Only 5% of rare diseases have treatment. Access to new medicines can save the lives of many people; however, less than half of the drugs approved in Europe are available in Spain.
Around three million Spaniards live with some of the different rare diseases that exist, pathologies that mostly (95%) lack treatment.
In these cases, the arrival of an innovative drug is often the only hope, especially when the pathology is serious.
Los orphan drugsintended to treat these rare diseases, were recognized in Europe in the year 2000.
Just last year the European Medicines Agency (EMA) approved 24 drugs for rare diseases, the 41% of the total number of new active ingredients authorized during the year.
However, in Spain only the 44% of orphan drugs that are approved in Europe, according to the latest annual report on indicators of access to innovative therapies in Europe.
In this context, Farmindustria has released the document Proposals for improving patient access to orphan drugs.
A document that contains sweet specific measures to ensure that Spanish patients with rare diseases have the maximum opportunities available for treatment, in line with Europeans.
“Patients with rare diseases cannot wait to have all the evidence to finance a drug that already has a European authorization with a favorable benefit-risk balance,” says the Director of the Farmaindustria Access Department, Isabel Pineros.
Spain, in inequality
As Farmaindustria points out, in Spain orphan drugs follow the same evaluation and authorization processes as the rest of the drugs, unlike other European countries where their specific circumstances are already taken into account, such as the number of patients affected or the lack of of alternative treatment, among other factors.
This generates that in Spain there is inequality in this type of treatment, limiting the access of the people who need it.
In fact, a study published in Journal of Rare Diseases in 2020 concluded that the 75 % of 32 countries in Europe, Canada and New Zealand analyzed had a different approach to these drugs and recognized that they could not be evaluated conventionally.
“We are facing a situation of clear inequality with respect to patients from other countries such as Germany, Italy or France. We need to specifically address access to orphan drugs to achieve a good position in the European context, as we already are in clinical research”, explains Pineros.
Image taken from the document ‘Proposals for improving patient access to orphan drugs’ by FarmaindustriaTwelve proposals to improve access
Thus, from Farmaindustria they list twelve proposals to increase and accelerate the arrival of new treatments to patients with rare diseases:
- To establish a early dialogue with the Administration once the drug has received a favorable report from the EMA.
- And accelerated financial assessment procedure.
- Create a specific external advisory committeewhich includes scientific societies, patient associations and experts of recognized prestige in pathology who analyze the circumstances of each drug.
- Adopt concrete criteria of funding for orphan drugs.
- Establish criteria for the definition of longitudinal models of financing.
- understand and accept the specificities and limitations of orphan drugs in terms of the level of data that, by their very nature, they can provide.
- make a first therapeutic evaluation and later the economic evaluation.
- Regarding the Therapeutic Positioning Reportsit would be recommended that if an economic evaluation is made, it be carried out once the financing price has been determined.
- Improve information collection systemswith further optimization and automation that takes advantage of the opportunities of the future Digital Health Strategy.
- A new approach that allows improve the evaluation and financing process of orphan drugs, taking into account the boom in research in this field.
- An orphan medicinal product must be subject to a special financingso it should not be compared with other drugs that lack these features.
- approve a specific referral financing procedurewith the aim of balancing the care impact generated by these drugs in the autonomous communities.