Professor Michael Barry:

I thank the committee for the opportunity to speak to it today.

The mission of the NCPE is to facilitate health care decisions on the reimbursement of technologies, usually pharmaceuticals, by applying clinical and scientific evidence in a systematic framework, in order to maximise population wellness.

The NCPE considers the clinical effectiveness and health related quality of life benefits in addition to all relevant costs, including costs that arise from savings from reduced health care resource use, for example, hospitalisation, following the new technology. We also then try to assess whether the price requested by the manufacturer is justified. The NCPE will then advise the HSE on the cost effectiveness or value for money and the budget impact associated with the specific product.

A medicinal product is designated as an orphan medicinal product if it is intended for the diagnosis, prevention or treatment of life threatening conditions affecting no more than five in 10,000 persons in the European Union at the time of submission of the designation application. The NCPE has a standardised process and criteria for the evaluation of pharmaceutical products, including orphan drugs. All assessments are conducted in accordance with published health technology assessment, HTA, guidelines issued by the Health Information and Quality Authority, HIQA. These guidelines were first developed in 2010 and updated in 2014. They are available on the HIQA website.

The NCPE HTA process is well established and usually commences when the relevant pharmaceutical company receives notification from the HSE corporate pharmaceutical unit, HSE-CPU, of the requirement for a rapid review. A rapid review is a quick look see, as it were, looking at the benefits and otherwise of the medication, to assess if we must examine it in more detail. The manufacturer submits the rapid review document, and the template is available on our website. That will include a range of information, for example, the regulatory status, the clinical condition, the proposed licensed indication, anticipated place in therapy, comparators, clinical evidence, safety, efficacy in addition to economic considerations. It is a wide range of information. The NCPE reviews this document within four weeks, and we usually are on time with that, to determine whether a full pharmaco-economic assessment is required. If it is not required the medication is usually reimbursed. The data from 2010 to 2015, which was published recently, shows that approximately 50% of products do not require a full assessment and therefore go straight through to reimbursement. That means 50% will have to be looked at in detail.

If a full pharmaco-economic assessment is required the manufacturer is asked to submit a full dossier to the NCPE. The application forms are on the website. A full HTA investigates in detail the value for money proposition associated with medications. Orphan drugs are assessed in the same way as other medications. The assessment includes a description of the relevant condition, its management and a detailed outline of the intervention under assessment. The clinical evidence supporting the efficacy of the product is reviewed. The manufacturer is required to outline in detail the health economics relating to the product and provide an estimate of the incremental cost effectiveness of the drug, in other words, what the added benefit is for the increased cost associated with that product. A budget impact analysis is also required in respect of the cost effectiveness analysis.

The HTA submission process also facilitates submissions by patient groups who wish to have their views taken into consideration. The patient group submission template is also available on the NCPE website. Having reviewed all the available documentation the NCPE submits its report to the HSE corporate pharmaceutical unit following a check for factual accuracy by the manufacturer. The manufacturer gets our report before we send it to the HSE to ensure that we are factually correct.

Examples of orphan drugs that have been considered by the NCPE include: agalsidase alpha, Replagal, and agalsidase beta, Fabrazyme, for Fabry disease; eculizumab, Soliris, for paroxysmal nocturnal dyspnoea and haemolytic uremic syndrome; ivacaftor and Orkambi for cystic fibrosis; elosulfase alpha, Vimizim, for Morquio A syndrome; human alpha 1 proteinase inhibitor, Respreeza, for emphysema in adults with documented alpha 1 proteinase inhibitor deficiency; and more recently migalastat, Galafold, for Fabry disease.

The HSE-CPU will forward the NCPE report and any other relevant information to the HSE drugs group. The final decision on reimbursement of any drug, including orphan drugs, is made by the HSE, not the NCPE.