SIMPATHIC
for the one living with rare disease
Incorporate patients’ perspectives and preferences at every step of drug development
What sets the SIMPATHIC-project apart is the emphasis on the patient’s perspective from the outset and a global collaborative approach on 9 different rare diseases*. The project aims to create a blueprint with standardized guidelines, based on patients’ perspectives, enabling efficient future references and research.
In this journey, patients are the primary focus and pivotal partner, steering our endeavours towards meeting their unmet medical needs and achieving outcomes that hold significance for them.
More specifically, we will develop a training program for patient associations, include their perspectives and preferences during the clinical trial design, and develop a patient-centred ethical framework.
Ready to join the project?
Do you want to contribute to the SIMPATHIC blueprint?
We will involve patients and patients’ representatives through:
– the beta-test of our training,
– the development of the PROMs (Patient-Reported Outcome Measures) to be used in the clinical trials,
– the definition of the ethical framework.
If you want to share your experience as patient or caregiver, please do not hesitate to get in touch with us.
We are currently preparing a basket trial to test a selected drug in molecularly confirmed Leigh syndrome (due to mt-ATP6 mutations) and Myotonic Dystrophy, using Goal Attainment Scaling and personalized outcomes measures. If you are a clinician with patients with Leigh syndrome or Myotonic Dystrophy, we are interested to hear from you.
