Here's to a Happier New Year?

Just as 2011 was drawing to a close—and, by coincidence, on the same day that the fate of Europe's single currency was under discussion at yet another make-or-break summit of Europe's leaders—the Director General of EFPIA made an ostentatious bid to persuade European policymakers to adopt a more ambitious approach to considering the future for healthcare and medicines.

Richard Bergström, still new in his post, lacks nothing in terms of style. He had issued hundreds of printed invitations to the "first annual EFPIA Director General's lecture," in one of Brussels' largest and most prestigious venues. His chosen theme was, "Is society ready for the new science," and his discourse ranged across the opportunities that pharmacogenomics and personalized medicine offered. But his optimism was shot through with warnings of unfulfilled potential, unless a better fit could be engineered between the challenging new developments in medicine and the inertia and lack of imagination that current regulatory systems suffer from.

Much of Bergström's thesis is well known and uncontroversial. Disease patterns are changing, development costs are rising, payers remain increasingly cautious about reimbursing innovations, many diseases remain untreated ... But he departed from the customary narrative with his flat rejection of the widely held view that there is any crisis in discovery. Research output remains good, he said, brandishing recent examples of effective new therapies in fields ranging from hepatitis to melanoma.

The real crisis, in his view, is that the collective efforts of the pharmaceutical industry are not meeting the collective needs of society, because the signalling between the two is deeply imperfect. The organizations that make regulatory or funding decisions on behalf of society are ill-coordinated between one another within countries, and across the international market from country to country, and their approaches to innovation are clumsy and insensitive. In consequence, industry reacts to ill-judged constraints and incentives, and the pathway to the clinical exploitation of new science is obscured or strewn with unnecessary and inappropriate obstacles.

Bergström provided fewer answers than questions. His chief objective, it seems, was to issue a cry of alarm, and to provoke deeper reflection.

What's in Store?

As 2012 opens up before us, what are the chances of a happier new year? There is certainly no lack of activity programmed at a European level in relation to healthcare and the provision of pharmaceuticals. Brussels bristles, it might be said, with plan and projects.

Among the first to emerge—perhaps this month, even as you read this—will be the legislative proposal to update the European Union's rules on pricing and reimbursement procedures: the Transparency Directive. Delayed from 2011 by last-minute haggling among EU officials over how far it is reasonable to oblige national authorities to come clean (and move more quickly) on reaching their decisions, this measure will offer no panacea to the sort of problems that keep Bergström awake at night. It is largely procedural in effect, since it leaves intact the autonomy that each EU member state enjoys over what prices and reimbursement levels it chooses to set for the products on its market.

However, this largely administrative measure will catalyze and focus much wider discussion during the year on some of the fundamental issues of healthcare economics. It might be legitimate, for instance, for national authorities to take a little longer to reach a reimbursement decision if they are going to conduct a fuller assessment of the overall value to the healthcare system of a particular innovative treatment. But that raises questions about what approach they might be using, how valid its methodology, how rigorous its application, how comparable or compatible it may be with methodologies being employed by national authorities in other countries (and, a fortiori in the EU, in other EU member states—that in theory constitute a single market).

Step forward at this point the EU's network on health technology assessment, EUnetHTA, which has been struggling over the last few years to come up with harmonized—or, at any rate, soundly based—approaches to evaluation. The same day that Bergström was stoking the fires of controversy in Brussels, EUnetHTA was holding its annual conference in Gdansk, Poland, to debate European collaboration in policymaking on health technology assessment in national and cross-border healthcare. The industry is cautious about the developments in this area, and at the very moment Bergström was addressing his audience, his federation (along with half a dozen other European health industry federations) released, in Gdansk, a highly skeptical analysis of the process, accompanied by an unambiguous demand for industry to be admitted to the magic circle that is developing and applying health technology assessment. 2012 will see battle joined across a broad front in this fight for influence over the way that decisions vital to the industry are made, and on what basis.

Getting Personal

But the prospects for personalized medicine that Bergström evoked are also going to be governed in part by how regulation relating to product authorization is adapted. And again, a major discussion will start early in 2012, when the EU is due to make its proposals for revising its 2001 rules on clinical trials.

In this area, even the EU's commissioner for health, John Dalli, recognizes the need for change. "We will have to look into whether improvements are needed to make personalized medicine products more rapidly available to patients," he admitted last month. "For example, does a defined or limited patient population pose additional challenges for conducting clinical trials?" he mused. The revision of the current clinical trials rules will open a Pandora's box of fevered debate on the very essence of what testing methods are appropriate for selected patients—and how such trials can make effective comparisons with standard treatments, or how far their results can be extrapolated to wider populations.

Developments in personalized medicine will also depend increasingly on new approaches to companion diagnostics—and that too imposes the need for radical changes in the regulatory environment. Here too, Dalli accepts the principle and anticipates upcoming debates: "The regulatory pathways for the placing on the market of medicinal products and of diagnostic medical devices are different because the nature of the products regulated is different," he noted, looking ahead to the planned 2012 revision of the EU's medical devices legislation as "an opportunity to ensure that diagnostic medical devices used in the context of personalized medicine offer the appropriate level of safety and performance." In addition, his officials are already engaged in preparing a major report that Dalli is committed to present in the middle of 2012 on the use of 'omics' technologies in pharmaceutical research and development.

At the same time, 2012 will see crucial reports presented on some of the other broad Brussels-sponsored programs already under way in Europe. The EU's self-styled "flagship" program on exploiting innovation to assure healthy aging will come of age with the implementation of a strategic implementation plan agreed to in late 2011 by the pharmaceutical industry and other organizations in the partnership. So too will the EU's e-health program, which is dignified by the personal participation of no lesser a figure than GlaxoSmithKline's Andrew Witty. A major new public/private partnership to fill the gaps in the antibacterial armamentarium will get under way. And a dozen working groups set up by the European industry commissioner—on subjects ranging from biosimilars to ethics, and from OTC switching to managed entry schemes—are due to finalize their work.

It will be a turbulent year for pharmaceutical policy in Europe. Whether Bergström's hopes will be realized for a profound rethink of the approach to new science is doubtful—but rather like his speech, 2012 is a year that will certainly pose a lot of new questions. As to whether the EU's single currency will survive long enough to pay for the exploitation of any of these new opportunities, this columnist declines to speculate.

Reflector is Pharm Exec Europe's anonymous columnist, a commetator so close to the action in Europe that his identity must remain secret