PP154 Funding Of Treatments For Rare Diseases In Singapore

Abstract

Introduction

A national multi-stakeholder charity fund has been established in Singapore to provide targeted support to patients with rare genetic diseases whose treatment costs remain unaffordable despite government subsidies and insurance. This presentation will provide an overview of the evaluation, price-setting, and stakeholder engagement processes established to inform the first list of drugs eligible for funding under the Rare Disease Fund (RDF).

Methods

The local prevalence of “rare” and “ultra-rare” conditions was defined in line with international rates (≤4 in 10,000 and <2 in 50,000, respectively) to facilitate an analysis of the rare disease landscape in Singapore, and to identify patients most likely to benefit from the RDF. Public healthcare institutions proposed drugs for consideration, which underwent technical evaluation and were then assessed in line with eligibility criteria by an expert clinical group and prioritized by decision makers for funding.

Results

The number of patients with select rare diseases in Singapore was lower than global estimates contextualized to the local setting. Supporting clinical evidence, funding decisions from overseas health technology assessment agencies, reference pricing considerations, and local budget impact analyses informed the first tranche of drugs (n = 5) recommended. Extensive engagement with pharmaceutical companies was needed to negotiate fair drug prices relative to overseas countries. Additional treatments will be included in the RDF once sufficient funds are raised.

Conclusions

As the evaluation process evolves, wider considerations of disease and treatment experiences from a multi-stakeholder standpoint should be included to inform RDF listings. There is also a need to balance the sustainability of the fund in the longer term with the number of emerging treatments that may require coverage in the future.