CEO Fiona Smythe is no amateur in the health-tech space. She and her husband co-founded mscripts, a patient communication platform for pharmacies, that sold to Cardinal Health in 2019. Following that, Fiona started her new digital health startup – MedaSystems; an expanded access software for health care professionals helping patients with serious and life-threatening conditions.
With this new venture, Fiona and her team are focusing on the growing opportunities to help patients get access to investigational therapies.
A self-described water-girl, Fiona finds her own therapeutic benefits from rowing and open water swimming in the San Francisco Bay. Very cool.
M.R. Rangaswami: You’re building software for a nascent market space. What are the trends in pharma that are driving the need for this? Why now?
Fiona Smythe: This is an exciting moment in the biopharma industry. The development of new treatments is exploding in several therapeutic areas driven by discoveries in gene therapy, immunotherapy, and technologies like CRISPR. We hear about experimental therapeutics in areas that were previously very difficult to treat—like oncology and rare diseases—on an almost daily basis.
Meanwhile, there’s a developing understanding that some patients require access to investigational therapies before the FDA approves them. Unfortunately, there’s a gap between the population seeking access to clinical trials and those who are actually eligible. For example, less than 5% of oncology patients are enrolled in clinical trials, although an estimated 70% of potential participants are interested in doing so. Additionally, the Covid pandemic has made it clear that we need more efficient pathways to therapies, and that we need to streamline the administrative burden of physicians.
This is where Expanded Access comes in. If you are a patient who has exhausted other options, your physician can request a promising experimental therapy directly from a pharmaceutical manufacturer. However, the process is inefficient and burdensome to the patient, the physician and to pharma. Recent regulatory guidance established requirements that US pharmaceutical companies provide patients with information about their EA policies. The internet and advocacy groups now allow patients to be minutely informed about new developments in drug therapies, adding to the pressure that physicians and pharma feel to help them get access. Finally, the FDA has been very public in its support for Expanded Access. Applications for Expanded Access have grown significantly in response. For example, Novartis received over 10,000 requests last year.
M.R.: The pharmaceutical industry operates internationally, across multiple regulatory jurisdictions. What are some of the challenges posed by working across borders? How can you help?
Fiona: Every country has its own regulatory process for dealing with investigational therapies. On top of that, processing a request from another country poses challenges in terms of language, time zones, shipping and labeling. If you’re trying to track an urgent request with a spreadsheet and email, you can imagine the challenges and opportunities for failure.
We realized that the core elements of Expanded Access overlap, and they cross international boundaries. The physician must get an agreement to treat from the pharmaceutical company, seek ethical and regulatory approval, and finally have the product delivered to the treatment center. The process requires speed and coordination among multiple parties and often across countries, and this type of process lends itself well to automation.
We built the platform as a networked hub that manufacturers and physicians can subscribe to and use to communicate. This allows us to pool industry knowledge. At the moment, there are many disparate entities solving the same problem individually – our goal is to solve the problem once for all of them. By designing our platform from a global perspective, we’re able to integrate features such as messaging, document upload, workflow routing, data capture and shipment tracking in a way that feels seamless to Expanded Access program administrators. Combining these previously disparate elements lets us offer vastly improved visibility compared to the status quo.
M.R.: While this is a for-profit enterprise, you’re also aiming to improve patients’ lives. What’s different about building a mission-driven enterprise?
Fiona: As you may know, another name for Expanded Access is Compassionate Use. There’s a humanitarian motivation at its core, with widespread agreement in the industry around putting the patient’s need for treatment first. Several of our team have a personal impetus for helping seriously ill patients find treatment options, whether it’s a partner, sibling or spouse who is coping with illness. That serves as a source of personal motivation and alignment for us. I’ve also found that potential partners and employees value this sense of mission – we all want to find meaning in our work.
Part of the reason for the FDA’s support of the Expanded Access pathway is that clinical trials aren’t always suited for conditions such as cancer or rare diseases, where unique disease pathologies affect a small number of patients. These patients tend to have a complex treatment history, making it difficult to include them in clinical studies. While not a substitute for clinical trials, the potential to gather real-world data through Expanded Access offers a new paradigm for the way we learn about novel medicines. It can help companies learn about secondary indications for existing products, or shine a light on a promising avenue for a new clinical trial. The opportunity to help contribute to the clinical development of promising therapeutics is tremendously exciting.
As a whole, our team has decades of experience in healthcare and other highly regulated industries, so we understand the particular complexities of this mission, from designing products to protecting patient privacy to ensuring that the organizations we work with can comply with regulatory requirements. Our desire to help further the research and clinical development of new therapies for patients with few remaining options is a powerful motivation for working in this area.
M.R. Rangaswami is the Co-Founder of Sandhill.com