EU HTA: the next great competition tool between Pharma Companies

EMA approval, then EU HTA Joint Clinical Assessment – JCA, then Country HTA, then Country P&R. This is the future market access process, requiring Manufacturers to produce and provide data satisfying, for type and quality, all those stakeholders in the respective required timelines.

The new centralized EU HTA starting next January 2025, will deeply change the market access dynamics, modifying and increasing the competition among Companies

The future EU HTA will shift good part of the tasks and responsibilities of the actual clinical HTA from Country level to the EU Coordination Group and to the operational Subgroups receiving the commitment of each single assessment - JCA

Likewise, the national HTA bodies will need to amend their organization, activities, and missions in readdressing good part of their actual resources and tasks to participate in the Coordination Group and Operational Subgroups (I commented that in an article here some months ago and we recently discussed it in published papers and proceedings of EAA – European Access Academy)

Manufacturers will need to adapt their strategies and operations, as the new framework will represent a new crucial competition.

Despite the lack of effective involvement of Manufacturers in setting up the EU HTA rules and in the governance of the future system (see also the recent disappointed statement from EFPIA and EUCOPE), the EU published documentations, HTA Regulation and delegated acts, clearly define which will be their operational tasks to compete in that new arena with the above mentioned centralized and local stakeholders. Here following a summary:

Early consultation: Manufacturer can request a joint scientific consultation from the Coordination Group when the clinical studies are still in the planning phase. It can be about PICO: the trial design, choice of comparator, endpoints, interventions, health outcomes, patient populations, etc.?

Submitting the dossier: Manufacturer compiles and submits a dossier containing all the necessary information. The content is further defined by Annex II of the HTA Regulation and delegated acts.

Planning phase: Manufacturer can be actively consulted in the planning phase of the JCA, like in the preceding pilots of the various Joint Actions

Timelines: Manufacturer will submit its dossier 45 days before CHMP opinion. The JCA will be published at the latest 30 days after the EPAR release. As the JCA report is set to be published just 30 days after the CHMP decision, national procedures may start very rapidly

Quality of evidence: Manufacturer will provide evidence following the international standards of evidence-based medicine, randomized, blinded, controlled, direct comparative studies where appropriate. It is still to be defined the relative weight given to the different PICO components, today quite different between Countries HTA respective agencies (i.e.: H2H, QoL, surrogate EPs, etc.)

Updating the dossier: Manufacturer, if new clinical data becomes available during the Joint assessment must proactively inform the Coordination Group and providing documentation when request

Country level: Manufacturer, if additional information, data, analysis, and other evidence is needed for complementary clinical analyses, will be asked by Member States all the necessary information, data, analyses, and other evidence.?

Manufacturer may not submit evidence at a national level that is already available at EU level.

The request to Manufacturer from some Countries of additional data and evidence will be due to the divergence between Countries agencies about different methodologies one another and between what is required for the future JCA

Those divergences between Member States requiring extra data will influence the launch sequence of EU countries which could delay the access of drugs in some of them.?

There could be rapid access in countries who base their pricing/access negotiations largely on the JCA, which could have a knock-on effect on launch strategy considering what comparators, endpoints, and subpopulations a JCA may require when planning clinical trials to avoid additional work later.

Manufacturer should anticipate any additional data that may be needed on a Country level.?

Manufacturer should hence ensure that their clinical trials are up to satisfy quantity, quality and timing of provided data for JCA, but at the same for EMA and single Country HTA bodies. The future market access competition is here?