Italy shakes up early access programmes

By Richard Wang, Senior Analyst & Andrea Bernardini, Senior Analyst

Email: [email protected] [email protected]

On 10th of November, the Italian Medicines Agency AIFA announced that it will be temporarily suspending its orphan drug research and access fund ‘fondo AIFA 5%’ for a re-think of its criteria and funding. The fund, set up in 2003, was created with contributions from pharmaceutical companies corresponding to 5% of the annual expenses for promotional activities. 50% of the fund is dedicated to reimbursing rare disease treatments that are awaiting commercialisation at a price set by the manufacturer through the named patient early access programme known as Law 326/2003. The other 50% is used to support independent research, drug information programmes and pharmacovigilance costs.

The reason for this suspension comes about as the amount of resources allocated annually to the fund has reduced over the past decade, from €20 million to around €10.5 million in 2021, resulting in demand overtaking resources. This temporary suspension would allow for an extensive review and simplification/optimisation of the system, with a priority to redefine the rules and criteria used. Indeed, the suspension of the fund comes amidst a flurry of other updates from the Italian market, revealing further the potential direction of the overhaul for early access programmes in Italy.

With the recent announcement that Italy’s hospitals have overspent €1 billion above the drugs budget in the first half of 2021, it seems clear that Italy’s drug reimbursement spending is “chronically underfunded” according to Massimo Scaccabarozzi, head of the lobby group Farmindustria. However, Italy plans to alleviate some of the pressure by increasing the overall funding for drugs from 14.85% to 15.20% of the National Health Fund (FSN) in 2022, to 15.35% in 2023 and to 15.50% in 2024, translating to a €2 billion year-on-year increase (as discussed here). With this draft budgetary plan, signalling an overall increase on NHS expenditure, it seems unlikely that fondo AIFA 5% will be restricted, but more in how much value is squeezed from it.

Another change is the likely merging of AIFA’s scientific (CTS) and pricing (CPR) committees which conducts the HTA and pricing assessment of novel treatments respectively (discussed previously here). Speculation has been circulating for quite some time coupled with the recent extension to the time in office of current CTS and CPR members may further cement the expected restructuring. The merging of the two committees could streamline market access, resulting in shorter waiting times for the final pricing and reimbursement decisions in Italy. However, in terms of early access through Law 326/2003, which is currently assessed only through the CTS, this could translate to both a clinical and economic evaluation being carried out, with the potential of price negotiations. This would not come as a surprise given the foreshadowing changes in March 2021 to the similar cohort early access programme of Law 648/96, where price negotiations have started taking place.

Piecing the above changes together, when the fondo AIFA 5% is refined and resumed, applications could face much more scrutiny and pressure, especially from an economic perspective. By involving price negotiations, whether that may be in the form of direct discounts or paying back the difference in negotiated price after launch (as seen in France), would rein in public health spending and allow for greater resource utilisation.

This latest announcement to suspend the fund comes as a major blow for patient access in Italy, at least temporarily. Patients faced with a high unmet need and requiring access to non-approved and/or non-reimbursed treatments would now have to rely on the other access pathways including Law 648/96, or Compassionate Use (Law 94/98). However, these alternatives are inherently different from Law 326/2003, as law 648/96 provides access opportunity before EMA approval for a cohort, and has longer timelines, while law 94/98 is for off-label use and is paid for by the patient or hospital. Previously, Italy has been considered a good opportunity in Europe for paid for early access of treatments by manufacturers as a source of early revenue. This view may change though, if pricing negotiations are on the horizon, accompanied with the impact that this may have on timelines. It will be interesting to see how the rules and criteria will be specifically defined in the future, but manufacturers should stay alert in order to best react and adapt to the changing Italian market.


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