The Long and Winding Road of Biologic Drugs

by Bryan A. Liang

A recent American Cancer Society review has found that cancer deaths among American men dropped nearly 20 percent between 1990 and 2005. The death rate among American women also fell dramatically.

These victories in the fight against cancer can be attributed to several factors, including key new medical treatments developed over the last few decades.

Such advances represent years of painstaking research supported by billions of investment dollars. This year, Congress has the opportunity to ensure that medical researchers continue to develop breakthrough treatments for diseases like cancer by protecting the intellectual property rights of biotechnology researchers while simultaneously promoting patient safety.

The path from the research lab to the patient is long and tortuous. For example, consider the work of cancer researcher Dr. Napoleone Ferrara. In 1989, while working at an upstart biotech company, Dr. Ferrara identified a biological mechanism integral to the growth of certain malignant tumors that had potential for a new, highly effective cancer treatment.

Translating these findings into viable medicine was challenging, but seven years later– 1996 — a treatment to inhibit tumor growth was developed.

The treatment was a special kind of medicine known as a “biologic.”

Like all biologics, this therapy was created using an elaborate procedure involving genetically engineered living cells. The final product was an antibody that specifically targeted tumor growth — a cancer-fighting smart bomb, of sorts.

However, before the biologic could be administered to patients, the Food and Drug Administration required the drug to undergo three separate clinical trials over the course of eight years. This requirement was key to ensure the safety of the drug in human patients.

In 2004, fifteen years after Dr. Ferrara’s initial discovery, the biologic drug Avastin was approved for medical use. Avastin has since been approved for the treatment of certain kinds of lung and breast cancer, as well.  And it’s currently undergoing some 450 clinical trials for other uses.

Avastin is a success story in biologics research, not only because of the scientific breakthroughs and research required, but also because it is one of the few drugs in human trials that make it to treatment with an appropriate safety profile.

It’s also an expensive proposition. It takes hundreds of millions to billions of dollars and sometimes decades to develop a successful biologic therapy.

In order to ensure that investment into research for drugs like Avastin, policymakers must support and protect biologics research while also ensuring patient safety.

Lawmakers can act this year to simultaneously expand the availability of today’s biologics, which are often expensive, and guarantee that the next generation of patients will have access to even more advanced biologic therapies that have yet to be invented. They can also act to create a system of safe copies of biologic drugs, known as “follow-on” or “biosimilars,” to ensure that the maximum number of patients get the benefits of biologics.

Current debate surrounds protecting data confidentiality surrounding development of new biologics to allow those investing in their development to recapture their investments and reward them for their assumption of risk for a potential billion dollar failure. However, as well, an approval process for “follow on” versions of these drugs is also part of this debate.

Policymakers must take into account the significant time and deep investments in new biological research and drug development to ensure that future generations obtain the benefits from these cutting edge drugs. Political pandering cannot be the basis of decisions that will impact this and future generations of patients who are relying upon these advancements; instead, rigorous economic analysis must be used to ensure continued investment in new biologics research.

Further, increasing access to biologics to allow the benefits of these amazing drugs to be disseminated to as many patients as possible is critical. But just as critical is a system of review that ensures that safety of follow on biologic forms is established. Adverse drug experiences in Europe have led to a requirement of clinical trials to ensure safety of these drugs, and should be adopted here.

The truth is that we need all the research we can get so that as many patients as possible obtain the benefits of new biologics and follow-on forms. Let’s hope our leaders in Washington recognize this and move quickly to safeguard biologics research while ensuring patient safety.

(Bryan A. Liang is Executive Director, Institute of Health Law Studies, California Western School of Law; and Co-Director, San Diego Center for Patient Safety, University of California, San Diego School of Medicine.)

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