More European Big Hitters Demanding Drug Evidence Evolution

Unapologetically, this column returns to the theme of demonstrating drug efficacy, evoked as recently as a month ago in “Bringing Evidence Back into Fashion in European Drug Rules” after the head of the French government’s health technology assessment (HTA) triggered a discussion of how much evidence was enough evidence—and made clear that at the moment, the evidence is often not enough.

The latest trigger comes from a different quarter—the organizations that pay for medicines in Europe. In Europe’s complex web of healthcare systems, the regulators and their approvals are the initial gatekeepers for medicines, and the HTA bodies are key influences on who will get them, but ultimately the decision in each country depends on whether the payers decide to pay for them. No matter what is coming down the pipeline toward patients and physicians, it is the payers who stand at the end and control the on-off tap. And they are far from happy, to judge from a meeting last week in Brussels.

As the July heat begins to drive away the last officials and diplomats and lobbyists from the airless committee chambers and meeting rooms, some of the tough talk from that meeting continues to echo down the empty corridors.“There is a significant evidence gap,” one prominent figure declared, listing some of what is missing in terms of supporting evidence for new drugs. “There has been an enormous increase in uncertainty,” exclaimed another. “We must explore how to get better supporting studies from companies,” insisted a third. And so it went on, for an hour. And what emerged most clearly was not just dissatisfaction, but a determination that the situation must change.

What raises this event above the normal run of critical exchanges between drug innovators and authorities in Europe was the combined fire-power of this assembly—and the fact that they have decided to go very public with their message. A brief digression will suffice to identify this college of cardinals of drug budgeting (readers wishing chapter and verse can watch the entire discussion on video here). They come from the Medicine Evaluation Committee (MEDEV), which brings together 22 national authorities for assessment, pricing and reimbursement from 18 EU member states plus Switzerland, and from the European Social Insurance Platform (ESIP), comprising 45 national social security organizations insuring citizens in 17 member states (and Switzerland).

The timing of the meeting is also a factor in its importance. As most ACT readers are well aware, the European Commission is scheduled to propose sweeping revisions of the EU legislation on pharmaceuticals by the end of 2022, intending to address shortcomings in terms of access to and availability of new treatments. MEDEV and ESIP have prepared their own detailed shopping list of the changes they want to see, and their emphasis is on “robust and timely evidence generation.” They are dead against any shifting of the assessment of safety, quality, and efficacy from pre- to post-marketing authorization. They continue to insist that randomized controlled trials “are the gold standard” and they believe that conditional approval pathways should be strictly limited, with tighter requirements for performance of any post-launch studies. The fast-track mechanisms for vaccines introduced as an emergency measure during the COVID-19 pandemic are not a valid model for normal circumstances, they say.

The legislative review is, say the payers, “an unprecedented opportunity to effectively mitigate the negative consequences of the growing weak evidence-high prices conundrum.” They see stronger evidence as the route to an improved world of “informed, timely decisions, better deals, better priority-setting, better chances for the renegotiation of contracts, healthier competition, and better targeted incentives.” They want to see the new pharmaceutical rules give them an earlier—and more influential—voice during the regulatory approval process. And to judge from the tone at their meeting, they are prepared—and preparing—to take their message to the top. Not only are they making their pitch to the European Commission before it makes its proposals, but they are also aiming to recruit the influence of their own national governments to exert pressure. It is clear that evidence is not just coming back into fashion. If payers get their way—and they usually do—it looks like it is here to stay.