Look behind the lecture: patient engagement in health technology assessment
In this feature, Samuel Thomas (Rose Li and Associates, MD, USA) discusses the workshop he moderated during the HTAi Annual Meeting (15–19 June 2019, Cologne, Germany) on the importance of stakeholder engagement in health technology assessment (HTA).
Interview segments:
Please can you introduce yourself and your institution?
What is the importance of social engagement in health technology assessment (HTA)?
What are some of the challenges associated with achieving successful and representative social engagement in HTA?
How may we work towards improved translation of stakeholder experiences to achieve better outcomes in HTA?
How may patient engagement vary according to the type of technology being assessed?
What methods do we need to develop to ensure we bridge the gap between the collection of patient numerical data and its translation into meaningful patient experiences?
How do you see social engagement changing in the future?
Please can you introduce yourself and your institution?
I am Director of Programs at Rose Li and Associates (MD, USA). We are a US-based consultancy that provides research, policy analysis and communications support to life science companies, government agencies and nonprofit organizations such as patient groups.
What is the importance of social engagement in health technology assessment (HTA)?
Social engagement is crucial for HTAs to ensure that the needs and priorities of patients and society are accurately reflected in assessments. HTA is a systematic process for evaluating the value of using a health technology in a given context. Although clinical benefits and costs are two of the main criteria in HTAs, assessments also consider implications for patients and caregivers. These include ethical, social and legal factors, and cultural and organizational contexts. Because of the breadth of factors considered, input from patients, caregivers and lay populations is necessary to inform certain HTA criteria.
“Social engagement is crucial for HTAs to ensure that the needs and priorities of patients and society are accurately reflected in assessments.”
Moreover, because HTAs influence policy decisions that affect patients and society, we must understand what these populations consider important in order to make policy-relevant assessments. Thus, social engagement is critical not just to understand implications for patients — external to clinical benefits and cost assessments — but also to understand patient perspectives on clinical evidence. Although HTA practitioners can evaluate evidence from multiple sources, they cannot infer patient values without engaging patients.
What are some of the challenges associated with achieving successful and representative social engagement in HTA?
Two primary types of patient engagement exist. The first is patient-based evidence; this is research derived from studying patient preferences, behaviors or statements. Second, there is patient input; this involves the provision of direct patient contributions — such as through writing, speaking or voting — to a given HTA assessment. Challenges differ based on the type of engagement.
Key challenges to patient-based evidence include difficulty reaching the target population, ensuring that assessments adequately capture patient heterogeneity and controlling for potential researcher biases.
“Although HTA practitioners can evaluate evidence from multiple sources, they cannot infer patient values without engaging patients.”
Key challenges to patient input include identifying patient representatives willing to submit input, providing adequate training to patient representatives, and designing appraisal processes and educating committee members to consider patient input seriously.
How may we work towards improved translation of stakeholder experiences to achieve better outcomes in HTA?
A challenge central to both patient-based evidence and patient input concerns translating information about patient experiences into actionable HTA criteria. Two main strategies can help. First, educating patients and their representatives about HTA processes so that they better understand the type of input that has the most value for appraisal committees. Second, developing appraisal processes that explicitly define how different types of patient experience information can, and should, be considered. More transparency and better documentation of how HTA bodies use patient preference and experience information in specific appraisals would allow all stakeholders to evaluate the effectiveness of different patient engagement methods in different contexts.
How may patient engagement vary according to the type of technology being assessed?
All HTAs should engage patients, regardless of the technology being assessed. That said, both HTA processes and patient experiences do vary across different types of technologies. Common across technology types is the need for HTA bodies to understand — from patients and caregivers — the impact of the health condition on patients, caregivers and their families. This information can be collected through either patient-based research or direct patient input. However, it can be challenging to understand the heterogeneity of experiences using the latter method.
“All HTAs should engage patients, regardless of the technology being assessed.”
Patients have more intimate experiences with some health technologies compared with others. For example, patients undergoing a minimally invasive surgery may have radically different experiences than patients undergoing conventional open surgeries — even if the long-term clinical outcomes are similar. On the other hand, patient experiences with two diagnostic blood tests may be virtually identical when undergoing the procedure. Patient input is still critical in this latter case; significant differences in false-positive or -negative rates may exist, and patients may provide unique perspectives on the relative merits of each test compared with HTA scientists.
Finally, some HTA agencies conduct rapid reviews on certain types of technologies. It can be difficult in these cases to adequately consider patient perspectives. Conducting broad patient-based research in a given disease area before evaluating a specific technology is one way to ensure at least some consideration for patient-provided context in these cases. Retaining a set of experienced patient representatives who participate on rapid review appraisal committees may also help.
What methods do we need to develop to ensure we bridge the gap between the collection of patient numerical data and its translation into meaningful patient experiences?
Training for patients, HTA practitioners and committee members will likely provide the strongest benefits in the short term. In terms of patient-based evidence, more methods are needed to aggregate perspectives of large numbers of patients, without reducing important details to a summary level that is no longer useful. Large-scale and passive data collection, such as mining online forums and social media, is an emerging field that requires additional methods of development to ensure data validity and protect against potential conflicts of interest. Finally, many HTAs rely on quality-adjusted life years (QALYs) for economic cost-benefit analysis. QALYs may, however, insufficiently consider “quality” and “value” from a patient perspective. Developing new patient-derived measures that can be used for economic analysis and decision making should be a high priority for HTA scientists.
How do you see social engagement changing in the future?
Many HTA agencies, technology sponsors and patient groups around the world are working to improve patient engagement processes. Therefore, I expect increasing opportunities for patient engagement throughout all stages of HTA processes. One trend that I expect to continue is patient engagement occurring earlier in the HTA process. This is achieved both by HTA organizations selecting and prioritizing technologies for assessment and by companies engaging patients in the early design of clinical trials.
“Developing new patient-derived measures that can be used for economic analysis and decision making should be a high priority for HTA scientists.”
Another area that I hope will continue to change is transparency. Although many HTA processes have become more transparent in recent years, not all decision factors are published. Decision makers review available evidence and listen to patient representatives, but the weighting of different factors in the final decision is usually not known. HTA bodies should increase the transparency of their decision-making processes and provide more feedback to patients regarding the impact of their contributions. Implementing additional transparency would not require any new sophisticated tools or processes. All it would take is goodwill and dedication from decision makers to become even more accountable toward the patient community and larger society.
Disclosures:
Mr. Thomas is a full-time employee of Rose Li and Associates, Inc. (RLA), a professional services firm that conducts paid contract work for life science companies, government agencies, and nonprofit organizations, including on topics related to health technology assessment and patient engagement. Mr. Thomas’s participation in the 2019 HTAi Annual Meeting and general work on patient involvement in health technology assessments is supported through an RLA contract with Eli Lilly and Company.