There are 130 orphan drugs for rare diseases and tumors authorized by the European Medicines Agency (EMA) in 2021. Of these, 122 are already available in Italy: 80% is reimbursed by the National Health Service and the remainder 20% is in range C or awaiting negotiation. In terms of access, our country is second only to Germany, which leads the European ranking. Italy’s gap is mainly attributable to drugs that arrived on the market in the last year due to the different access mechanism in force in Germany, which does not provide for prior negotiation. This is what emerges from the VI Annual Report of the Orphan Medicines Observatory (Ossfor), presented this morning in the Senate, which takes a national and regional snapshot of the orphan drug market in our country as at 31 December 2021.
On the occasion of the event, which obtained the patronage of the Senate of the Republic, institutions and important experts in the sector discussed the topics covered in the 2022 report, such as the governance of rare diseases, post-marketing evaluation, the revision of the regulations for orphan and pediatric drugs and their use in trials, but also the legislation that regulates the sector of rare diseases and the National Recovery and Resilience Plan (Pnrr), focusing on the possible proposals that can offer solutions to the problems that patients with rare diseases they are facing.
The report – states an Ossfor note – is built on two main guidelines: the first relates to the data and their analysis, the second instead contains the proposals. The data relating to access is confirmed by the Patients Wait (Waiting to access innovative therapies) study, conducted by Iqvia in collaboration with Efpia (European federation of pharmaceutical industries and associations), the largest European study on the availability of innovative drugs and related access times of patients, which currently considers 38 countries (27 EU and 11 non-EU). In this year’s report, unlike the previous ones, an entire chapter is dedicated to regional expenditure data, produced by the Italian Medicines Agency (Aifa), and another – edited by Iqvia – analyzes availability and access times of orphan drugs in Italy compared to other European countries. The region analyzed this year is Basilicata, whose data can therefore be compared to those of Tuscany, Lombardy and Lazio.
“The VI Ossfor Annual Report sees the start of the collaboration between the Orphan Medicines Observatory and Aifa. This is an important recognition that strengthens the transformation process of Ossfor from a Study Center to a multi-stakeholder platform on orphan drugs and rare diseases – explains Francesco Macchia , coordinator of Ossfor – The VI Report is published in a phase marked by the search for a normalization after the solution of continuity represented by the Covid-19 pandemic peaks of 2020 and 2021: an interruption that we hope has at least triggered the conditions for a relaunch of the National Health Service and, therefore, for overcoming the critical issues that still afflict the sector of rare diseases”.
From the document “positive aspects emerge – underlines Macchia – Italy in fact, as can be seen from the high number of orphan drugs available compared to those authorized by EMA, confirms itself as a country with wide access to therapeutic opportunities. Observing the regional data, it emerges however that equity, understood as the possibility of uniform access conditions for patients with rare diseases to pharmacological therapies, and also of the economic impact on family budgets, is an objective that has not yet been fully achieved and certainly should represent one of the main points of attention of health policies in the sector, bearing in mind the important opportunity represented by the Pnrr”.
The analysis carried out by Aifa shows that the expenditure incurred by the NHS for orphan drugs absorbs around 8% of the total public pharmaceutical expenditure and it is confirmed that the price of orphan drugs is a function of sales volumes, with an exponentially negative trend : the lower the number of people who will be able to use it, due to the rarity of the disease, the higher the price will be.
Furthermore, the Ossfor report highlights a substantially linear growth in the average expenditure for Ddd (defined dose per day, ed), which at least in part can be attributed to the increase in drugs for ultra-rare molecules, which have low sales volumes . The region with the highest expenditure for Ddd is Lombardy with 203.50 euros, followed by Emilia Romagna (199.43 euros) and Umbria (193.03); at the other extreme we find Friuli Venezia Giulia (159.68) and Pa di Trento (160.90), followed by Abruzzo (166.50).
The document, presented today in the Sala Zuccari of the Senate, was produced by Ossfor in collaboration with Aifa, the Rare Disease Alliance (Amr), Iqvia, Pharma Value, the Rare Diseases and Orphan Drugs Working Group of the Italian Society of Pharmacology (Sif) , Italian Society of Hospital Pharmacists (Sifo) and Sperimentazionicliniche.it, with the unconditional contribution of Amicus Therapeutics, Amryt Pharma, Chiesi Global Rare Diseases Italia, Janssen, Kyowa Kirin, Roche, Sanofi, Takeda and Ucb. The one with Aifa is an unprecedented collaboration of which the VI Ossfor Report is the first, important, result.
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