Bill Sibold, Executive Vice President and Head of Sanofi Genzyme, highlights the achievements of the company’s Rare Humanitarian Program over the last 30 years, bringing treatments to patients in developing countries with lysomal storage disorders who would otherwise not have access. Sibold outlines the persistent obstacles facing people with rare diseases and the work that still needs to be done on infrastructure and framework conditions for better patient access.
This year marks the 30th anniversary of Sanofi Genzyme’s Rare Humanitarian Program – an initiative that began under the stewardship of Henri Termeer, a visionary who recognized not only the need to discover and develop groundbreaking treatments for patients who had been left behind but also to help ensure access to treatment and care for those patients regardless of their location or ability to pay. From the very beginning, providing humanitarian support to the rare disease community has been a vital element of our mission to improve patients’ lives and our commitment to helping achieve equitable access across the globe. It’s in our DNA.
A thriving rare ecosystem is an indicator of healthy, sustainable healthcare
As we celebrate 30 years of the program and what it has accomplished, it’s also the time to reflect on lessons learned and how we in the healthcare industry can have the greatest impact moving forward – particularly in the face of the long-term effects of the COVID-19 pandemic. Because ultimately, we believe that a thriving rare ecosystem is an indicator of healthy, sustainable healthcare – we’re all in this together.
30 Years of the Rare Humanitarian Program
Sanofi Genzyme’s Rare Humanitarian Program is the first, largest and longest-running humanitarian initiative of its kind for people with lysosomal storage disorders – a group of rare genetic diseases that create enzyme deficiencies responsible for a wide range of symptoms. Established in 1991 when our first treatment for Gaucher disease was approved by the FDA, it has since expanded to support five different lysosomal storage disorder communities across six continents.
The program has evolved over the past 30 years while retaining our core commitment – to provide access to free treatments for patients that meet the program’s criteria, who otherwise would not be able to attain them. Since its inception, the initiative has brought free therapy to more than 3,300 people in over 100 countries. Today we support around 1,000 patients at no cost to them in parts of the world where patients would not otherwise have access to treatment.
We also know that the barriers to attaining quality healthcare for people with these diseases are complex, that they vary at the local level, and encompass more than the ability to afford therapy. That’s why the Rare Humanitarian Program is a key component of Sanofi Genzyme’s broader support for patient diagnosis, treatment monitoring, patient advocacy and physician education.
Persistent Obstacles Facing People with Rare Diseases
There are many factors that contribute to disproportionate barriers facing people with lysosomal storage disorders around the world. These include low, or in some cases virtually non-existent, disease awareness. Because these conditions are rare, without specialized education and training, many HCPs may not recognize the first symptoms, extending the time to proper diagnosis for patients and families in need. Access to treatment, inability to pay or travel to receive care are also common and significant barriers preventing people with rare diseases in underserved communities from attaining the treatment and care they need.
Tackling these challenges at the local level requires a deep understanding of the unique needs of each community we serve. That’s why we partner with organizations like Project HOPE, the China Charity Federation and many others who can offer invaluable insights into local factors that prevent treatment from reaching these areas, and the challenges patients and families are facing within the distinct socio-economic, cultural, public health and political constructs of their communities.
Reaching Patients in Need
The past year and a half underscored the importance of partnership and persistence in successfully confronting these obstacles, particularly in already hard-to-reach areas.
For example, in Bolivia we’ve worked closely with a local medical team and Global Health Partners since the program began operating there to ensure the medication we provide arrives safely, compliantly and gets directly to patients. Changes in the Bolivian government in 2019, coupled with the effects of the COVID-19 pandemic in 2020 exacerbated challenges in reaching this community, but through partnership and nine months of organization and planning, treatment was successfully delivered to Cochabamba, Bolivia in July of last year.
More Work to Do
As we mark the 30th anniversary of the Rare Humanitarian Program and celebrate the patients, families, healthcare providers and partners who we’ve had the privilege to come to know through this initiative, we also know we can and must do more.
Because while much has been accomplished over the past 30 years, today, 95% of known rare diseases still lack an approved treatment – and even when there is an approved therapy, access to these medicines still varies considerably across different countries and regions. Many persistent barriers to treatment and care were exacerbated by the COVID-19 pandemic, and some of these effects will be long-lasting.
While much has been accomplished over the past 30 years, today, 95% of known rare diseases still lack an approved treatment – and even when there is an approved therapy, access to these medicines still varies considerably across different countries and regions
But, as I think about the future, I know that there’s much to be optimistic about. Over the past 30 years, we’ve seen that when prioritized, and with all rare ecosystem stakeholders working together, it’s possible for developing countries to support stronger infrastructure and healthcare systems, to raise awareness, expand diagnostic testing, and increase patient support to provide sustainable options for rare disease patients.
We are dedicated to finding innovative ways to make a difference and will continue to build on our mission and three-decade tradition of working to improve access and healthcare infrastructure for rare disease communities around the world. By elevating our ambitions and commitment, I know we can do more for the global rare disease community. I hope you’ll join us.