Access to orphan drugs in Spain: 49 unfunded drugs and 26 months of waiting
The figures seem to have improved in recent months, but they are still far from being the desired ones. Access to orphan drugs in Spain continues to be an issue that, despite being considered a priority, is advancing slowly.
It is enough to analyze the data from the latest quarterly report of the Spanish Association of Orphan and Ultra-orphan Drug Laboratories (Aelmhu), which shows the lights and shadows regarding the availability of these drugs in our country.
With data updated as of August 31, the document reveals that although 85% of orphan drugs authorized in the European Union arrive in Spain and have a national code (CN), only 52% of them are financed. Along with this, it is reflected that 49% of the drugs have been financed with restrictions. That is to say: with limited indications or with unfunded indications.
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From these data it is also clear that there are 22 medicines that have marketing authorization in the EU, but not with a national code in Spain: 15% of the total.
The figures referring to these last four months are an example of how the trend is improving, but not at the pace that would be desired: between the months of May and August, 2 new national codes were assigned and the financing of 9 new orphan drugs was approved, until reaching 126 orphan products with national code and 77 financed products.
– There are about 202 products with orphan designation and trade name. Of which, 148 orphan drugs have a marketing authorization (MA).
– Likewise, of this figure in Spain, 126 have a national code and 77 are financed by the National Health System (SNS).
– From 2020 to this year, the time elapsed between the designation of the national code and incorporation into the SNS has been reduced from 33 to 26 months.
Average waiting time
Regarding the average waiting times between obtaining the national code for a drug and its incorporation into the SNS, the report points out that the figure “remains unchanged”, being the same as that of April 30: 26 months. If this figure is compared to last year, which was 34 months, the situation seems to have improved; but not if compared to 2021, in which the average waiting time was 24 months.
Along with this deadline, Aelmhu highlights that there are 49 orphan drugs with a national code not financed by the health system: 28 not financed by resolution and 21 under study or without a request for financing.
Furthermore, a notable fact is that of those 49 drugs still without financing, 21 of them had the NC before 2020. This is a clear example of why there is an urgent need to improve in this area.
Regarding this, and making a classification by therapeutic areas, the majority of unfunded orphan drugs fall within the oncological category (35%), followed by metabolic (27%), nervous system (8%), hematological (6%), anti-infectives (6%) and hormonal preparations (6%). The remaining percentage is divided between dermatological, musculoskeletal, immunomodulatory, sense organs and others.
- 35% oncological
- 27% metabolic
- 8% nervous system
- 6% sense organs
- 6% hematological
- 6% anti-infectives
- 4% immunomodulators
- 2% musculoskeletal
- 2% dermatologists
- 2% hormonal preparations
- 2% others
In reference to advanced therapies with orphan designation, Aelmhu's quarterly report reflects that, as of August 31, 2023, there are 15 treatments with marketing authorization, 14 with a national code and 5 financed by the SNS.
Regarding the sources used to prepare this report, the institution indicates that they are the European Medicines Agency (EMA), the Community Registry of Orphan Medicines, the Spanish Agency for Medicines and Health Products (Aemps) and the Ministry of Health. .
From the association
Taking stock of the numbers provided by this document, Aelmhu points out to this newspaper that although “an improvement is perceived in several of the indicators” such as, for example, the number of new drugs financed by the SNS, there are others, such as waiting time until financing, which remains “very high”.
“Knowing the evolution of the indicators, at Aelhmu we are committed to making the reduction of access times a priority; advocating to request, as we have been doing for years, a change of model for the evaluation and financing of orphan and ultra-orphan drugs. In other words: an agile, predictable and transparent financing and evaluation mechanism that, in a manner agreed upon by all the agents involved, takes into account the characteristics of this type of medicines; allows a reduction in waiting times with the common objective that patients have early access to treatments, and consolidates itself as a good tool to reduce uncertainty and restore Spain's attractiveness for the arrival of innovation already approved in the European Union,” the association explains to CF.
For this reason, they advocate that, beyond the numbers, we continue to “work together” to improve the availability of treatments for patients with a rare disease, reduce waiting times and ensure that research and innovation arrive as soon as possible. , eliminating bureaucratic access barriers.
“In short, within the change of model that we request, we must always keep in mind that for patients with a rare or ultra-rare disease, in most cases, the equitable availability of an innovative drug is the only therapeutic alternative to stop his illness”, they conclude from Aelmhu.