Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy

Figures & data

Table 1. Reimbursement routes.

Routine Commissioning: Assessed through NICE Technology Appraisal (TA) programme, will analyse cost-effectiveness, affordability, and the level of uncertainty from clinical trial data. If NICE issues a favourable recommendation for adoption, the NHS is mandated to fund the therapy within three months [8]. This TA can be issued with or without a Patient Access Scheme (PAS). PASs are independently reviewed by the Patient Access Scheme Liaison Unit (PASLU) [11], but subsequently implemented by NHS England (NHSE). This review includes an estimate of their administrative burden on the NHS. PASs come in two forms; Simple (discount) or complex (financial and/or element of value-based reimbursement). A complex PAS is likely to require additional data collection frameworks.

The Cancer Drugs Fund (CDF): Administered by NICE. Funding is subject to data collection, for re-assessment at a set time point. This option has existing data collection frameworks in place.

NHSE and NICE Highly Specialised Technologies Evaluation (HSTE): Managed by NHSE and typically permitted when other routes are deemed unsuitable, and patient numbers are very low. They do not necessarily have existing data collection frameworks available for routine adoption and may require significant infrastructure.

Table 2. Segmentation criteria.

Joint Accreditation Committee-ISCT & EBMT (JACIE) accredited and had stem cell transplant capability, or Actively treating paediatric ALL, or A centre of excellence for specialist oncology treatment

Figure 1. Treatment arms and monitoring frequency.

Table 3. Direct and indirect costs.

Direct Costs relate to and are sensitive to ‘per patient activity’. In most cases, these are variable costs that increase proportionally to the number of patients within the system.

Indirect Costs are not patient number sensitive but nevertheless are required by an MEA. These often relate to set up costs and infrastructure that would be required for the system to run. Whilst most cases are fixed costs, there are some variable costs contributing to this.

Table 4. Assumptions made using PAG and participant advice.

• Time horizon of the analysis was 10 years • Number of new patients treated per year was 50 for each treatment arm • The assumed 10-year survival rates (applied linearly) for rB-ALL were: ° CAR T-cell therapy = 75% ° Standard of Care = 21% [5] • The monitoring frequency for the three treatment arms is shown in Figure 1, where the CAR T-cell arm has a higher frequency of monitoring during the first year due to the higher uncertainty around the clinical efficacy and safety data provided at launch

Table 5. Quality assurance tests.

• Validation against original entry • Identification of potential duplicated tasks and tasks with missing details • Calculation of a minimum, maximum and variance value • Variance analysis to check for erroneous entries

Figure 2. The four stages of the treatment pathway timeline.

Table 6. 10-year total estimated administrative burden (£) of CAR-T cell therapy, with and without an MEA, and SoC (rB-ALL) split by treatment stage.

rB-ALL Therapy	Setup	Intervention	Monitoring	Exit	Total Cost (£)
CAR T-cell (without an MEA)	£46	£23,355	£221,916 Year 1: £150,024 Years 2–10: £71,892	£0	£245,317
CAR T-cell (with an MEA)	£3,269	£122,215	£974,717 Year 1: £572,904 Years 2–10: £401,813	£16,823	£1,117,024
SoC	£675	£236,362	£209,434 Year 1: £69,178 Years 2–10: £140,256	£882	£447,353

Table 7. Estimated total direct and indirect activity costs over 10 years.

	Direct Activity Costs	Indirect Activity Costs	Total costs (direct and indirect activities)
SoC	£445,905	£1,448	£447,353
CAR T-cell without an MEA	£234,391	£10,926	£245,317
CAR T-cell with an MEA	£1,095,180	£21,844	£1,117,024

Figure 3. The estimated 10-year total administrative cost and total personnel working days.

Table 8. Estimated incremental burden to an English NHS Trust associated with implementing an MEA for CAR T-cell therapy for 50 new patients per annum.

	Additional Cost/Patient		Additional Working Days/Patient
Comparator	Over 1 year	Over 10 years	Over 1 year	Over 10 years
SoC	£181	£1,814	1.1	11
CAR T-cell therapy without an MEA	£240	£2,403	1.5	15

National institute for Health and Care Excellence (NICE). Technology appraisal guidance. 2018 [cited 2018 Jul 27]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance

 Google Scholar

National institute for Health and Care Excellence (NICE). Patient access schemes liaison unit. 2018 [cited 2018 Jul 27]. Available from: https://www.nice.org.uk/about/what-we-do/patient-access-schemes-liaison-unit

 Google Scholar

Hettle, R., et al. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal. Health Technol Assess. 2017;21(7):1–8.

 PubMed Web of Science ®Google Scholar