The New Year Brings Same Old Battles

January 1, 2011

Applied Clinical Trials

Applied Clinical Trials, Applied Clinical Trials-01-01-2011, Volume 20, Issue 1

An alliance between two European leaders gives hope for unison but falls short on solutions.

As the glitz and warm goodwill of the festive season evaporate, drug firms and drug regulators are once again faced with the chilly reality of their differences of view, still largely unresolved as we move into the second decade of the 21st century. The clinical trial community is one of the principal victims of these differences, pincered between ever-increasing demands for demonstration of value and ever-tighter budgetary constraints from cash-strapped sponsors.

Peter O'Donnell

The problem is depressingly familiar: growing healthcare demand, rising healthcare costs, tougher drug-launch criteria, squeezed company revenues, and pressure on R&D budgets—at the same time, drug development becomes harder and more expensive. What makes it all the harsher in this unhappy new year is that the economic downturn is biting severely into both public expenditure and private-enterprise profits.

Two luminaries of the European pharmaceutical scene—one from each side of that traditional divide—made an attempt to forge a new approach as the old year came to an end. Andrew Witty, the CEO of GlaxoSmithKline and President of the European Federation of Pharmaceutical Industries and Associations, and one of Europe's top regulators, John Dalli, the European Union's Commissioner for Health, issued a sort of joint manifesto that tried to square the classic vicious circle.

Their starting point was that policy-makers and the pharmaceutical industry across the European Union share the goal that all citizens and patients in Europe should enjoy equal access to the right medicines and vaccines. They offered a vision of the pharmaceutical industry, policy makers, health insurers, patients, and others, working together to achieve that objective. Success, they claimed, would result in healthier citizens, enhanced competitiveness, more balanced budgets, and fair reward for innovation. "Valuable innovation can transform lives," they agreed.

A noble aspiration. At the broadest level, the need for greater equality is clear—as Dalli and Whitty remarked. They pointed to the beneficial impact of earlier diagnosis and new treatments on cancer, where incidence is increasing in the EU, but mortality is decreasing. "There is significant variability in patient access and in relative survival rates," they went on to note. "Similarly, we see wide variability between member states in the speed with which valuable innovative medicines become available to patients, and then further variability in uptake between member states and between disease areas."

Aspiration is one thing. Delivery is another. As the two European pharma champions acknowledge, the task of delivering equality across Europe is not easy. "Health services everywhere face enormous challenges. Governments are understandably struggling to cope with rapid demographic change, the pace of technological change, and unbridled demand for healthcare. The current financial crisis exacerbates the difficulty of balancing desire for healthcare equality with the need for financial management."

Against this background, they go on, "governments need to maximize public health outcome for a given budget, and to make choices."

This is the difficult bit—for pharma companies struggling to get new products onto the market, and equally for health authorities harangued by their publics.

European governments are focussing on value for money, and rely increasingly on processes like health technology assessment for decision-making. Dalli and Witty both say that this is appropriate, but there are reservations and hesitations.

"HTA requirements in different member states vary considerably, which causes complexity for industry. The ongoing fragmentation of healthcare delivery in Europe introduces further challenges," they remark, in a bold display of diplomatic harmony.

But as they advance in their argument, the underlying tensions inevitably emerge.

"In this challenging climate, it is vital to achieve the best results for patients across the European Union, for public budgets and for industry," they say. They speak of "an equilibrium" between the needs of these different players—but in the same sentence they add: "with patients put first"—immediately suggesting some disequilibrium in the priority to be allocated to each player. Reading this, it is impossible to resist the thought that all players are equal, but some are more equal than others.

From that point onwards, their joint manifesto starts to fray, with its separate threads stitched tenuously together only by the deployment of well-meaning generalizations that studiously avoid confrontation on the difficult details.

"All stakeholders need to change the way they think and act, with 'health for all' achieved collaboratively at local, national, and European level," they chorus blithely—and innocuously, since the appeal is so vague as to be meaningless.

"'All for health' should be the key enabler—the more institutions work in a trusting environment, the more likely the right approaches can be developed," they warble happily, allowing sound to triumph over sense.

Where the manifesto addresses the distinct needs of each "stakeholder," it does so in the same well-meaning and inoffensive terms. "Industry must be a genuine partner to governments and their agencies," it trills. Who could take exception to its brotherly expressions of mutual understanding? Industry "must deliver new medicines and vaccines that address unmet patient need and have demonstrable value." Well yes, of course. And how? "This means getting R&D right—reversing the decline in productivity and delivering medicines that match the health needs of the population." Not wrong, of course, but not sufficiently right either to bring any illumination into the dark realities of this debate. Similarly, "industry must increase the transparency of what it does and be a source of ideas to governments, including on increasing funding and more efficient use of budgets." Insofar as this highly-nuanced and carefully selected language conveys anything at all, it seems to amount to little more than an offer to industry to connive with governments in engineering its own downfall.

As a concession, as it were, to industry sensibilities, the passage concludes: "The pharmaceutical industry is hugely innovative. If governments work to support innovation, the industry will deliver the next era of revolutionary medicine."

That blandly optimistic formulation is followed by another: "Governments have the key responsibility for delivering equitable healthcare. It is possible for them to encourage and reward therapeutic progress, while maintaining core characteristics like equity, public funding, and strict regulation." It may indeed be possible. What would be more interesting would be to know more about how it is to be done.

The recipes proposed for success remain largely at the same level of pious exhortation and comfortable banality. "We must create in Europe the right policy and regulatory framework." OK, what's next? "This requires that effective prevention and wellness programs are in place." Safe, but vague. "We must shift focus from cost to value, and set a strategic agenda that addresses unmet needs." Difficult to fault, but difficult to decipher, too.

There is some attempt to grapple with the difficulties in establishing a sense of value—but again, without much real commitment to a discernible course of action. There is talk of "allowing industry to understand the type of added value that matters to patients and will be funded by payers," and of "a shared understanding of value" through "high quality dialogue between industry and authorities." Dalli and Witty (a coupling of names that faintly suggests a comic duo from the age of music hall, even if their routine often seems short of a punch line) see an important role for health technology assessment in ensuring that "the government does not pay for medicines that do not work but that equally, patients get access to medicines that may help them." But how it should play this role is less clear. "Closer cooperation" is one recommendation, immediately countered by the remark that "a single approach in the EU is undesirable, since member states will want to make decisions according to their own priorities."

At the same time, "the current diversity and overlap leads to unequal access for patients, complexity for industry, duplication of work, and unnecessary red tape." This tangled series of observations is hardly clarified by the conclusion that "While the final decision has to remain national, in particular on price and reimbursement, we should explore a common methodology on the clinical aspects of health technologies in Europe which might then be used for assessments of relative efficacy and effectiveness at European level."

So if clinical trial and pharmaceutical professionals were hoping that this unusual outburst of ecumenism in Brussels was going to make things easier—or even just clearer—in the new year, well, I'm very sorry. But happy new year anyway.

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

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