- Support and guide R&D
- Inform and refine patient support services
- Identify and educate patients
- Influence regulation and policy
White papers represent the synthesis of ClearView's perspective on key areas of interest within the broader biopharmaceutical industry.
Dramatic growth in the immuno-oncology field suggests a paradigm shift across oncology in the coming years. This evolution will impact decision-making for all novel oncology assets, and in particular for non-IO therapies. To compete in this future environment and ensure success within the future standard of care, cancer drug developers must think strategically regarding enduring white space opportunities and how to best position novel agents to capture these opportunities.
In this article published in In Vivo, we review key strategic considerations across clinical development, market access, and post-launch commercialization that will be important for current and future oncology drug development.
Orphan drug development is attractive to drug developers for many compelling reasons: tax advantages, extended market exclusivity, accelerated approval timelines, historically favorable access at premium prices, potential for lower cost clinical trials, and recent commercial success stories such as Soliris and Kalydeco. However, orphan diseases do not necessarily offer an easy win. Manufacturers must be prepared for the unique challenges associated with orphan disease drug discovery, clinical trials, and market development, as well as the eventual likelihood that U.S. payers will actively manage pricing and market access for orphan products. Ultimately, considerable opportunity remains for drug developers in orphan diseases, but success will require companies to develop an orphan disease mindset for strategic decision-making and invest in capabilities commensurate with the anticipated nuances and challenges.
In our new white paper, we lay out the clear areas of opportunity and the unique challenges associated with orphan disease drug development to help companies be best prepared to succeed in this exciting, but nuanced, space.
Informed drug development in an era of “precision medicine” requires a clear understanding of the risks and benefits that accompany the pursuit of companion diagnostics, a difficult objective given the complexity and rapid evolution of the field. While business cases for companion approaches typically focus on pricing and volume considerations, there is also a need for careful analysis of the strategy associated with managing clinical development, securing access and reimbursement, and generating product demand among target physicians. Without a full accounting of and appreciation for these factors, it is challenging, if not impossible, to direct an efficient, forward-looking drug-diagnostic program capable of maximizing commercial potential.
The breakthrough therapy designation has generated substantial interest since its creation one year ago, as this new pathway represents the most comprehensive set of clinical development benefits available for a pipeline agent. However, the breakthrough therapy pathway may also lead to potential commercial downsides and regulatory risk. Most notably, the limited dataset from the breakthrough clinical program may be insufficient to garner favorable pricing and reimbursement, particularly in Europe. In addition, the abridged clinical trials may not be considered satisfactory for regulatory approval in ex-U.S. markets. Taken together with several other commercial considerations, it is critical for manufacturers to develop a comprehensive understanding of these advantages and disadvantages prior to pursuing the breakthrough therapy pathway.