An Essential Balance: Preserving Our Commitment to Patient Safety

If one thing is true about biopharmaceutical research, it's that the quest to bring safe and effective new medicines to patients is anything but a one-size-fits-all model.
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Coming to work at PhRMA a year and a half ago required a bit of a crash course in the incredibly complex and always evolving biopharmaceutical research sector. In that time, I've come to learn that many industry critics seek to generalize (and sometimes mischaracterize) the development of new medicines, our sector's commitment to safety and the role of investment. And if one thing is true about biopharmaceutical research, it's that the quest to bring safe and effective new medicines to patients is anything but a one-size-fits-all model.

Think about biopharmaceutical R&D for a moment -- the more than $1 billion price tag for the development of a new medicine, the 10 to 15 years the process can take, or the 5,000 to 10,000 compounds that will be screened in order to eventually bring one new medicine to the market.

These numbers are illustrative, but tell only part of the development story. No biopharmaceutical research program is ever the same as the ones that came before. From the baseline science to the testing methods to the clinical trial design, R&D is unique and often unpredictable. No one, not the longest-serving industry veteran nor our harshest critic, can accurately characterize the development of new medicines in one broad stroke.

But within this always moving innovation ecosystem are some important constants -- none more so than the bedrock principle of patient safety. We do not waver when it comes to safety as we continue to innovate, develop new medical technologies and use science to treat patients in novel ways.

So we have a complex, rigorous, scientific development process that is ever-changing and a priority commitment to safety. How do we best serve these two important goals? By striving for modern regulatory systems that move forward in tandem with science and development.

This pursuit is especially relevant this year. With the reauthorization of the Prescription Drug User Fee Act (PDUFA) a must-pass piece of legislation in 2012, we as an industry are evolving to meet the needs of rapidly changing science, as our understanding grows and the tools at our disposal expand. There are many situations in the biopharmaceutical development process that must be addressed with balance, not sweeping general approaches. For example, careful consideration of the benefit-risk balance is essential, especially when evaluating patients with unmet medical needs.

Another point of discussion in the quest for modern regulatory frameworks, Senator Hagan's TREAT Act, would enhance the FDA's accelerated approval procedure for particularly novel development-stage medicines, balancing safety and efficacy with timely patient access to such medicines. We continue to participate in the important dialogue on this proposal.

Throughout these important policy conversations, we never forget our central mission as an industry -- to bring safe, effective new medicines to patients.

Where does investment fit in to pursuing complex scientific R&D, working to enhance regulatory flexibility and focusing on safety? I understand that investment in biopharmaceutical R&D can be a daunting proposition. It's lengthy, it's costly, and it's risky. And in an industry in which venture capital investment has historically played an important role, we certainly do not want to dissuade investment that can help lead to both medical advances and job growth.

As is, that investment already requires a bit of flexibility -- or at least faith. Today, the average hold-time for a publicly traded stock is seven months. And yet the average cost and complexity of clinical trials continue to rise, with the overall time to conduct trials through Phase 3 up to at least six years.

But at the end of the day, America's biopharmaceutical research companies don't just serve investment timelines, we serve patients; that's why we do not back away from our commitment to patient safety. Not after the clinical trials, not after a medicine comes to market, not after the investors have long moved on to other things. Never.

This is not a simple argument. R&D is important. Safety is important. Investment is important. We in the biopharmaceutical sector do our very best to make them all work together.

This post originally appeared on PhRMA's Catalyst Blog.

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