“Difficulties in access to orphan drugs in Spain are detrimental to patients, the healthcare system and pharmaceutical companies”

The director of Farmaindustria’s Access Department, Isabel Pineros, explains the situation of these drugs in Spain at a conference organised by the Kronos Foundation, the Hospital Clínico San Carlos and the Royal National Academy of Pharmacy

Treatments for rare diseases account for one in four approvals by the European Medicines Agency in 2022

In Spain, only half of the medicines authorised for the treatment of rare diseases are available, according to the report ‘Indicators of Access to Innovative Therapies’

Source: farmaindustria.es

In recent years, biomedical research on rare diseases has grown steadily. In the heat of the specific regulation of drugs to treat rare diseases, approved in Europe in 2000, the number of approved drugs has risen from eight to 147 available by 31 May 2023. That is more than 230 if one takes into account the loss of this orphan drug designation over time.

Nevertheless, the situation of rare diseases remains a challenge for the system. In Spain, there are approximately three million people suffering from a rare disease and only 5% have access to treatment. This was the subject of the third edition of Singulares, united for rare diseases, a conference organised by the Kronos Foundation, the Hospital Clínico San Carlos and the Royal National Academy of Pharmacy to discuss the importance of research and progress in the fight against these pathologies.

“One out of every four clinical trials carried out in Spain in 2022 was to treat a rare disease”, stressed the director of Access at Farmaindustria, Isabel Pineros, during her intervention in the debate, where she underlined that Spain is nevertheless far behind neighbouring countries in terms of the availability of new drugs. The latest W.A.I.T. Report (Indicators of Access to Innovative Therapies in Europe) establishes that in Spain 51% of the medicines authorised in Europe are financed. In France 79%, in Italy 82% and in Germany 90%.

The harm caused by barriers to access

“Spain is a leading country in clinical research, which offers patients the opportunity to have medicines available in trials earlier than in other places where they are not. In fact, it is even more valuable that 55% of this research is carried out in the early stages, when there is still a long way to go before the drug is authorised. And those access opportunities have to continue into the authorisation and funding phase,” Pineros explained. “Because the difficulties in the availability of orphan drugs generate not only a loss of opportunity for Spanish patients with rare diseases who need these drugs, but also damage to the quality of the healthcare system and harm to pharmaceutical companies, who see that the effort involved in researching and developing a new drug is not followed by swift access to the market”.

Although, according to the W.A.I.T. Report, the availability of orphan drugs has improved in recent years (from 44 to 51% of those authorised in Europe), the arrival of these drugs is delayed and the time from authorisation to funding in Spain was 786 days, more than two years.

“Particularly worrying is that Spain is, compared to France, Italy, Germany and England, the country with the most restricted availability. Forty-eight percent of orphan drugs that are financed do so with some kind of limitation on their use,” said the Farmaindustria spokeswoman.

In addition to Pineros, other participants in the colloquium included César Hernández, Director General of the Common Portfolio of NHS Services and Pharmacy; Daniel de Vicente, President of the ASMD Spain Association; Juan Carlos Valenzuela, Regional Pharmacy Coordinator of the Castilla La Mancha Health System; and José Luis Muñoz, neurologist at the Gregorio Marañón University General Hospital in Madrid.

The experts jointly stressed the importance of decentralising clinical trials, so that they reach more territories, as well as the need to accompany the patient with a rare disease and his or her family from the moment of diagnosis to treatment and subsequent follow-up. “From Farmaindustria we propose taking into account the particularity of their research and development, which includes a small number of patients and the lack of comparators as there are no therapeutic alternatives in many cases; and that once authorised, the processes are streamlined, that early access is prioritised and that the particularity of their nature is taken into account, which includes a small number of patients, the lack of comparators as there are no therapeutic alternatives in many cases, and the importance of having clear and predictable procedures and counting on the patient”, concluded Pineros.