 - generating EU costs of around $1 million in 2001 (half for fee exemptions, and half for meeting costs and staff time).
The incentives would also include a degree of market exclusivity for 10 years. However, this would be subject to review after six years if member states felt the price being charged was too high, supplies were insufficient or a better product emerged.
There is a growing European consensus on the principle that patients with rare disorders are entitled to treatments of the same quality, safety and efficacy as other patients, and if the market does not provide such treatments, it is the role of society to take appropriate steps, said the EC.
At a meeting May 27 the European Parliament's (EP) special all-party group on pharmaceuticals was highly critical of the lack of progress to date. The Italian Euro-MP, who is preparing a report on the subject for the EP, won applause for a fierce attack on EU delays and for his querying as to “whether there was any European Commission intention of making a serious proposal“ to back research on rare diseases.
The EU's research program now offers some aid to fundamental, clinical and epidemiological research on rare diseases. And an EU program for public health action on rare diseases for 1999 to 2003 has just been proposed by the EC. But even the EC accepts that national initiatives in the EU member states aimed at backing “uneconomic“ drugs have led to no significant progress in research on rare diseases.
Drug Industry Wants Financial Incentives
The European drug industry has backed the initiative, but its main European lobby group, the European Federation of Pharmaceutical Industries and Associations (EFPIA), said the reason there isn't greater research into orphan diseases is that “research is extremely difficult and risky - with chances of success very limited.“
Neither public institutions carrying out fundamental research nor private industries (especially the pharmaceutical industry) which conduct specific research into treatments can “justify the investment in resources in the absence of foreseeable returns.“
What is needed, the EFPIA said, is “recognition that intensive and expensive R&D by part of the pharmaceutical industry is needed to address orphan diseases - that this is risky in terms of results, profits and legal responsibilities.“
It called particularly for “financial, fiscal, scientific and regulatory incentives to industrial R&D in orphan medicines.“
Meanwhile, the European drug industry is still awaiting progress on the EU's proposed clinical trials directive, which would bring some standardization into national approval procedures. The EP is due to draw up a report on the EC's 1997 proposal, but plans to hold discussions in June have been discarded.
The report draftsman, Euro-MP Amadeo Amedeo, still has not produced a preliminary working paper. A staffer on the EP's public health committee, Rosemary Opacic, told BioWorld International in late May there was little prospect of a draft report appearing in the coming weeks, since “Amedeo is waiting for discussions in the Council of Ministers to advance further.“ *