Rewriting European Regulations on Rare Disease Treatments

The major review of European pharmaceutical legislation that will start later in 2022 has brought together two major healthcare players who have, by their own admission, still a lot of differences. Driven by a shared sense of urgency, European drug companies in the European Federation of Pharmaceutical Industries and Associations (EFPIA) have joined forces with the European Organization for Rare Diseases (EURORDIS), the European rare diseases lobby, to craft a series of joint policy proposals on orphan medicines. “The fact that EURORDIS and EFPIA have for the first time issued a joint statement is testament to the importance of the proposals put forward,” says the introduction to the proposals.

The trigger for this unprecedented alliance is the declared intention of the European Union to revise its incentive schemes for treatments for rare diseases—a crucial issue for both. For EURORDIS, new legislation is a seen as a major opportunity to improve the prospects for innovation and for boosting availability of new drugs—with the risk of a major disappointment for millions of desperate Europeans and their families if the EU gets the new rules wrong. For the industry, changing the current legislation constitutes a huge threat to what it depicts as a still-precarious and inadequate system of support for risky and high-cost R&D projects. The industry’s sense of threat is all the greater since the mood music coming from EU officials and national ministers has focused heavily on clamping down on alleged industry abuses and what are seen as unacceptable monopoly pricing practices.

Against this background, both sides have found common ground “while acknowledging areas of divergence.” And top of the list of what they have come up with is a radical concept for a new European pricing framework—equity-based tiered pricing (EBTP). The essence of this approach is to align medicines’ prices with countries’ ability to pay. In other words, poorer countries—such as Bulgaria—will be offered lower prices than the wealthiest, such as Germany or Sweden. This, they say in a joint statement, can “help address affordability constraints in lower-income EU member states without diminishing incentives to invest in new medicines.” This dramatic new approach is wrapped up in a series of conditions and reservations—and closely reflects a proposal that the EFPIA made earlier this year on its own initiative and predicated on counterpart measures by national governments to preclude any member state cheating on such a novel system.

Another of the joint proposals is to tweak health technology assessment (HTA) to enhance patient access to orphan drugs. These new EU rules on coordinating HTA, adopted in January but not fully in operation until 2025, envision giving new priority to orphan drugs. This represents “a significant opportunity to streamline value assessment processes through cross-country collaboration,” says the joint position, adding, in another echo of earlier EFPIA insistence, that the opportunity can be seized only “if duplication is avoided and methodologies are fit for purpose.” Yet more EFPIA fingerprints can be seen in recommendations for adjusting EU drug authorization frameworks: “More routine use of adaptive pathways and real-world evidence can help mitigate evidential uncertainty at launch, allowing timely patient access while additional evidence is collected,” say the partners in this position—coming very close to attitudes being heavily promoted by the drug industry over recent years. And in comments on pricing systems, the proposals have an almost eerie ring of earlier EFPIA arguments. Country-level HTA and pricing and reimbursement processes can be better adapted “by supporting greater use of novel payment and pricing models,” says the joint statement. And in a final introductory remark, the joint paper candidly and explicitly sets out an industry position about the need for “sustained innovation for tomorrow”—for which “EFPIA proposes to launch a moonshot for rare diseases, whereby more coordinated, targeted, and collaborative basic and translational research would allow to unlock a new wave of innovation.”

The stakes are high for both partners. Since the EU incentive scheme was introduced 20 years ago, more than 200 products have been authorized, with another 2,000 designated eligible for support. The emergence of at least half of these medicines—and the benefits they have brought to more than six million rare disease patients across Europe—are attributed to the scheme. If the EU pares the incentives back too far, in the interests of economy or to prevent—as some EU ministers have called it—“price gouging,” that flow of innovation may slow to a trickle, with bleak consequences both for research-based companies and for rare disease patients.

Reflector is Pharmaceutical Executive’s correspondent in Brussels.