Considering possible PICOs early on in clinical development is crucial

There are more and more drugs that address a specific subset of patients, not least within oncology. In parallel, the centralisation process of determining clinical benefit is approaching fast, and Joint Clinical Assessment (JCA) as part of EU HTA regulation will be implemented for new oncologics and ATMPs (advanced therapy medicinal products) in January 2025. As several emerging therapies could be treatment game-changers, quick patient access without delay is of outermost importance. 

The scope of the JCA entails surveying all 27 EU member states on what population, intervention, comparators and outcomes (PICOs) should be addressed by the evaluation procedure. This process does not involve dialogue with the manufacturer and will subsequently not take data availability into consideration.
Once the PICOs have been defined and communicated, the manufacturer will just have 100 days to complete and submit its evidence dossier answering the evaluation scope. Consequently, there is little or no room for data generation at this point.

Hence, not to delay patient access to ground-breaking treatments, considering possible PICOs early on in clinical development is crucial. Assessing potential heterogeneity regarding population (P), comparator (C) and/or outcome (O) to inform and plan the clinical trial program could mean a smoother and shorter process ahead. To support the design of pivotal clinical trials and ensure collection of qualitative data, Joint Scientific Consultations with the EMA and HTA bodies will be offered in select cases, e.g. first-in-class therapies, high unmet medical need or expected high impact on healthcare systems. For therapies already in later phases, a PICO simulation of the treatment landscape can still guide the scope for data analysis or highlight potential need for complementing clinical trial data with, for instance, real world data or indirect treatment comparisons.

The consolidated PICO(s) that will be drawn up within the framework of the JCA is intended to encompass variations in treatment guidelines at the time of the scoping process. Ultimately though, the population(s) and comparator(s) considered relevant for subsequent national HTA processes will also depend on positioning of the therapy. For this reason, an early PICO assessment can also be important to ensure that trial data will allow for relevant stratifications in statistical analyses. 

In short, an early peek at the PICO to prepare for JCA is strongly advisable to make sure you have the right (C) and (O) for your (I), to reach your (P) without delay!