OBJECTIVES:

NICE involves patient organisations in its technology appraisals, enabling them to inform decisions on which technologies will be available in the NHS. In NICE’s appraisal process, systematic reviews of scientific research are combined with submissions of evidence from industry, clinical experts and patient organisations. This multi-stakeholder approach may be especially useful when new drugs for rare conditions are assessed, due to limited clinical data and expertise available.

The objective of this research is to analyse previous NICE technology appraisals and highlight the criteria supported by patient groups in the evaluation of new drugs for rare diseases.

METHODS:

We analysed NICE technology appraisals between July 2016 and June 2017. From the 58 appraisals, 11 were related to rare diseases. We identified criteria used by patient groups in the appraisals as follows: 1. Description of the disease, complications and impact on patient QoL; 2. Social costs, loss of productivity for patients and family; 3. Treatment benefits; 4. Economic aspects of drug use.

RESULTS:

In 40% of the documents analysed, patient groups supported evidence related to the disease, societal costs, improvement in QoL and life expectancy for patients that can access new treatments. In 50% of the HTA reports, patient experts highlighted the psychological distress for patients affected by rare disorders, the need to improve disease management and the adherence to treatment, as well as the patient pathway in primary and secondary care settings. In 10% of the reports, patient groups supported the economic aspects and the potential cost-savings of the new drugs.

CONCLUSIONS:

Patient groups’ role in NICE technology appraisals is highly important. Their contribution is not limited to informing HTA decision makers on the features of the disease, patient QoL and potential benefits associated to new treatments, but can also provide support to evidence on the economic burden to the wider society.