It’s widely accepted that seeking patient input into their healthand care improves outcomes for them and their communities, improves services and drives efficiencies in healthcare utilisation. And learning from this, many pharmaceutical and device companies have adopted a range of approaches to seeking patient input throughout the clinical development and commercialisation of their products. However, what does this look like in practice? And, in the absence of the widespread adoption of rigorous approaches to undertaking and evaluating this, is the industry at risk of patient involvement becoming just a tick box exercise?
The industry is not alone in these considerations. The need for robust evidence means that healthcare technology assessment (HTA) agencies have been grappling with the challenge of seeking meaningful patient involvement for many years, supported, and indeed encouraged by organisations such as Health Technology Assessment International’s (HTAi) Patient and Citizen Involvement in HTA Interest Group (PCIG), a leading organisation advancing the patient and citizen involvement agenda.
So, can industry learn a thing or two from HTAi PCIG and those HTA agencies that are so often considered a hurdle to patients getting access to the medicines they need. The answer is a resounding yes!
HTA and meaningful patient involvement
Health technology assessment supports decisions about the reimbursement of new health technologies. At its heart, it is a policy-making process that assesses if a health technology is clinically and cost-effective in the real world. It may also consider social and ethical aspects of using the technology.
Given that HTA is a policymaking process, HTA agencies seek – or are assertively being encouraged to seek – meaningful patient participation in their decision-making process. The rationale for this is clear. Just like prescribers and researchers, patients are experience-based experts. Indeed, no one else can explain the impact of a disease better than a patient. And here is the crux of the matter. An individual patient can share what matters the most to them, what day-to-day challenges they face, and what they need for a better future. Yet, it is simply that. A person’s views and experiences.
If the patient ‘voice’ is to add real value to their decision-making process, HTA agencies are looking for evidence on patients’ experiences that they can use to plugs the gaps in peer-reviewed data on patients’ needs and preferences that they may rely on. This necessitates patients, carers and those that represent them to bring to the HTA decision-making table evidence from other patients; evidence that they need to be encouraged and supported to source.
So what can industry learn from HTA agencies?
Patient input: Patient input is exactly that, it is input provided by patients, their carers or those who advocate for them, such as patient organisations. It may be written, or verbal, such as taking part in a patient advisory board or similar. As a form of input, it must be two-way and never simply a one-off. This enables individuals to share their experiences and thoughts at multiple stages in the decision-making process, and during implementation of the decisions made.
Evidencing patients’ experience: While an individual’s experience can provide valuable personal perspective, industry needs to encourage those they seek input from to come to the table with evidence from multiple patients. As most of those industry engage with are representative of patient organisations, this is an ask that should not be too challenging.
Insight into the needs, preferences and experience of patients can come from a range of sources. These may include: data from calls to a telephone helpline; a survey of patients’ experiences; focus groups; round-table discussions; individual discussions with patients; insight from other groups in a network, support groups that can be tapped into; insight from support workers; issues that have been discussed on websites, closed chatrooms; and issues raised and discussed at meetings.
However, in informing decisions, this form of evidence should be seen as complementary to, and not a substitute for, any peer-reviewed patient-based evidence that may be available, or indeed, any similar data that a company should be generating.
Values for meaningful patient involvement
In addition to encouraging patients to bring additional evidence to the table, industry might benefit from considering the values for meaningful patient involvement. HTAi PCIG has defined five values for involvement in HTA (https://htai.org/interest-groups/pcig/values-and-standards). These could be adapted by an organisation to help set a new framework for patient involvement, while aligning internal stakeholders behind its value.
Relevance: Patients have knowledge, perspectives and experiences that are unique and contribute to essential evidence for HTA.
Fairness: Patients have the same rights to contribute to the HTA process as other stakeholders; have access to processes that enable effective engagement.
Equity: Patient involvement contributes to equity by seeking to understand the diverse needs of patients with a particular health issue, balanced against the requirements of a health system that seeks to distribute resources fairly among users.
Legitimacy: Patient involvement facilitates those affected by HTA recommendations/decisions to participate in the HTA, contributing to the transparency, accountability and credibility of the decision-making process.
Capacity building: Patient involvement addresses barriers to involving patients in HTA and builds capacity for patients and HTA bodies to work together.
First step to advancing meaningful patient involvement
Advancing meaningful patient involvement across an organisation will not be without its challenges. However, start by asking the following simple questions: does patient input involve seeking the opinion of a representative from a few select patient organisations alone?; does patient involvement focus on engagement at the beginning of planning, but not thereafter?; and does investment in understanding patients focus on bespoke research, that after informing development of a patient personas, is rarely referred to again?
Answering yes to one or more of the above is a clear indication that now may be the time to reconsider approaches to patient input. Taking a look at the academic rigour encouraged by HTAi PCIG may just be a good place to start
