New and promising horizon in addressing rare diseases – it is estimated that there are more than 7,000 – that affect three million Spaniards. For the first time in five years, Spain finances more than half of medicines orphans -innovative treatments for minority ailments- authorized by Europe. “The results achieved in 2023 represent a considerable improvement if we compare them with those of previous years,” highlights El Periódico de España, from the Prensa Ibérica group, María José Sánchez Losadapresident of the Spanish Association of Orphan and Ultra-orphan Drug Laboratories (AELMHU).

The association has just published its annual report – weeks before the commemoration of World Rare Disease Day, on February 29 – which analyzes all medicines with trade names that have the European orphan designation in force as of December 31, 2023. The situation of these drugs in Europe and their availability for Spanish patients is detailed. At the European level, indicates the report, there has been a decrease both in the number of new orphan designationsgoing from 29 in 2022 to 19, as in the number of new commercial authorizations, which have gone from 24 in 2022 to 12.

Best results

The good news is that, during this time, Spain has improved its access data, increasing the number of drugs financed and reducing the time to pay for them. In 2023, 21 new orphan drugs were covered, 12 more than in the previous year, and a total of 78 financed by the National Health System (SNS), of the 147 that have marketing authorization from the European Medicines Agency (EMA). That is, a 53%.

Of the total of 21 new drugs financed, 24% are immunomodulators19% are metabolic, another 19% are oncological, 9% affect the nervous system and the remaining 29% belong to other types of therapeutic areas.

Change of scenery

If these data are compared with previous years, it is the first time in five years that Spain finances more than half of the orphan drugs authorized by the European Union. “It must be remembered that, in recent years, the data showed a wide gap between treatments that were authorized in Europe and their effective access in Spain, in addition to significant delays in financing times, especially in 2022″, indicates Sánchez Losada.

The president of the association highlights that “greater awareness of rare diseases by the public administration” has been generated.

Over the past year, the entity has intensified dialogue with the different public administrations. The president of the association highlights that “greater awareness on rare diseases by the public administration“. This, added to “the commitment and flexibility of all the parties involved” has led to an improvement in its access and in their financing times.

The average time for financing in the last year has been reduced to 23 months, 11 less than in 2022

“If we evaluate the results obtained in 2023, one could say that the improvements achieved are asymmetrical. On the one hand, there has been a significant growth in the number of new orphan drugs funded. On the other hand, the average financing time has been reducedgoing from almost 3 years in 2022 to 23 months in 2022 (eleven less than that year)”, he adds.

Prices’ check

Of course, the association that brings together the laboratories clarifies: almost half of these new products are financed with restrictions. In addition, 100% obtained a conditional price: 5 have follow-up conditions, 12 are subject to a sales and price review, one is subject to maximum cost per patient and 9 have a spending ceiling, among other conditions.

Because, remembers the AELMHU representative, the scenario has not been easy in recent times. “In Spain, as in other countries, the National Health System was strained due to the pandemic and without a doubt, this is one of the reasons why it occurred. an increase in financing times“, indicates Sánchez Losada.

However, it continues, according to different indicators from the European Commission and the Organization for Economic Cooperation and Development (OECD), it can be seen in general terms “adequate progress” in the functioning of our health system. Likewise, it highlights that, although Spain is currently slightly below the European average in health spending, since 2014/15 public spending on health “has not stopped increasing and we understand that with European funds this trend will continue to rise.” .

The immediate future

If things continue this way, In the short term, the financing terms could be adjusted to the times of 2019, which were around 14 months on average, although taking advantage of the current momentum, we should be more ambitious and get them under one year“Sanchez Losada abounds.

Because he considers that, despite the moderate optimism that laboratories are now experiencing, take almost two years on average to finance an orphan drug “is not optimal, since rare diseases have hardly any treatment options or alternatives. Furthermore, They are pathologies whose diagnosis is complex and it takes an average of 5 years to achieve, generating in patients, and their entire environment, enormously difficult situations“.

Advanced therapies

Regarding the number of funded advanced therapies, in 2023, as in 2022, none have been subsidized. In these two years, explains the president of the association, Spain has now had six treatments of this type.

The laboratories ask to develop a differentiated financing model for orphan drugs, which recognizes their specificities

“We believe that the next step to continue progressing and to prevent the achievements achieved from remaining a mere one-time event, is to develop a differentiated financing model for orphan drugs, that recognizes its specificities and its characteristics and that it incorporates mechanisms to streamline accessincluding possible advance agreements, so that patients suffering from rare diseases can have access to these therapeutic innovations as soon as possible.

AELMHU reminds that there are still a total of 45 orphan drugs that are not financed, which correspond to therapeutic areas such as oncology – 40% – and metabolic areas – 24%. Furthermore, 49% of these products have been waiting for that subsidy for more than three years. Of the medicines authorized by the EMA, 16% They had not arrived in Spain in 2023.