Participation throughout the HTA process

Technology or topic selection

Most HTA bodies established to inform reimbursement or coverage recommendations review all new drugs and therefore do not need processes for identifying and selecting technologies for assessment. However, some jurisdictions require a sponsor to make a submission to trigger the assessment. Usually, the manufacturer is the sponsor. But when manufacturers do not submit a drug for assessment, other stakeholders, such as patient groups, may seek to make a submission so that patients can access the drug. Some HTA bodies, such as the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia, accept submissions from the public and patient groups. In practice this is rare because of the complexity of the technical process, but PBAC has supported and considered 1 submission from a patient group, Rare Cancers Australia.

However, HTA bodies with a mandate for assessing non-drug technologies do need processes for identifying and selecting technologies for assessment. For example, the Swedish Agency for HTA and Assessment of Social Services (SBU) and the Scottish Health Technologies Group (SHTG) accept topic proposals from patient groups and other stakeholders. Patient organisations have the right to request Germany’s Joint Federal Committee (G-BA) for a decision on non-medicine technologies. This means that not only can they propose a topic, but a request must be discussed, and if it meets the conditions for an HTA, G-BA must conduct the HTA. Instead of using a form, a specialist team, available to patient organisations regardless of funding, skills or size, helps with drafting the request (Haefner and Danner 2017).

Additionally, IQWiG (Germany‘s Institute for Quality and Efficiency in Healthcare programme) ThemenCheck Medizin (TopicsCheck Medicine) allows anyone to propose a topic for an HTA. IQWiG uses a 2-stage selection process to determine up to 5 citizen proposed topics each year. Usually completing proposal forms can be challenging for any stakeholder even with support from the HTA body. However, IQWiG has developed a simple online process designed to enable people without medical or research knowledge submit a question.

In terms of prioritisation of work, SBU has involved patients and carers in prioritisation methods. Its process is based on work by the James Lind Alliance and is used to identify the 10 most important uncertainties for condition areas with many uncertainties (Werkӧ and Andersson 2017).

However, most HTA bodies need to develop opportunities for patient input into decisions around which technologies to assess. This may include the participation of patients and their families in the development of criteria used to select technologies for HTA.

Scoping

Patients and patient groups may be involved in scoping, which generally uses the PICO (population, intervention, comparator, outcome) framework. For example, the National Institute for Health and Care Excellence (NICE) elicits patient input by publishing its draft scoping documents for comment and then publishes all the comments and the new scope. An examination of the NICE’s public involvement webpage reveals patient groups provide important input, particularly about patient subgroups, comparators and outcomes that matter.

Another example may be found in Australia’s Medical Services Advisory Committee’s (MSAC) PICO Advisory Sub-Committee. This group circulates the scope and a consultation survey to targeted patient groups.

EUnetHTA is a network of European agencies that collaborate in the joint production of HTA reports. The network uses the HTAi template (described in the section on HTA tools for patient input) to involve patients in EUnetHTA’s patient input in Relative Effectiveness Assessments (reports that assess clinical effectiveness). The intention is to gain patient input to inform the development of the PICO table and provide the assessment team with insights into patient experiences.

Patient input at scoping has been shown to be valuable for highlighting outcomes that matter to patients and identifying appropriate comparators for HTA submissions.

HTA tools for patient input

With the adoption of more rapid HTAs, especially for drugs, many HTA bodies began accepting submissions in the form of a written template. Some, for example the Centre for Drug Evaluation (Taiwan), the National Committee for Health Technology Incorporation (CONITEC, Brazil), and PBAC and MSAC (Australia), accept submissions from any member of the public, including patient groups. Whereas others, such as NICE (England and Wales), the Scottish Medicines Consortium (SMC) and CADTH (Canada), apply criteria that usually limit submissions to patient groups and include a declaration of funding basis. Submissions contain insights from the lived experiences of patients and their families that may challenge assumptions or address gaps and uncertainties. Templates generally ask questions about the daily lives of patients and families affected by the particular condition or illness, current treatment options and their impact, as well as the experiences of those who have had the technology being assessed.

The PCIG has worked with HTA bodies and patient organisations to review patient group submission forms. They have developed 3 standardised HTAi patient group submission templates for medicines, non-medicines and diagnostics. These tools have been consulted upon internationally and translated from English into French, Spanish and Mandarin. The template for patient group submissions to the non-medicine’s HTA could be adapted for clinical guidelines. This is accompanied by a cover note for HTA bodies, which stresses the need to adapt the template to suit their own circumstances, processes and the technology being assessed. In 2021, the EC-funded IMPACT HTA project used the HTAi work to develop a new patient group submission template for re-appraisal after data collection. This aims to document patients’ experiences during the data collection period and identify unexpected effects not captured in the clinical evidence.

The HTAi templates highlight the information that is valued by decision makers but patients need to be well supported by training, guidance and feedback to realise their value. This is because patients may not know which areas of their lived experience knowledge are being assumed, misunderstood or missing in the evidence. Some HTA bodies have dedicated staff who provide support to patient organisations during the completion of submissions. SMC, for example, provides feedback on draft submissions and CADTH gives feedback in a letter after the assessment.

The PCIG has adapted a guide developed by CADTH‘s pan-Canadian Oncology Drug Review that helps patients complete a patient group input submission template. The HTAi’s guidance for providing patient input using the templates helps patient groups understand what kind of information will have most impact and gives guidance on how to undertake surveys and conduct interviews with patients and report findings. Recognising that patient groups usually conduct this work without an ethics committee review, the PCIG also developed an HTAi short guide and a long guide on ethical issues for patient groups to consider when collecting and reporting information for HTA submissions.

Additionally, in 2020 the PCIG released a Summary of Information for Patients template and guidance based on a research project and SMC’s experience in providing information to patient groups from the sponsors about the medicine being assessed. The rationale for the template is that people, especially those in smaller or less HTA-experienced patient groups, require information about the product being assessed to target their input. The PCIG intends that HTA bodies will adapt the template.

Patient input in submissions has helped HTA bodies understand:

the trade-offs patients might make
the consequences of variations in service provision
the potential real-world value of small clinical benefits to patients
the consequences of treatment pathways
how a treatment is administered for patient subgroups not identified in clinical trials.

Berglas et al. (2016) studied how patient input was integrated in 30 assessments by CADTH’s Common Drug Review. They found that CADTH reviewers used patient insights about health status achieved, progress of recovery, and longer-term consequences of illness and treatment to frame the assessment. The CADTH Canadian Drug Expert Committee uses these insights to aid the interpretation of evidence. They also found patient input identified outcomes that are important to patients, but which may not be measured in clinical trials.

Committee membership

Patients and their representatives may be included on advisory and appraisal committees. To overcome ongoing confusion about the roles of patient and public members, Street et al. (2020) found identifying members by the interest and values they are tasked to represent provided clarity. They defined a patient member as someone ‘who has been selected to support the inclusion of the interests of patients in HTA processes on a committee’, whereas a public member ‘supports the inclusion of the interests of the society at large’. Patient members may be nominated for a committee to give specific expertise based on their lived experience. As committee members, they can also present the wider experiences and perspectives of their patient communities on a particular condition or issue. Furthermore, patient and public members play an important role in ensuring that patient involvement processes are appropriately enacted, can reflect on improvements, and provide training to those providing patient input. G-BA appraisal committees are an example of HTA bodies using this approach. They include patient representatives throughout the appraisal process and in all sessions of the committee.

Hearings

Australia’s PBAC conducts consumer hearings when there is greater uncertainty in interpreting benefit and harm evidence, such as some medicines for rare diseases. In Brazil, public hearings are legally provided for and the first hearing took place in March 2021. It was for spinal muscular atrophy. The intention is that public hearings be held before the final decision is taken for cases in which the secretary of the Ministry of Health’s Secretariat of Science, Technology and Strategic Inputs determines that the relevance of the matter justifies a hearing. The hearings are envisaged as a face-to-face consultative mechanism open to anyone and participants will have the opportunity to speak. (Silva et al. 2019.)

Consultation

Several HTA bodies, including the Italian National Agency for Regional Healthcare Services (AGENAS), CONITEC (Brazil) and NICE (England and Wales), publish consultation reports to seek feedback from a wider range of stakeholders, including patients and patient groups. Because these reports can be quite technical, patient involvement is better supported if patient-friendly versions are prepared and workshops or meetings are held to discuss the issues with relevant patient communities.

Dissemination

HTA bodies use patient-friendly versions of HTA reports and recommendations to communicate how recommendations were formed and what this means for patients. For example, working with Health Improvement Scotland’s public partners and drawing on the guidance from the DECIDE project, the SHTG produced a patient guide to its HTA on wound dressings.

Beyond individual HTAs

The use of patient participation beyond individual HTAs is less described in the literature. Examples include the formation of advisory groups at SMC, NICE and Health Technology Wales who involve patients in developing and reviewing patient involvement processes. Another example is CADTH’s involvement of patients in shaping and contributing to the agenda of its key capacity building activity, that is, its annual symposium. PCIG is currently undertaking a study to describe patient participation at the organisational level and may develop tools to support this area if a need is identified.

PCIG’s resources to involve patient groups and individual patients in HTA, include the HTAi Online Resource Directory. The directory aims to make it easier to locate useful resources shared by HTA bodies, not-for-profit organisations and other relevant organisations.

Patient involvement in HTA