The guide provides clarification concerning the regulatory framework surrounding the assessment of the therapeutic indication. According to Article 8.3 (j) of Directive 2001/83/EC and Article 6.1 of Regulation (EC) 726/2004, a Summary of Product Characteristics (SmPC) prepared in accordance with Article 11 of Directive 2001/83/EC must be drafted and included in the application for marketing authorisation. The SmPC is the basis for information to health care professionals and patients.
Section 4.1 of the SmPC guideline contained in Volume 2C Notice to Applicants provides that the therapeutic indication should be stated clearly and concisely. In addition, the therapeutic indication should define:
- the target disease, or condition distinguishing between treatment, prevention and diagnostic indication;
- when appropriate, the target population, especially when restrictions to the patient population apply;
- when relevant, any mandatory conditions of product usage not covered more appropriately in other parts of the SmPC.
Elements to be considered when assessing the indication
The guide provides that the definition of therapeutic indication requires the analysis of several aspects that influence the benefit/risk assessment. The assessment of the indication should take into account the data submitted as part of the application. It should also take into account the therapeutic context and whether the benefits and risks in the studied population are applicable to the proposed target population.
The guide provides a number of components to consider when deciding on the wording of the indication:
- Target disease or condition. This includes the effect of the medicinal product and information on duration of treatment;
- Target population. This includes the evaluation as whether the benefit/risk balance is positive or not in relevant subgroups of the target population;
- The place in therapy to evaluate if there is a defined treatment pathway ;
- Use in monotherapy or combination therapy;
- Mandatory condition of product usage.
The guide provides that even though these components can be applied to most therapeutic areas, they may be more or less relevant depending on the medicinal product. Other components not covered by the guide may be relevant as the exact wording of the indication remains a case-by-case evaluation.
Authored by Elisabethann Wright and Gregoire Paquet