Time to market is improving nationwide. In terms of access, our country is second only to Germany, where availability for patients is almost immediate
Those suffering from a rare disease (so defined because it affects a small number of people, which does not exceed 5 cases out of 10,000 in the European Union) in the majority of cases must deal with thelack of specific therapy. In 2021 in our country the availability of orphan drugs, i.e. those intended for the treatment of these pathologies, concerns 122 molecules, equal to 94% (130) of all those authorized up to that moment by the European Medicines Agency (EMA). If you take into consideration i 57 products approved by Ema between 2017 and 2020a January 2022 Italy among the top of the class, and precisely in second place, tied with Denmark, with 43 orphan drugs available to patientsafter Germania (54). The gap justified by the fact that in Germanyunlike the rest of Europe, the price of these products is not subject to prior negotiation between the German regulatory agency and the pharmaceutical industry and therefore their almost instant access explains Francesco Trotta, manager of the pharmaceutical expenditure monitoring office and relations with the Regions of the Italian Medicines Agency (Aifa).
This photograph was taken from the sixth annual report of the Orphan Drugs Observatory, founded in 2016 by the Center for Applied Economic Research in Health and the Observatory for Rare Diseases, which AIFA also contributed to drafting for the first time.
The investigation
The survey shows that the time to market of orphan drugs improves nationwide since they got the green light from the EMA: in the period 2012-2014 they expected an average of 24 months while in the three-year period 2018-2021 20 months. In line with the European average. The process after the European authorization provides for the implementation of the decision, the definition of the price and the conditions of reimbursement by the national regulatory agency, through a decision published in the Official Journal.
Good news also on the expenditure front of the National Health Service for orphan drugs (80% of the 122 molecules reimbursed by the state): from 1.4 billion euros in 2020 to 1.53 billion, equal to 6.4% of all public pharmaceutical expenditure. And it almost realigned itself to that carried out in the pre-pandemic year (2019), of 1.55 billion. Consumption also increased: from 8.1 daily doses to 8.4 (in 2019 they were 10.1). Since 2013, the trend of both spending and consumption has been growing, underlines Trotta.
The slowest Regions
The trend of orphan drugs is encouraging. The really sore point is the social and health care of rare patients, which remains deficient and uneven throughout the territory, comments Francesco Macchia, coordinator of the Observatory on orphan drugs.
At the regional level – underlines Trotta – theaccess to orphan drugs both for access times and for the overall homogeneous number of drugs available. From the date of transmission of the determination in the Official Gazette to the drug being made available to the patient in the area, in the period 2016-2021, according to the report, an average of 4 months pass. The slowest Regions, considering the median time for dispensing the first pack (the national median of 113 days), are Molise (455), Valle d’Aosta (372), the Autonomous Province of Trento (215) and Basilicata ( 167).
Interest on the rise
But – clarifies the Aifa manager – the regional differences I am attributable to different needs, determined by the resident population in the area, and therefore by a greater or lesser incidence of cases of rare diseases, and by the presence or absence of specialist treatment centres. On orphan drugs, concludes Trotta, theincreasing interest from pharmaceutical companies. Today there are 53 companies in Italy that sell at least one molecule for the treatment of a rare disease and the related total turnover is 1.4 billion. But only 13 companies exclusively produce orphan drugs and 33, that is, the majority, produce only one.
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February 2, 2023 (change February 2, 2023 | 2:04 pm)
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