Written answers
Tuesday, 7 November 2017
Department of Health
Rare Diseases Strategy Implementation
Billy Kelleher (Cork North Central, Fianna Fail)
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673. To ask the Minister for Health the action taken to implement recommendations 30 to 39 of the national rare disease plan on access to medicines and technologies, in tabular form [45915/17]
Simon Harris (Wicklow, Fine Gael)
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A National Rare Disease Plan for Ireland was launched by the Minister in July 2014. This is a generic policy framework for rare diseases. The scope of the plan is broad given that there are 8,000 rare diseases approximately affecting millions of EU citizens; and consequently, there can be a dearth of expertise and knowledge about some rare diseases, simply because they are so rare. Earlier this year, the Department published a Progress Report on the Rare Disease Plan.
The Steering Group Recommendations on access to medicines and technologies for people with rare disease in the Republic of Ireland and the actions taken to date are outlined as follows:
| Recommendation | Action |
|---|---|
| 30. The HSE develop a Working Group to bring forward appropriate decision criteria for the reimbursement of orphan medicines and technologies. The approach should include an assessment system similar to that for cancer therapies established under the National Cancer Control Programme and link with the CAVOMP at European level. | The terms of reference have been developed for a Rare diseases technology review committee and this will largely follow the approach in place for technology review of cancer treatments. Michael Barry has been appointed Chair of this committee. |
| 31. The HSE undertake a preliminary economic evaluation of current activity and costs for orphan medicine and technologies for rare disease patients across all hospitals settings. | The Acute Hospital Drugs Management Programme is undertaking a project to understand the patient numbers on enzyme replacement treatment, the cost of this treatment and the place of treatment. |
| 32. Applications for the use of orphan medicines and technologies in hospitals be dealt with in the context of a national budget, rather than through individual hospital budgets, and that the HSE take account of this. | The Acute hospitals drugs management programme is examining how approved enzyme replacement treatment might be funded via a centralised mechanism in a similar way to multiple sclerosis. |
| 33. The HSE develop a publicly available annual report documenting the use of both existing and new-to-market orphan medicines and technologies in Ireland and a summary of applications received and decisions relating to those applications. | Not commenced |
| 34. The existing horizon scanning between pharmaceutical companies and the HSE, including clinical value assessment authorities, continue and be enhanced so as to improve information available regarding orphan medicines in the pipeline and the future needs for these medicines. | There is a formal horizon scanning process in place via the corporate pharmaceutical unit in the HSE. |
| 35. The capacity to prescribe all orphan medicines and technologies for ultrarare conditions be limited to specialist teams designated through the Centres of Expertise. | Enzyme replacement treatment this is in place. |
| 36. The HSE apply a set of guidelines on the prescribing of orphan medicines and technologies in Ireland. The HSE should evaluate clinical outcomes regarding use of orphan medicines. | This is a function of the Rare Diseases Technology Review committee. |
| 37. Clinicians should provide data necessary to the monitoring of prescription patterns and pharmacovigilance, so as to ensure patient safety and high-quality healthcare. | This is already in place for some orphan conditions and clinicians are obliged to report any adverse events for drugs which they prescribe. |
| 38. Early dialogue between the HSE and companies who are running clinical trials in Ireland with Irish patients where license approval is imminent. | Not commenced |
| 39. Sponsors could be offered an incentive to run trials in Ireland increasing access to innovation for Irish patient | Not commenced |
The National Centre for Pharmacoeconomics (NCPE) conducts the economic evaluation of new and existing technologies (e.g. pharmaceuticals, vaccines, diagnostics) to determine their cost effectiveness or value for money.
A new Committee called the “Rare Diseases Medicinal Products/Technology Review Committee” was established in June 2017 by the NCPE. The terms of reference for the Committee have been finalised and the responsibilities of the Committee include the review of proposals for funding of new products for rare diseases or expanded indications for existing products for rare diseases.
The Committee will also contribute to the development of clinical guidelines for relevant Orphan Medicinal Products and support the implementation of same.
Dr. Michael Barry is the Chair of the Committee since 13 September 2017. It is expected that Committee will begin its work in early 2018 after appointment of the necessary support staff.
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