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). Yet for thousands of patients worldwide, these treatments have been transformative when no other treatment option was previously available.
In the interest of promoting patient access to treatment globally, Genzyme takes a highly responsible approach in establishing the price of our products. 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 provide our products free to patients to the best of our ability.
Ensuring Patient Access
Genzyme believes that developing transforming 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 in need.
Learn more about our Free Drug Programs
Nevertheless, 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.
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.
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.
The cost of therapies also makes it possible for Genzyme to continue research to 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 Niemann Pick disease type B.
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 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. The most common LSD, Gaucher disease, affects only about 10,000 people worldwide; the figures are smaller for the other LSDs. And the number of patients actually on our treatments worldwide is even lower.
Thus 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.
The sustainability of 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
- Investing in continuous improvement in our product manufacturing facilities and processes
- Providing educational materials for patients and physicians
- Sponsoring research conducted by academic labs and patient support programs led by advocacy groups
We offer our perspective on several common questions raised about the cost of our products.
How does 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, development and manufacturing cost of the drug, the size of the patient population, 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, Genzyme doesn't report the profitability of individual products.
Why can’t Genzyme lower the cost of these products?
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. We have also invested more than $200 million in gene therapy research, an approach that holds promise for LSD patients.
Why aren't generics available for these products?
Biologic products such as our ERTs are extremely sensitive to small changes in the manufacturing environment and process. Thus it's impossible to produce a true generic – and very difficult to create an acceptable biosimilar (also called a follow-on biologic). Regulatory bodies like the FDA have complex, rigorous requirements for approval of follow-on biologics.
Genzyme supports the creation of a pathway for the regulation and approval of follow-on biologics, so long as they incorporate processes and requirements to ensure patient safety and treatment effectiveness.
What is the price of Genzyme’s oral therapy for Gaucher disease compared to its ERT for the same disease?
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 development costs, the value these therapies bring, and the rarity of the disease. Both therapies have been shown to slow the progression of Gaucher disease, so they offer similar clinical benefits to patients and to the health care system.