Table 1 Access to medicinal products, through 'compassionate use', 'off-label' use and randomised clinical trials
'Compassionate use' European regulation | Off-label use | Randomised clinical trial | |
|---|---|---|---|
Purpose | Serves the needs of patients where no alternative treatment exists | Serves the needs of patients with an indication other than that the product is marketed for | Serves the needs of society and future patients and may benefit some of the included participants |
Party involved | Patients | Patients | Participants |
Disease | A life-threatening or chronically or seriously debilitating disease | Any indication for which the product is not authorised | Any |
Informed consent | Required in some member states | Not required | Required |
License | Medicinal product is not yet licensed | Medicinal product is licensed for other indication(s) | Medicinal product can be licensed and not licensed |
Responsible party | Prescribing physician with approval from the regulatory authorities | Prescribing physician | Sponsor with approval from the regulatory authorities |
Control group | Without control group | Without control group | With control group |
Data | In some member states, some data are reported to the regulatory authorities | Spontaneous adverse events may be reported | Outcome measure and adverse event data are reported to the regulatory authorities |
Access to the intervention | Medicinal product accessed through the programme, afterwards those patients can have access before the product is licensed | Medicinal product available on prescription | Declaration of Helsinki stipulates that participants "are entitled to...share any benefits that result from the trial, for example, access to interventions..." |