The role of the NCPE, the HTA process and European collaborations
Dr Caroline Walsh, SPHeRE cohort 2016-2020
Health Technology Assessor, National Centre for Pharmacoeconomics (NCPE)
Health Technology Assessment (HTA) involves the assessment of evidence of comparative effectiveness, safety, cost-effectiveness and potential budget impact of health technologies for use by patients. In Ireland, the National Centre for Pharmacoeconomics (NCPE), located at St. James’ hospital, Dublin, are mandated with providing recommendations to the Health Service Executive (HSE) on the clinical and cost-effectiveness of medicines in Ireland. The NCPE, which is an independent HTA agency, aim to facilitate decision-making on the reimbursement of medicines by applying clinical and scientific evidence in a systematic framework, in order to maximise population wellness. The NCPE Review Group are a highly-skilled multidisciplinary team, with expertise in medicine, pharmacy, statistics, epidemiology and health economics. HTA guidelines produced by the Health Information Quality Authority, with input from the NCPE and other stakeholders, outline the principles and methods used in assessing health technologies for use by patients in Ireland(1).
The Corporate Pharmaceutical Unit of the HSE (HSE-CPU) are responsible for pricing and reimbursement of medicines in Ireland. The HSE-CPU commission the NCPE to appraise new medicines, or those that have received approval by the European Medicines Agency (EMA) for new indications, following an application for reimbursement by the manufacturer. All medicines are subject to a preliminary assessment called a Rapid Review, which takes around four weeks from commissioning. Upon completion, the NCPE may or may not recommend the medicine for reimbursement, or the NCPE may recommend that a formal HTA is necessary to assess the cost-effectiveness of the medicine. This may happen when the medicine is very costly, or there is uncertainty over the medicine’s benefit or it is estimated that there is potential for a large budget impact should the medicine be reimbursed. In a formal HTA, the NCPE consider a number of the criteria that must be considered by the HSE in their reimbursement decision of all medicines, as set out in the 2013 Health (Pricing and Supply of Medical Goods) Act; including efficacy, effectiveness and added therapeutic benefit; cost-effectiveness and; budget impact. Ireland currently have two willingness-to-pay (WTP) cost-effectiveness thresholds for medicines; €20,000 per quality-adjusted life year and €45,000 per quality-adjusted life year. This means the medicine is considered cost-effective if it costs €20,000/€45,000 or less to achieve one additional year of a perfect quality of life. These explicit thresholds were agreed upon by the Department of Health and the Irish Pharmaceutical Healthcare Association (IPHA) in 2016. Consideration is also given to the affordability of the medicine through the presentation of estimated gross and net budget impacts. A summary detailing the NCPE Review Group’s appraisal of the formal HTA and the rationale substantiating the recommendation made to the HSE is published here, once the appraisal is complete.
The NCPE also have an academic and research function (affiliated to the Department of Pharmacology & Therapeutics, Trinity College Dublin), which aims to support evidence-based decision making and inform national guidelines on the conduct of economic evaluations. Areas of research include evidence synthesis and methodology, drug utilisation, health outcomes and economic evaluation (further information available here). More recently, the NCPE lead the COVID-19 Evidence Review Group established by the HSE, which aims to provide rapid evidence reviews of potential treatments for COVID-19. The Rapid Evidence Reviews are updated frequently and are available to download here.
In addition, the NCPE are involved in a number of European collaborations, including EuNetHTA and BeNeLuxa. The BeNeLuxa initiative was established in April 2015 by the Netherlands and Belgium. Ireland, represented by the NCPE and the HSE-CPU, joined the initiative in 2018. The BeNeLuxa initiative aims for sustainable access to, and appropriate use of, medicines in the participating countries (Belgium, the Netherlands, Luxembourg, Austria and Ireland). Areas of co-operation include horizon scanning, information sharing and policy exchange, joint HTAs, collaboration on pricing and reimbursement. The first BeNeLuXa assessment involving Ireland has recently been completed for onasemnogene abeparvovec (Zolgensma®), with reimbursement in Ireland not recommended unless cost-effectiveness can be improved relative to existing treatments (summary available here)(2). On the 4th June 2021, BeNeLuxa released a joint statement with the Nordic Pharmaceutical Forum, which set out the intention to examine the following issues together:
- The importance of cost-effectiveness in the assessment of the value of medicines;
- The availability of robust clinical evidence for decision makers in a timely manner.
The full statement can be accessed here. Regarding the second point, the need to facilitate earlier access to medicines from a regulatory perspective has introduced increasing challenges to HTA processes, whereby a lack of data may contribute considerable uncertainty to cost-effectiveness results. Methodological challenges to HTA include; the use of immature data to support clinical effectiveness; a lack of direct comparative evidence (versus relevant comparators); and the use of surrogate endpoints as primary endpoints in clinical trials. HTA methods are constantly evolving in order to address these challenges, for example, through the development of sophisticated evidence synthesis and survival extrapolation techniques. Despite the utilisation of advanced techniques, uncertainty can still remain in the absence of direct and unbiased, long-term comparative data. Both BeNeLuxa and the Nordic Pharmaceutical Forum agree on the need to collaborate to address the challenges facing decision-makers in relation to achieving value from medicines.
- Health Information Quality Authority. Guidelines for the Economic Evaluation of Health Technologies in Ireland. Dublin, Ireland: HIQA; 2020.
- Beneluxa Review Group. Beneluxa Review Group Assessment Summary: Onasemnogene abeparvovec for the treatment of patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene: Beneluxa; 2 April 2021.
|Dr Caroline Walsh is a Health Technology Assessor at the National Centre for Pharmacoeconomics. She is a graduate of the School of Pharmacy, Trinity College Dublin and holds a Masters in Pharmacy degree from the Royal College of Surgeons in Ireland (RCSI). Caroline completed her pre-registration training in the pharmacy department at Tallaght University Hospital (TUH). After qualifying as a pharmacist, she worked for one year as a hospital pharmacist in TUH, mainly on the oncology service and in the aseptic compounding unit. In September 2016, she joined the SPHeRE PhD programme as a RCSI PhD scholar under the supervision of Prof Kathleen Bennett and Dr Caitriona Cahir. Funded by a Health Research Board Research Leader Award, she was awarded a PhD from RCSI in November 2020 for her thesis entitled|
‘The association between medication adherence across multiple medications and health outcomes in ageing populations’.
Caroline is a former co-chair and chair of the student council of the International Society for Pharmacoepidemiology. She was previously president of the RCSI ISPOR student chapter. Research interests include pharmacoeconomic evaluation and the use of real world data in health technology assessment.