The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories(AELMHU) publishes the results of its Access Report 2023 in which it highlights how for the first time in the last 5 years, Spain finances more than half of the orphan drugs authorized for marketing in the European Union.
For more than 8 years, the association has been publishing a report analyzing all orphan drugs with a trade name, which have a European orphan designation in force as of December 31, 2023.
In 2023, 21 new orphan drugs were financed, 12 more than in the previous year, bringing the total number of orphan drugs financed by the National Health System (NHS) to 78, out of 147 with marketing authorization from the European Medicines Agency (EMA), i.e. 53% . Despite this, it should be borne in mind that almost half of these new products are financed with restrictions and 100% have obtained a conditional price: 5 have monitoring conditions, 12 are subject to a sales and price review, 1 is subject to a maximum cost per patient and 9 have an expenditure ceiling, among other conditions.
It is also important to highlight that there are still a total of 45 orphan drugs that are not funded, corresponding to therapeutic areas such as oncology at 40% and metabolic at 24%, the main areas without funding. In addition, 49% of these products have been awaiting funding for more than 3 years . The criteria for not financing them are mainly two: for reasons of rationalization of public spending in pharmaceutical provision and for their therapeutic and social value and incremental clinical benefit, taking into account their cost-effectiveness ratio.
In this regard, no advanced orphan therapies were approved for funding last year either, something that already passed in 2022.
At the close of the report, Spain only had 123 orphan drugs with a National Code out of the 147 that were authorized for marketing in the EU. This means that 24 authorized orphan drugs did not reach our country (16% of those authorized). This is the same percentage as last year, which is the highest figure in the last 5 years.
One of the determining factors that could lead to innovation not reaching EU Member States, once a marketing authorization has been obtained, are delays in funding times. In 2022, funding for orphan drugs reached an average waiting time of 3 years, which could partly explain why 16% of the drugs authorized in Europe have not arrived by 2023.
However, the average time to funding in the last year has been reduced to 23 months, 11 months less than in 2022. This undoubtedly represents significant progress compared to the delays that have occurred in recent years. However, it is necessary to continue this momentum in order to reach the 2019 funding times, whose average waiting time was at 14 months, or even reduce them.
According to María José Sánchez Losada, president of AELMHU, "during 2023 and despite the intensity of the elections, AELMHU has intensified the dialogue with the different public administrations to raise awareness of the slowdown and decline in access to orphan drugs in recent years. The improvements in both funding and waiting times during the last year have brought about a significant change which, from our Association, we value positively and we hope that this will become a trend in the coming years". In this sense, the Association highlights the joint effort of all the actors in these changes, led by the Ministry of Health. Ministry of Health and reinforce the organization's commitment to "continue working and providing recommendations on the evaluation, access and financing of orphan drugs with the agents involved in improving the quality of life of patients with rare diseases", concludes the president.
You can consult the executive summary of the report here and the infographic in this link.
