EUnetHTA 21 PICO exercises provide further insights into JCA requirements

Concrete effort estimation for the JCA Submission Dossier is now possible

Fri, 2023 / 09 / 29
The starting point for the Joint Clinical Assessment (JCA) of health technologies according to regulation (EU) 2021/2282 is the scoping process, in which the research questions of the assessment are defined in the form of PICO schemes. A central question here is how many different PICO schemes are to be addressed by pharmaceutical companies in a European benefit dossier and how reliably these PICO schemes can be anticipated even before the scoping procedure.

„JCA without HTD submission" – Behind this name hides the deliverable of EUnetHTA 21, which contains the so-called PICO exercises. These exercises were recently published as a substitute, since no actual JCA submissions for medicinal products were made by pharmaceutical companies within the framework of EUnetHTA 21. Consequently, these exercises were conducted using exemplary drugs without the participation of the associated companies. Below, we have summarized the key findings of the PICO exercises.

The PICO exercises were performed on the three products POMBILITI, PLUVICTO and EBVALLO, which already had a positive CHMP opinion. The purpose was to test the D4.2 Scoping Guideline, which contains the methodological principles of the scoping process, on the selected drugs and to adapt it according to the "lessons learned". Accordingly, the PICO schemes were first defined by the individual member states and then consolidated using the current guideline:

  • PLUVICTO (177Lu lutetium vipivotide tetraxetan, oncology drug) for pretreated patients with a progressive form of prostate cancer: 6 consolidated PICO regimens (2 for the label population and 4 for subpopulations) with 16 endpoints (participation of 8 member states).
  • EBVALLO (tabelecleucel, ATMP and orphan drug), a T-cell immunotherapeutic for patients with Epstein-Barr virus positive post-transplant lymphoproliferative disease: 5 PICO regimens (1 for the label population and 4 for subpopulations) in 16 endpoints (participation from 10 member states).
  • POMBILITI (cipaglucosidase alfa, orphan drug) as an enzyme replacement therapy for patients with the late-onset form of Pompe disease: 9 consolidated PICO regimens (4 for the label population and 5 for subpopulations) with 16 endpoints (participation from 10 member states).

For the exemplary scoping procedures within oncology and advanced therapy medicinal products (ATMPs), the identified number of PICO schemes is in line with EUnetHTA 21's previous estimate of an average of about 6 PICO schemes per JCA procedure. SKC has already considered in detail the expected number of PICO schemes in the scoping process for additional oncology drugs in the form of a PICO simulation.

As expected, rare diseases may result in a large number of PICO schemes for the European benefit dossier. This may be due to the heterogeneous standard of care in Europe, particularly for orphan indications. Interestingly, no off-label therapies were named as comparators in the PICO exercises – however, according to the D4.2 Scoping Guideline, it cannot be excluded that such comparators will be considered in the future.

In addition to the recently published deliverable D5.1 Submission Dossier Template, there is now the possibility of a first concrete estimation of the scope and effort for the European benefit dossier. The high number of required endpoints results in a large amount of data analyses. Further, it cannot be estimated how many PICO schemes will actually result if more than just 8 to 10 member states participate in the scoping process. Finally, it remains to be seen how the guidance document on the scoping process will be adapted based on the lessons learned in the PICO exercises.

We will continue to keep you informed about these and all other relevant developments regarding the EU HTA process. If you are interested in gaining access to European advice via the G-BA regarding the expected PICO schemes, among other things, or if you are looking for a strategic partner to assess risks and opportunities for the EU HTA process of your product at an early stage, please do not hesitate to contact us. We are the market access special forces.


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