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Biopharm Development & Manufacturing Guidelines For Launching A Successful Biotech Company By James Smith, Ph.D. very young start-up wants to make the next blockbuster product. We analyze the market, identify a huge patient population with an unmet need, and attempt to create a product that will meet that need. It sounds feasible at first glance; after all, what better way to get a return for investors E than to deliver a product with a huge, sustainable customer base that has no other alternatives for treatment? Of course, this is easier said than done. Such a grandiose endeavor comes with a tremendous development cost and often a very high regulatory bar. The drug development process is inefficient, almost seemingly by design, tolerating countless dead-end iterations during the molecule discovery phase. And the larger the market, the larger the clinical trial burden will be, with ever growing numbers to reach statistical significance, particularly if the product only has a modest clinical benefit. While this paradigm may have worked in the past for established pharma companies with deep pockets and vast research and development infrastructures, it is a recipe for disaster for a budding start-up. Time and again, young companies fall into the unfortunate trap of overextending scarce resources to develop a product for a huge market, not realizing the unrealistic burden that they have undertaken, and ultimately running out of funding before they can finish what they started. There are various ways that entrepreneurs can improve their chances of success. The successful start-up will have a well-defined commercialization strategy that is efficient and flexible, balancing commercialization progress with innovation, and leveraging partnerships and collaborations 58 LifeScienceLeader.com with industry experts. With funding most commonly being so tight during product development, an efficient commercialization strategy can prove to be the difference between failure and completing the only goal that really matters: crossing the line of market approval and introducing the product to the public. DESIGN AN EFFICIENT AND FLEXIBLE DEVELOPMENT PLAN As desirable as it sounds, a single, straight path to market simply does not exist. Inevitably, companies will encounter challenges and roadblocks that will delay progress. The setbacks could be seemingly endless: finding the right drug profile during discovery may not happen quickly, lead candidate prospects may have unfavorable safety profiles, or the product may have other undesirable characteristics or not be stable enough to meet expected requirements. However, you can mitigate these risks through careful comprehensive planning in which multiple possible pathways to market — with their own relative risks and benefits — are mapped out and evaluated in advance. This "guided flexibility" approach allows a company to maintain a development strategy that remains efficient in the face of obstacles while capitalizing on new opportunities as they present themselves. NanoSmart Pharmaceuticals, Inc. for example, has employed a highly flexible commercialization strategy as it takes on the challenge of developing a tumorNovember 2013 targeting drug delivery platform. The plan to achieve regulatory approval of the platform is to reformulate an existing FDAapproved drug by enclosing it within the antibody-targeted lipid nanoparticle. NanoSmart considered various APIs with which to commercialize its platform and began formulation efforts. When the first formulation candidate presented unacceptable stability issues that were found to be inherent to the API, the company was able to continue development of the platform by elevating the priority of other APIs that exhibited more favorable biochemistry. Due to the initial design of the project and adequate contingency planning, the unanticipated flaws of the API have not substantially delayed development of the platform. FIND A COST-EFFECTIVE REGULATORY PATH The regulatory path of a drug product has critical relevance in the clinical phase of development, as it directly affects the most costly aspect of commercialization: clinical trial size and length. For example, per capita Phase 3 clinical trial costs exceeded $40,000 in 2011, a staggering 70 percent increase from 2008 (Cutting Edge Information, 2011). Careful clinical trial planning is therefore crucial to the survival of the company. Perhaps the best option to leverage existing regulatory pathways is the orphandrug pathway. Orphan indications — rare diseases that have very small patient popu-