How Decentralized Clinical Trial Methods can be Leveraged to Overcome 5 Common Challenges in Clinical Trials
As anyone involved in clinical trials knows, the process to develop a new medicine is long and expensive, and many drug trials fail. It takes an average of 10 years to bring a drug from discovery to market, with the clinical trials phase taking an average of six to seven years1. The median cost of a clinical trial to support FDA approval of a new drug is $19 million2, and of all drugs in clinical trials, only 14% receive FDA approval3.
With so much at stake for patients and sponsors, research organizations involved in drug development must do all they can to overcome the challenges in clinical trials and increase the likelihood of success. Decentralized clinical trial (DCT) methods can help.
Five common challenges in clinical trials include controlling costs; getting FDA approval; recruiting participants; retaining participants; and completing the trial on time. Below is more on each challenge and how DCT can help overcome it.
1. Controlling costs
The average total cost to research and develop a drug that receives FDA approval is estimated at $2.6 billion4.The cost of clinical trials varies widely depending on several factors, such as the type of study, the nature of the drug being developed, and the study’s duration and number of participants. One analysis found that trials with fewer than 100 participants cost an average of $6 million, compared with $77 million for trials with more than 1,000 participants5.
The potential to mitigate the challenge with DCT
A significant part of the cost of clinical trials involves the set-up and use of brick-and-mortar sites and their associated costs, including staffing, pharmacy, administration, and overhead. These costs are avoided in a decentralized clinical trial that brings the trial to the participant’s home. For example, the largest Parkinson’s disease study ever attempted is relying on the services of certified mobile research nurses provided by PCM Trials. A lead investigator on the TOPAZ study estimates a cost savings of 80% per participant compared with a similar trial conducted in a clinical site. And a survey of individuals involved in the pharmaceutical and healthcare industries found a plurality of respondents expect DCTs to be less costly than site-based trials6.
2. Getting FDA approval
Investigators must submit an Investigational New Drug (IND) application to the FDA to get approval to conduct a clinical trial.The application includes information on previous animal study data and toxicity, manufacturing, background on the investigator, and details on the clinical protocol. The FDA has 30 days to review the application, after which time it may place a clinical hold to delay or stop the investigation, for any number of reasons.
The potential to mitigate the challenge with DCT methods
Reviewing and following FDA guidance documents when designing the study may enhance the likelihood of receiving FDA approval to proceed with a trial. For example, FDA guidance for Enhancing the Diversity of Clinical Trial Populations7 discussed the burden placed on participants who must make frequent visits to sites and, as a solution, recommended that sponsors consider using mobile medical professionals, such as mobile research nurses and phlebotomists, to visit participants at their homes.
3. Recruiting participants
There is widespread agreement that recruitment is one of the biggest challenges in clinical trials, though assessments vary as to the scope of the patient enrollment challenge. The Tufts Center for the Study of Drug Development’s (CSDD) data shows that despite substantial improvement over the years, nearly a quarter (23%) of studies were still not able to meet or beat their planned timeline8. The ability to attract and enroll patients who meet the study criteria will likely remain an ongoing challenge.
The potential to mitigate the challenge with DCT methods
A decentralized clinical trial enables sponsors to enroll participants from a much wider geography than can typically be done with a site-based trial. Many would-be participants lack the time or financial resources to travel to distant clinical sites, whereas a study that brings the trial to the patient is much more feasible. In addition, casting a wider recruitment net makes it more likely that participants will better represent the population who would benefit from the drug once it’s approved. Lack of diversity in clinical trials is a well-documented problem and of concern because drugs may react differently in people of different genders and ethnicities. A study of all 371 new drugs and biologics approved for sale in the U.S. from 2007 through 2017 found that women were under-represented in the studies by 7.3%; men were over-represented by 7.5%; and Blacks or people of African descent were under-represented by 65%9.
4. Retaining participants
As important as it is to enroll patients, it’s equally important to retain them. Unfortunately, data show clinical trial dropout rates have taken a turn for the worse in recent years, from 15.3% in 2012 up to 19.1% in 201910.
The potential to mitigate the damage with DCT methods
Some participants drop out for reasons that are to be expected and beyond the control of investigators, such as disqualification due to lab tests or other complications (20%), or the side effects of the study drug (14%)11. But other reasons, like the location of the study site, poor communication with the study site, and an overly burdensome time commitment, could be averted with decentralized clinical trial methods. The patient-centric focus of home-based studies makes participation much more convenient and less burdensome for participants. In addition, mobile research nurses are a powerful retention tool. Nurses often build a strong personal rapport with trial participants during their home visits and can help educate and motivate them to stay involved.
5. Completing the trial on time
One challenge for study sponsors is that other novel therapies promising similar benefits may be under investigation at the same time, creating a competitive environment. At the time of this writing, there were nearly 400,000 studies listed in clinicaltrials.gov, representing an eleven-fold increase over 2007 when roughly 35,000 studies were listed12. Being the first-to-market with a safe and effective new drug can give a company a competitive edge by gaining early patient adoption and avoiding price competition.
The potential to mitigate the challenge with DCT methods
Site-based trials carry some capacity constraints; for example, the site can handle only as many patient visits at one time as operating hours and staffing levels will allow. This may cause smaller numbers of participants to be enrolled in phases over a longer period, extending the length of the study. Decentralized clinical trials are free of those constraints, enabling more participants to be enrolled more quickly, potentially expediting the path to commercialization. The flexibility of the DCT model also can be beneficial if trial results should start to trend unexpectedly such as by making it easier to pivot quickly to scale up.
About PCM Trials
PCM Trials supports sponsors and CROs and, since 2008, has worked with more than half of the top 20 global biopharmaceutical companies. For more information, please visit PCM Trials Mobile Research Services.
To discuss an upcoming trial and how to leverage a decentralized or hybrid strategy to address challenges, call 888-628-9707 or visit https://pcmtrials.com/request-for-proposal/ to submit your RFP.
References
1. PhRMA. “Biopharmaceutical Research & Development: The Process Behind New Medicines.” (2015)
2. Johns Hopkins Bloomberg School of Public Health. “Cost of Clinical Trials for New Drug FDA Approval Are Fraction of Total Tab.” (2018)
3. CenterWatch. “New MIT Study Puts Clinical Research Success Rate at 14 Percent.” (2018)
4. Tufts Center for the Study of Drug Development. “Research Milestones.” (2016)
5. Johns Hopkins Bloomberg School of Public Health. “Cost of Clinical Trials for New Drug FDA Approval Are Fraction of Total Tab.” (2018)
6. Pharmaceutical Technology. “Industry expects DCT costs to be lower compared with traditional trials: Survey.” (2021)
7. U.S. Food and Drug Administration. “Enhancing the Diversity of Clinical Trial Populations” (2020)
8. Tufts Center for the Study of Drug Development. “Drug Developers Are Making Strides in Streamlining Patient Recruitment and Retention for Clinical Trials, According to Tufts Center for the Study of Drug Development.” (2020)
9. Tufts Center for the Study of Drug Development. “Drug Developers Are Making Strides in Streamlining Patient Recruitment and Retention for Clinical Trials, According to Tufts Center for the Study of Drug Development.” (2020)
10. CenterWatch. “Recruitment Rates Rising, but Retention Rates Fall, According to New Study.” (2020)
11. CISCRP. “2019 Perceptions and Insights Study.” (2019)
12. National Institutes of Health. “Ethical issues in competing clinical trials.” (2019)
