The United States (U.S.) represents the largest pharmaceutical market in the world. Thanks to its global market size ($105.3bn in 20211), strong protection of intellectual property rights, transparent regulatory framework that encourages innovation, as well as a mature clinical development industry, it is no surprise that many companies based outside of the U.S. are setting sights on this lucrative market.

Like the U.S., many countries have been increasing investments in biotechnology research and development over the past few decades, laying the foundation for potential clinical applications. For example, the number of biotech patents granted in China rose from over 1,600 (12% of global total) to more than 9,000 (24% of global total) from 2009 to 20192. Consequently, an ever-increasing number of non-US based companies are looking to bring their innovative investigational products to the U.S. for clinical development. Clearing the high bar set by the U.S. Food and Drug Administration (FDA) not only ensures access to the largest market, but also may provide a smoother path to marketing approval in other countries and markets.

Barriers to Entering U.S. Market
Companies based outside of the U.S. who aim to bring a drug to market in through the FDA face a set of unique clinical development and operational challenges:

• Design appropriate studies with an understanding of the local therapeutic landscape: Clinical practice and standard of care can vary significantly among different regions of the world. A protocol designed to identify a target patient population and meet standards of care in some countries may need to be amended before implementation in the U.S. For example, a study of advanced hepatocellular carcinoma in patients who have failed first line tyrosine kinase inhibitor (TKI) will be more difficult to enroll in the U.S., as the standard of care is shifting from first line TKI to first line immunotherapy. Similarly, an early phase non-Hodgkin’s lymphoma study may mandate only one line of previous treatment in some parts of the world, however, in the U.S. where many advanced therapies are available, most patients will have had at least two prior lines of treatment before considering such a study. A rational study design taking into consideration local therapeutic landscape will not only facilitate IND clearance, but also ensure access to the targeted patient pool. Collaborating with a CRO that has broad experience in the therapeutic area will help set a study on the right track from the start.
• Fierce competition for clinical trial sites: The U.S. is one of the most active clinical research markets, with many studies competing for the limited availability of top tier trial sites. Companies developing therapeutics in a crowded space such as immuno-therapy or precision medicine must be prepared to differentiate and justify their investigational products; using the science, therapeutic potential, and study design to drive investigator interest. Selecting the right sites is one of the key steps towards successful trial execution. Smaller biotech companies with limited resources or few key U.S. site relationships can utilize a CRO partner with large and relevant clinical trial site networks to support strategic site identification to ensure efficient trial delivery.
• Knowledge of local clinical trial process and timelines: Companies planning to bring their clinical studies to the U.S. need access to a network of vendors for supportive services such as drug depot, central labs, central imaging, etc. They also must understand local regulatory and oversight body requirements and timelines. For example, trials of cell and gene therapies will most likely be required to be reviewed by a local institutional biosafety committee (IBC) in addition to ethics review by their institutional review boards (IRBs). This additional review process extends the study start-up timeline. Having local resources at their fingertips is critical in the planning and execution of clinical development activities. Partnering with a CRO with an experienced team that understands the nuances of specific therapeutic areas and indications can provide a roadmap for key activities – including accurate estimates of cost and timeline factors.

Partnering with the right CRO to accelerate clinical development and commercialization
It is clear that to overcome the hurdles of entering the U.S. market, choosing the right CRO is paramount. By partnering with IQVIA Biotech’s clinical delivery team, sponsors gain expertise from two decades of planning and running clinical trials exclusively for biotech companies. Our dedicated teams have extensive experience in oncology and an understanding of the complexities and nuances of all phases of clinical development.

Contact us to discuss how we can help you navigate entry into the U.S. market.

References
1. https://www.ibisworld.com/industry-statistics/market-size/biotechnology-united-states/
2. WIPO IP Statistics Data Center, World Intellectual Property Organization, updated January 2021, https://www3.wipo.int/ipstats/index.htm