A Note from Joel S. Marcus, Co-Founder, Alexandria Summit; Chairman, Chief Executive Officer & Co-Founder, Alexandria Real Estate Equities, Inc./Alexandria Venture Investments; Lynne Zydowsky, PhD, Co-Founder, Alexandria Summit; Chief Science Advisor to the CEO, Alexandria Real Estate Equities, Inc./Alexandria Venture Investments; and Ellen Sigal, PhD, Chair and Founder of Friends of Cancer Research:
Since 2011, Friends of Cancer Research (Friends) and Alexandria Summit have worked together and formed a close strategic partnership to successfully host the Blueprint for Breakthrough series, which examines the foremost scientific regulatory issues for the breakthrough therapy designation. Our partnership continues to be successful due to Alexandria Summit gathering a global life science network in a unique and highly collaborative platform to catalyze transformative discussions and collaborations that will help drive the development of novel, cost-effective therapies that will save lives, manage illness, and reduce the burden of disease on society, while Friends has the ability to convene the right people to come to a consensus solution and truly enact change for patients’ benefits.
Our ongoing partnership plays a critical role in bringing together researchers, sponsors, advocates, and the FDA to develop regulatory roadmaps that drive forward novel treatments for patients who need them most. By leveraging Alexandria Summit’s impactful collective voice and Friends’ role as the foremost patient advocacy group in Washington DC, we can effect change and help drive better outcomes for patients and their families.
This year marks the 5th annual Blueprint for Breakthrough Forum and the use of real-world evidence (RWE) in clinical trials will be the main focus of this year’s event. Prior year’s event topics may be found by visiting http://www.focr.org/friends-alexandria-blueprint-forum.
We had a dialogue with Dr. Maria Koehler from Pfizer and Dr. Jane Perlmutter with the Gemini Group because we wanted to discuss the upcoming Blueprint for Breakthrough forum with a couple of our esteemed panelists who are both experts in their field and provide a unique perspective. The conversation that took place is below.
Maria and Jane, thank you for taking the time to chat with us ahead of next week’s Blueprint for Breakthrough Forum. We are thrilled to have you both participating in this innovative event. First things first. What do you plan on discussing on your panel at the Forum next week?
Dr. Koehler: For me, this workshop is about the FDA and oncology community coming together to discuss the idea of accepting alternative sets of evidence for both scientific and regulatory purposes – evidence that would be gathered from settings in which patients are treated in their local oncologist practice rather than the traditional clinical trial setting, which can be quite cumbersome. Transforming the way we conduct clinical trials to make them more seamless for patients has been a passion of mine since I left clinical practice and joined the pharmaceutical industry. It’s exciting to see that others also recognize the need for change, and I’m looking forward to a productive discussion on the 16th.
Thanks, Maria, we agree that clinical trials need to be more seamless for patients and we are also looking forward to a productive discussion next Thursday. Jane, what do you plan to discuss?
Dr. Perlmutter: I plan to discuss the potential use of real world experience as a way to increase the speed of improving treatments for cancer patients, as well as identifying low probability and long-term side effects that would otherwise be nearly impossible to find. I realize some patients may have concerns about privacy, but I have found most cancer patients welcome the opportunity to have their experiences contribute to the improvement of treatments for future patients.
We have also found that most cancer patients would like the opportunity to have their experience help improve treatments for future patients. Based off what you’ve told us you plan to discuss, how does that correlate with patients being critical to drug and treatment development?
Dr. Koehler: The patient’s perspective, well-being, and voice are vitally important to the development of new medicines. We’re looking beyond the topics of safety and tolerability, although those are and always will be critical. Now it’s about enabling patients to access the most promising new drugs in development without the level of disruption to their daily lives that comes with clinical trial participation. Today, only about three percent of eligible adult cancer patients participate in clinical trials – that’s a very alarming statistic.
Only three percent of eligible adult cancer patients participate in clinical trials? You’re right that is a very alarming figure. Jane, how do you think your panel discussion relates to patients being critical to drug and treatment development?
Dr. Perlmutter: Although research with cell lines have been valuable tools in drug and treatment development, until we see how real people respond, we cannot be confident that new drugs and treatments will be safe and effective. Traditionally, we have relied on carefully controlled randomized trials, but such trials are very expensive and take a great deal of time, and unfortunately only a small proportion of patients actually participate in trials. Learning from every cancer patient’s experience has long been a dream, and that dream can soon become a reality with the potential use of real world evidence.
We think you’re right, Jane that we need to see how real people respond in the real world to be confident that new drugs and treatments are both safe and effective. One last question: why do you think this event and topic are important to discuss?
Dr. Perlmutter: With the increasing use of electronic health records and social media, as well as the advancements in big data methods in other fields there is now great potential to leverage these data for improving treatment for cancer patients. I find there are also some low hanging fruit that we should take advantage of immediately. However, there are also many challenges to fully take advantage of real world evidence, and in this regard, I think it is timely to discuss steps that can be taken to collect data in a way that will be most useful.
We agree that leveraging data to improve patient care is what we see the end goal being. Maria, as a leader of industry what is your perspective?
Dr. Koehler: We’re fortunate to be at a time when we are developing medicines that allow patients to live their lives more fully than ever before, and we need our clinical trials to match. This will require collaboration across industry, academia, and government, which is what makes the Blueprint Forum so important. I think this event has the potential to bring about real, tangible progress. I see it as more than an opportunity to discuss the issue of using real-world evidence to facilitate the regulatory approval of promising new therapies – it’s a critical point in time for us to define and come away with a path forward.
We agree that there needs to be some sort of consensus solution to pave a path forward for use of real world evidence. Thank you both again and we look forward to furthering this conversation with you and all the other panelists next week.
The Blueprint for Breakthrough: Exploring the Utility of Real World Evidence event will take place on Thursday, June 16, from 9 am to 3 pm at the JW Marriott. For more information and to register please go to: http://www.focr.org/events/blueprint-breakthrough-exploring-utility-rea….
Ellen Sigal, PhD, is the Chair and Founder of Friends of Cancer Research.
Joel S. Marcus is a co-founder of Alexandria Summit and Chairman, Chief Executive Officer & Co-Founder, Alexandria Real Estate Equities, Inc./Alexandria Venture Investments.
Lynne Zydowsky, PhD, is a co-founder of Alexandria Summit and Chief Science Advisor to the CEO, Alexandria Real Estate Equities, Inc./Alexandria Venture Investments.
Maria Koehler, MD, PhD, is the Vice President Oncology Strategy, Innovation and Collaborations of Pfizer Oncology.
Jane Perlmutter, PhD, MBA, is with the Gemini Group and a cancer survivor.