info@rareimpact.euEvidence generation for ATMPs: Building consensus among stakeholders to accelerate patient access.
Project scope
Advanced therapy medicinal products (ATMPs) are innovative, potentially curative therapies for patients with high unmet medical needs. However, the evidence to support the value of ATMPs is often incomplete. Clinical trials involve only a small number of patients, and long-term outcomes demonstrating that a single treatment with an ATMP is transformative or curative are not established at the time of decision-making. As a result, these evidence gaps translate to uncertainty for decision-makers, resulting in inefficient patient access.
The goal of this workstream was to engage in a dialogue with stakeholders to better understand these challenges and to discuss the potential solutions to improving the evidence base for ATMPs, particularly through the collection of complementary, real-world data. Recommendations were proposed to address these issues, in order to support efficient decision-making and to facilitate timely patient access to ATMPs.
Through the collective exploration of the existing initiatives on registries in the area of ATMPs and the engagement of stakeholders in the field, the workstream proposed a description of the current situation and touched up issues helping to put in place a common vision on efficient patient access to ATMPs. Moreover, the workstream proposed recommendations and action points helping to redefine the value of ATMPs and with the role of stakeholders as co-designers of the future process.
Stakeholder engagement
This workstream involved engagements with stakeholders such as include industry, regulators, HTA bodies, payers, clinicians, and patients.