Member State [MS]

The EMA Committee for Orphan Medicinal Products (COMP) reviews applications for orphan-medicinal-product designation.

Information on medicinal products is provided to patients and healthcare providers in different, carefully regulated ways – including package labelling, package leaflets, and the Summary of Product Characteristics.

Doctors were not very clear in their communication about MS. I didn’t understand them. It was necessary to find easy and understandable Information. I focused on patient groups, looked for sites about patients’ rights. I started to collect all that know-how and started to sort out information, the good vs the bad. I realised how important patient information is. And I noticed that there was not much understandable and useful information for patients. Patient-level information is important for good decision-making.

HTA systems in Europe differ, but usually involve assessment and appraisal steps.

In the current Clinical Trials Directive, the definition of conditions for inclusion of vulnerable populations is limited to clinical trials with minors and incapacitated adults who are not able to give informed legal consent. Released in April 2014, the new “EU Clinical Trial Regulation” (EU Regulation 536/2014) expands on information given for the above populations. Additionally, it also goes further and defines the conditions, especially the informed consent process for clinical trials in pregnant and breast feeding women as well as in emergency situations. Although, the objective is to harmonise the performance of clinical trials in Europe, certain opt-out options for Member States are foreseen and thus might lead to exclusion of certain countries from some trials.