P72: Trends in the Use of Novel Trial Design Master Protocols in Clinical Research
Poster Presenter
Bradley Smith
Vice President, Translational Medicine
IQVIA United States
Objectives
The objective of the research was to characterize the use of master protocols in clinical trials including sponsor, drug class and therapeutic area over the last 14 years plus recent use in the development of COVID-19 treatments to test the hypothesis that use has increased in areas of most need.
Method
Clinical trial information was collected from the Citeline Trial Trove Database for umbrella, basket or platform studies for the last 14 years. Trial characteristics were summarized to determine if use of master protocols has changed to address areas of highest need for innovation.
Results
Our survey identified 110 master protocols using a strict criterium that excluded routine early phase cohort expansion studies that include multiple indications. Most studies were sponsored by cooperative groups (24%), academic organizations (18%) or government institutes (15%) with the remainder sponsored by industry (44%). Most industry sponsors are top 20 pharma (81%). 41%, 60% and 76%, of basket, umbrella and platform studies started in the last 4 years, respectively. 68% of master protocols are ongoing or planned. 54% of master protocols are early phase studies with less than 400 patients. 78% are phase 2 studies regardless of sample size. 48% of master protocols target more than one indication and 17% are in non-oncology therapeutic areas. 82% are in oncology, 13% in infectious disease, 3% in CNS and 2% in endocrinology. Immune-oncology trials represent 80% of industry studies. Adaptive study designs are used in 83%, 72% and 80% of basket, umbrella and platform studies, respectively. These results confirm the hypothesis that use of master protocols has increased in areas of most need in oncology and phase II studies especially in industry immune oncology research. However, use is also found in additional therapeutic areas as highlighted below.
We identified 13 COVID-19 master protocols with all but 1, sponsored by academic or cooperative groups. 4 are phase I/II or II while 5 are phase III and 1 phase IV. 4 studies are seamless design I/II or II/III. The mechanisms of action of the therapies vary greatly but most often are immunosuppressive (12 drugs), some with anti-viral activity as well (3 drugs) with 1 drug with only anti-viral activity. 7 studies target moderate to severe patients while 5 studies include a broader patient population. Only 1 study is for exposure prophylaxis. These results highlight the rapid adoption of platform studies in COVID-19 treatment development for specific rapid testing of immunosuppressive drugs in various patient populations.
Conclusion
Our survey identified 110 master protocols using a strict criterium that excluded routine early phase cohort expansion studies that include multiple indications. Most studies were sponsored by cooperative groups (24%), academic organizations (18%) or government institutes (15%) with the remainder sponsored by industry (44%). Most industry sponsors are top 20 pharma (81%). 41%, 60% and 76%, of basket, umbrella and platform studies started in the last 4 years, respectively. 68% of master protocols are ongoing or planned. 54% of master protocols are early phase studies with less than 400 patients. 78% are phase 2 studies regardless of sample size. 48% of master protocols target more than one indication and 17% are in non-oncology therapeutic areas. 82% are in oncology, 13% in infectious disease, 3% in CNS and 2% in endocrinology. Immune-oncology trials represent 80% of industry studies. Adaptive study designs are used in 83%, 72% and 80% of basket, umbrella and platform studies, respectively. These results confirm the hypothesis that use of master protocols has increased in areas of most need in oncology and phase II studies especially in industry immune oncology research. However, use is also found in additional therapeutic areas as highlighted below.
We identified 13 COVID-19 master protocols with all but 1, sponsored by academic or cooperative groups. 4 are phase I/II or II while 5 are phase III and 1 phase IV. 4 studies are seamless design I/II or II/III. The mechanisms of action of the therapies vary greatly but most often are immunosuppressive (12 drugs), some with anti-viral activity as well (3 drugs) with 1 drug with only anti-viral activity. 7 studies target moderate to severe patients while 5 studies include a broader patient population. Only 1 study is for exposure prophylaxis. These results highlight the rapid adoption of platform studies in COVID-19 treatment development for specific rapid testing of immunosuppressive drugs in various patient populations.
