Navigating Drug Development Through More Turbulent Waters

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Applied Clinical Trials

Applied Clinical Trials, Applied Clinical Trials-06-01-2019, Volume 28, Issue 6

The World Health Organization tries to find a balance with its Road Map for Access on how to best approach research and pricing in Europe.

No one with an interest in drug development will have missed the hostile tone of the exchanges over recent weeks as U.S. congressional hearings pursue their investigations into the price of drugs and the merits of innovation. Critics of the drug industry in Europe are seizing on the opportunity to drive home their message that the rules need changing here, too, to get better control of prices and research.

The fundamental question in U.S. discussions has been how drug firms justify the price they charge and how the price relates to their spending on research. The exchanges have frequently gone beyond the costs of the research investment to question whether the research is focused on the right areas, or even the merits of the research itself. The spill-over has extended to ambitions to limit the monopolies conferred by patents and to promoting alternative access via generics. The vigor of the discussions has provoked pushback from many supporters of the industry, such as Rep. Jim Jordan (R-Ohio), who cautioned that government action to nullify patents to promote generics would have a chilling effect on research.

In late May, the annual World Health Assembly convened to debate a plan for the next five years that leads off with a reference to "the high prices of new pharmaceuticals, and rapidly changing markets for health products that place increasing pressure on all health systems." It highlights the need for a "research and development agenda defined and research coordinated in line with public health priorities" and for "fair and affordable pricing."

The World Health Organization's “Draft Road Map for Access To Medicines, Vaccines, and Other Health Products 2019–2023” warns against weak policy interventions to manage expenditure, “such as the ineffective use of policies for generic and biosimilar medicines,” and urges “avoiding waste that occurs when health products are priced higher than is necessary, or when less expensive but equally effective alternatives are not used.” The relationship between government and the pharmaceutical private sector “requires particular attention,” especially in “avoiding the risks of undue influence.”

At the same time, the Road Map notes the “inadequate investment in research and development” that leaves many neglected diseases without adequate treatment options, and identifies the challenges in “setting priorities for research and development needs and incentivizing research and development for health products that have a potentially limited return on investment.”

WHO sets out its approach to getting the right balance. It says it is already “coordinating the efforts of different actors, setting research and development priorities, identifying associated gaps, defining desired product profiles, and facilitating the development of affordable, suitable health products.” Its new proposals now include creating “unifying principles for biomedical research and development,” and the “promotion of transparency in research and development costs.” The thorny questions of patent and other rights can be eased, it says, by promoting “public health-oriented licensing agreements and transparency regarding the patent status of existing and new health technologies.”

To “encourage more transparent and better policies and actions to ensure fairer pricing,” governments are urged to use “global and regional collaboration to increase price transparency,” and to adopt generics more widely. A list of “milestones” over the lifetime of the Road Map includes creating “target product profiles for missing health products to guide research and development priority-setting for unmet public health needs in areas of market failure.” The publication of guidelines on pricing policy and on monitoring price transparency for pharmaceuticals is foreseen for 2021.

Although WHO’s principal preoccupation is with the needs of the poorer countries in the world, the Road Map explicitly includes Europe. In the days before the World Health Assembly opened, the richest European countries were in the crosshairs of drug industry critics for allegedly attempting to impede the adoption of resolutions harmful to the interests of the industry.


More direct demands

The Road Map is not the only document on the assembly’s agenda. A much sharper resolution on access to medicines has been drafted by the health minister of one of Europe’s biggest countries-Italy-with a more critical tone and more explicit demands for new controls on industry. Giulia Grillo proposed “minimum standards for transparency regarding information from clinical trials and the costs of research and development for drugs and vaccines,” in a draft resolution. She champions sharing information via a government-led international database on “drug prices, revenues, R&D costs, public sector investments, and marketing costs.”

This approach has won support from other European countries who find themselves at a disadvantage in their negotiations with drug firms, including Greece, Portugal, Serbia, Slovenia and Spain. The backers of the resolution include Turkey, Brazil, South Africa, and Uganda. Germany, the UK, Denmark, Austria, and Sweden have all been trying to water down the draft.

The intervention by these countries-mainly with strong domestic drug producers-has provoked vigorous attacks by health campaigning groups. Médécins Sans Frontières said it was “disconcerting to learn that Germany, the UK, Sweden, Denmark, and five other OECD countries are attempting to derail this important effort to create transparency in medicines pricing and medical research and development.” Instead, “these governments should stop favoring pharmaceutical corporations’ profit maximization agenda and ensure the right to affordable medicines and treatment for their people.”

The debate and its outcome will have little direct effect either in the U.S. or in Europe, since WHO has no power of enforcement of any of its resolutions. But to dismiss the debate’s significance would be to underestimate the influence that will be felt from the high-profile articulation of growing hostility to high prices and rising demands for tighter controls on research budgets and strategy. Those already hostile to industry practices will be emboldened by strong support from the assembly, and those who are neutral will be tempted to entertain new doubts.

In the midst of all of this, those who are engaged in drug R&D and aiming to plan ahead effectively will have to face additional questions-and not just from critics, but from increasingly suspicious authorities responsible for regulation, research, reimbursement and payment, and ultimately from increasingly cautious financial backers, bosses, or shareholders.


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