Shorter approval times for medicines after Aifa reform

The timing of drug price negotiations is one of the parameters on which the performance of regulatory agencies is assessed, perhaps the one with the greatest emotional impact when considering the expectations and hopes of doctors and patients. The numbers giant Iqvia periodically publishes statistical data comparing different countries. Given the relevance of the subject, the statistical analysis adopted in these reports is very simplified: for each procedure, a start date is considered (in the EU area, the publication of the drug approval in the European Journal), and a final date (for us in Italia, the publication of the price and reimbursement determination approved by the Aifa Board); the average (standard deviation) and median of the duration in days of the procedures are then calculated.

This simple descriptive statistics approach leaves out of the analysis those procedures that have started but are not yet completed at the time of analysing the data. To overcome this limitation, my collaborators and I came up with the idea of applying the time-to-event statistical technique to this type of data. This approach is very common in medicine in the field of oncology, where the event being measured can be, for example, the progression of the disease, and extending the time to the event becomes a clear indicator of the effectiveness of a treatment.

In our case, the event to be measured becomes the allocation of the price to the new drug. This approach has the advantage that it can also include procedures that have not yet been priced at the time of the analysis. When this project started, we had at our disposal a database provided by the company Intexo, which included all drugs approved in Ema in the period 2021-23, and also the possibility of comparing Aifa's performance before and after the reform that came into force in March 2024. Applying time-to-event analysis to this sample, we found that the duration of negotiations, calculated from the date of publication in the European Journal to the price determination in Italia, fell from 483 days before the reform to 388 days after the reform, a reduction of 95 days. Considering only the time between the submission of the price and reimbursement application to Aifa by the pharmaceutical companies and the conclusion of the procedure, thus net of the time attributable to the companies, the negotiation time was 188 days shorter in the post-reform period.

Sensitivity analyses were also conducted, considering time periods in which Aifa's pre-reform operations could be reflected in performance in the immediate post-reform period, and in each case we confirmed a reduction in negotiation times. Now these data are being published in the journal Global and Regional Health Technology Assessment, and we are very interested to see the impact on industry experts. We are fully aware of the media value of this data, but we would like to emphasise the fact that the primary objective of this study was the validation of a new methodology for analysing price negotiation times, to be proposed as an alternative to the simple descriptive statistics still in use today, and the results that have emerged have a case-study character for us, and are therefore entirely incidental.

* Full Professor of Pharmacology, Director of the Pharmacology Section of the Department of Translational Medicine and Surgery, Faculty of Medicine and Surgery, Catholic University of the Sacred Heart in Rome