New approach gives patients a stronger voice in HTA

Patient involvement is increasingly recognised as a cornerstone of effective Health Technology Assessment (HTA). To support this, an international Summary Information for Patient Groups template was developed, offering plain language summaries of new medicines under assessment. Pilot programs using the template were conducted in Australia and England, allowing HTA bodies to trial the approach within real-world assessments.

Pilots conducted by the National Institute for Health and Care Excellence (NICE) and Pharmaceutical Benefits Advisory Committee (PBAC) confirmed the positive impact of providing plain language summaries, supporting patient organisation input. As a result, the template has now been incorporated into the NICE HTA process.

In the Australian context, the Consumer and Community Engagement Unit (CEEU) at the Office of HTA, within the Department of Health, collaborated with the PBAC to conduct an evaluation using an adapted HTAi template. The initial pilot involved a partnership with Bristol Myers Squibb (BMS), producing summaries for two submissions.

The five patient organisations involved in the PBAC pilot responded positively. They found the summaries highly useful, with one commenting that “it’s gold!,” noting that the document substantially reduced the time spent searching for information. In some cases, the summaries enabled organisations to gather targeted insights from patients and carers. For example, one group designed surveys focused on quality of life and patient perspectives regarding the medicine under assessment.

Patient organisations also reported increased confidence in contributing to HTA processes. Information regarding the treatment, comparators, place in the management pathway, and setting was particularly valued when presented in a clear, concise format. Feedback suggested that improved use of diagrams, tables, and formatting could enhance clarity and reduce summary length.

Pharma industry representatives tasked with completing the summaries were broadly supportive. A Medicines Australia survey confirmed strong feasibility, with thirteen of fourteen companies indicating they could complete the template.

Reflections from the CEEU highlighted that providing patient organisations with clear, concise information enabled them to better contribute perspectives on the impact of new medicines and identify additional considerations for PBAC. The pilot led to seven additional summaries involving various products and patient organisations.

Evidence from both Australia and England indicates that the summary improves submissions by providing context to patient organisations, although further accessibility enhancements are required. Yet, both industry and HTA bodies highlighted resource challenges in implementing and reviewing summaries, despite widespread support for the approach. The pilots revealed substantial resource demands, including staff time for implementation, stakeholder engagement, and evaluation. Future pilots should address these needs, and securing buy-in from industry, senior leadership, and committee members is critical for successful resource allocation.

Governance and mitigating bias are key considerations, particularly as submissions involve information prepared by companies. Guidance emphasises that summaries should be no longer than twelve pages and written in plain language. If not properly completed, the HTA body returns the summary for revision. To ensure equity of access, both Scottish Medicines Consortium (SMC) and NICE have made completion of the summary mandatory for company submissions.

To optimise patient contributions, information must be provided as early as possible. However, regulatory barriers may prevent sharing data with patient organisations before a company receives regulatory approval.

In Australia, the summary was provided to patient organisations when the agenda for the upcoming PBAC meeting was published. Over approximately eight weeks, “consumer comments” could be submitted ahead of the meeting. The CEEU and PBAC’s consumer nominee then collated and presented the information to PBAC. While eight weeks may not always be sufficient for patient experts to gather comprehensive input, this timeline balances the need to avoid delaying PBAC decision-making.

NICE and PBAC will continue refining pilots, guidance, and requirements. Meanwhile, the HTAi Patient and Citizen Involvement in HTA Interest Group (PCIG) will update international templates and guidance based on global learnings.

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