Pharma Reaps Billions Off Orphan Drug Designations, Says New Report By Dutch Investigative Group Intellectual Property 30/07/2020 • Svĕt Lustig Vijay Share this:Click to share on Twitter (Opens in new window)Click to share on LinkedIn (Opens in new window)Click to share on Facebook (Opens in new window)Click to email this to a friend (Opens in new window)Click to print (Opens in new window) Orphan drugs receive ten years of market exclusivity in the EU to promote R&D for rare diseases. Big pharma has reaped billions in profit off the back of European orphan drug regulations that are supposed to incentivize R&D for rare diseases, according to an analysis of 120 orphan medicines registered in the EU over the past two decades. Instead of incentivizing R&D for rare maladies, the EU’s orphan drug legislation has turned into a “corporate cash machine” that has enabled market exclusivities to extend well beyond the ten years set by the EU’s regulations, preventing generic competitors to enter the market, said The Investigative Desk, a Dutch cooperative of investigative journalists that undertook the research. The findings were published in the British Medical Journal (BMJ) and the Dutch Journal of Medicine (NTvG) on Wednesday. The number of orphan drugs earning over $1 billion in sales has skyrocketed in the past decade The Investigative Desk’s research comes almost four months after researchers from international consultancy Technopolis Group presented an EU-commissioned review of the region’s orphan legislation to pharma experts from EU member states. The review suggests that the region’s regulations could be overcompensating big pharma for orphan drugs. Surprisingly, the review also shows that an overwhelming majority of orphan drugs approved by the EU over the past twenty years would have been rolled-out regardless of receiving the EU’s orphan drug designation, suggesting the 21-year old approval process may not effectively spur R&D for rare diseases. “The number of new orphan medicines which may be attributed to the EU Orphan Regulation, equates to 18 to 24”, said the review. “While these products would not have been available without the Regulation, the others [about 118 products] would have been introduced anyway, also thanks to their development in other regions, like the US.” These findings, which will be officially published by the European Commission in a report later this summer, “may raise voices for reform” in the European Parliament, said The Investigative Desk in a press release. Daan Marselis, Reporter for The Investigative Desk in Holland, and lead author of the BMJ report The European Commission and the European Medicines Agency declined to comment on the findings until the European Commission’s official report is published, said Daan Marselis from The Investigative Desk, and lead author of the BMJ report, to Health Policy Watch. Currently, EU legislation makes it “impossible” to withdraw or shorten the ten year market exclusivity once it has been granted to a pharmaceutical company, even if unfair prices are spotted in time by regulatory authorities, said The Investigative Desk in a press release. Another problem is that companies can apply for multiple “orphan indications” on the same medicine to successfully extend their market protection period beyond ten years, said Marselis to Health Policy Watch. Since 2001, 14 drugs have enjoyed fifteen or even twenty years of market exclusivity, according to The Investigative Desk’s research. Since 2001, 14 orphan drugs have enjoyed more than ten years of market exclusivity Orphan Drugs Are More Profitable Than Ever, But 95% Of Rare Diseases Are Still Untreated Orphan drugs have become five times more profitable in past decades, as annual sales revenues have ballooned from €133 million in 2001 to €723 million in 2019, according to the Holland-based investigative group. Rare cancer drugs are the most lucrative, with an average turnover of € 1.1 billion, or twice as much as other orphan drugs that do not treat cancer. Although orphan drugs are becoming more profitable, 95% of rare diseases are still untreatable, said the EU-commissioned review. Meanwhile, over two thirds of the 146 orphan drugs introduced in 2001-2016 in Europe were developed for diseases that are already treatable. In other words, most rare diseases are still untreatable, and the majority of drugs are developed for rare diseases that are already treatable. “Over time, the [EU orphan drug] Regulation seems to have become less effective in directing research to areas where there are no treatments: only 28% of the 142 authorised orphan medicines targeted diseases for which there were no alternative treatment option (95% of rare diseases remain still without a treatment)”, said the review. Image Credits: WHO, BMJ, Daan Marselis. Share this:Click to share on Twitter (Opens in new window)Click to share on LinkedIn (Opens in new window)Click to share on Facebook (Opens in new window)Click to email this to a friend (Opens in new window)Click to print (Opens in new window) Combat the infodemic in health information and support health policy reporting from the global South. 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