Spain takes, on average, two years to approve a drug for a minority disease
There are 146 orphan drugs approved by the European Medicines Agency. Spain has only 81 in its catalogue. It has rejected funding for 25 and is studying 17 others.
ondacero.es
There are two main anxieties for those suffering from a so-called rare or minority disease and their families.
One is to name the disease, and it is not easy. The World Health Organisation estimates that there are more than 7,000 and the number is growing all the time. It takes an average of four years for patients with one of these diseases to be diagnosed. In 20% of cases it takes more than 10 years. Many are not diagnosed at all.
The second of the anxieties has to do with treatments. Only 6% of rare diseases have any kind of treatment and only 20% of these diseases are being investigated. The small number of patients works against them, both in the research field (they are not profitable) and in the clinical field (it is complex to group a sufficient number of patients to start a clinical trial) as well as in the field of funding.
Patients should not have to wait so long, they have no alternative.
In Europe, the European Medicines Agency maintains marketing authorisation for 146 orphan drugs. Of these,” Isabel Pineros, Director of Farmaindustria’s Access Department, told Onda Cero, 123 have shown interest in being marketed in Spain, but not all of them are funded. 81 are included in the SNS funding, 25 have received a resolution of non-funding and 17 are in the process of being studied”. The downside,” he adds, “is that these 21 new medicines took an average of two years to be included in the funding.
“The patient should not have to wait so long, they are medicines that have no alternative”
This delay has led all those involved – patients, doctors, industry – to call for an improvement in the approval procedures. The Law on Guarantees and Rational Use of Medicines and Medical Devices has been in the process of being amended since the last legislature. It aims to shorten times and incorporate both patients and clinicians in the approval committees. Farmaindustria is also calling for “a proposal for accelerated financing, early access. We need to work on a process that is predictable, that has clear timelines and includes patients and clinicians at all times, who in the case of rare diseases are the great experts who need to be listened to,” explains Pineros.
Spain, a research powerhouse
Good news comes, and this is no exception, from research. Spain is a leader in clinical trials, and 23% of the thousands initiated each year in Spain are aimed at minority diseases. In recent years, moreover, the percentage of research in early stages of the disease has risen to 51%.
It is estimated that in Spain there are around three million patients with a rare disease (with fewer than five cases per 10,000 people). Approximately 80% are caused by an alteration in the hereditary material and 65% are severe and disabling. Half of these diseases are life-threatening.
