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Study on the applicability of the NEED framework to rare diseases

The NEED framework and assessment procedures are intended to apply to all diseases. However, rare diseases are often considered a separate category of diseases that need to be approached differently in healthcare policy. For example, rare diseases are by definition considered by the European Medicines Agency (EMA) as characterised by high unmet medical needs. According to EMA’s definition, a rare disease is a disease that is life-threatening or chronically debilitating, with a prevalence in the European Union (EU) below 5 in 10 000 and for which no satisfactory method of diagnosis, prevention or treatment is authorised in the EU. The definition is used, alongside other criteria, for orphan designation to pharmaceutical products. There are currently about 2000 medicinal products that have an orphan designation. The number of requests for orphan designation is increasing. There are more than 6000 rare diseases.

The objective of this study is to assess to what extent the NEED framework and assessment procedures (see Figure below) for identifying patient and societal needs, is applicable to rare diseases, how the evidence collected on rare diseases according to the framework could be used by decision makers and whether/how the framework and/or procedures need to be adapted to be applicable to rare diseases.  

Figure: NEED’s assessment process


For more information, see here.

About NEED

NEED is financially supported by the Belgian Federal Science Policy (BELSPO) through the INFRA-FED call.

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Project coordinators

dr. Irina Cleemput



prof. dr. Robby De Pauw

Sciensano, Department of Epidemiology and Public Health, Service Health Information