The director of Farmaindustria’s Access Department, Isabel Pineros, participates in the XVI International Congress on Rare Diseases: “In recent years we have seen great advances in orphan drug research, but much remains to be done to ensure that this benefit reaches all patients who need it when they need it”.
Farmaindustria has presented a document of proposals to improve patient access to treatments for rare diseases, as well as a specific pathway to accelerate the arrival of certain drugs in emergency situations.
In 2022, the European Medicines Agency authorised 16 innovative medicines (with a new active substance) for rare diseases, 39% of all new approvals. This means that one in three of the new medicines is indicated for these pathologies.
Rare diseases are increasingly the focus of the innovative pharmaceutical industry, which in Spain promotes 96% of clinical trials. Moreover, one in four trials conducted in Spain in 2022 (234) were for these pathologies, according to the Spanish Clinical Trials Register (Reec).
“In recent years we have seen great advances in orphan drug research, but much remains to be done for this benefit to reach all patients who need it when they need it,” explained the director of Farmaindustria’s Access Department, Isabel Pineros, at the XVI International Congress on Rare Diseases, which is being held this Wednesday and Thursday in Murcia organised by the D’Genes Association and the Catholic University of Murcia.
Pineros referred to the year 2000, when the European Union passed the Orphan Drug Regulation, which for the first time stimulated the development of treatments for rare and mostly hopeless diseases. Since then, up to 240 drugs in this category have been approved.
“Twenty-five percent of clinical trials last year were for rare diseases and 58% corresponded to early stages of research, an enormous advance and opportunity for Spanish patients who participate in them, as they can access drugs that are often the only option,” said Pineros. It is estimated that there are three million patients with rare diseases in Spain, of which only 5% have treatment.
More than two years for new drugs to reach patients
While more and more drugs for rare diseases are authorised, the obstacle lies in reaching the healthcare system. According to the latest Access to Innovative Medicine Indicators report (WAIT 2022), only 51% of drugs for rare diseases are available in Spain and the average waiting time for patients is 786 days, more than two years. “The absence of an agile and efficient procedure encourages the use of alternative, individualised routes that were not designed for this purpose, such as medicines in special situations, which results in more uncertainty and sometimes more inequity, including territorial inequity,” said Pineros.
Farmaindustria’s Director of Access also pointed out that the professionals involved in clinical research in our country – a leader in clinical research – find that once completed, they cannot use the drug until the decision on funding is made, which means a loss of opportunity for patients with rare diseases who need these drugs, damage to the quality of the healthcare system and a brake on the pharmaceutical companies that have promoted clinical research in our country.
In this regard, Pineros explained the proposals to improve access to orphan drugs that Farmaindustria has developed based on the need to give recognition to their peculiarities that allow patients, in many cases with no other treatment alternative, to access new drugs.
In addition to this, Farmaindustria has recently presented a proposal to improve early access to medicines that cannot wait and whose benefit is more relevant, a situation that frequently occurs in this type of disease. “It is a two-phase procedure that allows the drug to be available as soon as it receives European authorisation, with an accelerated financing process and, therefore, that they can reach the patients who need them in conditions of equity for the entire NHS,” said the Farmaindustria spokeswoman.
This requires collaboration between all stakeholders: administration and industry, but also including patients and clinical experts in the disease, which is even more relevant for these pathologies.
The Farmaindustria spokeswoman spoke about the difficulties of access to orphan drugs together with the director of the Spanish Association of Orphan and Ultra Orphan Drug Laboratories (Aelmhu), Marián Corral López; the head of the Pharmacy Service of the Vírgen Macarena University Hospital in Seville, Miguel Ángel Calleja, and with the moderation of the director general of Planning, Pharmacy and Health Research of the Region of Murcia, Jesús Cañavate.