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closer to accessing new treatments

Only 5% of rare diseases have treatment. Access to new medicines can save many people’s lives; however, less than half of the drugs approved in Europe are available in Spain.

Twelve measures to improve access to new treatments for patients with rare diseases

Image taken from the Farmaindustria document ‘Proposals for improving patient access to orphan medicines’

Around three million Spaniards live with one of the different rare diseases that exist, pathologies that mostly (95%) have no treatment.

In these cases, the arrival of an innovative drug is often the only hope, especially when the pathology is severe.

The orphan drugsintended to treat these rare diseases, were recognized in Europe in 2000.

Only last year the European Medicines Agency (EMA, for its acronym in English) he approved 24 drugs for rare diseases, the 41% of the total of new active principles authorized during the year.

However, in Spain they are only available on 44% of orphan medicines 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 to improve patient access to orphan drugs.

A document that collects twelve specific measures to ensure that Spanish patients with rare diseases have the maximum available opportunities to be treated, 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 Access Department of Farmindustria, Isabel Pineros.

Spain, in inequality

According to Farmaindustria, in Spain orphan drugs follow the same evaluation and authorization processes as the rest of the drugs, unlike other European countries where specific circumstances are already taken into account, such as the number of affected patients or the lack of alternative treatment, among other factors.

This leads to inequality in this type of treatment in Spain, limiting the access of 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 some different approach to these drugs and recognized that they could not be assessed in a conventional way.

“We are facing a situation of clear inequality compared to patients from other countries such as Germany, Italy or France. We need to address access to orphan drugs in a specific way in order to be placed in a good position in the European context, as we already are in clinical research”, explains Pineros.

Rare diseases treatments medicines
Image taken from the Farmaindustria document ‘Proposals for improving patient access to orphan medicines’

Twelve proposals to improve access

Thus, from Farmaindustria they list twelve proposals to increase and accelerate the arrival of new treatments for patients with rare diseases:

  1. Establish a early dialogue with the Administration once the drug has received a favorable report from the EMA.
  2. one accelerated financial evaluation procedure.
  3. Create a specific external advisory committeethat includes scientific societies, patient associations and renowned experts in pathology who analyze the circumstances of each drug.
  4. Adopt specific criteria of funding for orphan drugs.
  5. Establish criteria for the definition of longitudinal financing models.
  6. Understand and accept them specificities and limitations of orphan drugs regarding the level of data that, by their nature, they can provide.
  7. Make one first therapeutic evaluation and later the economic evaluation.
  8. As for the Therapeutic Positioning Reportsit would be advisable that if an economic evaluation is made, it is done once the financing price has been determined.
  9. Improve information collection systemswith better optimization and automation that takes advantage of the opportunities of the future Digital Health Strategy.
  10. A new approach that allows improve the evaluation and financing process of orphan drugs, taking into account the boom in research in this field.
  11. An orphan drug must be subject to a scheme of special financingso it should not be compared with other drugs that do not have these peculiarities.
  12. Approve a specific referral financing procedurewith the aim of balancing the healthcare impact generated by these medicines in the autonomous communities.
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