The Critical Path Institute (C-Path) is pleased to announce a new peer-reviewed publication, titled “Transforming Drug Development for Neurological Disorders: Proceedings from a Multi-Disease Area Workshop,” is now available. NeurotherapeuticsJournal of the American Society for Experimental Neurotherapeutics.
A distinguished group of C-Path scientists and patient-advocates, led by C-Path Executive Director Diane Stephenson, PhD, Executive Director of Critical Paths for Parkinson’s Consortium (CPP), presented their learnings from C-Path’s 2022 Neuroscience Program annual workshop. The publication can be accessed in its entirety here.
Neurological disorders have long posed a formidable challenge to the development of effective therapeutic solutions. These disorders not only contribute significantly to global mortality and disability rates but also present distinct challenges in securing drug approval. Developing novel therapies for neurological indications is challenging, but there is renewed hope as significant progress is underway, transforming the drug development paradigm for both rare and common neurodegenerative diseases.
In October 2022, C-Path and the US Food and Drug Administration (FDA) joined forces to host the Neuroscience Annual Workshop. The gathering brought together various stakeholders from the drug development industry, academia, the patient community, and government and regulatory agencies. Its purpose: to chart a transformative course for the development of tools and therapies to address critical gaps and unmet needs in neurology research and drug development.
This annual workshop focused on five chronic progressive diseases that have long-term effective treatments: Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, Duchenne muscular dystrophy and hereditary ataxias. Over three impactful days, participants engaged in deep discussions, exchanged critical insights, and developed recommendations with the potential to spur the development of innovative therapies and essential tools to address these complex disorders.
Key highlights of the workshop include:
- Collaborative synergy: The workshop emphasized the power of collaboration, as representatives from various sectors pooled their expertise and resources to create a united front against neurological disorders.
- Collective Insights: Participants exchanged invaluable knowledge and experience, fostering a deeper understanding of the complexities and nuances surrounding this critical condition.
- Recommendations for future actions: The workshop provided a set of pioneering recommendations aimed at advancing the development of novel therapies and essential drug development tools to address specific chronic progressive diseases.
“The 2022 Neuroscience Annual Workshop demonstrated how interdisciplinary collaboration can deliver significant advances in the fight against neurological disorders,” said C-Path Chief Science Officer Klaus Romero, MD, MS, FCP. “The combination of diverse perspectives, shared insights, and visionary recommendations is poised to drive innovative solutions and, ultimately, reduce the burden of this condition on individuals and society.”
Excerpts from patient-advocates participating in the 2022 workshop:
In the past, I have experienced first-hand where patient-centered drug development (PFDD) is a trendy buzzword and only included when convenient. Back then it was often tokenism to include the patient’s voice. These C-Path workshops, together with regulatory agencies and industry sponsors, have shown that a real transformation is unfolding before us. Participants understood that those of us suffering from neurodegenerative diseases and valued our work as partners.
Kevin Coke, Pharm.D., Parkinson’s patient-advocate
“I was impressed with how the FDA and C-Path are working with industry and drug development experts to examine patient-centered drug development gaps and unmet needs in neurology and to incorporate the voices of people with a disease into a clinical. – to develop trial design and drug development processes; and integrated -; Effective solutions that benefit everyone.” – Joe Montminy, Alzheimer’s patient-advocate
“It’s not enough to just have a seat at the table. The voices of patients, care partners and those at risk of neurodegenerative diseases should have considerable weight in decision-making that affects the design, implementation and outcomes of research. We can tell when our involvement is tokenistic and empty, but That was not the case at this workshop. It is clear to me that the message from the regulatory body is that input from patients and their families is mandatory.“- Jesse Kevany, Parkinson’s lawyer
C-Path’s 2023 Neuroscience Annual Meeting set for Thursday, November 30, in Washington, DC is an important gathering that members of the CPP and CPAD consortia will not want to miss. Interested participants can know more by emailing [email protected].