A Guiding Hand
June 2007
Helen Davies, senior vice president of development at NovaQuest, outlines a practical process to optimize the development of emerging biotech therapies.
As with many aspects of biotechnology, the process of shepherding an emerging product from the laboratory to the patient is full of uncertainties. In the case of an emerging biotech, many of these uncertainties can be minimized by properly managing the development process from the starting point of discovery to the end point of marketing. Above all else, biotech companies poised to embark on a development path must remain focused on the product and committed to meeting key milestones along the way.
The majority of biotech products in development meet a medical need, such as new treatments for CNS disorders and cancer for example. When studying biotech compounds early in the development process, researchers may be tempted to look at a broad spectrum of possible indications, and that can be a costly mistake. When companies try to study too many indications at one time, they often increase the complexity of the program to the point where it becomes difficult to properly evaluate the effectiveness of the drug in any of the desired areas.
The focus at this early stage should be on achieving that first milestone—the proof of concept—by choosing endpoints or biomarkers that clearly establish the compound’s mechanism of action. In order to drive development in the early stages of a study, the biotech should have a clear vision of not just the clinical endpoint, but the ultimate intended use as well. The best way to retain this focus throughout the study is to ensure that clinical development plan outlines a simple, sustainable approach.
Biotech CEOs must think about the eventual product label in the very early stages to avoid studying the wrong drug on the wrong patient population. By carefully considering the label from the beginning and assessing similar products already on the market, executives are better able to match the product profile to the current market landscape. This assessment will lead to a better sense of whether the drug presents a realistic opportunity or a bad investment. Bad investments are not limited to development programs only, however. A common biotech mistake is over-investing in corporate infrastructure, such as real estate and non-critical technology. Emerging biotech companies should build the product first, then let the product lead the critical business decisions that will affect the company’s growth. Furthermore, investing in personnel with medical, regulatory and commercial strategy expertise provides key knowledge capital, which is a critical factor at this stage.
In the emerging stage, biotech companies also should discuss their platforms and therapeutic objectives with regulatory agencies early and often. Both the European Medicines Agency and the U.S. Food and Drug Administration are encouraging new standards and tools to promote efficient and accurate drug development. Given declining clinical trial success rates and a precipitous drop in new drug approvals, these agencies are very interested in providing advice and guidance in the early stages. For first-in-human studies in many conditions, regulators are encouraging the first use of therapies in patients, as opposed to healthy volunteers, which provides early efficacy and safety data that can drastically reduce a trial’s timeline. Planning this type of trial well in advance is a great strategy to maximize the Phase I package, because it can reduce the timelines so significantly. We recently conducted a cystic fibrosis study in Europe that we executed in stages—from single-dose safety, to multiple-dose safety and then to a multiple-dose efficacy study. In essence, we completed not only a first-in-human study, but also two proof of concept studies in less than 18 months. That’s an enormous savings of time, resources and, of course, money.
In order to ensure that an emerging biotech company is spending its time and money wisely, the company’s leadership should consider all these issues well before launching a study. Failure to carefully consider each step in the development cycle could derail even the most promising new drug. Furthermore, biotech companies are assessed by potential partners on the quality of the product, the management team and the caliber of the existing financial syndicate. Should the company be deficient in any of these areas, a partner may choose to invest elsewhere.
At NovaQuest, we are very interested in biotechnology and have invested in more than two dozen companies representing approximately 140 potential products over the past two years. Our involvement in any partnership is more than just a funding mechanism; the biotech companies in which we invest consider us a strategic development partner. And when we do invest in a biotech company, we are active investors. We form development committees with the partner company in order to view a drug’s development from both a design and an execution perspective.
The primary value of our partnerships is the expanded resources they yield. Our partner companies may draw on the expertise of Quintiles’ more than 16,000 employees. If one of our partners is looking to run a hepatitis program in Southeast Asia, for example, it’s easy for us to leverage development to support the right delivery, since we have a leading presence in that region. We also put together a dedicated alliance management team, which allows the partnership to continue to develop and serves as a governance structure to maintain an effective and fruitful partnership.
Although a strategic development partner can help optimize the development of an emerging biotech, leaders of these companies must first demonstrate a clear vision of the product’s clinical and financial potential. By taking a simple, sustainable approach to development and focusing on achieving the first major milestone, biotech executives can better position their company’s chances for success and ultimately increase their chances of delivering effective and innovative medicines to patients.
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