Implementation aspects of expensive (gene) therapies (working title)

Research area: HTA-methods & steering instruments

Project management part 1: Kathrin Wohlhöfner
Project management part 2: NN
Duration: February to June (part 1), July to November (part 2)
Language: English (with German summary)

Background:
It is predicted that by 2022 there will be about 16 Advanced Therapies Medicinal Products (ATMPs) on the market, about 2-4 new therapies per year. Currently, 11 ATMPs are approved, which includes CAR-T cell therapies. According to the Alliance of Regenerative Medicine (ARM) annual report, more than 1,000 clinical trials worldwide are investigating ATMPs for various diseases at the end of 2019, 94 of which are already in phase 3 trials. Since there is (mostly) little knowledge about actual patient-relevant benefits and long-term effects at the time of approval, there is a need for fair access to potentially effective therapies on the one hand, but also a review of feasible and realized short-, medium-term benefits for patients on the other hand.

Objective:
The content of the project is, on the one hand, to develop an organizational model (work steps, processes, responsibilities, data infrastructure and data ownership) for data monitoring (outcome based managed-entry agreements)  on very expensive (gene-, ATMP-) therapies and, on the other hand, to test it on a case study. 

Method: 
Part 1: systematic synthesis of models for the introduction of therapies under data documentation, synopsis of international experiences.

• Identification of international models
• Partly-structured interviews
• Analysis of work steps, processes, responsibilities, data infrastructure and -ownership, etc.

Part 2: Piloting of the model based on examples

• Either retrospective analysis based on national examples (Spinraza, Zolgensma, CAR-T cell therapies).
• Or: prospective set up of an organizational model for a new therapy.

Timeline:

Part 1: February to June 2021

Part 2: June to November 2021