The Cost of Enzyme Replacement Therapy
As a pioneer in the development of treatments for rare disorders, Genzyme recognizes our responsibility to maintain transparency and an open dialogue around the factors affecting the cost of therapy.
Products, Pricing, and Patients

One of the realities of modern health care is that medical treatment can be expensive. This is especially so for rare diseases such as the lysosomal storage disorders (LSDs) treated by Genzyme's enzyme replacement therapies (ERTs). Yet products like these are crucial to thousands of patients worldwide, transforming and even saving lives 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 charge a single price throughout the world for each of our enzyme replacement therapies, and we work closely with national health services and private insurers 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.

Ensuring Patient Access

Approximately 10% of patients on Cerezyme receive their treatment for free, and we have charitable access programs around the world to make all of our ERTs available 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.

Factors Affecting the Cost of Treatment

Genzyme produces five ERTs. While each product treats a different disease, they are all similarly affected by several factors that contribute to their cost – described below.

Product Development Costs

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. For example, Genzyme's investment in Myozyme® has totaled $1.3 billion to date.

Moreover, regulatory agencies often require post-marketing studies, so development costs may continue for years even after product approval. And at Genzyme, we continue to invest significantly in next-generation versions of treatment that could further benefit patients; such efforts are currently underway for both Pompe disease and Gaucher disease.

Product Manufacturing Costs

The manufacturing process is one of the biggest cost differentiators for a biologic product like an enzyme replacement therapy over a traditional pharmaceutical product. For ERT, the process involves manipulating the DNA of sensitive living cells, then carefully harvesting and purifying the enzyme they produce for use in humans. Tiny changes in the manufacturing environment and process can significantly affect the final product. It is a time-, labor-, and resource-intensive process, far more so than creating a chemically based drug.

Manufacturing Enzymes: An Inside Look
From cell cultures to final product, discover the process of manufacturing enzyme replacement therapies through video and animation.

Genzyme manufactures these products at our own sites. We have invested tremendously in state-of-the-art facilities, technologies, and quality-control measures. We continue to devote hundreds of millions of dollars to upgrade our manufacturing plants and build new ones to keep up with patient needs while maintaining product quality.

Disease Rarity and Small Patient Populations

The most significant factor affecting the cost of our enzyme replacement 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 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.

Frequently Asked Questions

We offer our perspective on several common questions raised about the cost of our products.

Is it worth the cost to treat these diseases?

We strongly believe so. The diseases our products treat are severe, progressively debilitating, and in some cases potentially fatal. They have a profound impact on patients and their families. Untreated, these diseases are still quite costly because their symptoms and effects require significant medical intervention. Our ERTs have proved to be effective and safe and have helped many patients lead a relatively normal life. The products have broad reimbursement support by insurers and governments globally precisely because of the clinical value they provide to patients.

A few considerations related to these costs:

  • Most patients receive ERT infusions twice a month, throughout their entire lives
  • Dosage for all the products is based on a patient's weight, meaning that the cost of treatment is less for children, higher for adults
  • Because so few people require these products, the cost to individual health care systems and insurance plans is very small

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 ERTs, 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 and MPS I. These in turn supported the development of treatments for Pompe disease. 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 phase 3 studies of an oral drug for Gaucher disease. This product, which could offer patients greater treatment convenience, has been in development for more than ten years. 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?

Reproducing a traditional small-molecule pharmaceutical drug as a chemically equivalent generic is fairly straightforward. For biologic products such as enzymes, however, production is far more complex. These products 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.

Did Genzyme benefit from publicly funded research when it developed a treatment for Gaucher disease?

In developing our ERTs, Genzyme was able to build upon initial discovery work on a potential treatment approach for Gaucher disease conducted by the U.S. National Institutes of Health (NIH). Genzyme partnered with the NIH very early on to produce the enzyme for research and clinical studies. Any company could have pursued this opportunity but no others came forward.

Genzyme also took tremendous financial risk to develop this Gaucher treatment – not only with the clinical development, but also with a complicated manufacturing process. We made a $200 million investment to build a manufacturing facility even before Cerezyme was approved, and with little evidence that a sustainable business model could be established. We literally bet the company to take this step for patients, recognizing it would be years (10 or more in some cases) before any insurer or government might reimburse us for the treatment. And we bore considerable clinical development costs for many years after approval from various post-marketing studies required by regulatory agencies.

Has Genzyme encouraged physicians to prescribe higher doses of Cerezyme?

No. We have been very clear in our position that physicians make decisions about patient care, including decisions about dosing. Among Gaucher physicians, it is now widely accepted that because Gaucher disease is highly variable from patient to patient, Cerezyme doses should be tailored to each patient based on disease severity. Genzyme strongly supports this individualized approach, which is based on a substantial body of clinical evidence developed over the past 20 years.

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