Dáil Éireann Debate, Thursday - 10 February 2022
390. Deputy Colm Burke asked the Minister for Health the actions that are being taken to ensure that patients and carers are involved in the health technology appraisal process of new treatments for rare diseases; and if he will make a statement on the matter. [7126/22]
Amharc ar fhreagraHealth technology assessment (HTA) is a multidisciplinary process that summarises information about the medical, social, economic, and ethical issues related to the use of a health technology. Examples of health technologies include medicinal products, medical equipment, diagnostic and treatment methods, rehabilitation, and prevention methods. Its aim is to inform the formulation of safe and effective health policies, investment and reimbursement decisions that are patient focused and seek to achieve best value.
The National Centre for Pharmacoeconomics (NCPE) and the Health Information and Quality Authority (HIQA) are the two agencies primarily responsible for the undertaking of HTA in Ireland. The NCPE assesses medicines for reimbursement. HIQA conducts HTAs to inform national health policy and national health service decisions at the request of the Minister for Health and the HSE.
The National Centre for Pharmacoeconomics and HTA for rare diseases
The National Centre for Pharmacoeconomics (NCPE) are committed to transparency in their work and clear communication to all stakeholders. They have provided a submission process for Patient Organisations since 2016 and undertook a major review of the process in 2018. This review included a public consultation with Patient Organisations.
The Patient Organisation Submission of Evidence Template is a document that enables patient organisations to provide patient and carer input to the assessment of a particular medicine. The purpose of the submission is to identify important aspects of the medicine that are:
- not identified or well presented in the published literature, or
- not well captured in quality of life measures or other outcome measures that have been used in clinical trials and other research studies, or
- not well known and/or understood by experts in HTA and decision makers
The submission is also an opportunity to identify the priorities and preferences of patients and what the added value of a particular medicine may be to them. This information is then used as part of the HTA of the medicine.
Since the current Patient Organisation Submission Process was launched in 2018, the proportion of HTAs for orphan drugs where patient organisation submissions were received has increased every year. The NCPE currently have seven ongoing HTAs for drug treatments for rare diseases/orphan indications. A patient organisation submission has been submitted or is planned for all seven of these HTAs.
To support this process, the NCPE deliver a 10-week long patient education module annually, in conjunction with the Irish Platform for Patients' Organisations, Science & Industry (IPPOSI), entitled “Understanding the Principles and Practices of Health Technology Assessment”. This aims to equip patients and their representatives with the background information and knowledge to actively participate in HTA decision making processes in Ireland.
The NCPE also provide ongoing support to patients and organisations throughout the submission process, particularly focusing on smaller or less experienced organisations with fewer resources available to facilitate participation in the process.
The NCPE have also created a dedicated section on their website for patients, which includes information on the submission process, the drug reimbursement process and how to interpret NCPE recommendations. Updates on the NCPE website in respect of all steps in the HTA process are provided in real time from the date the rapid review is commissioned.
In 2019, the NCPE also engaged the National Adult Literacy Agency (NALA) to co-design Plain English summaries of full HTA reports, which detail the recommendation and next steps in the reimbursement process.
HIQA
HIQA undertakes health technology assessments of other healthcare interventions and healthcare programmes, which may include interventions or procedures to screen for, diagnose or treat rare diseases. HIQA constitutes an expert advisory group (EAG) with a multidisciplinary membership, including relevant patient and public representation, to provide expert input to each health technology assessment. EAG members participate in regular meetings throughout the process, and review the draft HTA report and HIQA’s associated advice.
The Rare Diseases Technology Review Committee
In 2018, the Rare Diseases Technology Review Committee (RDTRC) was introduced by HSE Leadership (as recommended by the National Rare Disease Plan for Ireland) with responsibility for:
- Reviewing proposals received from industry or expert groups in Ireland for funding of new products for rare diseases, or expanded indications for existing products for rare diseases and making recommendations as to the implementation of the relevant recommendations from the National Rare Diseases Plan.
- Providing contributions to the development of clinical guidelines for relevant Orphan Medicinal Products (OMPs) and supporting the implementation of guidelines in conjunction with the National Drugs Management Programme Office where applicable.
The RDTRC facilitates the input of patient and clinician perspectives and assist the Drugs Group in its reimbursement recommendations to the HSE EMT. The RDTRC complements the existing process in the case of orphan medicines, rather than replacing any of its stages.
