Pharmaceutical Commerce - September/October 2012 - (Page 1)

SEPTEMBER/OCTOBER 2012 2012 Pro duct Security Report P. 20 Brand Marketing & Communications Brand Marketing & Communications Building bridges to rare disease patients Orphan drug development and marketing have important distinct requirements for R&D, regulatory and marketing By Wendy White, Siren Interactive Grappling with the long-term care market The geriatric-dominated LTC market is growing, but has unique constraints By Lena Anthony Have you noticed that interest in rare disease product development isn’t as “rare” as it used to be? Currently about one-third of the drug development pipeline is targeting rare (or “orphan”—the two are used interchangeably) diseases, which are defined as those that affect fewer than 200,000 people in the US. Approximately 10% of the US population has one of the orphan or rare diseases. Congress, with the encouragement of the FDA and patient advocates, created the Orphan Drug Act of 1983 to help motivate and incentivize pharmaceutical companies to invest in developing treatments to better meet the needs of rare disorder patients. Rare disease patients are interested in being Logo of the Global Rare Disease Day, emphasizing a collaborative stakeholders in drug development, and collective holding of hands to address rare disease needs. patient communities are a potent factor in conducting Credit: Eurodis research and clinical trials, and partnering with pharma for a drug’s commercialization. Patient insight, feedback, and data relative to science, medicine and quality of life are critical to identifying gaps and opportunities that are unique to the rare disorder patient perspective on the disease. The FDA Rare Disease Unit already involves patients directly through its Patient Representative Program, in which patients work with the FDA and sponsors throughout the product development process. I believe that this patient-centric model is the future of healthcare as we move toward personalized medicine that is customized to patient populations based on their genetic information. The long road to diagnosis The conventional expectation is for a patient to rely on a physician for an accurate diagnosis of a common disease and an effective treatment. Often neither of these presumptions applies to a patient with a rare disorder. Given the small universe of patients, a typical physician might never encounter a rare disorder patient over the course of his or her entire career. Physicians may not have the expertise to recognize or diagnose a rare disorder. A biotech or pharmaceutical company can play an important role in education—not only for patients but also for physicians. It can take several years for a patient to receive a correct rare disorder diagnosis. Misdiagnosis often results in poor quality of life for the patient and, potentially, side effects from medication that’s incorrectly prescribed. Even when the correct diagnosis is made, the majority of patients are faced with the reality that there is no treatment. An FDA-approved therapy is available for only about 5% of the 7,000 rare disorders and diseases. Facing endless healthcare questions without many answers, rare disorder patients often take the lead in driving and managing their own diagnosis and treatment. Understandably, they rely heavily on the Internet for information that may not be available anywhere else. “Dr. Google” is frequently consulted when searching symptoms or treatment options, verifying a physician’s diagnosis, or finding information about a prescription drug. For rare disease-focused pharmaceutical companies, there’s a need for digital expertise rather than a reliance on continued on page 14 The long-term care (LTC) market is being whipsawed by many of the more prominent trends in healthcare: the aging of the Baby Boom generation; the efforts to rein in healthcare costs (which drives more patients to LTC alternatives, like home-based care); and, as a result of Obamacare, the simultaneous expansion of national healthcare coverage and threatened contraction of state-level Medicaid programs. For the pharma industry, LTC presents a special set of opportunities and challenges: the elderly are the biggest proportion, demographically, of medicated patients, while the LTC distribution channel has constraints not common to other channels. This year, approximately nine million Americans over the age of 65 will need long-term care (LTC), and by 2020 that number will grow to 12 million, according to CMS. In 2030, when the last of the baby boomers turn 65, and the first wave are well into their 80s, the number of older adults requiring LTC will increase even more. According to the HHS, people who reach the age of 65 have a 40% chance of entering a nursing home. About 10% of the people who enter nursing homes will stay there at least five years, with the average stay being about two-and-a-half years. This market—in which healthcare services are delivered in a variety of settings, including skilled nursing homes, assisted living facilities and retirement communities, as well as in the patient’s home—represents a sizable growth opportunity for drug manufacturers and the pharmacies that dispense their products. In 2011, prescription drug spending in the continued on page 16 Legal & Regulatory Ensuring the quality of comparative effectiveness research As payers seek higher-quality data from realworld treatment settings, the importance of reliable measures becomes critical By Nancy Dreyer, MPH, PhD, Quintiles Outcome Randomized clinical trials will always play an important role in the drug development process, and rightfully so. Yet as physicians’ prescribing choices are becoming increasingly constrained by payers, persuasive data from randomized clinical trials are no longer sufficient to ensure adoption of effective new products. Payers and physicians are coming to realize that data obtained from the randomized clinical trials—which will remain key to the regulatory approval process—cannot be relied upon to give insights to a product’s real-world value. For dossier submission to payers, biopharmaceutical companies must now submit other types of data to assist payer organizations in their formulary decisionmaking. A new drug’s success on the most important measures—improvement in the clinical outcomes of the patients who use these products and cost effectiveness for the organizations that are paying for them—are what drive formulary decision. Comparative effectiveness research (CER)—an approach in which products are evaluated in a true-to-life setting against the current standard continued on page 32 September | October 2012 Visit our new website at www.PharmaceuticalCommerce.com 1 PRSRT STD US POSTAGE PONTIAC, IL PERMIT 268 PAID http://www.pharmaceuticalcommerce.com http://www.PharmaceuticalCommerce.com

Table of Contents for the Digital Edition of Pharmaceutical Commerce - September/October 2012

Pharmaceutical Commerce - September/October 2012
Editorial
Contents
Op-Ed
Top News
Business/Finance
Brand Marketing & Communications
Supply Chain/Logistics
Information Technology
Manufacturing & Packaging
Legal & Regulatory
Meetings and Editorial Index

Pharmaceutical Commerce - September/October 2012

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