Background and Importance Access to innovative medicines requires extensive and careful pharmaco-economic evaluation.

The inclusion of patients in Clinical Trials (CT) allows early access to new experimental medicines and considerable economic saving for the healthcare system.

Aim and Objectives Evaluate the economic benefit of including patients with hereditary transthyretin amyloidosis (hATTR) in clinical trials between 2018 and 2023.

Material and Methods Retrospective analysis of paramyloidosis-related clinical trials taking place at the centre since 2018. The data collected were the number of paramyloidosis-related CT, the number of patients included the time of participation in the CT and the average price of conventional treatment.

Results At our Clinical Trials Unit there are currently 6 Paramyloidosis-related CT underway, involving a total of 65 patients.

In economic terms, patient participation on ongoing CT related to Paramyloidosis has led to a cumulative saving of 15,667,487.98€, compared to the costs of conventional therapy (tafamidis¹,inotersen² and patisiran³).

The distribution of annual savings was:

• 2019: 644.396,70€

• 2020: 2.447.335,64€

• 2021: 4.465.670,09€

• 2022: 4.206.997,00€

• August of 2023: 3.903.088,55 €

Conclusion and Relevance Participation in CT allows early access to new experimental therapies and contributes to the development of new drugs and/or new therapeutic indications. In Paramyloidosis, new agents like TTR stabilisers, subcutaneous antisense oligonucleotides and iRNA therapies are potential new alternatives.⁴

By participating in CT, centres obtain an extra source of funding. The participation of patients in CT also allows for a reduction in costs, through the preservation of financial resources and medication.

The savings generated by the participation in CT help to provide better care and an efficiency healthcare system.

References and/or Acknowledgements 1. European Medicines Agency – Vyndaqel® Public assessment report. Available at: https://www.ema.europa.eu/en/documents/product-information/vyndaqel-epar-product-information_pt.pdf

2. European Medicines Agency – Tegsedi® Public assessment report. Available at: https://www.ema.europa.eu/en/documents/product-information/tegsedi-epar-product-information_pt.pdf

3. European Medicines Agency – Onpattro® Public assessment report. Available at: https://www.ema.europa.eu/en/documents/product-information/onpattro-epar-product-information_pt.pdf

4. Gertz MA, Mauermann ML, Grogan M, Coelho T. Advances in the treatment of hereditary transthyretin amyloidosis: A review. Brain and Behavior. 2019;9(9). https://doi.org/10.1002/brb3.1371

Conflict of Interest No conflict of interest.