Dáil Éireann Debate, Tuesday - 6 October 2020
745. Deputy Éamon Ó Cuív asked the Minister for Health when CAR T-cell therapy will be available here; when he expects it will be approved for reimbursement; the arrangements that are currently being worked on in relation to facilities for the therapy; and if he will make a statement on the matter. [28766/20]
View answerThe HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.
In line with the 2013 Health Act and the national framework agreed with industry, a company must submit an application to the HSE to have a new medicine added to the reimbursement list.
Reimbursement is for licensed indications which have been granted market authorisation by the European Medicines Agency or the Health Products Regulatory Authority.
In making a relevant reimbursement decision, the HSE is required under the Act to have regard to a number of criteria including efficacy, the health needs of the public, cost effectiveness and potential or actual budget impact.
HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds, on the advice of the National Centre for Pharmacoeconomics (NCPE).
The HSE advised that on 18 September 2018, it received an application for pricing and reimbursement of tisagenlecleucel (Kymriah®) for both its licensed indications:
- for the treatment of paediatric and young adult patients up to and including 25 years of age with B-cell acute lymphoblastic leukaemia (pALL) that is refractory, in relapse post-transplant or in second or later relapse; and
- for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.
Following the NCPE’s completion of Rapid Reviews for both of these indications, on 22 October 2018, the HSE commissioned full HTAs for both indications.
On 27 August 2019, the NCPE’s HTA report for the pALL indication was received by the HSE The NCPE recommended that tisagenlecleucel not be reimbursed for pALL unless cost-effectiveness could be improved relative to existing treatments
On 20 September 2019, the NCPE HTA report for the DLBCL indication was received by the HSE. The NCPE recommended that Tisagenlecleucel not be reimbursed for DLBCL unless cost-effectiveness could be improved relative to existing treatments. The HSE has confirmed that it engaged in commercial negotiations concerning Tisagenlecleucel with the applicant.
The HSE also advised that it received an application on 6 September 2018 for the pricing and reimbursement of axicabtagene ciloleucel (Yescarta) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy.
Following the NCPE’s completion of a Rapid Review with respect to this indication, on 22 October 2018, the HSE commissioned a full HTA.
On 25 February 2020, The NCPE’s HTA report was received by the HSE. The NCPE recommended that axicabtagene ciloleucel not be reimbursed unless cost-effectiveness can be improved relative to existing treatments. Subsequently, the HSE engaged in commercial negotiations concerning axicabtagene ciloleucel with the applicant.
The Drugs Group is the national committee which the HSE has in place to make recommendations on the pricing and reimbursement of medicines. The membership of the HSE Drugs Group includes public interest members. In October 2020, these three indications are expected to be included on the agenda of the next HSE Drugs Group meeting.
The decision-making authority in the HSE is the HSE Executive Management Team (EMT). These three indications remain under consideration with the HSE, in line with the 2013 Health Act.
CAR-T is a novel treatment for patients with cancer. It is a patient-specific, individualised cell therapy that consists of genetically modifying the patient’s own T lymphocytes. Both CAR-T therapies described above were licensed as medicines by the EU in 2018. Although licensed as medicines by the EMA, there are technical processes required to support the use of CAR-T therapy which are more multifaceted than standard drug administration. These involve a complex supply chain, laboratory accreditation and other specific supports to be in place in addition to the drug reimbursement approval. As a result, CAR-T can only be carried out at designated, accredited centres which have been assessed and accredited from a quality control and a process management perspective. The designated site would not manufacture the CAR-T, which is a separate process that occurs off-site.
The NCCP have designated the National Stem Cell Transplantation (SCT) unit at St James’s Hospital (SJH) and the Haemopoetic Stem Cell Transplant (HSCT) unit at Children’s Health Ireland (CHI) Crumlin as the initial sites to develop/deliver this service for adult and paediatric patients on the island of Ireland.
The companies providing the licensed CAR-T products require hospitals to be technically ready to provide these products through an established CAR-T service. Each of the designated hospitals has completed a gap analysis, considering these technical readiness requirements and the service requirements and have identified areas where investment is required.
