Orphans Under Attack Again

There have been months of subterranean rumblings of concern across Europe that the generous scheme to promote orphan drugs is being abused by drug firms keen to maximize profits rather than promote innovation. Now the rumblings have turned to shouts – and in the most august of Europe's healthcare forums, the Council of Ministers of Health of the European Union.   These ministers met informally in Amsterdam in mid-April, with an agenda that included a serious review of whether orphan drugs are value for money or organized banditry. And the pressure for such a contentious discussion came from the country that holds the rotating presidency of the EU for the first half of this year, the Netherlands.

  Edith Schippers, the country's combative health minister, was already one of the most outspoken critics of industry behavior. Now she is the president of the EU health council, she has wide scope for turning her personal views into a public campaign – and at the Amsterdam meeting she demonstrated her determination to make the most of her six months in this position.   As ringmaster for the meeting, she circulated a background paper to her counterparts from the other 27 EU countries that contained a clear exposition of her concerns. Under the politically-correct title of "Innovative and affordable medicines: Innovations for the benefit of the patient," she said that in terms of pharmaceutical policy, "It is time to set a new course."   Part of her narrative related to the challenge that national health authorities face in deciding on the price and reimbursement status of all medicines, particularly since governments' negotiating power is limited by operating as separate countries in the face of global companies. Here her suggestions related mainly to remedying the asymmetry that works to the advantage of drug firms. She encouraged national authorities to work together and share their information, so as to strengthen their hand when the deals are done. "With a share of about 27% of the global pharmaceutical market, the EU member states could have major leverage to influence the behavior of pharmaceutical companies operating globally on the price of their products," her paper said.   But she was at her most incisive in relation to orphans. The costs of many new medicinal products are "extraordinarily high, in some cases up to €500,000 per patient for one year." And pharmaceutical companies are protected by mechanisms that were designed to encourage innovation, she said, notably by market exclusivity for orphan drugs. "The industry now sometimes makes undesirable use or even misuse of these protection mechanisms in order to maximize profit on a product."   For Schippers, the question arises ever more frequently as to whether the current supplementary European incentives - in the form of supplementary protection certificates, data exclusivity and market exclusivity - have not "overshot the mark." She answers her own question with the unambiguous remark: "Market protection of orphan drugs leads to unwanted effects."   As ministers of health, she says, "we do have leverage to influence supplementary measures that are currently in place in the EU and we can demand social responsibility from companies."   Her analysis of the EU rules governing orphans is that "the first medicine to come on the market for such a disease has a monopoly, even if its effectiveness may still be unclear and regardless of whether or not it is the best medicine for the patient." The result, she says, has been "a hefty increase in products with an ‘orphan’ designation" – based on a "broad definition of the concept ‘orphan drug.’"   She accuses manufacturers of registering a single drug for numerous sub-indications, and by this ‘cutting up’ of indications and the related disease prevalence, they can win orphan drug status for the product. "This results in monopolies and the associated prices. And this is not what was intended by the EU regulation."   Schippers is proposing a clearer delineation of the ‘unmet medical need’ that is central to the EU orphan drugs rules, and a review of whether orphan medicines’ market exclusivity still makes sense. Potential applicants for orphan drug status have been warned!