Susan Desmond-Hellman is president for product development at Genentech. A former practicing oncologist, she joined the pioneering biotechnology company as a clinical scientist in 1995.

Tell us about Genentech.
Genentech is the first biotechnology company. We’re 32 years old this year and we use the technical science called biotechnology to make medicines. The mission of the company is to meet unmet medical needs. We go after the really hard problems in medicine—things like cancer, cystic fibrosis, rheumatoid arthritis, systemic lupus—and increasingly focus on diseases that many baby boomers are worried about, such as Alzheimer’s disease.

What does it mean to be the first biotechnology company?
In the mid-’70s, recombinant DNA technology was co-discovered by Herb Boyer, one of the co-founders of Genentech, who was a biochemist at the University of California, and Stanley Cohen, who was at Stanford. This technology was thought to allow a company to be able to take living cells and make them into factories to produce medicines.

Genentech was started in 1976 based on this new technology, and the first product the company worked on and brought to market using this recombinant technology was human growth hormone.

So you use biological products as factories to produce treatments for human ailments?
That’s exactly right. One of the most exciting products that we have now is a medicine called Avastin, which is a cancer medicine. Avastin is anti-VEGF (anti-vascular endothelial growth factor), which basically blocks cancer cells’ ability to form new blood vessels. We use biotechnology to make the antibody that is anti-VEGF. Because that antibody is like what you and I would make, it isn’t rejected by the body. The science of biotechnology enables us to do things in medicine that weren’t possible using chemistry.

Most biotechnology companies don’t actually make money.

What are your biggest obstacles to innovation?
One of the big challenges is our ability to predict the outcomes. If you make a discovery in research and it’s at a very basic level, it is not at all easy to predict how that discovery translates into a medicine for human beings that is safe and effective. The balance in a company like Genentech is to make something that people can trust, to really innovate, and to take enough risks so that we can do something substantially better for patients. One of the most important things in allowing a company like Genentech to innovate is having a really strong, transparent Food and Drug Administration as well as the European equivalent of the FDA. Innovation in a highly regulated environment is a particular challenge for a company like Genentech.

How is the biotech industry different today than it was at its inception?
I think first and foremost there’s a greater confidence in the medicines that we are able to produce using biotechnology. When we first launched recombinant human growth hormone, Nutropin, in the mid-’80s, there was a lot of worry about the use of a recombinant product in children, the population you worry about most.

Genentech did something incredibly innovative at that time: we started a patient registry. We enrolled every child ever treated with Genentech’s growth hormone to make sure that we didn’t have any surprises on safety. That registry’s been in place now for 25 years. We feel very confident that we understand the safety of recombinant human growth hormone for children. Now when I say to a cancer patient or a physician that Genentech’s going to bring out a new product using biotechnology, there’s enormous confidence that biotech’s a reliable, safe approach to making human medicines.

The other thing is that some biotech companies—Genentech, Gilead, Amgen, Biogen Idec, Genzyme, to name a few—are now profitable. In the early days of biotechnology, there was a lot of promise that you could make a business out of biotechnology, and the business model is now established—although it’s important to note that most biotechnology companies don’t actually make money.

Is it fair to say that the industry in its entirety has probably lost money over time?
It’s absolutely fair to say that. The lead times, the uncertainty, the difficulty of making important new medicines remain a huge challenge for the industry.

The biotech industry gets heat from critics who claim that they charge overly high prices for their products. What do you make of this critique?
We challenge ourselves to make important, valuable new medicines. We’re not trying to do something new for small problems. We’re trying to innovate and make a difference for big problems. And the investments necessary and the risk the company takes and the failure rates are such that the business model of biotechnology relies on premium pricing.

But we’re sensitive to the most important thing of all for us, which is patient access. My team and the folks at Genentech who work in research and development don’t just want to make drugs that make a difference. We want patients to have access to them. Genentech works very hard to have a strong support mechanism for patients who can’t pay or for patients who can’t afford their copayments, which is more and more common these days.

I understand that it has been harder for women in countries outside the United States to get access to Herceptin for breast cancer due to regulatory obstacles. Could you discuss this?
It’s a huge issue for cancer patients. HER2-positive metastatic breast cancer is a very special form of breast cancer. We made a remedy, Herceptin, specifically for the 20 percent of breast cancer patients who have this form of breast cancer that is especially difficult to treat.

When a company does trials, it isn’t enough just to do the trial; then one must go to the regulators, not just in the United States but outside the United States, and request approval. An extra layer is that in Europe one has to get approval for the pricing of the product as well. We really find that difficult for patients.

If you make a discovery in research...at a very basic level, it is not at all easy to predict how that discovery translates into a medicine for human beings that is safe and effective.

The FDA approved Herceptin in less than five months. It was really outstanding. Everyone knew patients were waiting. The sense of urgency that we had, that the FDA had, that physicians in practice had, was huge. That’s what patients deserve.

This is the balance that all of us are facing over the next several years as more and more pressure to control healthcare costs comes about. My plea is not to squash innovation. I would submit that in countries like the United Kingdom, where access to Herceptin was delayed because of concerns over the cost of healthcare, there needs to be a healthy public policy debate about patients waiting, their need for new medicines and for innovation, and balancing that with the cost of healthcare.

What are the areas of public policy that concern you at Genentech the most?
Right now one of the most important public policy areas for us is the importance of a strong FDA. As a former practicing physician, I can tell you that the FDA’s high standards and their ability to help the American public understand what’s safe and effective are a huge asset that we have in the United States. Having a strong FDA gives all of us confidence in medicines, and it’s widely known that the FDA has talked about their challenges of recruiting and retaining great employees.

The other thing that’s high on our list is making sure that new public policies don’t interfere with this move toward more personalized medicines. We were so excited about what Herceptin has enabled for patients with HER2-positive breast cancer. There should be more medicines like Herceptin.

What is your take on off-label prescription of drugs, particularly in the oncology sphere?
I feel very strongly that physicians and their patients deserve to have access to what the entirety of medical knowledge can offer them. Part of that is the opportunity to prescribe drugs off-label. That said, it’s a very different situation than when a healthcare provider prescribes on-label. There’s more uncertainty. There’s a need to make sure you’re clear with the patient about the unknown risks when you use a medicine off-label.

You spent two years in Uganda studying AIDS. How has that experience influenced your career?
I went to Uganda as a faculty member at University of California, San Francisco on loan for two years. The experience included patient care, teaching, and medical research. The most important thing I learned from that experience is to feel incredibly fortunate to have been born and raised in the United States and have access to a great education and everything we’re given here.

Everything I’ve done since I got back from Uganda for me is my small way of trying to give back. Having an experience early in your career that allows you to have more insight into how lucky we all are, how fortunate most of us are in the United States, is really a life-changing kind of experience. 

Photograph by William Mercer McLeod.