The Growing Orphan-Drug Paradigm - Pharmaceutical Technology

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PharmTech Europe

The Growing Orphan-Drug Paradigm
How niche strategies can offer mainstream potential for biopharmaceutical companies.

Pharmaceutical Technology
Volume 36, Issue 5, pp. 106, 105

Product-development implications

Orphan drugs have historically focused on a small, defined market need. Because the population of individuals likely to optimally benefit from any individual drug may be small, drugs produced will be higher cost because drugmakers will have to recoup their investment from a smaller population of patients. Proving the economic and clinical value of treatment is therefore more crucial for orphan drug. This value case can include elements such as high quality outcomes, better patient experience, sufficiently improved health to allow the patient to more effectively manage other conditions, or product characteristics that improve overall patient adherence. Each of these elements should be explored and planned for early in product development.

Because the target population will be smaller, clinical-trial participants may include some people suffering from comorbidities that would otherwise be excluded from the sample. In this regard, the trial participants begin to look more like people in the real world. As a result, careful tracking of all patient outcomes will be crucial. Preparing to track these outcomes will need to start early in product development and continue postmarket to develop the kind of longitudinal data required to make the case for premium reimbursement rates to payers.

Finally, developing more targeted drugs alongside similarly targeted diagnostics will be key. As medicine becomes increasingly personalized and diagnostics become more targeted, it will become easier for orphan-drug makers to identify the population most likely to benefit from their drug. Developing drugs and diagnostics together will not only improve the likelihood that the drug will be successfully used, but can also improve the likelihood that the drug will be approved. In 2011, two personalized drugs were approved for use in conjunction with a specific diagnostic test—Xalkori (crizotinib) for lung cancer and Zelboraf (vemurafenib) for melanoma (3, 4).

A model for success

The advantages and benefits of adopting an orphan-drug centric approach reach beyond product development because the smaller population doesn't require large marketing or promotional campaigns. As a result, companies pursuing orphan drugs have adopted different kinds of promotional campaigns, often partnering with patient advocacy groups to get in front of their target populations.

This kind of commercial model could become the model pharmaceutical manufacturers adopt increasingly across their portfolios. Orphan-drug manufacturers have historically had to develop a deeper understanding of that population's characteristics than drug companies seeking blockbusters. As diagnostics become more targeted and the use of personalized medicine grows, companies pursuing all types of drugs will need to understand their target populations better and partner with advocacy groups like orphan drug companies have done. Ultimately, the growth of orphan drugs is one element of a larger paradigm shift in the pharmaceutical industry—one that manufacturers will need to adjust to.

Rita E. Numerof, PhD, is president, and Michael N. Abrams, is managing partner, both with Numerof and Associates,


1. European Organization for Rare Diseases, "Rare Diseases: Understanding this Public Health Priority" (Nov. 2005).

2. FDA, "Risk of Acute Myocardial Infarction and Sudden Cardiac Death in Patients Treated with COX-2 Selective and Non-Selective NSAIDs" (Sept. 2004).

3. FDA, "FDA Approves Xalkori with Companion Diagnostic for a Type of Late-Stage Lung Cancer" (Aug. 2011).

4. FDA, "FDA Approves Zelboraf and Companion Diagnostic Test for Late-Stage Skin Cancer" (Aug. 2011).


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