Investigator Initiated Studies (IIS)
PTC is currently accepting IIS Requests aligned with our portfolio and research interests. Please complete the form below to register and apply.
At PTC Therapeutics, we are dedicated to transforming the lives of patients and their families. As part of our commitment, we provide support to independent Investigator Initiated Studies (IIS) that align with our portfolio and research interests. These studies offer us valuable opportunities to deepen our understanding of rare diseases and potentially improve patients’ lives.
PTC Therapeutics is proud to support innovative research that has the potential to make a significant impact on patient care and advance scientific knowledge aligned with our research areas of interest. We invite researchers to collaborate with us in our mission to better understand and treat such rare diseases.
Submission and Review Process
All IIS requests are reviewed by the PTC review committee, which comprises medical and scientific personnel. Proposals are evaluated based on scientific merit, alignment with our research interests, and available funding. A complete IIS proposal, as detailed in the IIS proposal worksheet (please see link below), should be submitted 60 days prior to the program start date. Following initial review, investigators may be asked to submit a full protocol for further evaluation.
Submission of a proposal does not guarantee approval. Financial and/or product support is contingent upon the full execution of a research agreement by both parties. Researchers are invited to submit their concept proposals via our submission portal after completing below registration to our portal.
Investigator Initiated Studies are unsolicited, independent research studies where the investigator independently conceives the research idea, develops the study protocol, and serves as the sponsor.
We are committed to promoting the advancement of medical and scientific knowledge through investigator-initiated studies. By supporting these studies, we aim to expand our understanding of our products and their potential applications. Data obtained from these studies can inform patient care and inspire new ideas for further disease-related research.
- Clinical studies of approved and investigational uses of our drugs or diagnostics assays, medical devices, and solutions (interventional studies phase I to IV).
- Clinical observational studies and real-world evidence (non-interventional studies).
- Health economics evaluations and analytical studies.
- Have the scientific, technical, and operational capabilities to conduct a study, including adequately trained staff.
- Have expert statistical support.
- Submit a scientifically well-designed and well-written study proposal.
- Fulfill all regulatory requirements and deliver reports within agreed timelines.
- Publish the study results in a scientific journal, where applicable.
- Agree to safety reporting to health authorities and to PTC, where applicable.
- A fully executed IIS agreement between the sponsor and PTC.
- Approval from an Ethics Committee/Institutional Review Board (EC/IRB) and/or health authority and any other local approvals that are required.