Development of new health technologies necessitates patient involvement at multiple points throughout the process, and this should include health technology assessment (HTA). Indeed, the scientific basis of HTA is not robust without the inclusion of patient-based evidence, complemented by patient participation in HTA. To uncover some of the basics behind patient involvement in HTA, Neil McGregor-Paterson, secretariat, Health Technology International´s (HTAi) Patient and Citizen Involvement in HTA Interest Group (PCIG) spoke to a number of the contributors to the world´s first academic book on the subject.
HTA is a complex academic subject. Is it possible to summarise HTA and its aim?
Karen Facey, co-editor of the book Patient Involvement in Health Technology Assessment: “HTA is a multidisciplinary field that addresses the clinical, economic, organisational, social, legal and ethical impacts of a health technology, considering its specific healthcare context as well as available alternatives.
“The scope and methods of HTA may be adapted to the needs of a particular health system, but HTA processes and methods should be transparent, systematic and rigorous. In health systems throughout the world, HTA plays an essential role in supporting decision-making.”
Is patient involvement in HTA really that important to the decision-making process?
Brian O’Rourke, chair of the International Network of the Agencies for Health Technology Assessment: “If you’re not involving patients, you’re not doing HTA! It’s that simple. Patient involvement improves the quality, relevance and value of HTA. It is difficult to conceive of HTA being conducted in a meaningful way in the 21st century without the involvement of patients.”
Neil Bertelsen, chair of Health Technology Assessment’s (HTAi) Patient and Citizen Involvement in HTA Interest Group (PCIG). “Healthcare systems are under intense pressure to contain spending, especially at a time when scientific innovations are accelerating. So, can healthcare systems achieve value for money in a health service system, while taking account of the needs, experiences and preferences of patients? The answer is yes. But only through transparent, systematic processes to which all stakeholders can contribute, and in which all evidence is judged fairly and rigorously. HTA is one such process and patients are one of the key stakeholders.”
Patient involvement would imply that it is only those with lived-experience of a condition that should input. Is there a broader definition of patients?
Neil Bertelsen: “It is really important that all stakeholders have a common understanding of what is meant by a patient within an HTA process. In essence, decision-makers need to understand the patient experience and how this relates to the provision of care and treatments. It is also important to remember that it is not always possible for those receiving care (the classical definition of a patient) to be involved directly in HTA processes, for example, those suffering from late-stage cancers, those with cognitive impairments or children. That’s why we often refer to patients as:
Individual patients – people with lived-experience of a condition
Expert patients – those with lived-experience who have also been trained in areas such as HTA
Carers – those who provide care and support for those living with a condition
Patient organisations – individuals within an organisation who are responsible for representing the views of their organisation and those they represent
Patient advocates – those who advocate for patients, but are not representing the views of a patient organisation.”
Involvement describes the ´act or process of taking part in something´. In terms of HTA, how does this definition relate to patient involvement?
Professor Sophie Staniszewska, lead for the Patient and Public Involvement (PPI) and Patient Experiences Programme, Warwick Research in Nursing at Warwick Medical School: “In HTA, patient involvement encompasses two distinct but complementary ways in which HTAs could be strengthened by taking account of patients´ perspectives.
“The first is robust research into patient aspects through, for example, qualitative and quantitative research into patient experiences, preferences and perspectives. This is key patient-based evidence that should have equal status to clinical and economic forms of evidence in HTA.
“The second is patient participation in the HTA process through, for example, contributing a written submission, or attending a committee hearing. Ideally patient participation needs to be a two-way dialogue. This enables real-world perspectives and experiences to be shared. Dialogue helps fill gaps in the literature and address uncertainties, while helping patients and HTA agencies understand each other better and work together to address issues as they arise.”
In terms of HTA agencies, it would appear that the National Institute for Health and Care Excellence (NICE) is one of the agencies that is leading the way in approaches to supporting patient involvement?
Lizzie Thomas, senior public involvement adviser at the National Institute for Health and Care Excellence: “Involving patients (and the public) is integral to NICE´s work, and there is a formal policy setting out this commitment. NICE has a dedicated team, the Public Involvement Programme (PIP), to support and advise on patient and public involvement across all its work programmes. We work with both individuals and patient organisations to include patients’ views and experiences, and we have an evidence-based approach to our patient involvement activities. NICE recently undertook a strategic patient and public involvement review, as part of our commitment to ensuring the quality of what we do and our commitment to continuous improvement of our patient involvement work.”
A two-way process, where patient and carer representatives are treated as partners that support the development of patient involvement in HTA, as well as providing input, is vital to ensure a collaborative and impactful approach. Is this reflective of HTA in Scotland?
Jennifer Dickson, public involvement coordinator at the Scottish Medicine Consortium (SMC): “Yes, over the past three years SMC has completely overhauled its approach to patient and carer involvement to achieve this.
“We have developed a Public Involvement Network (PIN) that formalises relationships with each submitting patient group through a registration process to become an SMC Patient Group Partner. Each submitting patient group is offered information, training and one-to-one support. This supports them to fully and effectively participate in HTA.
“Furthermore, our innovative PIN Advisory Group provides a forum to enable patient and carer groups to engage constructively and productively with SMC as a respected partner in developing and shaping all aspects of public involvement work. This provides advice to SMC that further strengthens our relationship with patient groups and ensures that the views of patients, carers and members of the public are effectively used to inform HTA decision-making. These activities are critical to ensuring that patient and carer representatives have the capacity and capability for meaningful HTA involvement.”
Arguably, the increased use of rapid HTAs, with their shortened review timelines, presents new hurdles for patient involvement in HTA. Are these challenges insurmountable?
Hervé Nabarette, HTA division, technical advisor, Hauté Autorité de Santé (HAS): “In recognising the challenges of securing the patient perspective in rapid HTA on drugs and devices, HAS has adopted a new approach that consists of written submissions with a time allocation of 45 days to do so. During the testing phase – within the first six months – 18 patient organisations participated, sending 24 contributions concerning 22 drugs.
“This demonstrates that through the adoption of new approaches for rapid HTA, patient involvement can be maintained. Nevertheless, HTA bodies must put in place quality improvement methods. They must also encourage stakeholders to integrate patient perspectives in the assessment and appraisal steps, and provide adequate answers to tough questions, for example, expression of expectations from reviewers and the committee concerning patient perspectives on a given product; the nature of the contact between patients and HTA bodies, and information given to patient organisations to allow for appropriate submissions.”
What role is there for the pharmaceutical industry in supporting patient involvement in HTA?
David Grainger, Global Public Policy, Eli Lilly: “In terms of supporting patient participation, some jurisdictions limit the communication from industry to patients to just the fact that a new technology is about to undergo an HTA review. However, where governments or other agencies do not provide adequate support, there is a role for industry in supporting capability development, education and training.
“This is particularly relevant where there are few public resources to support involvement. Providing it is at arm’s length and is transparent, this serves the need and enables patient involvement where it otherwise might not occur.
“Indeed, there are several examples of successful programmes supported by industry and delivered through third parties. One example is the annual programme delivered by the London School of Economics, which is supported by a number of industry partners and is attended by patient advocates from across Europe.
“Some companies work directly with experts in this area to develop on-line, or in-person resources, designed to introduce HTA concepts and help patients engage with their local agency. This agency engagement is a key output of these activities, as this is a critical step before patients consider if, when and how they can get involved.
“However, patient participation should ideally be accompanied by research into patient perspectives on outcomes reported in clinical trials supporting HTA. The industry has a critical role in generating robust peer-reviewed research that meets the requirements of HTA. As patients can contribute valuable information about important outcomes, as well as the practicalities of clinical trial participation and associated research, they should be involved at all stages of medicines development.”
Finally, given the significant resource implications on patients getting involved in HTA, is patient involvement a must have for all HTA?
Neil Bertelsen: “Yes, but this involvement needs to be carefully focused. Our experience of patient involvement in HTA is that the patient perspectives add most value when there are difficult value judgements that arise from a new intervention; when clinical and economic evidence is limited; or when added value is at the cusp of a predetermined threshold. It is at these points that the patient perspective can illuminate the data, pointing the decision-making committees to those aspects of the evidence that have most impact on the patient experience.
“One of the challenges is for patient organisations to collect all the evidence they would like to put forward in a very short space of time. There is a pressing need to pool resources globally, to share best practices and to share the evidence generated for patient group submissions to the various HTA bodies.
“However, patient involvement is not a substitute for poor-quality or incomplete data. Nor is it a lobbying process. It is unlikely to result in a positive recommendation for technologies that sit well above a willingness-to-pay threshold.
“As a final thought, patient involvement in HTA has come a long way in the last decade. Yet, there is still so much more to do if patient-based evidence and patient participation are going to maximise their value to HTA decision-making. This will necessitate all stakeholders with an interest in HTA and its outcomes to work together, respect each other’s boundaries and collectively drive forward the patient involvement agenda.”