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Biopharm Development & Manufacturing lations with unmet medical needs — offer substantial financial incentives such as tax benefits and additional patent protection. But more importantly, the orphan pathway allows companies to tap into rapidly growing support networks that increase a company's chance of success. Because products developed for orphan indications benefit a patient population that has no other treatment alternatives, the FDA has been historically flexible in evaluating efficacy, allowing data collected from smaller and/or shorter trials and allowing expedited review. In fact, a 2011 study performed by NORD (National Organization for Rare Disorders) found that 66 percent of approved noncancer orphan drugs between 1983 and 2010 received FDA approval with flexible evaluation criteria. This is an attractive incentive that could reduce a product's time-to-market. With few exceptions, the FDA has reviewed and approved orphan drugs much more quickly than non-orphan drugs (Figure 1). The orphan-drug pathway can be strategically used to accelerate a company's time-to-market while decreasing the overall commercialization cost both before and after FDA approval. Revenues can then be used to pursue further clinical trials to expand the drug's indications for use with additional orphan or non-orphan indications. Similarly, NanoSmart plans to commercialize its drug delivery platform by achieving its initial approval with a reformulation of an FDA-approved drug already approved for an existing orphan indication, such as a rare pediatric cancer. The initial approval will validate the platform's potential to improve therapies, thereby mitigating the regulatory burden of future orphan and non-orphan filings, and facilitating licensing agreements with other pharmaceutical companies looking to extend patent life. BALANCE PROGRESS VS. INNOVATION For a start-up, much of its true value lies in its intellectual property, not just the size of its potential customer base. While constant, measurable progress toward defined goals is a key element that drives investments, start-ups should also be concerned with simultaneously enhancing their valuation. Therefore, whenever practical, a company should focus on developing an IP portfolio that will facilitate additional regulatory approvals, increase future revenue potential, and extend the company's potential beyond the life cycle of any single product. Importantly, broad intellectual property also mitigates the investor's risk of any single failure or substantial challenge to product development. Focus on the prod- In the big world of clinical trials, it's the small stuff that counts. WZƐĞƌǀĞƐĂƐĂƚƌƵĞĞdžƚĞŶƐŝŽŶŽĨŽƵƌĐůŝĞŶƚƐ tĞƚĂŝůŽƌĞĂĐŚƉĂƌƚŶĞƌƐŚŝƉƚŽLJŽƵƌƐŝnjĞ ĐƵůƚƵƌĂůďĂĐŬŐƌŽƵŶĚ ƉŝƉĞůŝŶĞ ƐĞƌǀŝĐĞ ĂŶĚŽƉĞƌĂƟŽŶĂůƌĞƋƵŝƌĞŵĞŶƚƐ͘ clearlypra.com >ĞĂƌŶŵŽƌĞ about our ƚĂŝůŽƌĞĚƐŽůƵƟŽŶƐ WŚĂƐĞ/ /s &Ƶůů ^ĞƌǀŝĐĞŝŽƉŚĂƌŵĂĐĞƵƟĐĂůƌƵŐĞǀĞůŽƉŵĞŶƚ ŵďĞĚĚĞĚĂŶĚ&Ƶůů^ĞƌǀŝĐĞ^ŽůƵƟŽŶƐ dƌĂŶƐĨŽƌŵŝŶŐůŝŶŝĐĂůdƌŝĂůƐƚŚƌŽƵŐŚ KƵƌWĞŽƉůĞ /ŶŶŽǀĂƟŽŶ dƌĂŶƐƉĂƌĞŶĐLJ © PRA 2013. All Rights Reserved. 11.13 November 2013 LifeScienceLeader.com 59