While concrete changes to the PBAC procedures as a result of the Patient Voice Initiative are limited, several meetings with patients and patient advocates were held in September and October of 2016, at which a number of ideas were proposed to improve patient involvement in HTA processes.
While there have not been many updates to how England incorporates the patient voice within technology appraisals, it has remained a prime example for other countries in how patients might be integrated into decision making. The National Institute for Health and Care Excellence (NICE) not only includes patient input within the individual technology appraisal process but also utilizes a Citizens Council to address overarching moral and ethical issues NICE should take into account.
When considering the individual single technology appraisals (STAs), NICE seeks recommendations from consultees and commentators to nominate patient experts to be included in the assessment.5 Once selected by the Chair of the Appraisal Committee, the 2 patient experts submit a short personal statement on the technology and how it should be used in England, as well as attend the Appraisal Committee meeting.
Looking more broadly than individual HTA decisions, NICE utilizes the Citizens Council to provide a pathway for patients to have a larger role on what therapies are ultimately recommended to the National Health Service (NHS).6 The Citizens Council, a panel of 30 members of the public who reflect the demographic characteristics of the UK, is responsible for producing social value judgements and occasionally changes to NICE’s methodology. While many are familiar with NICE’s scientific value judgements which are about interpreting the “quality and significance of the [scientific] evidence available,” social value judgements relate to society instead of science.7 The Citizens Council meets once a year for 2 days to discuss topics that typically arise from the NICE guidance development procedure, after which a report is generated. To date, 18 reports covering a range of topics have been published.
In addition to involving patient groups in typical HTA submissions, Scotland also employs a unique alternative pathway for orphan, ultra-orphan, or end-of-life treatments in which patients have significant influence over the final outcome.
The Scottish Medicines Consortium (SMC), responsible for providing recommendations to the NHS of Scotland on clinical and cost-effectiveness of new medicines, leads patient advocacy efforts by sharing additional evidence from patient groups. In 2015, the SMC began formally registering patient groups for partnerships to gain information from the patient and caregiver perspective through patient group submissions. These submissions assist the SMC committee to capture relevant patient information as part of its assessment for new medicines. The SMC has partnered with over 80 groups since the launch of its patient group registration.8
Beginning in 2014, for therapies treating orphan, ultra-orphan, or end-of-life indications, pharmaceutical companies can tap into the Patient and Physician Engagement (PACE) pathway, which gives patient groups (and clinicians) a stronger voice in SMC decision making.9 To be eligible for this process, the manufacturer must provide supporting evidence that its product falls into one of the eligible categories. If the New Drugs Committee (NDC) arrives at a “not recommended” conclusion, then the manufacturer can request the drug undergo the PACE pathway, which adds an additional 1–3 months to the assessment timelines. Representatives are chosen from patient groups and asked to describe the added benefits of the medicine that are not fully captured within the typical drug assessment. These may include: