The innovative pharmaceutical industry has emerged shaken following last week’s compromise-driven agreement on joint scientific health technology assessments in the EU. The deal represents a significant diminution of the pharmaceutical sector’s expectations. The feeling created is that the concessions and opt-outs offered to national HTA authorities amount to a “missed opportunity”. For its part, EU authorities and member states heralded the agreement as a political breakthrough after 40 months of stalemate in negotiations. Undoubtedly, there is some justification in this point of view. But there is also no escaping the reality that the prize of an efficient one-stop-shop for joint clinical HTA remains elusive. Instead, the industry is left with a diluted version of the promised new regulation which provides for JCAs to be “annexed” to national HTA methods, but with risks attached that member state agencies will be able to decide on a voluntary case-by-case whether to utilize the jointly conducted clinical reports.
The optimism that greeted the European Commission’s unveiling in 2018 of proposals for binding joint clinical assessments (JCAs) has been steadily eroded in recent years. Gradually replacing this burst of hopefulness has been industry disappointment over what might have been. True, the shape of last week’s political compromise struck by the European Council and European Parliament will create the foundations for a permanent framework involving voluntary cooperative joint scientific HTAs for pharmaceuticals and medical devices across 27 EU member states and some 50 HTA organizations. To accentuate the positives, the delivery of the plan has the potential to result in a less fragmented HTA system in the EU. Greater collaboration offers the possibility of improving the speed and quality of HTA processes, particularly in smaller EU countries, accompanied by reduced duplication and more equitable access. But the provisions for ensuring the uptake of JCA reports in national HTA processes is not nearly as robust as the industry would have preferred. Political battles in this area were largely won by a conservative alliance, led by Germany, which rebuffed what were perceived to be attempted intrusions by the European Commission into the independence of national HTA structures and competencies.
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has drawn attention to the resulting “unnecessary administrative and regulatory barriers” it says characterize the compromise framework, amid warnings the “maximum flexibility” allowed in how member states apply non-binding JCAs will “inevitably” result in a “suboptimal” and “more inefficient system” than originally planned.
This perspective is countered by some EU political stalwarts who have insisted that the deal establishes a transparent JCA framework, complete with clear provisions regarding stakeholder selection of HTA topics and involvement in joint work, as well as safeguards governing the uptake of JCA, including in terms of reporting on use in national HTAs. This is supported by claims the agreement provides clear definitions for divergent scientific reports and institutes a voting mechanism by a coordination group when it comes to political strategy and technical scientific issues. The European Parliament’s rapporteur in the field, Tiemo Wölken, is quoted as saying that the safeguards enshrined in the agreement are capable of “[ensuring] that joint assessment cannot be ignored”. However, the full text of the deal has yet to be released, meaning that a proper analysis of the basis for these claims is difficult.
The slowness of the timeline for implementing the plan may also generate some frustration within the pharmaceutical industry. The current thinking is that the JCA will begin to be applied in stages, starting with new cancer medicines, about three years after the regulation is adopted. Assuming adoption is forthcoming later in 2021 – with the European Commissioner for Health Stella Kyriakides indicating that this was possible in the “coming months” – then this would trigger the first joint scientific HTA reports from about 2024 onwards. The idea is for JCAs to later be expanded to encompass ATMPs and then rare disease drugs. It is anticipated that all new medicine and medical devices will be added in stages, probably within a 3-5-year period, following the regulation being adopted in late 2021 or 2022.
The full details of the package of measures that have been agreed between the European Council and European Parliament have yet to emerge into the public domain. As such, some of the current interpretations surrounding the agreement may change. Ambiguities contained in the agreement also mean further fine-tuning is likely to be required in the years ahead. Furthermore, to some extent, best practices by national HTA organization, in terms of how fully JCA reports are utilized, may come to be refined by custom as much as by EU regulation. Nevertheless, the fundamental problem for the pharmaceutical industry remains that rather than adopting harmonized, binding scientific joint HTAs, the regulatory haziness that is emerging risks shrouding the endeavor in an aura of unpredictability.