PSR’s Mindset
Creating a true partnership with dedication to rare disease patients and our clients to make a positive impact on the lives of people with rare diseases is what drives PSR and its team. Both PSRs input on delivering new treatments for rare disease patients and our participation in fundraising activities exemplify our dedication to the rare disease field on a business as well as a societal level.
PSR is very privileged to work with many biotech and pharma pioneers over the last 16 years in assisting them with getting new therapies available for rare disease patients. We aim to accelerate the clinical development of orphan drugs and facilitate access to treatment for rare disease patients and their families.
We are proud to be experts in the regulatory and clinical development of orphan drugs, working closely with patients, companies, patient groups, regulators, key opinion leaders, government funders and other stakeholders.
WE CARE ABOUT RARE!
PSR’s Mindset
Creating a true partnership with dedication to rare disease patients and our clients to make a positive impact on the lives of people with rare diseases is what drives PSR and its team. Both PSRs input on delivering new treatments for rare disease patients and our participation in fundraising activities exemplify our dedication to the rare disease field on a business as well as a societal level.
PSR is very privileged to work with many biotech and pharma pioneers over the last 16 years in assisting them with getting new therapies available for rare disease patients. We aim to accelerate the clinical development of orphan drugs and facilitate access to treatment for rare disease patients and their families.
We are proud to be experts in the regulatory and clinical development of orphan drugs, working closely with patients, companies, patient groups, regulators, key opinion leaders, government funders and other stakeholders.
WE CARE ABOUT RARE!
Meet the Team
Science and Societal
Despite general acknowledgement of the benefits brought by innovative therapies for rare diseases, patients suffering from rare diseases continue facing difficulties in receiving a correct diagnosis and in getting timely access to adequate treatment.
This situation undermines the right to have equal benefit from the healthcare system between patients suffering from more common diseases versus patients suffering from rare diseases. This may lead to a negative discrimination among citizens which should be prevented on the basis of the principles of equity and solidarity (the core values of our society).
In this respect a joint effort with all stakeholders involved is necessary, taking into account, amongst others, the following aspects:
- Engage in a constructive dialogue with all relevant stakeholders to define a shared and consistent approach to the needs of the rare diseases’ sector
- Raising awareness around rare diseases, facilitating early diagnosis (including newborn screening) and limit the use of inappropriate treatments for patients.
- Further optimize the use of patient registries to fill the gap of data collection and strengthen available scientific knowledge and understanding (including natural history studies).
- Address ‘long-term’ policy environment (with continuous dialogue and political commitment) in order to achieve recognition of the needs of patients suffering from rare diseases & supporting the R&D infrastructure.
- Jointly develop innovative reimbursement strategies & price models to ensure ‘long-term’ access to effective new treatments for rare diseases.
- Achieve predictable and timely procedures by regulators to facilitate access to treatment for patients.
- Enhance patient representation in policy, regulatory and industry processes.