Research Proposal

Digital Technologies & Cell Therapies


To build a case study by identifying the real-world data gathered by digital technologies used in collecting outcomes data for CAR-T cell therapies and Luxturna approval and reimbursement.

Early Findings

  • "On Aug. 30, 2017, the U.S. Food and Drug Administration (FDA) approved the first chimeric antigen receptor T cell (CAR-T) therapy for Kymriah (tisagenlecleucel). With Kymriah, Novartis established a major milestone in the industry as the drug became the first therapy approved by the FDA that was based on gene transfer."
  • On November 27, 2014, VitalTransformation held an event that featured stakeholders in personalized medicine and digital technology companies ("Where Personalized Medicines and Health Technologies Intersect"). Digital companies within the personalized medicine filed include POW Health, Patient View, Handle My Health, and Patients Know Best. The agenda and personal bios from the sessions are here.
  • In December 2019, McKinsey published an article titled "Driving the next wave of innovation in the CAR T-cell therapies." Software companies noted in the article that have partnered with medical entities include Vineti and TrakCel.
  • Vineti is noted to be the leading cell and gene therapy platform.
  • McKesson and TrakCel have launched an innovative collaboration to support cell and gene therapy, late-stage developers.
  • In the United States, some market challenges regarding reimbursement of CAR T include certain policies set forth by the Centers for Medicare & Medicaid Services. "In the inpatient setting, the current diagnosis-related group payment is insufficient to cover the cost of CAR T care. Even with the outlier and new technology add-on payments (NTAP), hospitals have stated that they are expected to take a net loss on Medicare patients treated with CAR T. In contrast, in the outpatient setting, Medicare Part B would reimburse using the ASP plus model, which would ensure cost recovery for providers."
  • Gene therapies like CAR T have significant costs. "Novartis listed Kymriah at $475,000 per treatment, with experts estimating complications, additional medication, and hospital stays of these durable treatments could bring that cost up to $1 million or more. Yescarta’s price is set at $373,000 by Kite/Gilead, while Spark Therapeutics listed Luxturna’s price at $850,000."
  • "Various reimbursement strategies have been proposed in managed care for gene therapy, including outcomes-based and value-based pricing. Within the context of CAR-T therapy, ICER recommended setting lower launch prices for future drugs and increasing the price or offering rebates or refunds for a high initial price once clinical benefits are known. “Value-based pricing should be viewed in context with the affordability of a new treatment for different health insurers and payers based on the size of the population eligible to receive the therapy,” ICER researchers noted in a final report on CAR-T therapies. Additionally, ICER recommended entering patients of CAR-T therapy into a registry to track long-term outcomes and follow up."
  • One tangential digital technology used in gathering patient data uses social networking sites. "For example, a network developed by the company PatientsLikeMe has 23,000 patients who have signed up to share information regarding five different illnesses: mood disorders, Parkinson’s, multiple sclerosis, HIV/AIDS, and Lou Gehrig’s disease. Particularly for rare illnesses where it is hard to generate the patient numbers required for clinical trial, site organizers say "patients have been a tremendously underutilized resource."

ICER 2018

  • A report on the ICER 2018 noted that "due to the uncertainty surrounding CAR-T payment, we modeled an outcomes-based reimbursement strategy, with payment of CAR-T only for responders within different assessment time points, including one month and one year. We assumed the hospital mark-up was not included as part of the outcomes-based contract between the manufacturer and the payer, and thus that payment from the payer to the hospital for the mark-up occurred regardless of response status. We assumed payment for the mark-up occurred at infusion. Fourth, to account for uncertainties in the long-term effectiveness of CAR-T, we presented the incremental cost-effectiveness ratio for multiple model time horizons, from one year to lifetime. This scenario analysis provides decision-makers with the ability to make judgments around the duration and forecasting of the cure-related benefits observed in the single-arm trials."

NICE 2016

  • The NICE 2016 report noted 3 different levels of maturity in the evidence base for regulatory approval. One set involved 60-80 patients and a median followup of 10 months. A second set was 60-80 patients with a maximum followup of 5 years. The third set involved 120-140 patients with a maximum 5-year followup.
Prepared By
Amy R.
1720 assignments | 5.0