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The challenge of HTA - supporting patient advocacy groups

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The intersection of rare diseases with Health Technology Assessment (HTA) is a complex juncture in the modern healthcare landscape. As the demand for patient-centric decisions escalates, understanding the nuances of HTA becomes vital for rare disease communities to advocate effectively.

This piece, delves into the complex world of HTA, the challenges rare disease communities encounter, and the transformative potential of co-created educational initiatives.

The HTA Maze: Where Rare Disease Communities Often Get Lost

HTA, by design, is a comprehensive evaluation mechanism assessing the clinical, economic, social, and ethical aspects of healthcare interventions. However, its multifaceted nature often poses challenges for the rare disease community:

  1. Complex Evaluations: Understanding HTA's intricacies, which incorporate evaluations ranging from clinical effectiveness to cost-effectiveness, often becomes overwhelming for the uninitiated.
  2. Data Challenges: The rarity of these diseases implies limited patient data, complicating assessments when evaluated through the traditional HTA frameworks.
  3. Misalignment of Priorities: Rare disease patients' lived experiences and their insights into quality of life are often undervalued or overlooked in HTA's quantitative evaluations, leading to potential disparities in assessments.

The Transformative Potential of Co-created Educational Programmes

In the face of these challenges, the rare disease communities can significantly benefit from tailored educational resources designed to elucidate the HTA process.

The potential advantages of such programmes are manifold:

  1. Demystifying the HTA Process: Customised educational programmes can break down the complexities, offering a clearer understanding that empowers rare disease communities.
  2. Encourage Informed Advocacy: An understanding of HTA equips the community to advocate effectively, ensuring their needs align with HTA metrics and evaluations.

Real-world Triumphs of Collaborative Educational Initiatives

Examples illustrating the potential of collaborative educational initiatives:

  1. EURORDIS and HTA Training: Recognizing the knowledge chasm, EURORDIS (a non-governmental patient-driven alliance) collaborates with stakeholders to deliver comprehensive HTA training. This initiative enhances rare disease patients' abilities to contribute constructively to policy dialogues, embodying the potential of collaborative educational ventures.
  2. The Orkambi Journey in the UK: Equipped with an understanding of HTA, the UK's cystic fibrosis community played a pivotal role in influencing discussions surrounding the drug Orkambi. Their informed advocacy was instrumental in shaping outcomes, underscoring the power of knowledge .

The Fruitful Convergence: Depth, Tailoring, and Impact

Collaborative ventures between experts and patient communities foster a learning environment characterized by:

  1. Depth of Understanding: Collaborative programmes can delve deeper into the nuances of the HTA process, equipping participants with an in-depth understanding.
  2. Tailored Content: Such programmes, designed with insights from patient groups, ensure content relevance, addressing specific challenges, and concerns that are paramount to rare disease communities.
  3. Enhanced Impact: When education is tailored to the community's needs, the resultant understanding and advocacy are significantly more impactful.


For rare disease communities, understanding HTA is more than an academic exercise—it's a vital tool for effective advocacy, shaping their healthcare journey. The challenges of navigating the complex HTA process are undeniable, but with collaborative educational initiatives, these challenges are not insurmountable.

Co-created programmes, built on a foundation of shared expertise and lived experiences, promise a future where rare disease communities are not merely passive recipients but active, informed participants in the healthcare discourse.

Such partnerships not only demystify the HTA labyrinth but also empower the rare disease community to advocate for healthcare decisions that truly resonate with their needs and aspirations.

Swii.ch Health support a number of pharmaceutical clients with their patient advocacy training initiatives, including the HTA/health economic framework, processes, and tactical strategies.

A recent training programme delivered virtually with supporting eLearning resources, focussed on inter-country variations in the HTA process and the paramount importance of patient engagement.

Co-created with HTA experts and patient advisers the initiative was crafted to enable patients to lend their distinct voice to the HTA processes, aligned with specific country requirements.

If you would like to discuss how we could support your patient engagement needs please contact Rob Wyer - r.wyer@swii.ch


  • Drummond, M.F., Sculpher, M.J., Claxton, K., Stoddart, G.L., & Torrance, G.W. Methods for the Economic Evaluation of Health Care Programmes. Oxford University Press.
  • Facey, K., Boivin, A., Gracia, J., Hansen, H.P., Lo Scalzo, A., Mossman, J., & Single, A. Patients' perspectives in health technology assessment: A route to robust evidence and fair deliberation. International Journal of Technology Assessment in Health Care, 26(3), 334-340.
  • Hailey, D., Werko, S., & Bakri, R. Involvement of consumers in health technology assessment activities by INAHTA members. International Journal of Technology Assessment in Health Care, 25(1), 78-83.
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