New regulations in the legislative landscape impose a high level of attention on access to medicines in the near future.
From 1 January 2025, the new HTA regulation will become operational: it is a multidisciplinary process for the evaluation of health technologies such as drugs, medical devices, and medical apps. The European Union therefore recognises the importance of HTA and aims to formulate shared assessments, avoiding differences and duplication: a single dossier produced by the developer (pharmaceutical company) will have as its output the Joint Clinical Assessment (JCA).
The first medicines to be evaluated from 2025 are advanced therapies and cancer drugs.
The evaluation scope of the JCA includes all relevant parameters for evaluation in terms of:
- patient population,
- intervention,
- comparator and outcome (PICO).
In particular, the choice of comparator is a matter of debate. In fact, even Standard of Care or drugs with off-label use could be considered in the JCA, if proposed by one of the member states. In Italy, in fact, the 2019 DM Negotiating Criteria allows the comparison with drugs in 648/96 or with established therapeutic strategies.
Objective
Starting from the list of medicines included in the PRIME programme, based on the identified therapeutic indication, the #Team Pharma Value, together with Sveva Sanzone of Biogeno, identified for which drugs the competitor could be detected under the 648/96 list.
Materials and Methods
From the Horizon Scanning activity published by AIFA on 04/04/2023, the list of medicinal products that have had access to the EMA's PRIME programme, but for which a marketing authorisation application has not yet been filed, was analysed. The list, updated to 31.3.2023 and subsequently published on the EMA's institutional website, of medicines included in the PRIME programme was also examined. These drugs were then compared with the 648/96 list, which includes drugs reimbursed for an indication that is not yet authorised, in order to identify the existence of any comparators between off-label uses reimbursed by the NHS.
Results
On the list there are 68 medicines that have accessed the PRIME programme reported in AIFA's 'Horizon Scanning' for which a marketing authorisation application has not yet been filed. Four additional medicines were included in this analysis because EMA issued an update in the first quarter of 2023.
Among the 72 drugs identified by EMA as promising: 33 are ATMPs, 14 are chemically synthesised, 19 are biologicals and 6 are immunologicals. Of these 72 drugs, 16 are oncohaematological and of these 12 are ATMPs. There are 21 ATMPs for therapeutic indications outside oncohaematology. It means that if all 72 expected drugs receive a marketing authorisation from EMA, 37 of them would have a Joint Clinical Assessment as of 2025.
Based on the therapeutic indication of each drug, the work involved checking which drugs could have the comparator on the 648/96 list. Specifically, there are 5 drugs (approximately 7% of the total) that would find a potential 'off-label' comparator in that list. Of these, 2 drugs are oncohaematology (one is a biologic and another is an advanced therapy) and 1 is an advanced therapy in ophthalmology.
Conclusions
Although not exhaustive, the evaluation shows that as many as 37 oncohaematology and/or advanced therapy drugs could be included in the JCA evaluation and that among these, 3 would have a potential comparator in the 648 list as early as the first evaluation in 2025, being oncology and/or ATMP.
