European Union Gives Itself Top Marks for its Pediatric Medicines Regulation

There are advantages in being both judge and jury, particularly on contentious issues where dissident voices might disturb a hard-won equilibrium. The European Union appears to be on course to derive maximum benefit from that happy dual role, judging by the latest stage in its ongoing reflections on the incentives it offers for drug research.

In late October, the EU's executive, the European Commission, presented a glowing report on progress made in children's medicines since the Pediatric Regulation came into force 10 years ago. The report, prepared for the EU's legislative bodies, the European Parliament and the Council, concludes that positive advances-including the authorization of 260 new medicines-could not have been achieved without the EU legislation. It also said the regulation "gives a good return on investment."

The report highlighted some of the numbers to support its case: more than 1,000 pediatric investigation plans-the first step in developing medicines for children-agreed by this year, and 131 of them completed by the end of 2016; a significant surge in new treatments for children with rheumatologic diseases, an area where there were very limited therapeutic options before 2007; and a 50% increase in the proportion of clinical trials that include children between 2007 and 2016-up from 8.25 % to 12.4 %.

The regulation set up a system of obligations, rewards, and incentives to encourage manufacturers to research and develop medicines for children's specific therapeutic needs. It obliges companies to screen every new product they develop for its potential use in children. In return, they can qualify to recuperate the additional upfront costs, via a six-month extension of the product's supplementary protection certificate-and, when the product is a designated orphan medicine, an extra two years of market exclusivity. The Commission says it wants to do more, so it is going to explore how to combine the effects of the pediatric regulation and the EU's orphan medicines legislation "to increase the number and quality of medicines that treat rare diseases-including rare cancers, in children."

All very impressive and ambitious, but the positive approach seems to pre-empt the more strategic reflection on incentives that was set in train last year by health ministers. In June 2016, the EU Health Council invited the Commission to check on whether the incentives in the pediatric and orphan regulations and the supplementary protection certificate were really driving research, or simply giving brand-name manufacturers extra opportunities to boost their profits and to fight off generic competition. The council conclusions underlined “the importance of timely availability of generics and biosimilars in order to facilitate patients’ access to pharmaceutical therapies and to improve the sustainability of national health systems.” And that was merely the diplomatically polite terminology that appeared in the final formal text. The background to the debate featured some highly colorful and deeply critical ministerial remarks about profiteering and price-gouging among innovative drug firms.

So where does this leave that broader reflection? European Health Commissioner, Vytenis Andriukaitis will present the pediatric regulation report's findings to people working in regulatory affairs, patients' groups, and other stakeholders at a conference in Brussels in late November. A further report is due to appear from the Commission before the end of this year covering incentives in general and the orphan drugs scheme in particular. These are just "an essential intermediate step in the debate on a joint vision about the future parameters for pediatric and orphan medicines." Results of this reflection will be presented by 2019 to allow the next Commission to take informed decision about possible policy options. But despite all the anxieties expressed over recent years about abuse of drug research incentive schemes in Europe, the Commission's current mood music seems to be offering a less hostile approach to innovation than what the health ministers were looking for.

That could be good for research, but it could be less good if it springs from a political decision to ignore criticisms of the scheme, because criticisms will not just go away, and are being expressed by a growing number of influential organizations who have already shown they can modify the course of drug regulation history. Only last month, 33 civil society organizations-including Médecins Sans Frontières, Health Action International, Oxfam, Wemos, UAEM Europe, and the European Aids Treatment Group-wrote an open letter to the Commission urging abolition of the SPC mechanism, greater transparency, and more precise impact assessment of public health implications in the SPC review process, and to improve transparency of market exclusivity status. A botched review will store up trouble for the future, which may be more difficult to avert by acting as judge and jury.

Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.