The Challenge

While innovation in healthcare continues, these innovations cannot benefit patients until they reach them. The science is driving an increasingly targeted approach to the development of new medicines. However, in order to reward innovation and ensure the sustainability of the healthcare system, authorities need to evaluate the value to the patient, healthcare systems and to society.

The FIPRA Approach

In order to be able to effectively demonstrate the value of outcomes, all stakeholders need to work together. FIPRA has convened influential high-level stakeholder discussions, including national payers and HTA authorities, the EMA, clinicians and patient representatives. Working closely with leading academics, this work has contributed to national and European consideration of policies to enable sustainable access to innovation.

The Outcome

TRUST4RD  Tool for Reducing Uncertainties in the evidence generation for Specialised Treatments for Rare Diseases

Building on the work undertaken in relation to outcomes based approaches and use of RWE, the multi-stakeholder coalition 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.

Use of real-world evidence in assessing innovative medicines

The stakeholder endorsed Conclusions were presented during a rare disease conference that was part of Malta’s EU Presidency agenda and have been used by leading national authorities in the development of their policies on the topic. The conclusions were also published by the British Medical Journal.

Outcomes-based approaches to pricing and reimbursement

The roundtable conclusions were presented at a European Commission stakeholder meeting on Pricing and Reimbursement and were referenced in the Opinion of the Expert Panel on Effective Ways of Investing in Health on Innovative payment models for high-cost innovative medicines.

Improving access to personalised medicine

In 2013, when the potential of -omics based approaches was being considered by policy makers and regulators, FIPRA convened stakeholder input into a report which identified ‘Ten Actions to Stimulate Patient Access to Personalised Medicine’. These were presented at Luxembourg’s EU Presidency conference. This contributed towards EU Council Conclusions on personalised medicines for patients in 2015.

European Alliance for Personalised Medicine: FIPRA also conceived and initiated this multi-stakeholder coalition.

Resources

TRUST4RD: Tool for Reducing Uncertainties in the evidence generation for Specialised Treatments for Rare Diseases
TRUST4RD: Tool for Reducing Uncertainties in the evidence generation for Specialised Treatments for Rare Diseases
Case Study
Building on the work undertaken in relation to outcomes based approaches and use of RWE, Fipra convened the multi-stakeholder roundtable ‘TRUST4RD’.
The use of real world data throughout an innovative medicine’s lifecycle
The use of real world data throughout an innovative medicine’s lifecycle
PDF, 183kb
Outcomes based pricing and reimbursement of innovative medicines with budgetary limitations
Outcomes based pricing and reimbursement of innovative medicines with budgetary limitations
PDF, 193kb
Discussion document for the multi-stakeholders meeting on pharmaceuticals ( Meeting DG GROW 12th September 2017)
Ten Actions to Stimulate Patient Access to Personalised Medicine in Europe
Ten Actions to Stimulate Patient Access to Personalised Medicine in Europe
PDF, 159kb