Patients, their families and caregivers are increasingly being recognised as therapeutic area experts, given their day-to-day hands-on experiences with their respective conditions. Underpinned by the ever-greater availability of online medical information, patients are becoming more involved in, and engaged with, their healthcare.
Many patients have a clear desire to contribute to the drug development process. The same holds true for involvement with HTA activities, with many efforts undertaken by patient associations, advocacy groups and HTA agencies in Europe to facilitate this.
Alongside these efforts, there are opportunities for the pharmaceutical and medical device industry to help bring the patient voice into HTA discussions. Incorporating patients’ insights on the value an intervention could provide in a particular therapeutic area may help improve patient benefit and fulfil the unmet need.
“The ultimate aim for pharmaceutical companies is that patients have access to treatment. Market authorisation is the first step of the process, but it doesn’t ensure that the patient will be able to access a treatment. Access follows from reimbursement decisions made by HTA agencies,” says Krystallia Pantiri, a research consultant at Pharmerit International, a consultancy with groups specifically focused on market access and patient-centred outcomes.
She adds: “Cooperating with patients during the HTA process and exchanging knowledge with them helps companies to focus on patients’ needs.”
To dig deeper into how pharmaceutical companies can best engage with patients, Pharmerit recently undertook a qualitative study on the European landscape of patient engagement in HTAs.
Commissioned by the European Federation of Neurological Associations (EFNA), the project sought to explore the HTA processes in place, and these processes’ impact, from the perspectives of patients and HTA members/experts.
Building on the findings from literature for the UK, Germany, and France, Pharmerit conducted a series of patient and HTA stakeholder telephone interviews from the three countries, each of which have distinct patient engagement models.
In the UK, patients can attend HTA meetings held by the National Institute for Health and Care Excellence (NICE). If they disagree with the decisions taken by NICE, patients can appeal against them, but they cannot directly vote for or against those decisions.
Patients in Germany don’t have voting rights for reimbursement decisions. The Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) does publish assessment reports on the Gemeinsamer Bundesausschuss (G-BA) website and requests input via a public session. However, patient associations are very rarely approached by IQWiG.
In contrast, patient voting rights are present in France and its HTA body, Haute Autorité de Santé (HAS), publishes details of its future assessment plans. However, France’s seeming transparency can still present a challenge to patient associations, because the onus is put on them to check what kinds of assessments are happening and when.
When patients do not have a voice at the voting stage, they are treated unequally to the rest of the stakeholders, potentially undermining efforts for meaningful patient engagement in HTA.
A common issue in the UK, French and German systems is that there is often misalignment between formal HTA process and real-world practices. Across countries, patient and expert feedback highlights four areas for improvement in meaningful patient engagement to facilitate better integration of the patient perspective in HTA decision-making: shared purpose among the stakeholders, representativeness of the stakeholders, transparency in communication and capacity and capability for engagement.
Pantiri explains: “HTA authorities expect patients to generate evidence not available from other sources, help interpret the existing data and provide feedback on data relevance rather than replicating the evidence that somebody else has produced. Patient associations, on the other hand, want to impact the decisions and to make sure that their perspectives are heard. So patients’ focus is on being able to attend assessment meetings, vote on their decisions and be seen as equal to the rest of the stakeholders.”
In addition to a lack of shared purpose, there can also be an issue if the patients present in assessment meetings are not wholly representative of individuals with the specific disease being discussed. When this occurs, an opportunity is missed to understand the comprehensive experience of the condition.
HTA processes themselves can hinder patient engagement, perhaps through a lack of transparency in communication and documentation. For instance, NICE provides very detailed formal procedures and guidance for people who want to get involved and provide input to its assessments but without feedback to the patients on how their input is subsequently used.
Pantiri says: “They welcome the input but there is no mechanism to check if it was taken into account in any part of the process. Even if the input wasn’t useful, if NICE doesn’t give feedback to the patient associations that took the effort to get involved, they won’t know where they need to improve.
From a pharmaceutical perspective, companies need to be aware that patients often lack the skills or resources to navigate HTA processes and contribute effectively. “Many also face problems of sustainability – their volunteers engage in HTA without financial support and it takes time away from their home life, work or family engagement,” Pantiri notes.
To some extent, the solution to these problems is a financial one. “Without funding, patients are left with few opportunities to mobilise resources and organise information in a way that will reach the level of the rest of the stakeholders. In the end, the better the quality of information, the more likely it is that their evidence will be taken into account,” Pantiri says.
These challenges are then accentuated by Europe’s scattered HTA landscape, which requires pharmaceutical companies to tailor their approaches to patient engagement in HTA on a country-by-country basis.
Pharmaceutical companies can help maximise the effectiveness of their future patient engagement efforts within HTA in Europe. Starting with engaging patient associations earlier in the process – i.e. phase III trial design at the latest – can ensure that the endpoints captured in a clinical trial are relevant to the patients and reflect the comprehensive experiences of the condition.
“It is important to share knowledge and agree on quality standards in order to support the implementation of engagement practices and then to allocate resources to sustain change over time,” Pantiri recommends.
There are many different ways that pharmaceutical companies can involve patients at different phases of a product’s development, such as consultation interviews on the design of clinical trials, entry and exit interviews in trials, concept elicitation interviews to understand a patient’s journey with the disease and treatment, and cognitive interviews to evaluate and refine outcomes measurement tools based on patient input. The key thing here, Pantiri says, is to ensure that activities are relevant and meaningful to patients.
She concludes: “It’s very important that you have conversations with the patients to see if your outcomes are indeed aligned with patients’ actual experiences.”
Krystallia Pantiri is a research consultant in strategic market access and patient-centered outcomes, located in Pharmerit’s Rotterdam office, in the Netherlands. Her all-round experience includes (systematic) literature reviews to inform network meta-analyses, global value dossiers and HTA/reimbursement dossiers, patient/stakeholder engagement, preference studies and qualitative patient reported outcomes (PRO) research. Disease areas she has worked in include vaccines, inflammatory disease, respiratory diseases, oncology, hematology, neurological disorders and orphan diseases.
Pharmerit International is a global, premier health economics and outcomes research (HEOR) consultancy with 20 years of experience supporting pharmaceutical, biotechnology, and medical device organisations in health economics and outcomes research (HEOR) and market access worldwide. Pharmerit delivers quality research across four multi-disciplinary Centres of Excellence, Modelling & Meta-Analysis, Patient-Centred Outcomes, Real-World Evidence, and Strategic Market Access. Pharmerit recently announced that it will be merging with the OPEN Health Group. The combined entity will have approximately 700 people, 15 offices, and representation across three continents. The consolidation of publications, medical communications, HEOR, RWE, and market access creates a unique entity equipped to be a leading global HEOR and Medical Affairs Consultancy.