Patient engagement process in rare disease: how does it differ between countries?
Skeldon G, Adkins E, Ayre S, Langham S, Nicholson L. PRO95 Patient engagement process in rare disease: how does it differ between countries? Value Health 2020;23(Suppl 2):S706
Abstract
Objectives: The value of patient engagement in assessing new treatments in health technology assessment (HTA) is widely recognised. Its important is demonstrated in are diseases, where the patient voice can provide unique insight and evidence to help HTA agencies assess the value of new interventions. This study reviewed the different approaches to patient engagement in the HTA of orphan drugs (ODs) in four countries that include it as part of their HTA process.
Methods: HTAs of 34 ODs with marketing authorisation from January 206 to February 2020 were identified from HTA websites in Australia (Pharmaceutical Benefits Advisory Committee[PBAC]), Canada (Canadian Agency for Drugs and Technologies in Health [CADTH]), England (National Institute for Health and Care Excellence [NICE]), and Scotland (Scottish Medicines Committee [SMC]) and details of the patient engagement process were recorded.
Results: 88 HTAs were identified for 34 ODs: Australia (n=13), Canada (n=22), England (n=27), and Scotland (n=26). Of these, 10 (76.9%), 20 (90.9%), 24 (88.8%), and 24 (92%) HTAs included patient input, respectively. The patient engagement process differs between agencies. NICE, SMC and CADTH have structured templates for patient feedback; PBAC has a web portal for single input. NICE and SMC only accept input from patient organisations, while CADTH and PBAC also accept individual participation. NICE and SMC have appraisal meetings attended by patients/their representatives. In Canada, the Canadian Organization for Rare Disorders represents patients with diseases that lack a patient organisation. Patient input into PBAC appraisals varied, ranging from two individuals during one appraisal to 459 patient-related sources for another.
Conclusions: Whilst each of the go==four HTA agencies welcomed patient input, approaches varied significantly. This study illustrates the need to formalise and standardise the approach to patient engagement to ensure equitable access to ODs.
