Lay people as Decision Makers

Our initial method of patient involvement in HTA was to recruit lay members to our HTA committees. This follows best practice (Reference Kelson4), and lay membership of NICE committees has remained a constant throughout NICE's work. Lay members bring the perspectives of people who use health services to our HTA decision making. They do not bring condition-specific expertise (although they may have that experience) but take a general view on the topic at hand, reflecting on the patient evidence presented to them and highlighting that evidence in committee discussions.

In 2001, having one lay member involved with NHS committees was seen as good practice (Reference Hogg and Williamson5). From its inception NICE went further than this, stating every committee at NICE will have a minimum of two lay members, as full voting members of the committee, “who play a crucial role by providing a patient/carer/public perspective to the discussions and decisions taken” (6).

Lay members are recruited via an advertisement on the NICE website. They complete an application form detailing their relevant experience and knowledge, as well as declaring any interests which might conflict with the work of the committee. As with all committee members, lay members undergo shortlisting and interview in order to be appointed to a NICE committee. Lay members sit alongside the other members (academic, health professional, and management) as equals with full voting rights.

Enhancing the Role of Lay Members

In 2008, we piloted expanding the HTA committee lay member remit by incorporating a lay member as a member of the “lead team” for each topic that technology appraisal committees were considering. The lead team comprised three committee members who focused on clinical effectiveness, cost-effectiveness, and patient and carer evidence, respectively (Reference Amis, Littlejohns and Rawlins7). Having a lay member focusing specifically on patient and carer evidence meant that this evidence was treated with parity alongside the other evidence. The lay members took turns being the “lay lead” for each topic.

As part of the lead team, the lay members participated in work before committee meetings. They also presented evidence summarized from patient organization submissions at committee meetings (8). The role helped lay members to be more engaged and informed about the topic and the evidence they were considering, and be seen as an integral part of the team.

In 2010 and 2012, we evaluated this enhanced lay member role. Semi-structured interviews with lay members and committee chairs in 2010 identified five key themes: overall experiences, practicalities, expectations, potential improvements, and impact. All agreed the pilot should become routine procedure. Improvements included: setting out expectations of the role more clearly, especially during new lay member inductions; and enhancing the template that patient organizations used to submit patient evidence to give the lay member taking part in the lead team better information to draw from.

The 2010 themes informed the 2012 survey sent to all committee members (n = 117; 88 responses, including all lay members and all chairs). Key findings corroborated 2010 results. The presence of the lay member on the lead team impacted both visibility of patient evidence: 66 percent believed it increased the committee's awareness of patient issues and 74 percent said it improved how NICE addressed patient issues. There was also an impact on the lay members themselves—especially their understanding of topics—and engagement within the committee: “the climate within which a lay person works has greatly improved” [lay member] (unpublished data, 2010).

In addition to their role on NICE committees, lay members also help us to develop HTA methods and processes, and our approach to patient involvement. They act as a critical friend, working collaboratively with us to address issues either as part of working groups or individually, and provide feedback to us via an exit survey when their work with us comes to an end.

The Challenges of Diagnostic Assessments—a New Approach

In 2009, NICE expanded its HTAs to include evaluations of medical and diagnostic technologies (1).

Understanding the long-term clinical and cost-effectiveness of diagnostic technologies presented a challenge for NICE given that the impact of a technology on disease progression, for example, may not be known for some time after the technology is used. Extensive modeling of post-diagnostic care pathways was required to understand how the information provided by a diagnostic technology influenced care and outcomes, compared to standard care.

In addition to the two committee lay members, a patient (or carer) with specific knowledge of the condition or technology being considered is recruited to the committee for that specific topic with full voting rights as a committee member. Patients provide input at the early stages of assessment by participating in scoping workshops and offering their experiences of using a technology (if applicable) or identifying the key clinical and quality of life outcomes for patients which may not be captured in research evidence. The patient who has been appointed to the committee helps shape the technical assessment, including identifying and reviewing the evidence related to the technology, and presents patient evidence at the committee meetings. Patients are supported in this via an induction meeting, an information pack, and support from NICE's Public Involvement Programme and the committee team.

The Role of the Patient Expert

While lay members take a generalist view across many topics, we need to ensure that the experiences, views, and perspectives of people affected by the specific condition or technology that NICE is considering are captured. Since 2001, NICE has invited two individuals (known as patient experts) with that experience to submit a written statement and give testimony at committee meetings for medicines HTAs (8). Patient organization stakeholders nominate and support the patient experts with additional support being provided by NICE. Ideally patient experts balance the breadth and depth of experience of patients with a particular condition. For example, one patient expert may have experience as a member of a patient organization, bringing a range of perspectives, and another may have direct personal experience of the intervention being considered, or a comparator.

Improving How We Work With Patient Experts

We regularly gather feedback from patient experts about their experiences of working with us, to improve how we support them and provide the information they need to participate fully in HTA committee meetings. Evaluation projects, in 2008 (n = 61; 44 responses) and 2012 (n = 62; 49 responses), both recorded patient experts’ overall experience as either “excellent” or “good” in over 80 percent of responses, some “fair”, a few “poor”, and no “awful” responses. One respondent said they “felt [their] presence worthwhile not just tolerated. My contribution listened to with interest. Treated with consideration” [Patient Expert]. Other respondents however reflected a more negative experience: "I do not think that meetings are designed for really listening to patients or patient experts. For this reason I did not feel that I had the opportunity to raise some of the issues that should have been; and that NICE themselves state they want to hear" [Patient Expert].

The 2008 project identified the need for greater involvement of lay people in HTA processes (see above), and other improvements were also taken forward. Since 2012, we have sought feedback immediately after people's participation, to enable a more timely response to issues raised than is possible with large retrospective evaluations. The reported experiences of patient experts have continued to improve, with 96 percent (n = 26) of patient experts stating that their experience was “good” or “excellent” in 2019–2020.

In 2014, we started to involve patient experts in our early dialog (Scientific Advice) program. NICE advises sponsor companies on their evidence generation plans during clinical trial stages, aiming to ensure the best evidence is then available for regulators and HTAs later. Involving patients, or their advocates, during products’ clinical development allows patients to identify outcomes most important to them and ensure acceptability of clinical trial protocols.

Patient Involvement for Novel Interventional Procedures

In addition to medicines HTAs, NICE has developed HTA guidance on novel interventional procedures since 2003 (1). Interventional procedures guidance (IPG) considers the safety and efficacy of procedures used for diagnosis or treatment, and can include things such as making an incision to gain access to the inside of a patient's body; gaining access to a body cavity without cutting; or using electromagnetic radiation.

The procedures considered are normally new, and not yet established in clinical practice. This gives interesting challenges for evidence collection and interaction with stakeholders, as our experience suggests that knowledge and awareness is generally lower than for that of medicines. In the early years of the program, the main patient inputs were comments made at draft guidance consultation, by patient organizations and individual patients and carers.

In 2006, noting the unique insight that patients with experience of specific procedures have (12), NICE enhanced the input of patient evidence to IPGs by seeking their views to directly inform the committee's decision making.

This insight is obtained through anonymous questionnaires, tailored to each topic, distributed to patients via clinicians performing the procedure, and more recently, patient organizations. Questionnaires are returned directly to NICE and a summary report produced for consideration by the committee. Input from patients is not sought for procedures used only in research, and when it is sought it is not always possible to obtain it. An evaluation in 2014 showed NICE obtained patient input for twenty-four topics (69 percent of suitable topics). This input was valuable to cross-reference with other evidence sources considered by the committee, obtain patient quality of life information, and alert the committee to issues not raised in other types of evidence.

The Right Method for the Job—the Challenge of Medical Technologies

Medical technologies in this context include medical devices, active medical devices (powered devices), active implantable medical devices, and in vitro diagnostic medical devices (13).

Approaches to patient involvement needed to adapt to the new challenges posed by medical technologies. We recognized at an early stage that some medical technologies being evaluated would not lend themselves readily to meaningful involvement of individual patients. These are particularly those medical devices that a patient would be unaware of, for example, devices used during surgery where a patient is unconscious, where the main benefit of the device is to the user (such as a surgeon) or where the patient is seriously ill (such as esophageal Doppler monitors). Conversely, there are medical devices about which the views and experiences of individual patients are critical to our understanding of the benefits or disbenefits of the device. These can be devices that are used by patients or their carers themselves, devices which have a direct impact on a person's quality of life, or devices which have clinical outcomes of importance to patients.

We took a flexible approach to individual patient involvement, securing patient expert input or using patient surveys where we and our committees thought that input could be meaningful.

Responding to Feedback: Improving How We Work With Patient Organizations

We understand that, for patient organizations, working with NICE has not always been easy. In 2012, a representative from a patient organization gave evidence to the Health Select Committee (19) (of Parliament) noting uncertainty about what NICE is asking for when we invite submissions from patient organizations and for evidence from patient experts. In 2015, the Triennial Review of NICE noted that “overall there was a sense from [patient organization] stakeholders that patients felt second-best, that their input was not valued as highly as that of clinicians or specialist stakeholders and that NICE could do more to support patients who do get involved” (20).

In response, NICE has iteratively improved the resources and advice we give to patient organizations and evaluated the effectiveness of those changes. In response to the Health Select Committee recommendations, we updated the template patient organizations are asked to complete to submit evidence for medicines HTAs. In 2016 we evaluated this, using three parallel online surveys: patient organizations who had used the updated template, the committee lay members who had used submissions from patient organizations using the new template in their lead team role, and other committee members and chairs.

The survey results showed that a new section in the template was really useful. This asked the person completing the template to highlight five key points to draw to the attention of the HTA committee. Respondents said the template was generally easy to use and understand for both organizations completing it and decision makers. Committee members felt that the revised template had driven up the quality of patient evidence submissions.

Challenges remained for patient organizations in finding the evidence to contribute to the submissions; for decision makers in terms of the variability in the submissions; and for all in developing a shared understanding of the purpose and utility of patient evidence.

NICE has continued addressing these challenges, most recently in seeking comment from committee members about the impact of patient evidence for our Highly Specialized Technologies' HTA program. This assesses treatments for very rare diseases. A critical element of this work involved “completing the feedback loop.” We drew on the experience of our colleagues at the Canadian Agency for Drugs and Technologies in Health (CADTH) to develop feedback letters (21) for patient organizations about where their evidence submission had an impact or revealed new information (Reference Tonkinson, Livingstone, Upadhyaya and Leng22). By providing this feedback, patient organizations can see how the HTA committees have used their evidence and can focus on key areas in future submissions.

In recent years, we have further enhanced patient input into Interventional Procedures assessments by introducing submissions of evidence from patient organizations, using a template proforma, tailored to each topic.

As well as improving and evaluating our own resources, NICE has contributed learning, expertise, and staff resource to helping to develop international patient organization evidence submission templates for medicines, non-medicines (MedTech), and diagnostics HTAs (23), through our membership of the Health Technology Assessment International Patient and Citizen Interest Group.

In 2019, we further expanded our engagement with patient organizations by using a co-production approach to improving patient involvement in NICE HTAs. A core patient working group oversaw the project, and we captured a broad range of patient organizations’ views, experiences, and suggestions for improvement through an event and survey (unpublished data, 2020).