Orphan Drug market, how is Italy doing? The latest OSSFOR Booklet paints a favourable picture for the Italian landscape.

By Marco Sancandi, PhD; Analyst

On the 31st of January 2023, the 6th joint report from the Orphan Drugs Observatory and the Italian Society of Hospital Pharmacy and Pharmaceutical Services for Healthcare (OSSFOR) on the orphan drug (OD) market in Italy was published [1].

Of the 130 ODs approved by the EMA for rare diseases and cancers, 122 are currently available in Italy, with 80% reimbursed by the National Health Service (SSN). The remaining 20% are in the C-band or awaiting negotiation, placing Italy second only to Germany in terms of access to ODs. This data is also confirmed by the patient W.A.I.T. (Waiting to Access Innovative Therapies) indicator survey conducted by IQVIA in collaboration with EFPIA (European Federation of Pharmaceutical Industries and Associations).

Total spending on ODs amounted to €1.53 billion in 2021 (an increase of 9.4% compared to 2020), representing approximately 8% of the total SSN budget. In addition, 8.4 million doses of ODs were prescribed (+3.7% compared to 2020), representing 0.03% of total drug consumption. The trend, both in spending and consumption, has been consistently increasing since 2013 and is in line with the trend seen in total drug spending. The therapeutic areas with the highest spending are lymphoma, myeloma, and genetic diseases (37.9% and 13.6%, respectively), which also confirms last year’s trend.

The use of drugs that lost OD status in 2021 (n=4) was reported to have remained stable, suggesting that access to OD therapies is not hindered by the loss of OD status with no meaningful differences in use as a result of “exiting” the OD list.

It is interesting to note that while drug spending and consumption are increasing, access time is decreasing. The OSSFOR report noted that, unlike other European countries where approval procedures are lengthening. In Italy the time between EMA approval and AIFA pricing and reimbursement decision for ODs has decreased from 24 months (2012/2014) to 20 months (2018/2021). However, OD’s access time might change over the course of this year. With the merging of the scientific (CTS) and pricing (CPR) AIFA commissions into a single commission within the first half of 2023, it will be fundamental to monitor whether this improved trend of faster access times will be reversed. In fact, the new single commission will have 10 members, half the total number of the CTS and CPR. Therefore, doubts have been raised over the likely timelines given the workload this new AIFA faces.

With the merging of the 2 commissions, Italy would still be considered as a favourable market in Europe for OD manufacturers. Companies planning an Italian launch should always keep in mind AIFA’s three key criteria of therapeutic need, added therapeutic value, and quality of evidence to demonstrate the innovativeness of their product. In the case of uncertainties that may arise during the assessment, Italy also provides an opportunity to use AIFA registries to gather additional evidence post-authorisation and/or to pursue risk-sharing contracts.

At P4A we constantly monitor changes in the OD market and we will continue to do so in order to best advise our partners on how to react, plan, and adapt to the changing OD market. Contact P4A to learn more about our services to support with determining ideal access pathways and follow us on LinkedIn to remain up to date on how to seize opportunities in an ever-changing space.


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