Health technology assessment (HTA) is applied within the societal context of health care. Its methodologies are designed to ensure internal scientific validity and provide accountability, yet assessing value to patients in terms of acceptability of the technology, applicability within the everyday practice of health care, immediate and longer term outcomes, and social consequences is also important (Reference Porter1). The HTAi Patient and Citizen Involvement in HTA Interest Group glossary defines HTA as the systematic evaluation of the clinical effectiveness, cost-effectiveness, and social and ethical impact of a health technology on the lives of patients and the healthcare system (Glossary) (2). More traditional definitions do not refer specifically to patients but include social and ethical aspects (Reference Banta3). How and whether patients are involved in HTA is generally dependent on health policy decisions of a particular country, and the mechanisms to involve patients have changed only incrementally over the years (Reference Gauvin, Abelson, Giacomini, Eyles and Lavis4;Reference Wortley, Wale, Grainger and Murphy5). This is despite reports that HTA can be strengthened by inclusion of the patient perspective and committing resources to supporting patient participation (Reference Facey, Boivin and Gracia6–Reference Berglas, Jutai, MacKean and Weeks8).
Some countries already make substantial efforts to integrate patient involvement into their HTA processes. These include: the National Institute for Health and Care Excellence (NICE) in England (9), the Scottish Medicines Consortium in Scotland (10), the Canadian Agency for Drugs and Technologies in Health (CADTH) (11), and the pan-Canadian Oncology Drug Review (12). However, other countries are only just starting to consider or are yet to consider public involvement (Reference Hailey, Werkö and Bakri13). Although some HTA organizations provide reasons why patients are involved, the stated purpose of such involvement may not always be clear (Reference Berglas, Jutai, MacKean and Weeks8;Reference Kreis and Schmidt14;Reference Dirksen15).
In this commentary, we have consolidated key arguments why patients should be involved in HTA to aid discussion and future developments. We set out to provide arguments why patients should play a robust part in HTA, not just in some countries but as a broad principle of HTA. The arguments we present are not limited to the Western context, and should, therefore, also appeal to HTA agencies situated in non-Western countries, paving the way for broader uptake.
FOUR ARGUMENTS IN FAVOR OF PATIENT INVOLVEMENT IN HTA
Argument from a Patients’ Rights Perspective
The Alma-Ata Declaration in 1978 reaffirmed that health, with its physical, mental, and social aspects, is a fundamental human right; and that people have the right and duty to participate individually and collectively in the planning and delivery of their health care (16). The Declaration describes primary health care as essential health care based on practical, scientifically sound, and socially acceptable methods and technology at a cost that the community and country can afford to maintain. The World Health Organization adopted this challenge, and in May 2014 went on to approve a resolution on health intervention and technology assessment in support of universal health coverage (17).
As patients have a right to participate in the planning and delivery of their health care, and the HTA process typically determines the health services and procedures available to patients, it follows that patients have a right to be heard as part of the HTA process. Such patient involvement can take many forms, including providing submissions, generally through patient groups, detailing experiences with a particular health condition or health technology (Reference Berglas, Jutai, MacKean and Weeks8), as expert patients or providing patient stories or interviews (Reference Staley and Doherty18), and as patient representatives on an HTA committee (12). In engaging patients in these ways, HTA agencies can enable patients to be part of the policy level decision making for which healthcare technologies are available. Beyond respecting patients’ rights, such engagement has the added value of building public trust in the health system processes and in increasing transparency (Reference Drummond, Tarricone and Torbica19).
Argument Based on Values
The determination of the cost-effectiveness of an intervention is an important factor in the final recommendations of an HTA committee in countries that include it in their HTA processes and can predominate over other considerations that underpin a healthcare system (Reference Towse and Barnsley20). However, value determinations and value judgements are also needed as part of HTA decision making (Reference Hofmann, Cleemput and Bond21;Reference Henshall and Schuller22). This is because value judgements underpin which health technologies and treatments are, and are not, assessed; what comparators are selected; and which populations are considered eligible to access a treatment or procedure (Reference Hofmann, Cleemput and Bond21). The benefits of a technology and, therefore, its value to patients in terms of health outcomes are also dependent not just on the technology but how it is used, the effectiveness of other interventions throughout the care pathway, and interaction with multiple healthcare providers (11).
Moreover, due to cultural and belief system differences across countries, each country has its own values that underlie its health system and HTA processes. Therefore, patient and community values should be taken into account in the evaluation of health technologies so that the HTA decisions reflect the value to patients and the community. Involving patients and the public in the HTA processes is an important way to identify and integrate these values.
The NICE Citizens Council in the United Kingdom is an exemplar methodology for eliciting community values, as demonstrated by its determination of the values that need to be considered when making decisions about trade-offs between equity and efficiency (23). Although “patient values” and “citizen values” may differ, the Citizens Council is inclusive of those with health issues and includes a cross-section of the community. The Citizens Council uses deliberative methods to develop a consensus view of values, where the perspectives of subsets of the community may have widely differed at the beginning of the deliberations. This helps to ensure that decisions about treatments and services that are funded by the National Health Service are aligned with the core values and morals of those whom the health system serves.
Argument Centering on Evidentiary Contributions
HTA decisions are informed by scientific evidence in the form of clinical trials. However, clinical research alone often does not provide complete information about patient outcomes and the total costs of care (Reference Porter1). For example, the elements of benefit to the patient may not be captured in the measure of health gain, or they may be broader than the health outcomes assessed in the trial (Reference Hofmann, Cleemput and Bond21). Patient-relevant outcomes may also not be clearly reflected in quality of life data (Reference Kinter, Schmeding, Rudolph, dosReis and Bridges24).
However, patients can provide valuable insights to inform the HTA decision-making process. These insights include: how it is to live with the condition, what it is like to be treated for that condition within the health system, what their unmet needs are, and other information that is not obtainable from clinical trials or medical practitioners. Thus patients or carers can express the value to them of a drug treatment to reduce the need for repeated surgery in young children; a different class of drugs with potentially reduced side effects, where all existing drugs were of the same class and had serious toxicities; availability of a medical procedure that allows withdrawal of poorly effective medications to control heart rhythm; and the experiences of living with cardiac devices such as implanted defibrillators. Such insights can provide valuable context and understanding of the health technology under assessment that are not captured, or perhaps even capturable, within clinical trials (Reference Greenhalgh, Snow, Ryan, Rees and Salisbury25).
Work in this area has already begun. Patients are keen to be heard in health care, research, and policy decision making by providing input; capacity exists for the robustness of that input to be considerably strengthened (Reference Anderson and McCleary26). In one example, researchers partnered with a patient advisory board whose members were able to identify that their active presence at focus groups of patients added value to the researcher-led focus groups by instilling trust and confidence of the participants (Reference Johnson, Bush, Brandzel and Wernli27). Qualitative research methodologies exist and can be used to provide important knowledge based on a range of patient perspectives (Reference Gagnon, Desmartis and lepage-Savary7;Reference Hansen, Draborg and Kristensen28), and provide a depth of understanding of issues related to illness and the appropriateness and acceptability of interventions (Reference Tjørnhøj-Thomsen and Ploug Hansen29). Additionally, measurement of the stated preferences of patients in terms of outcomes of healthcare interventions is another research methodology being developed to bring a patient perspective to HTA (Reference Dirksen15).
Argument from a Methodological Perspective
HTA methodologies are not static but continue to evolve, and patients can be an important part of that evolution. Two new advances to meet the demands currently being made for timely HTA are: early scientific advice, and managed entry into the health system together with the accrual of real-world evidence. Patients have a role to play in each of these. Early scientific advice is offered by some HTA agencies. CADTH, for example, interviews patients to help inform that advice, and to identify patients’ needs (30). In addition, patients can give advice on study design, to maximize recruitment and retention of participants, address patient relevant endpoints, and ensure that quality of life is measured as an outcome (Reference Low31).
Patient input into data collection is also important. This may be in the form of patient-reported outcomes for clinical studies, where digital collection methods and large datasets are part of the changing paradigm (Reference Basch32). Finally, where evidential uncertainties remain when undertaking an HTA of an innovative health technology a managed entry pathway may be considered, to allow the collection of real-world evidence (e.g., through a registry) while providing early access to a health technology. The goals and benefits of using this pathway need to be weighed up against the potential risks for patients, the need for truly informed consent, and patient participation in data collection (Reference Basch32).
CONCLUSION
Countries such as Scotland, England, and Canada make a considerable effort to integrate patient perspectives into their HTA systems, and continue to develop their processes for patient involvement. Other countries have yet to develop this aspect of their HTA processes. We offer here key arguments for why patient perspectives should be part of HTA in those countries, centering on: patient rights, alignment with patient and community values, evidentiary contributions, and a methodological development perspective.
By refraining from listing specific patient values, or delineating how patient perspectives are to be obtained, our intent is that these arguments are universally applicable, that is, they can appeal to agencies situated in both Western and non-Western countries. Methodologies and approaches for person-centered HTA and inclusion of patient perspectives are still in development. Therefore, recognition of the importance of including patient perspectives and implementation internationally is a key first step toward continued advancement.
CONFLICTS OF INTEREST
Dr. Wale reports that she is an immediate past chair of the HTAi Interest Group on Patient and Citizen Involvement in HTA (PCIG). Dr. Scott reports that she is a member of HTAi's Interest Group on Patient and Citizen Involvement in HTA (PCIG). Doctors Hofmann, Garner, Low and Sansom have nothing to disclose.
