Transcript Presentation - American Society for Experimental NeuroTherapeutics

How Patient Collaborations Can
Drive Innovation
02 Mar 13
Bray Patrick-Lake, BS, MFS
Director of Stakeholder Engagement
Clinical Trials Transformation Initiative
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Disclosure
Duke University
Employee
PFO Research Foundation
President and CEO
Alliance for Headache
Disorders Advocacy
Board member
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Background
• Patients have long been frustrated by the lengthy
times and high costs of developing and
commercializing new therapies with low yields
• IOM recently identified better cooperation and
communication among stakeholders as a priority
to transforming the clinical research enterprise
• New models of patient collaboration are evolving
to drive innovation and transformation
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Traditional Patient Collaborations
• Disease-specific advocacy and education-based
patient organizations
• Perform fundraising for research
• Form partnerships with investigators or create
centers of excellence for research and treatment
• Become involved with industry to promote
clinical trials and investigate opportunities for
development of new therapies
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Evolution of Patient Collaborations Driving Innovation
•
Powered by internet,
social media and mobile
communications
•
Patients are getting
connected and organizing
globally
•
Impatience, limited
financial resources and
poor return on
investment have fueled
cross-sector relationships
and out of the box
thinking
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Model: Systemic Improvement and Transformation
CTTI is a private public partnership co-founded by
FDA & Duke University in 2007
Organization: 60-member diverse stakeholder collaboration
of academia, industry, government, patients, CROs and IRBs
Mission: To identify and promote practices that will increase
the quality and efficiency of clinical trials
Patient Leadership Council: 16-member council of patient
thought leaders forming a collective voice around clinical trial
issues to drive systemic improvement and transformation in
the clinical trial enterprise
www.ctti-clinicaltrials.org
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Patients Accessing Assets from External Sources:
Crowdsourcing
•
Prize4Life (www.prize4life.org) is a PAO whose mission
is to accelerate the discovery of treatments and a cure
for ALS by using powerful incentives to attract new
people and drive innovation
• P4L awarded monetary prizes to encourage
promising biomarker concepts and predict
progression
• Awarded treatment prizes to fill the drug
development pipeline with promising therapeutics
by encouraging researchers to extend the lives of
ALS mouse models by 25%.
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Model: PAO’s forming Partnerships to Innovate
•
•
•
Prize4Life partnered with the Northeast ALS Consortium
(NEALS) and the ALS Therapy Alliance (ATA) to develop the
Pooled Resource Open-Access ALS Clinical Trials (PROACT) database
• largest database of clinical data from ALS patients ever
created
P4L Partnered with InnoCentive for crowdsourcing, open
innovation and prize competition
• www.InnoCentive.com
Working groups and multi-sector collaborations on drug
development, e.g. FasterCures and Health Research
Alliance
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Creating Research Tools for the Public Domain
• Michael J. Fox Foundation for Parkinson’s Disease
(www.michaeljfox.org)
•
Clinical Trials Recruitment Best Practices Manual
•
•
To provide trial investigators with a comprehensive look at lessons
learned and best practices that have resulted in successful recruitment
for PD trials across many sites
Creating tools for bench scientists and putting them in the
public domain
•
Animal and cellular models, proteins, genetic resources, high-quality
antibodies against PD proteins
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Organizing Patients Online
•
PatientsLikeMe (www.patientslikeme.com)
•
Creation of longitudinal registries for common and rare diseases
•
Data-sharing platform harvesting patient-reported information
from online patient communities
•
PLM offers its services to the R&D organizations of their pharma
and device clients
•
•
Providing specialized information that might inform a target
product profile or give insight into the real-world use of their
candidate drug/device
Partnerships with Merck (psoriasis) and US Dept of Veteran’s
Affairs’ Epilepsy Centers of Excellence and UCB SA
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Patient-Funded Drug Development Companies
• DART Therapeutics, LLC (www.dartrx.com)
• Innovative, new-model biotechnology firm focused on developing
therapies for Duchenne Muscular Dystrophy (DMD)
• Co-founded in 2010 by two patient foundations in DMD, Charley's Fund
and the Nash Avery Foundation
•New model for research and drug development centered on the needs
of families affected by DMD and potentially transportable to other
pediatric rare diseases
•Accomplishments in first two years:
• Acquired a promising clinical stage drug candidate and spun out
a special-purpose entity focused on its development.
• Conducted research on a biomarker as potential new tool to
track muscle health that could enable lower costs and faster drug
development timeframes
•Structured partnerships between patient foundations and
investor capital to expedite promising programs in DMD
Paving the Legislative and Regulatory Pathways for New Therapies
•
Friends of Cancer Research (www.focr.org)
•
•
•
•
Cancer research think tank and advocacy organization
Develops partnerships and advocates for policies that will
get treatments and therapies to patients in the safest and
quickest way possible
Patients work with federal health agencies, congressional
leadership, academic research centers and private sector
industry
In follow-up to the 2011 FOCR event, The Advancing
Breakthrough Therapies for Patients Act was introduced
and included as a component of the 2012 re-authorization
of the Prescription Drug User Fee Act (FDASIA) to expedite
development of new, potential “breakthrough” therapies.
American Society for Experimental NeuroTherapeutics | 15th Annual Meeting
Conclusions
• Patients can drive innovation when viewed as partners
in rather than subjects of research
• A common theme of patient-driven stakeholder
collaboration has emerged in innovation embracing
clinical trials networks, open access and information
sharing models, and legislative overhaul
• New and promising models of patient collaborations
should be considered in order to drive innovation such
as crowdsourcing, online organizing, and patient-funded
drug development companies