The Challenge

Highly innovative technologies, such as immunotherapies and cell & gene therapies, often come to market early via expedited regulatory approvals to benefit patients. With a limited clinical evidence base, making decisions about the value of these products is difficult for HTA bodies and payers. Moreover, these highly innovative – potentially transformative – technologies often come with a high price.
These growing challenges have led HTA bodies and payers to become more receptive to the use of real-world evidence (RWE) to complement clinical trial evidence and evaluate the effectiveness of the innovations coming to market. However, doing so requires agreement at the earliest stages on the methodology of generating RWD and the key questions which need to be addressed by RWE to enable patient access.

The FIPRA Approach

FIPRA has advised, over a number of years, on the set up of payer/ HTA-led multi-stakeholder dialogues. Guided by a multi-stakeholder Steering Group and working alongside leading academics in the field, FIPRA facilitates the stakeholder contribution through roundtables and webinars, which aim to identify how to adapt the current approaches to enable the use of RWE  for value assessment and reimbursement of highly innovative technologies. Over time, a RWE Learning Network based on trust and collaboration was created, and includes HTA bodies and payers, regulators, public health authorities, clinicians, patient representatives, registry-holders, data analysts, industry and academics from Europe and beyond (US, Canada).

The Outcome

Two practical guidance papers were developed through this multi-stakeholder collaboration: 
1. TRUST4RD – Tool for Reducing Uncertainties in the evidence generation for Specialized  
Treatments for Rare Diseases – authored by Professor Lieven Annemans (University of Ghent), is designed to guide policy makers, HTA authorities, regulators and payers on a collaborative approach to optimise RWE. The paper was published in Orphanet and presented at various conferences including ISPOR. HTAi, and the World Orphan Drug Congress.  
2. RWE4Decisions Actions for Stakeholders – ‘Real World Evidence to support HTA/payer decisions about highly innovative technologies. Actions for Stakeholders’ – lead author: Dr Karen Facey. The paper was published in the International Journal of Technology Assessment in Health Care and presented at a Finnish EU Presidency conference on health systems for the wellbeing of People in the 2020s.

Over the years, RWE4Decisions has built a multi-stakeholder Learning Network on RWE, that provides a pragmatic and agile platform for Payers and HTA bodies to collaborate with regulators and other stakeholders to share case studies and learnings on the use of RWE – from Europe and beyond (US, Canada…). The aim is to address the evidence uncertainties specific to highly innovative technologies at the time of market launch, to inform decision-making and enable a learning healthcare system to improve patient outcomes.