Orphan Drugs: Commercialization, Clinical and Regulatory Challenges

Part of Gerson Lehrman Group, Inc.
Orphan Drugs
Commercialization, Clinical and
Regulatory Challenges FeaturingThomas Copmann, PhD, Owner of Drug Development AssociatesCandida Fratazzi, MD, President of Boston Biotech Clinical ResearchMichael Richardson, MBA, Chief Executive Officer at Radnor Pharma Moderated byDarien Kadens, PhD, MBA, Senior Director, Market Intelligence, INNOVUS
© 2011 Gerson Lehrman Group, Inc. All rights reserved. +1 212 616 8915 | GLG_Life_Sciences@glgroup.com | www.gplus.comPart of Gerson Lehrman Group, Inc.
Are orphan drugs the next act for big pharma? It's beginning to look that way, based on big pharma's 70% market share in 2009 for rare and genetically inherited diseases, up from a little over 50% three years earlier. But successfully competing in this space requires different strategies and skills than those necessary for blockbuster drug development. Gerson Lehrman Group, Inc.'s panel of experts discusses the commercial, clinical, and regulatory challenges unique to orphan drug development.
Q: It's been estimated that there are between MR: A lot of this comes down to whether you're 6,000 and 7,000 rare diseases. How are working opportunistically or whether you have companies prioritizing rare diseases the ability to select from the very long list of based on their relative commercial diseases. The first question is: Are you selecting attractiveness? What other factors are a disease area or are you taking advantage of an driving prioritization decisions? opportunity that's linked to your company's core competencies? Michael Richardson: I think a lot is driven by reimbursement and exclusivity. In categories where Depending on your situation, some tactics become there are multiple options available, as well as more important. For example, if you have a disease brands and generics, there's a lot of competition that a physician might see once a year or once and a lot of pressure from payers. With many a decade, the way to success is not by having orphan drugs you have more opportunity to have a sales force creating top of mind awareness. flexibility with pricing and some very clear-cut Instead what you want to ensure is that all the intellectual property to recoup your investment. data resources that a physician would go through while doing a differential diagnosis would be Companies have to put themselves in spaces accurate and up to date. You want to be sure they and therapeutic areas where need is very could find your product as they work through the high and there's more flexibility on pricing and more common treatments and options. It is highly reimbursement. A drug that's not nearly as large as inefficient to use the classic marketing tactics like a blockbuster can become incredibly profitable for you would do for a proton pump inhibitor. the volume it has. I would emphasize access to the patient groups. If the patients are already identified, it's very Q: What commercial capabilities are required important to have an association with the for a company to become orphan focused appropriate patient advocacy group. Otherwise, across multiple disease areas instead of unless a patient is routed to a subspecialist the staying in one disease area? incidence of a physician seeing a patient like that would be very rare.
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Orphan Drugs (continued)
Q:Is it easier to work on multiple drugs because Dr. Thomas Copmann: One of the major problems is you no longer have an investment in a large that the FDA and EMA (European Medicines Agency) sales force? waive part of the regulatory requirements on safety for orphan drugs but not on efficacy requirements. MR: That's right. What you're doing is more of a This complicates recruitment. A lot of time, rare ground up approach. It's important that every disease populations don't want to be on the placebo database you can think of gets people to the right end of things. That's that major hurdle I see on the place. It can be Epocrates, Sermo, WebMD. Even clinical development side.Wikipedia and Google are common ways that patients find their way to treatment. Q: Could you elaborate on the differences The same synergy or economy of scale that you between the regulatory environments in the would us... [download for more]