The Fight for Orphan Drug Research Reform in Europe

For anyone who thought the battle over reforming the European Union rules on stimulating drug research was nearing a conclusion, think again. Last summer, this column highlighted the official position on the legal frameworks for incentivizing the development of medicines for children and rare diseases: after some years of public discussion and internal reflection, EU officials had concluded that the system had some merits but needed fine-tuning. Now the EU’s most recent step toward that fine-tuning exercise has, it appears, reignited the entire debate with renewed intensity among some of the protagonists.

On the one side remains the drug industry, urging retention of the scheme with improvements. On the other side are a range of critics who broadly distrust the drug industry, are skeptical of the value of intellectual property protection, and want to see the scheme drastically limited and reduced in scope.

Over recent weeks, more than 100 opinions have been submitted to the EU by pharmaceutical companies, industry associations, national authorities, patient organizations, and citizen groups, counseling the EU as to how it should now move ahead. In reply to a public consultation on a possible EU regulation, the European Alliance for Responsible R&D and Affordable Medicines, for instance, warns bluntly that “enforcing intellectual property rights to control knowledge and access can be a key barrier to the progress of research itself, large-scale production of affordable health technologies, and equitable access.” At the same time, the major European drug industry association, EFPIA, insists the discussion “fails to understand the science behind biopharmaceutical research.”

An influential citizen group, Health Action International, demands that the reform becomes a “fight against the misuse and abuse of incentives, which is allowing some pharmaceutical companies to exact exorbitant prices while benefitting from the exclusivities” allowed by the current legislation. The leading public health lobby in Brussels, the European Public Health Alliance, alleges that patients are “held hostage of companies’ business and marketing strategies while companies benefit from the incentives generously granted by EU legislators.” And reimbursement agency, the Swedish TLV, weighed in with its view that “current systems for orphan drugs have been to at least some extent misused.”

Many critics of the industry continue to lament the lack of access to innovation, which they see as a consequence of the current scheme. “The regulation should tackle threats to affordability and access, such as incentives leading to overcompensation, indication stacking, and high drug prices even when the R&D investment of a company was very low,” says the Association of European Cancer Leagues. It adds that the problem is all the greater because of uneven healthcare opportunities across Europe’s patchwork quilt of national systems: “Children and patients with rare diseases must have equitable access to affordable treatment. It should not matter where you live.”

Industry associations and individual companies have lined up to defend the principle of incentives to remedy the market failure that leaves rare diseases still without adequate treatment, and to issue their own warning of the consequences of imprudent “curtailing the established systems of available incentives,” as the German BPI association puts it. That will not solve the issues of access and availability, it says, and runs the risk of hampering innovation: “Without incentives, companies are unlikely to take the necessary steps to develop and file for marketing authorizations.”

Pfizer finds “striking incongruity” in the EU’s recognition of the merits of the incentives and the policy options now being entertained, which would “remove many of the incentives and rewards for the important and difficult task of developing medicines for very small patient populations and conditions.” Sanofi says it is “deeply concerned about potential changes.” Pharmig, an Austrian drug industry association, perceives risks in “focusing to reduce important incentives, which will have a negative impact for many patients.” Merck KGaA is “strongly concerned that most of the options being explored would disincentivize innovation in areas of unmet needs where significant R&D efforts are still needed.” The US PhRMA has also entered the fray, anxious that some of the options under discussion “would limit the very incentives that have proven to be effective to date,” and “jeopardize the critical R&D that will enable tomorrow’s discoveries.”

The way the EU plays this debate out over the coming months will say a lot about how it will settle even more fundamental questions in the future about drug prices, research, and patents.

Reflector is Pharm Exec’s correspondent in Brussels