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, email@example.com
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).