Drug and device makers face daunting odds in seeking to bring a product from Phase I clinical trials through to market approval – according to the Massachusetts Institute of Technology’s Project Alpha, a mere 10.5% make it to market.
A new report from Clarivate™ offers some steps life science companies can take to de-risk their clinical trials and improve their odds of market approval.
Among these are:
- Understanding the nuances of target markets
- Engaging regulators early and often
- Strategically selecting participants and making trials patient-centric
- Understanding diseases and treatments from a patient POV
- Building resiliency into clinical trial protocols
- Utilizing a variety of data sources to mine insights
Understanding the nuances of target markets
Choosing which markets to enter when and in what order requires understanding more than just local regulatory and reimbursement processes. It also requires thinking about how the product stands to benefit the local population, what sites and investigators are available to conduct trials, and what the path to approval looked like for in-market competitor products.
In one recent study, nearly half of recent market launches observed missed their first-year forecasts due to market issues such as HEOR data that was too weak to substantiate pricing or higher than expected discounts and rebates. A solid understanding of local market practices, regulator and payer concerns can help companies build needed evidence generation into trials.
Engaging regulators early and often
The work of building a robust regulatory strategy is best begun as soon as candidates are identified. Companies can help to expedite the process by initiating discussions with agencies in target markets as early as possible in order to understand pathways to expedited approval, local agency collaboration with regulatory authorities in other markets, feasibility of real world evidence to support analysis and expectations around study populations.
Increasingly, regulators are insisting that products be studied in local patient populations before they will consider them for approval. One recent example of this is the FDA’s rejection of an Innovant/Eli Lilly and Company oncology candidate based on clinical trials conducted solely in Mainland China.
Strategically selecting participants and making trials patient-centric
Effective recruitment and retention of patients requires an understanding of which patients are most likely to benefit from an investigational product, where the pain points for participation are and how to reduce the burden on patients, and which sites and investigators are best suited to the trial. Choosing the right participant segments can realize greater efficiency and better study outcomes, while a more frictionless patient experience can boost retention rates.
“Choosing the right participant segments can realize greater efficiency and better study outcomes, while a more frictionless patient experience can boost retention rates.”
Johnson & Johnson has patients in 32 countries vet its clinical trial protocols to make it easier for patients to participate. A pioneer of patient-centered clinical trial design, the company was pushing into at-home direct-to-patient clinical trials even before the pandemic, and has invested in diversifying its pool of trial participants and making pediatric clinical trials easier for patients and caregivers.
Understanding diseases and treatments from a patient POV
Gathering deep patient insights at the outset of the development phase can help companies avoid pitfalls down the road. Knowing, for example, the burden of diagnostics and tests on patients, patient preferences on mode and frequency of administration, and perceptions of risk/benefit tradeoffs can inform product and protocol design, and ultimately even market access conversations.
“Gathering deep patient insights at the outset of the development phase can help companies avoid pitfalls down the road”
Most importantly, these insights can help ensure a product that meets patient needs, ultimately improving uptake, outcomes and quality of life while bolstering a company’s argument for access and reimbursement.
Building resiliency into clinical trial protocols
Protocol amendments can add greatly to the time and cost of a clinical trial. To avoid them, companies can work to anticipate possible impediments such as trial disruptions or changing regulatory demands and build in compensation measures, such as master protocols, less stringent inclusion criteria and adaptability of visit types and site responses.
The COVID-19 pandemic has put the need for flexibility into stark relief. Clarivate data show that since March, 2020, more than 1,450 trials have reported a delay due to COVID, with the biggest impacts felt in trials concerning treatments for rare and neurological diseases.
Utilizing a variety of data sources to mine insights
Triangulating traditional and emerging data sources, including real world data, social data and digital health intelligence as well as site and investigator benchmarking and epidemiological data, can help companies spot potential roadblocks and design flaws as well as opportunities.
Having the right people asking the right questions and equipped with the right tools early in the process of trial design can help to set companies up for success.
For more detailed insights into this topic, read the full report here.
 Project ALPHA, 2022. Estimates of Clinical Trial Probabilities of Success. [Online]
Available at: https://projectalpha.mit.edu/pos/
[Accessed 20 5 2022]
 Ford J, Fezza T, Elsner N, et al. Key factors to improve drug launches (March 20, 2020). Deloitte Insights. [online]
Available at: https://www2.deloitte.com/us/en/insights/industry/life-sciences/successful-drug-launch-strategy.html
[Accessed 20 5 2022]