1 2 aim

What we do

Care

Implementing the updated standards of care that are informed by best practices by experts in the field.

Research

Sharing research interests, exploring research opportunities, co-funding, sharing knowledge, avoiding duplication.

Advocacy

Encourage and inform patients and patient organizations to use their voice to inform policy in their country.

Regulatory

Enhance the relationship with regulatory and HTA & extend influence on policy makers.

Education

Engage and educate patients and patient organizations about diagnosis, standards of care, research, drug development and access to therapies.

Companies

Advocating to lessen the burden in clinical trials, expand inclusion criteria, thoughtful consideration of invasive procedures.

Clinical Trials

Including the patient voice in clinical trials by expanding inclusion criteria, lessen burden of participation, innovative designs, minimal exposure to placebo and fair pricing policies

Data

Invest in data & knowledge capturing, promoting open science, data interoperability and security in accordance with the FAIR principles.

Outcome measures

Support the need for sensitive outcomes measures, validated biomarkers, and harmonization of regulatory decisions and working with all stakeholders in delivering access to therapies.