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On Oct. 20, media organization and Fool content partner Xconomy organized a forum on oncology research and therapeutic development termed "Boston's War on Cancer." The event was hosted by Millennium: The Takeda Oncology Company. Millennium CEO Dr. Deborah Dunsire provided the opening on the state of the industry.
I had the opportunity to chat with Dr. Dunsire about some aspects and trends in cancer therapeutic research and development. A transcript of our chat is presented below.
Ralph Casale: This forum highlights that there are and can be multiple approaches taken toward developing pharmaceuticals designed to treat the many forms of cancer. We have seen presentations on developmental drugs with varied mechanisms of action targeting different cellular pathways and activities. Is there a need for more collaborative oncology research, earlier in the development stages, wherein synergistic effects of therapeutics with different strategic approaches are explored?
Dr. Deborah Dunsire: Absolutely! With targeted therapies there will generally be a need to combine two to three of them to overcome the "escape" mechanisms that cancer cells usually have. Very few cancers have only one driver pathway activated. Most cancers have multiple mutations and multiple drivers, or they have significant redundancy -- down-regulation of one active driver pathway results in up-regulation of another pathway. The cell uses this redundancy to escape the otherwise lethal intervention. Combining therapies or modalities is a way to countermand this.
Casale: The FDA has in many cases been understandably willing to consider cancer therapeutics under an accelerated approval process. Takeda/Millennium's VELCADE (bortezomib) was approved under such a process. There have, however, been recent setbacks in the required follow-up studies for some therapeutics approved under the accelerated, such as recently with Roche/Genentech's Avastin (bevacizumab) in breast cancer. Do you think the accelerated approval process may be in jeopardy, and how important is it for oncology therapeutic research?
Dunsire: The accelerated approval process is a critical way to get novel and active therapies to patients facing life-threatening illnesses. Many times these patients have run out of proven therapeutic options throughout the course of treatment and otherwise would not have access to these therapies. I believe the accelerated approval process is vital to patients with cancer and other life-threatening diseases. I don't believe it is in jeopardy per se. I do believe it is becoming more rigorous and demanding in the type of surrogate markers that will be acceptable to grant an accelerated approval upon. The more therapeutic options a specific disease has available, the higher the bar that is set for approval.
Casale: Increasingly it seems that approval by the governing agencies, including the FDA and EMA, is no longer the last barrier to a successful drug launch. Guiding agencies such as the U.K.'s National Institute for Health and Clinical Excellence (NICE) are recommending on issues like reimbursement. A recent example is the NICE recommendation against the use of GlaxoSmithKline's (NYSE: GSK ) Tyverb (lapatinib) to treat breast cancer based on cost-to-benefit analysis. The U.S. has also recently established a Council for Comparative Effectiveness Research. Is oncology research changing in response to these agencies?
Dunsire: Oncology research and development have been changing over the last decade -- mostly driven by the need to find therapies with a higher benefit-to-risk ratio than traditional chemotherapeutics had. The understanding of disease biology drove the possibility for more targeted therapies that have, in a few cases, revolutionized care for certain cancer patient populations. The challenge is that targeted therapies may only provide modest or moderate improvements over broader groups of patients (e.g., EGFR inhibitors for lung cancer patients who are not EGFR positive). Managing this more limited benefit can raise challenges for payers, and so we continue to seek out better biomarkers to select the right drug for the right patient to magnify the benefit-risk ratio. Where we can do that, it is easier for payers to see the value.
Casale: Is the funding environment for biotechnology also seeing an effect?
Dunsire: Certainly the decline in predictability of FDA approval in the face of positive trial data has increased investors' perception of the risk of biotech/pharma investments, and that does influence where they will invest their resources. We have seen approximately 25% of the biotech universe of companies disappear in the last 18 months, in part because of lack of funds to continue business operations.
Casale: Oncology research has led the movement toward personalized medicine with companion diagnostics, in some cases defining the most appropriate mode of therapeutic treatment. Do you believe that personal genomics, as well as the more widespread advent of electronic health records may revolutionize the pharmaceutical industry?
Dunsire: Unquestionably this revolution is underway! It may take years rather than months to accomplish our goal, but we will see patients receive care based on their unique genetic profile in the coming decade.
Casale: Biotechnology investors look back fondly at the great success stories of Amgen (Nasdaq: AMGN ) and Genentech, the latter now fully part of Roche. In 2008, Millennium Pharmaceuticals chose to accept an offer of acquisition from Takeda Pharmaceutical. Do you believe over the next decade that investors should continue to expect the acquisition model of successful biotechnology companies by their larger, "big pharma" brethren to continue to dominate?
Dunsire: As with any trend -- it will go on until the next wave replaces it. Nothing in life stays the same forever. Perhaps the next revolution will come from the success of the personalization of care. New technologies -- ones that we can only imagine today -- may eclipse the need for large Phase III trials and alter current paradigms of pharmaceutical marketing. Remember -- the dinosaurs once ruled the planet ... and IBM thought no one would need a small computer!
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