Patient Involvement Initiatives in HTAs: How Can We Do Better?

OBJECTIVES: This study aims to evaluate the role of patient involvement initiatives (PIIs) in health technology assessments (HTAs) within four European countries and recommend improvements.

METHODS: General methods and guidelines, published articles, HTA methods, and surveys of HTA agencies were analysed in England, Germany, Spain, and Poland. The assessment used a three-point score (low, medium, and high) across five domains: 1)’ transparency’: clear processes for PII involvement in HTA; 2) ‘involvement’ of patient representatives (PR); 3) ‘support’: appropriate resources for effective PR involvement; 4) ‘inclusion’ of all relevant segments of the patient community; 5) ‘consideration’: regular review of PII goals and best practice. A case study illustrated current good practices.

RESULTS: England scored ‘high’ for all five domains. Germany scored ‘high for ‘transparency’ and ‘support’, ‘medium’ for ‘involvement’ and ‘low’ for ‘inclusion’ and ‘consideration’. Spain scored ‘medium’ for ‘involvement’ and ‘low’ for all other domains. Poland scored ‘low’ for all domains. In England a PR group (the Sickle Cell Society) appealed NICE’s decision not to recommend voxelotor for haemolytic anaemia caused by sickle cell disease, citing the 2010 Equality Act. Following the appeal, and a reduction in price, voxelotor was recommended via NHS England’s Innovative Medicines Fund. The success of this PII was due to a formal framework for patient input into the HTA process, clearly defined legal responsibilities, and engagement from all segments of the patient community.

CONCLUSIONS: To improve PIIs in HTA, agencies should consider a mandatory review of engagement processes for existing PIIs and plan for improvement. We further recommend establishing consistent legal frameworks across European countries to clearly define the mechanism for patient involvement within HTA. To support PIIs, agencies should: 1) provide plain language training on HTA process and explain key discussion points during decision meetings; 2) focus on outreach to engage diverse patient perspectives.