KEY RESULTS. More than half of companies do not have a dedicated department or team responsible for innovative clinical trial design.

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2 OVERVIEW The drug development industry is increasingly pursuing innovative approaches to clinical trial design due to rising costs of conducting trials, greater emphasis on patient-centricity, and the logistical limitations of randomized clinical trials in areas such as oncology. Though many of these approaches are still in their early stages, they offer the potential to lower costs, enhance efficiency, increase flexibility and improve data quality while ensuring patients receive the most effective treatments. 1-3 Significantly, the U.S. Food and Drug Administration has signaled its willingness to consider innovative designs smoothing the way for these approaches and recently held a public meeting to discuss the agency s expectations for these designs and to promote their use. 4 To gather better information about the nature and effectiveness of innovative clinical trial designs, SCORR Marketing, in partnership with Applied Clinical Trials, conducted a survey in the spring of We specifically looked at many emerging designs, including adaptive, basket, umbrella and enrichment studies. In this report, we explain: Who has a team dedicated to innovative clinical trial design and who doesn t Most common emerging trial designs The two-year outlook for the use of clinical trial designs How these approaches are implemented Benefits realized by adopting innovative study designs Which kind of trials offer the best ROI How well these approaches have met operational and financial objectives Survey participant demographics include: Organization types those who work for sponsors such as pharmaceutical, biopharmaceutical and biotech companies; service providers such as contract research organizations (CROs) and technology providers; academic/ research institutions and hospitals, research sites and consultancies 55% 27% 9% Job functions those in clinical operations, research and development (R&D), and data and project management Locations more than half of respondents located in North America, more than one-fourth based in Europe, with respondents from six continents 3% 5% 1% Company size respondents from smaller (50 employees or fewer), medium (51 to 1,000 employees), and larger (1,000+ employees) companies 2

3 KEY RESULTS More than half of companies do not have a dedicated department or team responsible for innovative clinical trial design. Almost as many respondents (44%) said their company does not have a dedicated team or department as there were who claimed their company or institution did (46%). Company has dept/team primarily responsible for innovative clinical design Service providers North America Organization type and geography matter Company does NOT have dept/ team primarily responsible for innovative clinical design Academic/research Outside of North America Survey participants from service provider companies were twice as likely than not (67% vs. 33%) to state their company has a person, department or team specifically tasked with innovative clinical trial design. On the other hand, respondents from academia were almost twice as likely (58% vs. 33%) to say their institution does not have dedicated resources. North American respondents were almost twice as likely (57% vs. 32%) to say their company has a dedicated innovative clinical trial design department or team. Conversely, those from outside of North America were almost twice as likely (59% vs. 32%) to claim that their company does not. New clinical trial designs are most often implemented by therapeutic area Almost two in five survey participants (39%) said their company implements new clinical trial design by therapeutic area. Slightly less than one-third said implementation was a centralized companywide initiative. The response from most organization types, job functions, levels of responsibility, geographies and company sizes mirror the overall results. Two notable exceptions are: C-Suite/VP respondents were much more likely to say implementation was a companywide initiative (54%) as opposed to a therapeutic area (23%). Given that their responsibilities generally concern the company as a whole, it is possible that senior management interpreted this question somewhat differently than the other groups: They may perceive innovative clinical designs as a corporate initiative even though selection of the type of trial design may reside in the therapeutic departments. Respondents from small companies (50 or fewer employees) were somewhat more inclined to state that implementation was centralized (42%) rather than dependent on therapeutic area (26%). 3

4 KEY RESULTS (CONT) Adaptive studies provide the best returns on investment (ROI). A plurality of respondents (44%) selected adaptive trials defined in the survey as those where there is flexibility to alter the study based on how patients are responding as providing the best ROI. Basket studies were named by roughly one quarter of the respondents as offering the best investment returns. Basket studies are trials that allow for testing one drug on different subgroups. Among organization types, sponsors were equally inclined to select adaptive studies and basket studies (42% each) as providing the best ROI. Service providers were four times more likely to believe adaptive studies provided the best ROI (36%) compared to basket studies (9%), while academics were twice as likely (36% vs. 18%). Respondents from outside of North America strongly favored the ROI of adaptive studies over basket studies (56% vs. 17%). Those from North America were equally disposed to identify adaptive and basket studies (35% each). What do these organizations say provides the best ROI? Academia/ Research Sponsors Service Providers Familiarity with adaptive studies high across most groups Seven in ten (70%) respondents were familiar with adaptive studies. Most groups ranging from organization type to company size were more familiar with adaptive studies than they were with any other innovative clinical trial designs. Exceptions include survey participants from research sites (only 29% were familiar with adaptive designs), those outside of North America or Europe (33%), R&D (45%), and associates or coordinators (also 45%); these happen to be groups that are less likely to influence study design. Some groups are familiar with more types of innovative clinical trial designs than others Among levels of responsibility, C-Suite/VPs identified an average of 3.34 types of innovative clinical trials with which they were familiar, while managers identified an average of less than half that (1.55). On average, North American respondents recognized 2.72 types of innovative designs and Europeans were familiar with 2.49 types, while respondents from the other areas of the world were acquainted with only 1.30 of the options. Studies that use EHR data Tie (adaptive studies and hybrid studies) Tie (adaptive studies and basket studies Umbrella studies Adaptive studies Tie (studies that use semantic technology and data and "other") 4

5 KEY RESULTS (CONT) There is a cautious optimism about the future of study innovations. The caution may stem from uncertainty about whether these innovations have met operational and financial expectations. A plurality of respondents predict their company and the industry both will implement more study innovations in the next two years. However, a sizable minority believe the increase will be slight. Interestingly, respondents believed there will be broader adoption of innovative designs in the industry than in their own company. In two years, the utilization of study innovations at your company will? In two years, the utilization of study innovations in the industry will? Increase substantially 42% 50% Increase slightly 33% 44% Stay the same 25% 6% Decrease 0% 0% Big companies more optimistic overall but smaller companies more likely to make substantial changes One-half (50%) of respondents from smaller companies believe their company s utilization of study innovations will increase substantially in the next two years. About one in seven (14%) say the growth will be small. One-third (33%) of respondents from larger companies believe their company s utilization of study innovations will increase substantially in the next two years, while more than half (56%) think the increase will be slight. Uncertainty over whether clinical trial innovations have been worth it The most popular answer to whether clinical trial innovations have met expectations is unsure. Two-thirds of respondents (67%) either answered unsure or no. 45% 33% 22% 22% 33% North American survey participants were somewhat skeptical. Just one in five (22%) said their company s expectations were met. Yes Respondents from large companies were especially pessimistic. A mere one in nine (11%) stated that they believed their company s operational and financial expectations were met by the implementation of clinical trial innovations. No Unsure 5

6 OTHER KEY TAKEAWAYS Is there a person, department or team in your company primarily responsible for innovative clinical trial design? Please identify the therapeutic area(s) in which? (Select all that apply.) 10% 46% You work Your company implements new clinical trial design 44% Yes No Unsure Cardiovascular 49% 35% Oncology 40% 46% Immunology 33% 32% Neurology 33% 30% Infectious disease 30% 24% How are new clinical trial designs implemented by your company? (Select all that apply.) By therapeutic area Centralized companywide initiative By medical condition By geographic area Unsure Other 10% 12% 14% Percentages 29% 29% 39% Rare diseases 30% 22% Gastroenterology 28% 27% Alimentary/metabolic 26% 19% Dermatology 26% 14% Pediatrics 26% 11% Respiratory/pulmonary 26% 11% Musculoskeletal 23% 14% Other 14% 11% 6

7 OTHER KEY TAKEAWAYS Which of the following provides the best returns on investment? (Select all that apply.) Adaptive studies Basket studies Studies that use EHR data Enrichment studies Hybrid studies Umbrella studies Siteless studies Studies that use semantic technology and data Other 9% 12% 14% 16% 16% 26% 26% 44% 14% Percentages Which of the following types of study innovation(s) does your company use or plan to use? (Select all that apply.) EHR data Siteless studies Blockchain Semantic technology and data 17% 14% 31% 63% Other 9% Percentages Which of the following types of clinical trials? (Select all that apply.) Are you familiar with? Does your company implement? Adaptive 70% 43% Basket 59% 43% Master protocols 45% 32% Enrichment 39% 27% Hybrid 38% 27% Umbrella 29% 20% My company does not implement any of these n/a 20% Other 9% 11% 7

8 OTHER KEY TAKEAWAYS On a 1-10 scale (with 10 being the highest), please rate the extent to which your company collaborates with in designing clinical trials (ratings are represented as averages). 1 CROs 6.49 (average response) 5 Sponsors 7.73 (average response) 10 Does your company solicit or receive scientific advice from regulatory authorities prior to design? 22% 7% Yes No Unsure Does your company utilize a target product profile (TPP) a template for the summary of a drug development program described in terms of labeling concepts prior to designing a new clinical trial? 27% 48% Yes No Unsure 71% 25% 8

9 OTHER KEY TAKEAWAYS Better recruitment/retention On a 1-10 scale (with 10 being the highest), please rate the extent to which your company has benefited from each of the following in its implementation of new clinical trial designs. Cost savings Faster execution Better quality data AVERAGE RATING Regulatory compliance 0% 20% 40% 60% 80% 100% What does your company need most to improve the implementation of new clinical trial designs? 19% 19% Training Up-front planning 11% 17% Resources Technology Institutional support My company does not need anything 17% 17% 9

10 OTHER KEY TAKEAWAYS How does your company measure the performance of innovative clinical trial designs? (Select all that apply.) Better quality data 59% Better recruitment/ retention Cost savings 54% 54% Faster execution 44% Regulatory compliance 34% No measurement 22% Other 5% 0% 10% 20% 30% 40% 50% 60% Level of complexity Level of communication Training requirements Number of work responsibilities On a 1-10 scale (with 10 representing the most change), how much has your job changed as a result of the implementation of innovative clinical trial designs? AVERAGE RATING % 20% 40% 60% 80% 100%

11 SUMMARY & CONCLUSION While most companies have implemented innovative trial design, only half have a dedicated person, department or team responsible for overseeing it. Adaptive studies, which companies are most familiar with, have the best ROI. Better recruitment and retention has been the biggest benefit of innovative trial design. Conclusion The jury is still out on whether clinical trial innovations have met operational and financial expectations. Two-thirds of respondents either said they were uncertain these new designs accomplished what they had hoped or said outright that the innovations failed to meet their hopes. Indeed, the industry continues to have serious reservations about these innovations, including concern about the potential for drawing of erroneous conclusions and doubt that the infrastructure is yet fully capable of handling digital data. 5-6 The complexity that arises from the quantity and format of data and interoperability systems is also worrisome. 7 Yet despite the uncertainty and disappointment expressed by those who answered this survey, they still expect to see these innovative designs popularity grow both within their own company and in the industry as a whole. Especially now that at least some of these designs have the FDA s blessing, these innovations have potential to make a positive impact. The problems the industry faces, with inefficiencies and rising costs in particular, are simply too significant to think otherwise. 11

12 REFERENCES 1 Innovative Trial Design: Where Regulators and Payers Divide, Drug Development Technology, 11/15/ Is Patient Centricity Truly at the Core of Clinical Trials? KNect365 Life Sciences, July Illuminating Ideas: Innovative Clinical Trial Design, Roche website. 4 " Promoting the Use of Complex Innovative Designs in Clinical Trials, U.S. FDA, 3/20/ " FDA Advances Innovative Clinical Trial Designs, Applied Clinical Trials, 3/22/ " Digital R&D: Transforming the Future of Clinical Development, Deloitte Insights, 2/14/ " Leading CROs Form New Industry Standards Group to Improve Collaboration With Sponsors, Align Clinical CRO, 3/27/