The Challenge

Tackling rare diseases is a quandary for healthcare providers. The lack of available treatments mean that patients affected by these conditions have some of the highest unmet needs. This makes it vital that they gain early access to the new safe and effective therapies with a minimum of delay.

Yet when new treatments do become available, low patient numbers make that robust evidence of safety and efficacy conduct in randomised clinical trials remains limited. Without an approach that recognises the specificity of rare diseases in evidence collection and patient needs, it becomes difficult for industry to invest in developing treatments and for payers to make informed decisions on reimbursement.

The use of real world data (RWD) is becoming increasingly relevant to provide evidence evaluate the effectiveness of the innovations coming to market. However, doing so will 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

Building on the work undertaken in relation to outcomes based approaches and use of RWE, Fipra convened the multi-stakeholder roundtable ‘TRUST4RD’ (Tool for Reducing Uncertainties in the evidence generation for Specialised Treatments for Rare Diseases).

Fipra chaired and facilitated the stakeholder contribution through a series of roundtable and workshops including payers, HTA bodies, regulators, clinicians and patient groups and industry.

Working alongside leading academics in the field. the process identified the challenges posed by using RWD and identified how to adapt the current approaches to best allow the effective use of RWE to support robust decision-making for market access and reimbursement.

The Outcome

The Coalition ultimately developed the TRUST4RD tool, designed to guide policy makers, HTA authorities, regulators and payers on a collaborative approach to optimise RWE. A paper, outlining the approach has been submitted to Orphanet and final version of the tool was presented at ISPOR, HTAi, and the World Orphan Drug Congress. The TRUST4RD tool, alongside further work on developed providing recommendations for each stakeholder group (RWE4Decisions), was presented at a Finnish EU Presidency conference on health systems for the wellbeing of People in the 2020s. At this meeting policy makers, patients, healthcare professionals and academics gave consideration to how RWE can be used to address uncertainties and conclusions will contribute to the development of national and European policies.