Patient-Based Evidence for Antimicrobial Wound Dressings (SHTG)

SHTG, part of Healthcare Improvement Scotland, assesses nonmedicine health technologies, such as devices, diagnostics and changes to treatment pathways, predominantly in rapid HTAs of clinical and cost-effectiveness. It also undertakes some full HTAs that have time scales and resources that allow it to follow the Danish model of HTA (Reference Kristensen and Sigmund9) that includes thorough evaluation of patient aspects and organizational issues alongside clinical and cost-effectiveness.

Patient involvement in HTAs of nonmedicine technologies can be challenging. Often there is no clear patient group to participate in these HTAs and patients may be unaware of the specific intervention used. However, patients’ needs, preferences, and experiences can be essential to ensure nonmedicine health technologies are administered optimally.

SHTG's HTA of antimicrobial wound dressings in patients with chronic leg ulcers (10) demonstrates the way in which patient-based evidence can provide essential evidence to formulate conclusions and develop relevant advice. SHTG found wide variability in the type of antimicrobial wound dressings used in each health board in Scotland, and uncertainty in the clinical and cost-effectiveness of these dressings. To identify sufficient literature relating to patient perspectives and values, SHTG iteratively developed a literature search (Reference Hielkema and Wessels11), adding terminology to capture people living with chronic wounds and any type of wound dressings.

Additionally, SHTG conducted one focus group (n = 8) and six telephone interviews with people in Scotland. Thirteen of the participants had experienced chronic wound treatment with antimicrobial wound dressings and one participant was a relative of a person who had experienced the treatment. Participants were identified and recruited by a practicing tissue viability nurse in a National Health Service board following ethical approval for the research. Qualitative evidence synthesis (QES) was then used to develop a comprehensive patient aspects section for the HTA report (10). This section identified patient communication and education issues, as well as outcomes of importance to patients. For example, while wound healing was the most important outcome to patients, controlling the signs and symptoms, such as odor, exudate, and pain, and preventing infection and wound deterioration were also important outcomes.

This robust evidence base enabled patients’ perspectives and experiences to guide recommendations with three of the recommendations incorporating the needs and values identified in the QES. This additional evidence source also enabled HTA researchers to have sufficient evidence to develop consensus guidance and make recommendations (10).

The impact of patient involvement continued beyond publication of the recommendations as the patient-based evidence guided a short life implementation working group, the creation of a patient version of the HTA report (12), and guidance for Scotland's National Health Service (NHS Scotland) and staff training materials. While such an outcome is persuasive of the impact and potential value of patient involvement in HTA, it also highlights the urgent need to develop this methodology so that it can be used in more rapid HTAs which account for most HTAs in Scotland.

Patient-Based Evidence for Obstructive Sleep Apnea (CADTH)

Over the past few years, CADTH has made a concerted effort to enhance how it incorporates the patient voice within projects related to medical devices and clinical interventions (13). Direct engagement has been challenging as there are not always obvious patient groups to involve when conducting HTAs, for example when assessing diagnostic or screening interventions. As a result, CADTH has explored using QES of patient-based evidence in assessments such as the Optimal Use assessment of interventions for the treatment of obstructive sleep apnea (OSA) in adults (14).

An Optimal Use assessment aims to encourage the appropriate use of a health technology by considering the clinical and cost-effectiveness, safety, and patients’ experiences of the intervention (13). Additionally, it addresses ethical issues, implementation considerations, and any potential environmental impacts. Optimal Use assessments are reviewed by CADTH's Health Technology Expert Review Panel for recommendation

The QES considered the perspectives and experiences of patients, their family members, and their nonclinical caregivers about interventions for the treatment of OSA. This included positive airway pressure therapies such as continuous positive airway pressure (CPAP), oral appliances, surgical interventions and lifestyle modifications, such as diet and exercise. From the resulting thematic synthesis, two primary themes emerged. The first theme identified a range of factors that influence whether people seek and initiate treatment for OSA. For example, the review highlighted factors that might prevent people from seeking a diagnosis or starting treatment such as risk awareness and fear. It also stressed the importance of family, spouses or partners in encouraging diagnosis in the first place. The second theme reflected that all the interventions for OSA are inconvenient, uncomfortable, and require considerable adaptation to daily routines. Some people living with OSA can adapt and incorporate interventions into their lives, although others cannot. This leads to low compliance despite great effort. Support of family members and caregivers again emerged as important in this process.

The QES contributed to the HTA in three major ways. First, the findings allowed for a better understanding of the clinical findings, in particular, how the interventions were used and how this could affect the assessment of the effectiveness of the intervention. In particular, the QES challenged assumptions in the context of compliance as it remains unclear whether some people do not observe a positive effect because they cannot comply with their treatment, or whether they do not comply because they do not observe a positive effect. Second, the synthesis informed the recommendation generated by the expert committee, that is, people with moderate or severe OSA should first try CPAP and then if they cannot tolerate it, consider a mandibular appliance. Third, the review helped to identify important implementation considerations. For example, at times there is insufficient instruction provided on how to use and care for CPAP machines or oral appliances which can affect compliance and effectiveness.

Recognition of the added value of QES of patient aspects has resulted in its wider use in CADTH and an understanding that the influence of patient-based evidence varies across HTA topics. Currently, the syntheses are conducted in parallel with other evidence gathering which is partly due to aggressive timelines. However, CADTH is exploring whether this type of review should be done earlier, perhaps during the scoping phase to better inform the entire assessment. Using QES to explore relevant patient aspects is a rapidly evolving aspect of CADTH's work and has been very well accepted by expert committees.

Patient Input in Tumor Necrosis Factor-Alpha Inhibitors (NICE)

It is likely that HTA bodies and the patients and patient groups that take part in HTAs, may at times have different perspectives about how and when patient involvement added value to an HTA. An example is drawn from a case study prepared by a patient group, the National Ankylosing Spondylitis Society (NASS), following its participation in a National Institute for Health and Care Excellence (NICE) HTA of tumor necrosis factor (TNF)-alpha inhibitors for ankylosing spondylitis and axial spondyloarthritis (15). NASS recorded the case study to enable other patient groups to learn from their experience.

The case study suggests that to have impact, a patient group needs to undertake considerable work before, during, and after the initial assessment. NASS identified the need to keep up to date with therapy developments in its areas, including monitoring the medical advisory board and the latest clinical research papers and attending conferences. NASS also gained a solid understanding of the needs of people living with ankylosing spondylitis in the United Kingdom by conducting surveys every 3 years with their members, taking 5,000 calls per year on their helpline, speaking to members at an annual members’ day and awareness events around the United Kingdom, and interacting through social media. This preparation enabled NASS to provide an informed response within the short timelines when NICE announced the appraisal.

NASS drew on its knowledge base when completing the NICE template for patient group submissions to provide information about the impact on daily life of ankylosing spondylitis, but also ran an online survey with their members during the submission period to identify patient views on specific issues, such as intravenous infusion and sequential treatment. For example, the online survey used closed questions to gather statistics such as the number of patients currently on anti TNF therapy and open questions that could be analyzed to gain a greater understanding of the advantages and disadvantages of treatment options. The survey was open for 4 weeks and attracted 608 responses. When the data were analyzed, it provided NASS with useful statistics, themes, and illustrative quotes.

Following a review of the evidence and submissions, NICE published its draft recommendations in its Appraisal Consultation Document (16). At this point, NASS had 20 days to comment on the draft recommendations, which included two important areas for people with ankylosing spondylitis that were negative. First, infliximab was not recommended for the treatment of ankylosing spondylitis. Second, treatment with another anti TNF therapy was not recommended for people whose disease had not responded to treatment with the first anti TNF therapy, or those who had an initial response which was then lost.

In response to the draft recommendations, NASS conducted another survey with members on these two issues, particularly seeking the views of people who had switched anti TNF therapies, reasons as to why they had switched, and how beneficial this switch had been. The survey was open for only 8 days due to the consultation deadline, but attracted 858 responses which provided statistics and quotes to clarify patients’ experiences and needs.

From these data, NASS was able to provide a slide presentation (including graphs and quotes from patients) for the NICE expert committee on the sequential use of anti TNF therapies and the specific people/populations who might benefit from infliximab, because it was not a self-administered treatment. These included people with memory loss, learning disabilities, dexterity problems, or a fear of needles.

These real-life data resulted in the negative recommendations being changed as follows:

Patient Input – Submissions, Surveys and Representatives (CONITEC)

In Brazil, community participation is stipulated as a right in the Brazilian Federal Constitution and other laws, including those related to the Brazilian Public Health System (SUS). The Law 12,401/2011 created the National Committee for Health Technology Incorporation (CONITEC) and formalized public and patient involvement (PPI) in the SUS HTA process through: public consultations for all recommendations about the inclusion, exclusion, or modification of health technologies; public hearings, depending on the assessment (not yet undertaken); and the participation of the National Health Council, which represents citizens and users of SUS, as a member of CONITEC (17).

Usually, CONITEC recommendations are submitted for public consultation for 20 days, but consultation can be prolonged. Anyone can provide comments on the recommendations if they identify themselves.

In 2014, a consultation form specifically for the public was created to capture the patient and caregiver perspectives of the technologies being assessed. Currently, there are two forms for public consultation comments available online. The first, seeking technical and scientific information, is usually completed by health professionals, industry, and the general public. The second aims to capture opinions or experiences and is completed by patients, caregivers, and health professionals (Reference Silva, Petramale, Rabelo, Santos, Facey, Hansen and Single18).

The number of submissions received from patients and the public is one measure of patient involvement impact (Reference Silva, Petramale, Rabelo, Santos, Facey, Hansen and Single18). In 2014, CONITEC began to disseminate a list of upcoming public consultations on its website and e-mail lists and partner's social media. This resulted in a more than 400 percent increase in submissions annually; from 2,584 submissions in 2014 to 13,619 submissions in 2015. This figure was influenced by two topics, that is, beta interferon 1-a in multiple sclerosis (19) and cesarean section (20), which attracted 4,846 submissions and 3,706 submissions, respectively. However, an exponential increase can still be noticed if these two topics are excluded.

Between January 2012 and June 2017, CONITEC performed 219 public consultations, receiving more than 30,000 submissions with more than half of these from SUS users. Since 2015, CONITEC has produced summary versions of its technical reports for the public and patients in plain language to aid understanding of the reports and enable patients to contribute more easily. This tool may also have contributed to the increase in community participation.

To investigate patient needs and preferences, CONITEC began conducting patient surveys in 2015 to inform its Clinical Protocols and Therapeutic Guidelines (PCDT). By June 2017, 13 surveys had been completed. Patients provided information about their diseases and made suggestions, such as healthcare improvements, appeals for new technologies and aspects that in their perspective critically needed addressing in the PCDT.

In 2014 and 2015, patient representatives took part in CONITEC's plenary sessions aimed at solving issues related to the use of technology including “Budesonide and Formoterol in aerosol for the treatment of asthma” (Reference Silva, Petramale, Rabelo, Santos, Facey, Hansen and Single18). One of the patient representatives taking part in a plenary session, a guest patient representative of the Brazilian Association of Asthmatics (Associação Brasileira de Asmáticos, ABRA), reported the difficulties faced by patients to control the disease, the high cost of treatment and, most importantly, the educational process needed for safe and correct use of medicines. The patient representative ratified the similar efficacy of the analyzed technologies and identified education as the most important issue as patients were often unaware of how to correctly use medical devices in asthma.