Dr. Lesley Tilson:

I thank the committee for the invitation to attend the meeting. I am joined by my colleague, Dr. Emer Fogarty, who is a senior health technology assessor at the NCPE. I will briefly outline the role of the NCPE and the centre's involvement in European initiatives and I will discuss the implications of the proposed European regulation promoting co-operation in the area of health technology assessment.

The NCPE conducts rigorous, evidence-based health technology assessments to inform decision-makers on the reimbursement of pharmaceuticals and vaccines in Ireland. The NCPE assessment considers the clinical effectiveness and health-related quality-of-life benefits and all relevant costs, including potential savings from reduced health care resource use like hospitalisation, which a new treatment may provide and whether the price requested by the manufacturer is justified. A budget impact analysis is also required in addition to the status of HTA in other jurisdictions. The NCPE process facilitates submissions by patient groups who wish to have their views taken into consideration. The NCPE will then advise the HSE on the clinical effectiveness, cost effectiveness and budget impact associated with specific pharmaceutical products. The final decision on reimbursement of any drug is made by the HSE following consideration of all relevant evidence and recommendations in line with the Health (Pricing and Supply of Medical Goods) Act 2013.

The NCPE process is well-established and standardised criteria are used for the evaluation of pharmaceutical products. All assessments are conducted in accordance with published general HTA guidelines produced by HIQA and internal NCPE assessment guidelines. The pharmacoeconomic process is outlined in the 2016 framework agreement on the supply and pricing of medicines.

The NCPE is involved in several European initiatives. The NCPE is committed to, and heavily involved in, European co-operation by participating in various collaborations, including the European Medicines Agency joint scientific advice process, numerous Innovative Medicines Initiative-funded initiatives and the EUnetHTA collaboration in health technology assessment. The NCPE is a full partner to the current and final EUnetHTA joint action and is involved in the four main work packages. These involve co-operation in the following areas: early scientific advice to manufacturers and post-market evidence generation; production of joint relative effectiveness assessments; guidelines on development and quality assurance; and implementation. The NCPE provides meaningful contributions to many EUnetHTA outputs, such as the prioritisation of work tasks for Joint Action 3 across work packages and companion guides for the conduct of HTA. The centre is currently co-authoring the topic identification, selection and prioritisation horizon scanning programme for current and future models of co-operation. Furthermore, the NCPE has provided expert advice to the Department of Health regarding collaborative pricing and reimbursement initiatives at European level, including the Valletta and Beneluxa initiatives. Overall, the NCPE has invested significant resources in co-operation on European projects in recent years and the centre welcomes the publication of the Commission proposal on the regulation of HTA.

On 31 January 2018, the European Commission put forward a proposal to develop a regulation around HTA. Specifically, the proposal requires mandatory co-operation of member states in the area of joint clinical assessment. The Commission has described the intention and scope of the proposed regulation. The purpose is to address impediments to market access between member states, avoid duplication of work for HTA bodies and overcome the challenges of voluntary HTA co-operation by creating permanent infrastructure and funding to support joint activity. Specifically, the proposal focuses on joint work on clinical aspects of HTA while the assessment of more context-specific economic issues and decision-making on pricing and reimbursement will remain at member state level. Member states will continue to draw conclusions on the overall added value of the assessed health technology based on the joint clinical assessment report and the assessment of non-clinical aspects of the technology.

The NCPE welcomes the proposal and consider that there is potential for efficiency gains for our organisation and potentially the broader health service, if implemented in a timely manner. However, the NCPE considers that there are several important issues that require detailed clarification before these potential efficiency gains could be realised. These include whether it will be mandatory for pharmaceutical companies to submit evidence as part of this process. As much of the relevant clinical evidence is held by the companies, their participation is considered to be critical. Another factor is the co-ordination of the publication of the joint clinical assessments and the product launch date in Ireland. Product launches and reimbursement applications are often made in Ireland some time after receipt of EU marketing authorisation. Efficiency gains may be lost if the product launch is later than the date of marketing authorisation and reassessment is required due to the availability of new clinical evidence. Furthermore, efficiency gains may not be realised if the primary language of the new body is not English.

The NCPE understands that these clarifications may not be available until after the proposed regulation comes into force and are produced as part of the secondary legislation process. Thus, at this point it is not possible for the NCPE to confidently assume that the process will yield significant efficiency gains in the assessment of clinical effectiveness of new drugs.

The committee has specifically requested information on how the proposed regulation may affect the approval of orphan drug products in Ireland and the likely impact on drug pricing in member states. The draft regulation does not distinguish orphan drugs or propose any specific processes for orphan drugs. In its current format, the draft is unlikely to affect the approval process for orphan drug products in any way. Drug pricing and reimbursement is outside the scope of the proposed regulation, which contains no reference to formal co-operation in this area. The NCPE considers that the proposed regulation should not prevent or impede international co-operation on pricing and reimbursement. I thank the committee for the invitation to speak today.