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The Main Challenges Of Conducting Clinical Trials: How To Overcome Them
Clinical trials are essential for advancing medical knowledge and developing new therapies, yet they are complex endeavors fraught with challenges: From recruiting patients to navigating intricate regulations, the process requires careful planning, significant resources, and adaptability.
To overcome these hurdles, a strategic approach and collaboration with a clinical research organization (CRO) can make all the difference.
In this article, we'll explore the main challenges in conducting clinical trials and provide actionable strategies to address them, with a focus on improving recruitment, maintaining regulatory compliance, and managing data accuracy.
Understanding the Key Challenges in Clinical TrialsClinical trials are multifaceted processes, and several factors can impede their progress; some of their main challenges include:
Patient Recruitment and RetentionRecruiting and retaining participants is one of the most significant barriers; this issue arises due to:
Stringent Eligibility CriteriaTrials often have narrow inclusion criteria, limiting the pool of eligible participants.
Lack of AwarenessMany potential participants are unaware of ongoing trials or have misconceptions about them.
Logistical BarriersParticipants may face travel, time commitment, or financial constraints.
Dropout RatesRetaining participants through the trial's duration is equally challenging, as unforeseen circumstances or lack of engagement can cause participants to leave.
Regulatory and Compliance HurdlesClinical trials are subject to stringent regulatory oversight to ensure patient safety and data integrity.
However, navigating the difficult, region-specific regulations can lead to delays in approvals and increased costs.
Additionally, any deviation from compliance standards can compromise trial outcomes or result in legal consequences.
Data Complexity and AccuracyThe sheer volume and variety of data collected during trials pose another significant challenge.
Modern trials rely on data from electronic health records (EHRs), wearable devices, and multiple trial sites, which can lead to inconsistencies or errors if not appropriately managed; ensuring the accuracy and security of this data is paramount.
Rising Costs and TimelinesClinical trials are expensive and time-intensive: Delays in recruitment, regulatory approval, or data analysis can further inflate costs – bringing a drug to market through clinical trials can take up to 10 years and cost billions of dollars.
Strategies for Improving Patient Recruitment and RetentionAddressing recruitment and retention challenges is essential for the success of clinical trials.
Effective strategies include:
Community EngagementEngaging with communities through education and outreach can help build trust and awareness.
Community leaders, patient advocacy groups, and local healthcare providers can play pivotal roles in dispelling myths and encouraging participation.
Simplifying Eligibility CriteriaBroadening inclusion criteria without compromising the study's integrity can significantly expand the pool of eligible participants.
Leveraging predictive modeling to assess risk factors can also help identify potential participants.
Using Technology to Streamline ProcessesDigital platforms, such as trial-matching apps and social media, can be used to reach a larger audience.
Virtual trials, where participants can complete certain activities from home, also reduce logistical barriers and increase convenience.
Developing Patient-Centric ApproachesOffering flexible schedules, providing transportation or reimbursement for travel costs, and maintaining regular communication with participants can improve retention.
Listening to participants' concerns and addressing them proactively fosters a positive experience.
Overcoming Regulatory and Compliance HurdlesNavigating supervisory settings is a critical aspect of clinical trials – strategies to overcome these challenges include:
Early Engagement with Governing BodiesProactively consulting with regulatory agencies can clarify requirements and streamline the approval process.
Early engagement also helps ensure study designs meet all necessary standards.
Global Harmonization of ProtocolsAdopting internationally recognized trial protocols across multiple countries can reduce variability and simplify compliance.
Organizations such as the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) provide guidelines for coordination.
Training and EducationInvesting in thorough training for all team members ensures they understand regulatory requirements and can adhere to them; regular workshops and updates on supervisory changes are essential.
Leveraging CRO ExpertiseCROs are well-versed in traversing governing backdrops across regions – partnering with a CRO can expedite compliance processes and reduce the risk of costly errors.
Managing Data Complexity and Ensuring AccuracyIn clinical trials, data is the backbone of decision-making; ensuring its accuracy and integrity is non-negotiable.
Strategies to achieve this include:
Investing in Advanced Data Management SystemsModern clinical trials generate vast data, making advanced management systems necessary.
Cloud-based platforms and AI-powered tools can efficiently process, analyze, and store data while maintaining security.
Implementing Data StandardizationStandardized data formats and collection methods across trial sites ensure consistency; standardization minimizes errors and facilitates smoother data integration during analysis.
Conducting Regular AuditsRoutine data audits help identify discrepancies and maintain data quality throughout the trial.
Employing third-party auditors can provide an objective evaluation.
Training Staff on Best PracticesEducating trial staff on proper data collection, entry, and handling protocols is critical to avoiding errors.
A well-trained team ensures all data meets regulatory standards and can withstand scrutiny.
Conducting clinical trials is undoubtedly complex, but the rewards are immeasurable for advancing healthcare and improving patient outcomes.
Sponsors can navigate these obstacles by addressing recruitment challenges, simplifying regulatory processes, and employing robust data management strategies.
Partnering with a clinical research organization offers additional support, providing the expertise and infrastructure necessary for trial success.
Through collaboration and innovation, the future of clinical trials looks promising, paving the way for groundbreaking medical discoveries.
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Incretin drugs have been so successful in clinical testing they're akin to a pharmaceutical Swiss Army knife. Already, medicines from Novo Nordisk and Eli Lilly approved to treat obesity and diabetes have proven beneficial for heart failure, sleep apnea and kidney disease.
A trial expected to produce results sometime this year could determine whether incretins might even play a role treating Alzheimer's disease.
Known as Evoke, the study has been testing an oral version of semaglutide, the active ingredient in Novo's Ozempic and Wegovy, in more than 1,800 people with early Alzheimer's over two years. Novo aims to show semaglutide can help slow the cognitive decline associated with the condition.
A company spokesperson told BioPharma Dive results should be available sometime in 2025. Analysts from the investment bank Jefferies have estimated the findings could disclosed as early as the first half, although a federal database lists the trial's primary completion as September.
If results are positive, semaglutide could force researchers to consider how targeting gut hormones affect the brain and, possibly, change Alzheimer's care.
The only treatments shown in testing to alter Alzheimer's course, Biogen's Leqembi and Eli Lilly's Kisunla, have only modest effects on cognition. They have so far been used sparingly because of side effects that include brain swelling.
As a daily pill patients could take at home, oral semaglutide would be more convenient than Leqembi and Kisunla, which require periodic trips to healthcare facilities for lengthy infusions. — Jonathan Gardner
Link to trial
MASH Clinical Trials
Clinical trials for MASH are research studies that evaluate potential new treatments for a form of liver disease called metabolic dysfunction-associated steatohepatitis (MASH). In a clinical trial, researchers test whether an experimental treatment, such as a new drug or medical device, provides any health benefit to people, such as preventing or treating a disease. Clinical trials for MASH are usually designed to find out whether a treatment improves features of the condition, such as inflammation and the accumulation of fat and scar tissue (known as fibrosis) in the liver.
Determining whether an experimental treatment causes side effects is another important purpose of clinical trials. The goal of some clinical trials is to find out whether a new therapy is safer and more effective than existing treatments for a given disease or condition.
A good match for a MASH clinical trial is a person who has a confirmed diagnosis of MASH and meets other criteria established by the researchers conducting the study. Each study is different, but some common criteria you may have to meet to be enrolled in a MASH clinical trial include:
Age. Studies often limit participation to people within a certain age range, such as 18 to 75.
Weight. You may need to be overweight or have obesity to participate in a MASH trial.
MASH status. Scientists conducting a clinical trial of a new MASH treatment often require participants to have liver disease that meets or exceeds a certain level of severity. That's determined by the amount of fat, inflammation, and fibrosis in your liver, among other factors.
You may not be a good choice for a MASH clinical trial if you have certain other qualities. For example, you may not be eligible if you:
Before you can participate in a MASH clinical trial, you will undergo a pre-screening process to determine whether you meet the specific criteria that the researchers require. A member of the research team will explain what's involved in the study and what to expect. For example, they will tell you about the treatment being investigated, what benefit it might offer, and what side effects it could cause. They will also tell you how and where you will receive the treatment, how long the study will last, and other important information.
Remember, before you enroll in a clinical trial, you will be told if there's a chance that you will receive a placebo instead of the treatment being studied. A placebo is a substance that has no medical benefit, but is designed to resemble the treatment that's being studied – they are sometimes called sugar pills. In a type of study called a placebo-controlled trial, researchers compare a group of patients given an experimental treatment with a group who receives sugar pills or other fake treatments. This type of study is considered one of the best ways to learn if an experimental therapy is safe and effective.
If you are accepted into a clinical trial, you will be asked to sign a form acknowledging that you understand what's involved, including the potential benefits and risks. Once the trial starts, you may need to travel to another city or stay overnight in a hospital to participate. The sponsors of the trial may reimburse you for participating, as well as pay for your travel and lodging costs. If for some reason you feel unable or unwilling to continue participating in a clinical trial, you can leave at any time.
Clinical trials for MASH have provided critical information about how to control this form of liver disease. Some recent key findings include:
Diet and exercise. A number of clinical trials have shown that changing your diet, losing weight, and getting more physical activity can improve MASH. For example, in a 2015 clinical trial, researchers studied people with MASH who adopted a low-fat diet that included plenty of fruit, vegetables, and whole grains. Participants also increased their exercise with a goal of 200 minutes per week. After one year, 25% no longer had fat or inflammation in their livers, nearly half showed significant improvements, and about 1 in 5 reduced fibrosis, or scar tissue, in their livers.
Vitamin E and pioglitazone. A government-sponsored study found that taking 800 international units of a natural (not synthetic) form of vitamin E called RRR-alpha-tocopherol daily for about two years improved MASH in 43% of patients treated, while the diabetes drug pioglitazone was less effective. Another trial found that this form of vitamin E may improve MASH in children.
Resmetirom. The MAESTRO-NASH trial provided evidence that encouraged the FDA to approve the sale of resmetirom (Rezdiffra), which is the first – and currently only -- drug created specifically to treat MASH. In the trial, about 30% of patients treated with resmetirom (100 milligrams daily) had reduced liver fat and inflammation, while their fibrosis stabilized, compared to less than 10% of the participants in the placebo group. Resmetirom lowered LDL ("bad") cholesterol, too.
Semaglutide. You have probably heard or read about the popular weight loss medications Ozempic and Wegovy. Both of these drugs contain a drug called semaglutide, which belongs to a class called GLP-1 receptor agonists that was originally developed to treat type 2 diabetes. A preliminary 2020 trial found that up to 59% of MASH patients treated with semaglutide no longer had excess fat or inflammation in their livers, and their fibrosis stabilized. More data from this research is expected soon.
Tirzepatide. This drug is already approved for controlling blood sugar in type 2 diabetes (Mounjaro) and weight loss (Zepbound). Tirzepatide is like an enhanced version of semaglutide and other GLP-1 receptor agonists, since it acts on other cell receptors, too. In a preliminary trial, 62% of patients treated with tirzepatide (15 milligrams once weekly) eliminated excess fat and inflammation from their livers, while 51% had improved fibrosis without their MASH getting any worse.
Survodutide. Like tirzepatide, this experimental drug is a GLP-1 inhibitor, but it has enhancements that cause it to act on other cell receptors, as well. In a preliminary study, survodutide improved MASH and stabilized fibrosis in up to 62% patients given the medication.
Pegozafermin. Your body makes a hormone called FGF21 that helps control how much fat you burn in your liver, among other roles. Pegozafermin acts like FGF21 and has shown promise in ongoing research for treating MASH. In one study, 27% of patients given the highest dose (44 milligrams) had improved fibrosis, while MASH went away in 26%.
If you're interested in enrolling in a clinical trial for MASH, ask your doctor if they can recommend one or help you find one. You can also search for a clinical trial using these tools:
ClinicalTrials.Gov. This online database of clinical trials is maintained by the National Library of Medicine. The website has an easy-to-use search engine that can help you find clinical trials of MASH.
NIH Clinical Center. The "Search the Studies" website allows you to look for clinical trials being conducted by the National Institutes of Health (NIH) Clinical Center in Bethesda, MD.
ResearchMatch. A nonprofit program funded by NIH that connects people interested in volunteering for clinical studies with researchers at medical centers in the United States.
If you find a clinical trial that looks promising, talk about it with your doctor. They can help you figure out if you're a good fit, as well as weigh the potential benefits and risks.
Talk with your doctor to learn if you're a good fit for a MASH clinical trial. (Photo Credit: E+/Getty Images)
Taking part in a clinical trial can involve some risk, since an experimental treatment may cause side effects. However, many safeguards are built into clinical trials to protect you, should you decide to enroll in one. For example, the U.S. Government has rules and requirements for how clinical trials are conducted. Researchers who want to perform a clinical trial must describe the design of the study in a plan called a clinical trial protocol. An oversight committee, known as an Institutional Review Board (IRB), must examine and approve the protocol before a study can begin. All of these measures are in place to protect your safety if you enroll in a clinical trial, as well as help ensure that the study's results are accurate and reliable.
Clinical trials are scientific studies in which researchers test potential new medical therapies, including treatments for MASH. The purpose of a clinical trial is to find out whether a new treatment is safe and effective. Several clinical trials of treatments for MASH are currently underway. Enrolling in a clinical trial could give you access to effective therapy for MASH before it's available to the general public.
What is the difference between MASH and NASH?
MASH and NASH are different names for the same form of liver disease. NASH stands for nonalcoholic steatohepatitis, which was the term doctors formerly used to describe a potentially serious form of liver disease that occurs when fat builds up in the organ. In 2023, an international panel of experts agreed to change the name of this condition to metabolic dysfunction-associated steatohepatitis, or MASH.
What drug is used for MASH?
Until recently, drugs used for MASH were prescribed "off label," meaning that they were approved for treating other conditions but not MASH. However, in 2024 the FDA approved resmetirom (Rezdiffra), the first drug specifically designed to treat MASH.
What is the new FDA approved medicine for MASH?
Resmetirom (Rezdiffra), the first drug specifically designed to treat MASH, was approved by the FDA in 2024.
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