The balance of healthcare innovation lies in developing new treatments, while also ensuring that everyone has access and nobody is left behind. At Koneksa, our team has been busy pushing the boundaries of digital biomarkers to advance science and clinical trials, but we can’t lose sight of patient access and health equity.
Recently I participated in several World Economic Forum Open Forum events, as part of Partners for Patients’ Accelerating Global Health Equity Summit. PFP.NGO is a non-government organization dedicated to helping the United Nations’ 1948 declaration, “the right to health is a cardinal social and economic right,” come to pass.
I’m grateful to the Summit organizers for the opportunity to participate as a speaker, co-moderator and panelist on topics including sickle cell disease, clinical trial innovation and the representation and equity challenges we must address in the development of new medicines.
Presenting these topics among such a diverse group of international leaders was a reminder that clinical research often fails to appropriately represent the populations healthcare is intended to serve. Reflecting on the incredible promise of recently approved gene therapies for sickle cell disease (SCD) is a reminder that they are very far from being able to scale to the people and places that need them the most.
SCD is caused by a gene mutation most common in individuals whose ancestors are from sub-Saharan Africa. Barriers to access will exist even in the most developed markets where new therapies are being launched, but need to be delivered by specialized facilities and cost several million dollars per patient. Fewer than 200,000 of the nearly 8 million people living with sickle cell worldwide live in the markets where these new therapies are approved, and those living in sub Saharan Africa are about 9 times more likely to die from the disease.
Fortunately, these new therapies foreshadow additional innovations that should provide greater access. Improvements that help improve scale and reduce the cost of new modalities like cell and gene therapy are happening all the time; furthermore what we learn from these treatments may inform other types of drug therapies that can reach more patients in-need. At Koneksa, our teams have been working on a monitoring biomarker to better measure the full impact of sickle cell crises, an incredibly painful and debilitating experience for most patients, that should eventually enable better prediction of these events. Using digital health tools and mobile phone data collection ensures we are also able to scale globally as the footprint of clinical development in this disease expands to more areas of need.
In an essay published in the New England Journal of Medicine in November 2020, Drs. Alexandra Power-Hays and Patrick T. McGann wrote, “Unfortunately, the social construct of race in America requires the majority of patients with [sickle cell disease] not only to face the consequences of a serious health condition but also to navigate a society in which the color of their skin is often an unfair disadvantage.” Capturing the full burden of this disease on patients’ lives is essential to demonstrating the impact that new treatments can have on their well-being.
It’s important for us as an industry to continue innovating and advance new technologies to enable more medical breakthroughs. From gene therapies, to newer treatments for patients with rare diseases or the many common neurodegenerative conditions with no disease modifying treatments, digital biomarkers can enable clinical trials to reach more patients.
At Koneksa, our mission in building “health measurements that matter” is about improving our fundamental understanding of disease to accelerate drug development, so patients can receive novel therapies faster.
I came home from Davos, with a renewed sense of hope and optimism, and I made new connections that will help our team explore and access patient populations around the world. Together, we can build new digital solutions to advance science and medicine for all.