Accelerating the path from possibility to proof in the development of new medicines

Michelle Longmire, MD

Dr. Longmire is mission-driven to accelerate the development of new therapies for disease. As a Stanford-trained physician-scientist...

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We live in an era of exponential medicine. The potential to create new therapies that could treat or even cure disease has never been greater. mRNA technology in COVID vaccine development and siRNA technology in the treatment of common diseases such as high cholesterol to rare genetic conditions are indicative of an era where novel promising therapeutics can be synthesized on lab bench tops worldwide. The potential for disease eradication is revolutionary and achieving this should be a top priority for humanity.


So what does the path to eradicating the over 10,000 human diseases look like? Sadly, it appears painfully long. At the current pace, we will not have treatments for the majority of human diseases within any of our lifetimes. With only approximately 50 drugs approved per year by the FDA, if one new therapeutic was approved for each disease it would still require more than  200 years... The reality is that the limitation is not caused by a lack of breakthroughs in bench science but instead bottlenecks in the in-human science, also known as clinical research.


Why is this? How has basic medical science moved so much faster than the clinical application of these breakthroughs? Isn’t the primary purpose of basic medical science to improve human life?


At the heart of the issue is a problem common to industries that emerged before the digital era: The underutilization of digital innovation. The proven path becomes the only possible path, paralyzing innovation. And in domains that involve human health or safety the stakes are higher and the force of the status quo far stronger. But the potential value of the unrealized opportunity is the cost of the status quo and therefore we must force ourselves to answer the question: How can we solve this problem better with the technologies of today?


Let’s look at this. Safety and science are the heart of the clinical research process that seeks to answer the question: is the therapy safe and is it effective (determined by the clinical science)? As we have experienced with the COVID vaccines and therapies, the faster we answer this question, the greater the net benefit. Therefore, I would argue that speed should be the third variable in the clinical research equation. Innovation should strive to maximize the synergy of all three variables. Thus, the critical question becomes, How do we achieve safety, science, and speed in clinical research with the technologies of today?


It starts by leveraging the most meaningful tools: Decentralization, Data, and Digitalization. The convergence of these three technologies will drive new synergies at the intersection of safety, science, and speed and enable a new era of drug development.


  1. Decentralization: Decentralization removes the barrier of geography in clinical research, enabling access to any participant in the world compared to  only those who live in proximity to a limited number of clinics. Greater access improves scientific reliability by broadening the diversity of participants which produces  data that is reflective of the population affected by the condition. Decentralization also improves speed by  accelerating enrollment (which in some cases accounts for 60% of the total time of the trial and 80% of trial failures) and improves patient retention which  thereby reduces the number of additional patients that need to be recruited due to participant dropouts.


  1. Data: Leveraging real-time data and advancements in statistical methods we can push to determine safety and efficacy faster; real world evidence can harness synthetic control arms to improve safety and speed.


  1. Digitalization: Leveraging digital we can refine measures of disease to be more sensitive and specific, which leads to heightened safety and faster research through endpoints that take less time to measure; digital facilitates remote monitoring of  participants, which removes unmonitored areas that were previously blind spots in clinical research; digitization powers advanced analytics that can provide earlier insights into drug safety and efficacy.


At Medable, we believe that by innovating at this intersection of safety, science, and speed with the technologies of today, we can 10X the number of therapies in development, which potentially could increase the number of new therapies available per year from roughly 50 to 500, thereby potentially reducing the timeline for treatments for the majority of human diseases from 200 years to as few as 20 (Please read Key Assumption below*).


Will this be easy? No. Will this be accomplished? YES! It is simply a matter of when.


We know our goal is audacious and some would say impossible. But our journey has shown us that the time for change is now. Patients cannot wait for novel treatments. As an entrepreneur, I have learned that the greatest limitation in any pursuit is one’s imagination of what is possible. We are building a world where the hope for better medicine is measured in days, not decades, and our team will stop at nothing to make our vision reality.


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We are incredibly grateful to our new and existing investors who are helping us make our vision reality, even faster. Thank you to Blackstone and Tiger Global joining us on our mission and to GSR for your continued incredible support. We would not be here without our entire team of backers and believers: Sapphire, PPD, Young Sohn, Mark Armenante, Streamlined Ventures, WTI, HealthX, Launchpad Digital Health, and eXXclaim Capital.


* Key Assumption: The modeling assumes that current and near-future science harbors promising treatments for the majority of diseases. While this is certainly not unequivocally the case, when examining commonalities in disease classes and pathways (the molecular manifestation of genomics), I believe that reducing the barriers to clinical development could drive wider utilization of existing effective science and therapeutic strategies in the development of new medical treatments for untreated and suboptimally treated disease. Additionally, by lowering the cost barriers to clinical development there is more financial incentive to develop therapies for disease categories that have historically been underserved in drug development.