Health | Availability of medicines to treat orphan or rare diseases

To ask the Minister for Health the extent to which adequate provision continues to ensure the availability of medicines to treat orphan or rare diseases; the extent to which the provision is adequate based on experience; and if he will make a statement on the matter.

REPLY

The 2013 Health (Pricing and Supply of Medical Goods) Act is a clear, robust, statutory framework under which all new medicinal products must be assessed by the HSE. The HSE is the statutory decision-maker for the reimbursement of such products, and the Act ensures that these decisions are made on an objective and scientific basis.
The Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2021 seeks to remove any threshold incremental cost-effectiveness ratio (ICER) informed by the Quality-Adjusted Life Years (QALY) metric from the assessment of orphan medicinal products while retaining the responsibility for the HSE to consider cost-effectiveness under modified criteria.
The QALY is the economic evaluation system which is used to set a threshold of cost-effectiveness when assessing medicines. Using the QALY, the National Centre for Pharmacoeconomics (NCPE) can systematically assess new medicines for cost effectiveness. Disregarding a validated systematic evidence-based scientific methodology to inform decision-making would greatly limit the HSE’s capacity to determine whether the submitted price for orphan drugs represents value for money. In the absence of cost-effectiveness information on orphan drugs, the HSE would enter commercial negotiations in an uninformed, and therefore disadvantageous position.
The 2013 Health Act does not provide for a different ruleset when assessing orphan medicines. However, the criteria that apply to the evaluation process allow the HSE to have particular regard for the unique circumstances surrounding orphan drugs. The statutory framework in the 2013 Act requires the HSE to take account of a range of objective factors and not just cost-effectiveness under the QALY system.  In making reimbursement decisions, there is a list of criteria contained in the Act which the HSE is required to consider, including the magnitude of the clinical effect, cost effectiveness, budget impact, available resources, and unmet medical need.
Consequently, the QALY economic evaluation system does not restrict the HSE from applying discretion particularly in seeking to address unmet medical need. In making reimbursement decisions, the HSE often reimburses orphan medicines in excess of a cost per QALY of €45,000 in cases where the potential to address unmet need is supported by robust clinical evidence.
Budget 2021 allocated €50 million for the approval of new drugs and nineteen of the fifty-two new medicines approved by the HSE in 2021 were orphan medicines, representing over one third of approvals. Budget 2022 allocated a further €30 million for the approval of new drugs. The HSE has approved 36 new medicines/new uses of existing medicines to date in 2022 including three medicines  for the treatment of rare diseases.
Notwithstanding the concerns outlined above, the Bill is honourable in its intent and must serve as an opportunity to explore the policy options available to us. We all want an assessment process for orphan medicines which provides our most vulnerable patients with access to the innovative treatments which they deserve. The Bill represents an opportunity to comprehensively look at the current system and consider how issues such as transparency and communication can be improved, and I have asked my officials to do that.