As a rule, a medicine must be authorised before it can be made available to patients, and it is not possible to obtain the relevant authorisation before conducting pre-clinical and clinical studies to confirm the relevant efficacy and safety of the medicine. Therefore, it can take many years from the initial formulation of a medicine to its market launch.
The drug could help save the health
Or even the lives, of patients who have had no therapeutic options available to them to date? Is it permissible to speed up access to the drug in the name of helping seriously ill people, when this as yet unregistered therapy carries certain risks?
This issue of „compassionate use” is not currently addressed by Polish law; however, this does not mean that speeding up access to treatment because of the patient’s seriousness is not permitted. The basis for allowing patients to gain access to an unregistered medicine (not only in Poland, but in general anywhere in the world) is provided by Article 83 of Regulation 726/2004 laying down Community procedures for the authorisation of medicinal products for human and veterinary use; however, this provision applies only to products (covered by an application for marketing authorisation or undergoing clinical trials) that qualify for central registration. Also the provisions of the Polish Pharmaceutical Law allow applying for exceptional marketing authorisation for an unregistered medicine, in particular in the case of a threat to human life or health (Article 4(8) of the PF). However, none of these regulations contain detailed conditions that must be met before it is possible to market an off-label medicine in order to save the health or life of patients; for this reason, their application is difficult in practice.
Law of regulations governing the use of compassionate medicine.
The new regulations are to define the rules for running compassionate use programmes and the responsibility of individual national institutions and their cooperation with the European Medicines Agency, financing methods and safety procedures. In particular, the Rare Diseases Plan highlights the role of such programmes in improving patient access to treatment, including the need to ensure that participants in a clinical trial can continue treatment with a new drug while registration proceedings for the drug are already underway.
Indeed, depriving patients of treatment at the end of a clinical trial, particularly if it has proved remarkably effective, simply because of the protracted registration process raises serious ethical questions, which pharmaceutical companies are increasingly addressing through various compassionate use schemes that provide the drug to patients free of charge.
The Rare Diseases Plan also mentions another form of support for access to medicines that is used in practice by their manufacturers, namely, early access programmes. These programmes concern medicines that have already been registered but are not yet on the market (for example, due to ongoing reimbursement procedures). Sometimes such a medicinal product is made available more quickly to patients in particular need, at the manufacturer’s expense, so that patients who need urgent treatment do not have to wait, for example, until a positive reimbursement decision is taken. In light of the information contained in the Rare Diseases Plan, early access programmes are also to be regulated.
The new regulations for both compassionate use and early access programmes are to be implemented by the end of next year; which means that we should see the draft of the relevant changes in the law in the coming months.