The PSC Forum launches to accelerate PSC research
There has never been a more exciting time for PSC research
We are seeing clinical trials for drugs to treat PSC, some with such promising results that they are moving to Phase 3 trials. Yet people with PSC are still dying, and right now, there is no proven medical treatment for PSC whatsoever. People with PSC urgently need an effective treatment.
However, given the multifaceted nature of PSC, finding an effective treatment or combination of treatments is not easy.
The Last Black Box in Hepatology
PSC is described as the last black box in hepatology, and with good reason. PSC is rare and complex, and is highly variable between patients and unpredictable. There is no single blood test to identify PSC, and no clear way to confidently measure disease progression. There is no simple way to categorically state that a new drug has halted PSC progression and its associated complications.
Just imagine for a second that our researchers DO find a compound that is proven to be effective. Before patients can access a new drug, it must first be approved by regulatory authorities and payers, and that means the clinical trials must be designed right, first time. We know that if we are going to crack this disease in the shortest possible time, we need extend international research collaboration to include all stakeholders throughout the research process.
PSC Forum Launched
In March 2016, I spoke on behalf of patients at the ‘Trial Design and Endpoints for Clinical Trials in Adults and Children with PSC’ meeting sponsored by the U.S. Food and Drug Administration (FDA) and the American Association for the Study of Liver Diseases (AASLD). This meeting underlined the complexities of establishing a treatment and the massive burden of unmet needs of PSC patients.
Based on that work, the PSC Forum, launched today, was created, bringing together researchers, professional societies, patients, pharmaceutical companies and regulatory authorities with a view to accelerating the development of PSC drugs, right through to post-licensing. Our goal is to prioritise the gaps and barriers in current PSC research, address regulatory issues in collaboration with regulatory authorities, and facilitate collaborative research in order to accelerate the delivery of drugs and access to patients.
As a PSC patient I am honoured to have been invited to represent PSC patients around the world on the PSC Forum steering committee. It is tremendously exciting to have the right people sitting at the same table with a common goal: accelerating PSC research to make a safe, effective treatment available to patients in the shortest possible time.
19 June 2017
Read the PSC Forum Press Release from the Forum for Collaborative Research