During the BIO Patient & Health Advocacy Summit last week, a panel led by Julie Gerberding, Executive Vice President and Chief Patient Officer for Merck and Co-chair of BIO’s Patient Advocacy Committee, discussed the many successful collaborations which have led to impactful clinical trial partnerships between industry and patient advocates.
T.J. Sharpe, a melanoma patient blogger of the widely read ‘Patient #1 Blog’, was diagnosed with stage 4 melanoma 5 years ago, when his son was only 4 weeks old. Using his experience as an IT consultant in health care, he searched and discovered a location for an immunotherapy clinical trial. Unfortunately, that trial failed, and the disease progressed. T.J. then found a second clinical trial for a novel immunotherapy called anti-PD-1. After 75 doses over 4.5 years, TJ received a no metastatic disease diagnosis this summer.
Being a person in charge of his own health, T.J. described his own collaboration and how he forged his own partnership. He made his own decisions for treatment and reached out to friends who were patients in journeys before him. He was lucky to have industry friends to point/connect him to clinical trials and potential therapies, and lucky to have good physicians to help him navigate the right treatments for him. He mentioned that many patients often become the world’s greatest experts on their disease, to push back, challenge the system, and ask ‘why not’—to collect as much information and ammunition to continue the fight.
Sharon Tan, Global Project Head for Sanofi Genzyme, and Amy Kant, Reinvention Director for the National Niemann Pick Disease Foundation, came to the discussion from similar vantage points, both having experience with Niemann-Pick diseases. Bringing so much urgency to the ‘right now,’ Sharon and Amy had a unique perspective on how to move through a clinical trial, and how to get through to the FDA, particularly with a rare disease treatment that has taken 10-15 years of research and development. Their sticking point was that somebody has to bring all the parties together in partnership to move forward. Research, industry, clinical, patients and family – each being an important piece to the process.
Zachary Hallinan is Project Manager for Clinical Trials Transformation Initiative (CTTI), which is upwards of 80+ patient groups with a focus on clinical trials. The group looks directly at:
- how they can work better together, and
- attempting to come up with some real world best practices.
Part of the discussion to be had is not just financial, but there are multiple ‘values’ to be found. Looking at different philosophies is also incredibly important. For example: “(blank) might cost (blank) hundreds of dollars, but, if we are successful, you might in the end save like 4000%.” True ‘value’ can be found by bringing all parties to the table and talking through different philosophies.
Hallinan commented on patient registries, and how there is a lot that all of us can bring to help educate sponsors and point out where you can help along the continuum. A big barrier is lack of knowledge, and a gap between what patient groups think they bring to the process and what providers think the patient groups bring to the process. Gerberding added that the FDA has made a commitment to include an expert on the patient experience to review committees, coming at it from a different perspective and shifting to a strong partnership for industry to attract more patients in trials.
During questions, the conversation transitioned over to ClinicalTrials.Gov. Only ~3-4% of patients will even sign up for a clinical trial, so efforts to make this wide-open search tool friendlier are recommended. Suggestions include developing ways to help narrow down and geo-locate clinical trials; make the site multi-lingual and eliminate trials that aren’t active or are out of date / not recruiting; update in real time; and let patients make the best decision for their health.
The panel was a lively and passionate discussion of collaborations which are fundamentally changing industry’s approach to clinical trial design and recruitment of patients in today’s rapidly changing health care landscape.