Rare Disease Day: Our commitment to patients, science, and society

Every year at end of February, the global health community recognizes rare disease day to help raise awareness of rare complex, debilitating conditions that are often difficult to diagnose and treat. At Sanofi, we have longstanding heritage in developing treatments for rare disease and strive to push the boundaries of science to improve the lives of people around the world afflicted by these diseases.

Our company’s commitment embodies a triple commitment to patients, science, and society.

Patients

Some facts: There are an estimated 7,000 different types of rare diseases, and 65% of rare diseases are associated with multiple disabilities, while 50% of rare diseases affect children. It is estimated that worldwide more than 300 million people—and their families—live with a rare disease.

Given these numbers, the one counter-intuitive I want you to take away from this article it is that “rare is not so rare.” And whilst major progress has been made over the past decades in several regions of the world, the life for someone living with a rare disease can still look like an obstacle course. This can include just getting to a diagnosis. On average, someone experiencing the signs and symptoms of a rare disease might search for 5 years before receiving an accurate diagnosis.

The unique challenges faced by patients living with a rare disease has led to the development of an incredible community of advocates around the world – patients, family members, physicians – who are pushing healthcare systems and the pharmaceutical industry to not be complacent in their policy and R&D efforts. We at Sanofi are proud and humbled to have been a part of the rare disease community for nearly 40 years. And I strongly encourage you to tune in or watch the replay of our company’s LinkedIn Live event featuring inspiring rare disease advocates.

Science

Major progress has been made to better understand biology and the mechanisms behind some rare diseases. But we still have so much to discover: More than 90 percent of the estimated 7,000 different rare diseases have no available treatment option.

The complexity of biology, together with scarce data and limited understanding because of the small numbers of patients living with a given rare disease, creates a massive challenge that we can only overcome by focusing efforts on transformational science. That’s the ambition we have set forth in our R&D strategy.

And in the years to come, there are reasons to be hopeful that the pace of clinical research could accelerate. The progress of genomic medicine or synthetic biology could be game changers in discovering next generation medicines for patients living with rare diseases.

Of course, the progress of scientific research depends not only on technology or the availability of scientific data. Sometimes, it is about a hunch, when a committed team of scientists ask themselves “what if?” We had one of those moments recently, which ultimately led our team to kick off a clinical development program targeting GM2 gangliosidosis, a specific class of rare diseases.

Society

The pandemic has forced us to question some fundamentals of modern society: how we live and work but also how we connect with our ecosystems, our communities, and the planet. COVID-19 by itself will not change the world but our response to it at individual, community and societal levels will.

Last year, unrelated to the pandemic, we undertook a review of our corporate social responsibility strategy, or what I call our new “contract with society.” I learned a lot from the review process as we challenged ourselves about areas of commitment where we could uniquely push the boundaries and make a difference.

The months ahead will allow us to accelerate on the execution of our new strategy, and it is clear that our humanitarian programs in rare diseases will feature in our efforts.

2021 will mark the 30th anniversary of our humanitarian programs, which first started in the United States and have since been expanded to serve more than 3,000 patients in 90+ countries. As part of our contract with society, we have committed to donate 100,000 vials to treat rare disease patients every year free of charge.

Rare Disease Day is the moment in the year when all of us at Sanofi are proud and humbled to reiterate our #Pledge4Rare. I cannot think of a topic that better connects the dots of our ambition to advance science, improve people’s lives and help tackle societal problems.