One of the realities of modern health care is that medical treatment can be expensive. This is especially so for rare diseases such as lysosomal storage disorders (LSDs). The prices of our treatments are determined by the clinical value they provide to patients and the rarity of the diseases they treat. Because the LSDs our therapies treat are ultra-rare, the cost per patient to develop, manufacture and support a therapy for these diseases is much different than for medications that can be used to treat hundreds of thousands or millions of patients. Yet for thousands of patients worldwide, these treatments have been transformative.
In the interest of promoting patient access to treatment globally, we work closely with national health services and private insurers around the world to ensure that these products are accessible to covered patients. In developing countries, we help physicians and local authorities build sustainable health care systems that can pay for treatment – and where such systems do not yet exist, we offer a humanitarian program to provide, to the best of our ability, our enzyme replacement therapies (ERTs) free to patients with a demonstrated need. .
Ensuring Patient Access
We believe that developing transformative therapies carries with it the responsibility to increase access to these treatments for patients, regardless of their location, financial situation, or other circumstances. We have charitable access programs around the world to provide all of our enzyme replacement therapies for free to patients with lysosomal storage disorders and a demonstrated need in circumstances where access to treatment is limited.
Learn more about our Free Drug Programs
We recognize the concerns about the cost of these products, and we're committed to openly sharing our approach to pricing to help patients, physicians, insurers, governments, the press, and others understand the many complex issues.
Sanofi Genzyme produces four enzyme replacement therapies (ERTs) and one oral treatment for LSDs. While the products treat different diseases, they are all similarly affected by several factors that contribute to their cost – described below.
The most significant factor affecting the cost of our LSD therapies is the rarity of the diseases they treat. The U.S. Orphan Drug Act defines a rare disease as one affecting fewer than 200,000 people in the United States. For the most common LSD, Gaucher disease, there are only approximately 10,000 people diagnosed with this disease worldwide; the figures are even smaller for the other LSDs. The number of patients actually on our treatments worldwide is even lower.
Because of the rarity of these diseases, Sanofi Genzyme's development and product manufacturing costs are supported by a patient population that is a small fraction of those for most drugs. But we are committed to continuing to produce these treatments for even the relatively small groups of patients who need them.
Developing any medication is an expensive endeavor – costing from hundreds of millions to well over a billion dollars. The expenses include early research and development as well as years of clinical trials. Moreover, regulatory agencies often require post-marketing studies, so development costs may continue for years even after product approval.
Additionally, Sanofi Genzyme continues to research and develop new and improved treatments. Such efforts are currently underway to develop next-generation treatments for Pompe disease and Fabry disease, as well as therapies for rare diseases with no approved treatment options such as Acid Sphingomyelinase Deficiency (ASMD).
LSDs are complex, chronic diseases characterized by worsening progression if not treated. For many patients, our products can alter the course of their disease and alleviate symptoms. Our products deliver significant value to patients and the larger health care system by reducing the need for critical care services, costly procedures, and hospitalizations, and enabling patients to have greater independence and continue to work and care for a family.
Our therapies are personalized medicines, each developed to treat only one genetically determined disorder. As a result, our products offer a high degree of efficacy to those patients with a confirmed diagnosis who are most likely to benefit from treatment.
The sustainability of Sanofi Genzyme’s rare disease business model is dependent on generating sufficient revenue to support the following ongoing commitments:
- Developing new and better treatments to meet unmet medical needs
- Maintaining physician-led registries that capture information about the patient experience and provide data and insights that support ongoing research
- Continuing our patient advocacy initiatives which make sure patient needs are understood and addressed when we make important decisions related to our therapies
- Helping patients access medical benefits and reimbursement
- Providing educational materials for patients and physicians
- Sponsoring research conducted by academic labs and patient support programs led by advocacy groups
- Investing in continuous improvement in our product manufacturing facilities and processes
We offer our perspective on several common questions raised about the cost of our products.
How does Sanofi Genzyme set prices for products?
In setting the price of a drug, we consider all of the factors that can affect costs, including the value it delivers to patients and health care systems, the size of the patient population, development and manufacturing cost of the drug, and the essential need for Genzyme to be a viable business able to bring more drugs to patients who need them in the years ahead.
What is the profit margin on these products?
Like most companies, Sanofi Genzyme doesn't report the profitability of individual products.
Why can’t Sanofi Genzyme lower the cost of these products?
Sanofi Genzyme has established prices for our therapies, based on the factors described above, which allow us to operate as a sustainable business. Our business was built on providing a treatment for Gaucher disease, which then allowed us to invest in developing similar treatments for Fabry disease, MPS I, and Pompe disease. These in turn supported the development of an oral therapy for Gaucher disease, providing an important new treatment option for patients. These self-sustaining business practices allow us to continue making and distributing our life-saving treatments to patients around the world.
We're also committed to reinvesting in research on new treatments. We're currently conducting clinical studies of an oral drug for Fabry disease as well as an enzyme replacement therapy for Acid Sphingomyelinase Deficiency (ASMD). We also have invested more than $200 million in gene therapy research, an approach that holds promise for LSD patients.
What is the price of Sanofi Genzyme’s oral therapy for Gaucher disease compared to its ERT for the same disease?
Sanofi Genzyme’s goal is to price these treatments on par with one another to ensure that all patients are treated with the best medicine for their individual needs without considerations based on price. The price of our Gaucher therapies is based on the value these therapies bring and the rarity of the disease. Both therapies have been shown to slow the progression of Gaucher disease, offering similar clinical benefits to patients and to the health care system.