How do we shape a future with no one left behind? In many countries, health technology assessment (HTA) is a relevant consideration for the distribution of limited resources and is used to decide whether a health technology should be reimbursed or not. In the Philippines, under the Universal Health Care (UHC) Act, only health technologies with a positive recommendation from the Health Technology Assessment Council (HTAC) may be reimbursed by PhilHealth. This specific provision allows the government to prioritize scarce resources on health technologies that are proven cost-effective. For individuals with rare diseases, however, this provision puts them at an extreme disadvantage. Currently, PhilHealth’s benefit packages for rare diseases are limited to the Z Benefits for Children with Disabilities which include packages for hearing impairment, visual disabilities, mobility impairment, and developmental disabilities.

As part of HTA, the value of health technologies is often assessed through economic evaluation. Due to the low prevalence of rare diseases, we lack sufficient and robust clinical data about the course and management of these diseases. Many rare diseases are genetic, metabolic, and heterogeneous which makes it challenging to ascertain the value of treatment at a population level. Hence, the traditional HTA process may be considered less relevant given that technologies for rare diseases are often not cost-effective due to their high price and limited effectiveness.

If traditional HTA cannot be applied to health technologies for rare diseases, what else can be used to evaluate such technologies and provide the evidence to decision-makers? Some countries, such as Sweden, have accepted higher cost thresholds per health outcome for orphan drugs. In other countries, HTA agencies have revised their evaluation framework (e.g. Canada and the US), developed a separate review process (e.g. England) or established a separate funding program for rare conditions (e.g. Australia).

In our journey to UHC, we face a difficult trade-off between improving the health of patients in need of expensive orphan drugs and patients in need of other more practical health care interventions. While the HTAC uses multiple criteria in its decision analysis, the current framework by which health technologies are assessed may not be appropriate for rare diseases because the evidence base is limited. The HTAC should develop separate or modified processes to review and make decisions on technologies for rare diseases. Such new or modified evaluation processes can make expensive orphan drugs available, accessible, and affordable to individuals with rare diseases while ensuring that only cost-effective treatments are reimbursed by the government.