Neil Bertelsen, PFMD Board member, sees parallels in how Health Technology Agencies (HTA) and medicines regulators are working to incorporate patient perspectives into decision-making.

Health Technology Assessment (HTA) bodies play a vital role in modern healthcare systems. Based on expert clinical and economic assessments, they evaluate new technologies – including medicines and medical devices – and help to shape decisions about their reimbursement and use. In some countries, patients play an active and growing role in these evaluations, but others are just beginning to explore how to incorporate patient experiences in their decision-making.

Neil Bertelsen, a member of the PFMD Board and of the HTAi Interest Group on Patient & Citizen Involvement, has seen increasing interest among HTA bodies in understanding how patients experience their disease and how new technologies may impact patient care and quality of life. Hearing the voices of patients and caregivers can help improve the quality of HTA decisions by focusing on issues that are most relevant to patients.

‘The decisions HTA agencies make can profoundly affect the treatment and care available to patients,’ he says. ‘By bringing patients, carers and citizens into the process, it offers them the opportunity to input into those decisions and helps improve understanding of how decisions are made.’

Defining good data

At the same time, medicines regulators – like the US FDA and the EMA – are also exploring how they can take account of patient experience of disease and expectations of new treatments. The FDA already has several patient-engagement initiatives underway while the EMA’s 2025 strategy speaks of integrating meaningful patient relevance measures into their work and working more closely with HTA agencies.

‘HTA bodies and regulators are asking the same questions about how patients experience their disease,’ Bertelsen says. ‘There’s a lot of overlap in terms of their goals, but there is a need to define what good evidence looks like in terms of patient experience and patient preferences. PFMD could help bridge the gap by defining good practices on how data is collected and interpreted.’

Several global initiatives also have a role to play in defining patient preferences and patient-important outcomes, including Innovative Medicines Initiative (IMI) projects such as PARADIGM and PREFER.

The ideal situation, where companies and patient organisations systematically collect data on patient experiences and preferences for use in regulatory approval and HTA, is still some way off, but we are starting to see the beginnings of advice and guidance on this. Internationally, the main challenge for HTA is fragmentation. Individual countries control their own health policies and budgets and so HTA processes, as a national discipline, can use different methods and come to completely different conclusions. Even within the EU, there are significant differences and long-standing proposals for greater harmonisation have stalled.

However, the trend towards greater patient involvement in medicines development, approval and uptake is clear. The next step is to make the process more efficient. ‘The FDA is encouraging industry to submit patient experience data, but companies are exploring how they can use this in submissions to HTA bodies,’ explains Bertelsen. ‘This means HTA agencies have to think about how they will interpret patient experience data, as well as what kind of evidence they will accept and from whom.’

Patient data repositories

Patient advocacy organisations also have much to gain from a more systematic approach to how their input is captured and used in decision-making. Some HTA bodies already ask patient groups to submit evidence to the decision-making committee. These can include data from surveys and patient registries as well as patient stories and practical examples on the impact of a disease and its current treatment. However, this can be a resource-intensive task.

Health technology assessments do not happen simultaneously in all parts of the world. Patient groups in countries where a new technology is launched first will have already developed evidence needed for their national HTA. What is missing is a way of sharing this research so that other patient groups in countries can benefit from this work. This could be an opportunity for organisations like PFMD and HTAi, says Bertelsen. ‘Instead of having every national patient group collect more evidence, there may be opportunities to collaborate and share data in a repository – avoiding duplication and ensuring that smaller patient organisations can access the great data and evidence generated by other patient groups around the world.’

While there is still a long way to go in building patient-centricity into all HTA and regulatory decisions, great progress has already been made. In future, this should deliver new generations of medicines and medical technologies that have been developed, approved and evaluated to reflect patients’ needs, expectations and preferences. The only question is how quickly we can get there.