“Access: arriving or entering. Same as the entrance. “According to the definition presented by the dictionary, access appears to be a relatively simple and easily definable word. However, when we associate access to the word medicine, this definition becomes a complex process with several phases and actors until it actually occurs .
The most recent case of cystic fibrosis, which is widely circulated and discussed on social networks and in the media, is closely related to the issue of access to the drug. However, this link leads to another topic that deserves particular attention and debate in the health sector in Portugal and at European level: what is access to medicines and how is access to therapeutic innovations actually made?
The process starts at European level with the granting of a marketing authorization, which is often shortened to AIM. This evaluation phase is carried out by the Committee on Medicinal Products for Human Use of the European Medicines Agency with the main aim of evaluating the efficacy and safety of a new medicine. Following the positive opinion of this committee, a new procedure for the European Commission to approve the AIM follows. At the end of this European approval cycle, the new drug will receive a centralized marketing authorization and will be available in all countries of the European Union.
However, this first evaluation process at European level is not synonymous with access to new medicines. In general, medicines in all European health systems are financed in whole or in part by the state. Therefore, a procedure for evaluating the financing of the new medicine with the respective therapeutic and economic evaluation is necessary in each Member State. This process will culminate in a decision on the financing of the drug and only after it has been completed will access to the new drug actually take place.
In exceptional cases, in which there are diseases for which no therapies are available, access to the medicinal product is ensured during the assessment process through special authorizations (AUE), which are carried out as part of early access programs. Medicines are transferred to hospitals by marketing authorization holders and are exempt from National Health Service fees. Although the evaluation of the drug funding has not yet been completed, in exceptional cases patients may have access to innovative, exempted drugs. AUE requests are approved. Once the funding of a drug has been approved in the case of hospital drugs, its participation is guaranteed 100% by the state, that is, in its entirety.
The full process of evaluating the funding of an innovative medicine under current legislation must take place over 180 calendar days, which is six months. If we look at the data published at European level on the time of access to new medicines, i.e. the time that has passed between the decision by AIM at European level and the decision to finance it at national level, we see that the value for Portugal corresponds to 711 days, which corresponds to about 23 months. In addition, the data published for the last period between January and March 2020 show that more than half of the procedures for assessing funding for new medicines fail to meet the deadlines.
Access to medicines is therefore associated with a complex, time-consuming and bureaucratic cycle. The European Union is preparing a legislative proposal so that part of the process, therapeutic evaluation, can take place at European level, similar to the process of granting a marketing authorization. Despite being approved as one of the flags of the Portuguese Presidency of the European Union in the health sector, this document has been waiting for the green light since 2018.
In the case of pharmaceuticals, we therefore see that the definition of access at European level is complex and heterogeneous. Although there is a centralized European process, effective access is only achieved after a national process with different timings and methods between countries, which ultimately determines effective patient access to medicines. Action at European level is needed to help reduce these access times and to put the real beneficiaries of the medicine, the patients, high on the agenda.