In-depth Process and Product Expertise – This is Key to CDMO Support of Orphan Drug and Breakthrough Therapy Development & Commercialization

As older blockbuster drugs lose patent protection and generic competition increases, many pharmaceutical companies are focusing discovery efforts on therapies with the potential to treat multiple niche populations. Increasingly, innovative small and emerging pharma firms are developing new drug candidates with orphan or breakthrough therapy status that are ultimately licensed or sold to large brand manufacturers. These companies rely heavily on contract manufacturing and development organizations (CDMOs) that can provide in-depth scientific expertise and achieve under rapidly accelerated timelines the development of cost-effective, robust, reliable processes that consistently yield high-quality products.  

Until recently, most pharmaceutical firms were not interested in the development of small-volume drugs due to fears of limited returns. With the age of the blockbuster drug now history, many drug companies are finding that niche therapies, particularly those that may treat numerous indications, not only provide patients with life-saving medications, but also realize attractive financials if developed in a streamlined and cost-efficient manner. There are over 7,000 different types of rare diseases and disorders, yet only a couple of hundred approved therapies designated as orphan drugs. According to EvaluatePharma, although the average Phase III development time for orphan drugs is not shorter than that for non-orphan drugs, the Phase III drug development costs for the former are half those of the latter, and the anticipated return on investment for a Phase III/filed orphan drug is nearly twice that for a non-orphan drug.

As a result, EvaluatePharma estimates that the orphan drug market is growing at an annual rate of 11%, more than double that of the overall prescription drug market (5%), and by 2020 will reach $176 billion in annual sales and account for 19% of the total non-generic prescription market. In 2013 alone, 260 orphan drug designations were granted. In 2014, the FDA approved 15 NDAs and seven BLAs with the orphan drug designation, along with 24 supplemental approvals.

Many companies are also pursuing the new breakthrough designation established in 2012 by FDASIA, the Food and Drug Administration Safety and Innovation Act. A candidate qualifies for breakthrough therapy designation if preliminary clinical evidence suggests that the drug may have substantial improvement over available therapies on at least one clinically significant endpoint. The development and approval times for breakthrough therapies are typically half that of the seven years for conventional drugs, and both the sponsor and CDMO benefit from greater FDA guidance and communication with the agency. FDA’s CDER approved 14 breakthrough therapies in 2014 and nine in 2015 as of August 21. 

Of the firms pursuing the development of orphan drugs and breakthrough therapies, many are small or emerging pharmaceutical or biopharmaceutical companies focused on niche, small molecule therapies. These companies often have limited resources in terms of laboratory, analytical, and manufacturing equipment (indeed, some are virtual companies in that respect) and depend heavily on service providers to perform crucial process and formulation development, validation, regulatory compliance, and manufacturing activities. The choice of CDMO can therefore have a direct impact on the success or failure of the new drug.

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