Orphan drugs (ODs) for rare diseases (RDs) present pharma companies with the opportunity to address a substantial unmet medical need. More than 500 orphan drugs have been approved for the treatment of rare diseases since the passage of the Orphan Drug Act (ODA) in 1983, and the pace is quickening. Commercialize an Orphan Drug, more than 230 new orphan drugs have received FDA approval. In 2015, FDA approved 21 novel drugs with a rare-disease designation, out of 45 new drugs overall.
Per the ODA, this designation provides for multiple incentives, including R&D tax credits, waiver of the Prescription Drug User Fee Act (PDUFA) filing fee ($2.3 million per filing in FY 2016), seven years of marketing exclusivity, and more. The ODA provisions were designed to spur innovation by helping drug sponsors recover development costs and to provide a pathway to profitability for products that are usually limited to a small patient population.
Stronger efforts are needed by pharma companies pre-launch to ensure a successful OD launch (Commercialize an Orphan Drug). The small number of patients for each OD means the margin of error regarding accurate forecasts based on projected diagnosed patients, from a financial standpoint, can be substantial. An inaccuracy of just a small number of patients can have significant financial implications. Further, epidemiology-driven forecasts must estimate testing rates, diagnosis rates, as well as trends and leverage points to drive testing and diagnosis rates. Further, there may be an initial one-time “bolus” of untreated patients who had exhausted other treatment alternatives. This places greater importance on the validity and data used in prevalence and patient flow models to develop accurate patient forecasts.
PAGs have always been an integral part of customer engagement in the pharmaceutical industry. However, their role was long marginalized to rather being add-on support for general marketing and sales activities. Their role and benefit is a completely different story in orphan diseases, where many research and development initiatives have not only been influenced but also substantially initiated, driven, and funded by them.
Pharma companies must go beyond the drug when it comes to providing patient support. For example, the majority of RDs affect children, so the role and needs of caregivers are paramount. Social and economic support programs for caregivers will be necessary for the continued engagement and treatment of RD patients. In some cases, diseases may be progressive and eventually terminal, and treatments may be palliative. Great sensitivity to patients and caregivers must be applied in the design of patient materials and their touchpoints with patient support infrastructure, such as patient hubs and clinical educators.
