Endocrine disorders and metabolic dysfunction impose enormous costs on populations worldwide – personal, societal, medical, and financial – and chronic conditions are multiplying rapidly. Obesity, in particular, has serious effects on mental health, causing grave concerns, especially for young people. In an alarming projection, more than half of adults and a third of children and adolescents worldwide could be overweight or obese by 2050.1 As these trends accelerate, the role of an endocrine & metabolic CRO becomes increasingly vital in driving targeted clinical research that supports innovation, therapeutic development, and improved patient outcomes in these high-burden areas.
Yet hope is on the horizon. For example, since the GLP-1 (glucagon-like peptide) receptor agonist, semaglutide was first approved for type 2 diabetes and subsequently for weight loss, we have seen a meteoric rise in the use of GLP-1 and related compounds with a high efficacy for weight loss and improving glucose-related parameters. For drug developers, the opportunity to bring innovative therapies to market is enormous; in fact, more than 100 anti-obesity drug candidates are in various stages of development. Along with opportunity, however, comes intense competition and risk.
By 2050, more than half of all adults worldwide will be either obese or overweight and a third of children will also be obese or overweight. It is a pivotal moment in endocrinology clinical research because the GLP-1 drug class, as well as other related compounds, affect multiple aspects of endocrinology and metabolism. We were at first testing these drugs in diabetes but then we realized that the same drugs that we were using for diabetes could also be useful for obesity patients. We also found that it could be useful in reducing liver fat so then it became useful for patients with MASH. And now we’re looking at whether the GLP-1 drugs could affect cravings that people have, so it’s becoming important in addiction research. It will be interesting to follow this space forward and see where it leads, which molecules actually succeed, which molecules may not do as well, and we are gaining insights. It’s very important for us to ensure that clients are developing these drugs in a way that could actually reach approval and ensure that patients are benefiting from these drugs.
Navigating GLP-1 Evolution and Market Differentiation in Endocrine & Metabolic Trials
Companies with established diabetes and obesity therapies must defend their market position from competitors by expanding their portfolios to treat different patient segments – possibly with precision medicine strategies – beyond obesity indications. On the other hand, companies seeking to enter the market need guidance about strategies for developing second-generation GLP-1s, combination therapies, and next-generation products for obesity and related cardiometabolic diseases, such as metabolic dysfunction-associated steatohepatitis (MASH).
Parexel is uniquely well positioned to support drug developers in these endeavors, with experience that is both broad and deep. Our endocrine & metabolic CRO team including board-certified adult and pediatric endocrinologists, and top-quality regulatory, market access, real world evidence, study design and biostatistics experts can fully support pharma clients with precisely defined market positioning, optimal trial designs and regulatory strategies. From small studies in rare metabolic diseases to large studies in obesity, osteoporosis and type 2 diabetes, our dedicated project teams identify the right sites through our strong global site networks and provide customized patient recruitment and retention strategies aimed at engaging the study participants and minimizing their study burden. We manage clinical trial delivery through partnership with our key vendors, as well as providing eCOA and sensor solutions, including continuous glucose monitoring and glucose-related data capture for diabetes clinical trials.
Obesity and Type 2 Diabetes Mellitus
From our experience in adult obesity and type 2 diabetes trials, we have found that there remains a high level of interest in obesity trials even in the U.S. where there are many competing trials. While this results in rapid patient enrollment, it is essential to ensure that the right patients are being screened who meet eligibility criteria and are motivated and willing to remain in the trial. Proactive discussions with sites throughout the enrollment period to evaluate and adjust site-specific strategies are key to recruiting the target patient population and accelerating processes. Our project teams have utilized dedicated patient retention strategies for obesity trials, addressing the key risks of discontinuation due to gastrointestinal side effects or to perceived lack of efficacy for patients who might be on placebo.
These strategies have resulted in a high rate of success in meeting trial milestones ahead of schedule. For example, in a global Phase II trial for adults with T2DM and adults with obesity, Parexel achieved rapid enrollment, with the T2DM arm completed in 3 months (5 weeks ahead of projected timeline). This seminal trial set a precedent for methodology, design, and operational delivery in obesity research.
Experience in the past 5 years
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Phase I to IV obesity and obesity-related clinical trials
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patients enrolled
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unique sites
Metabolic Dysfunction-Associated Steatohepatitis (MASH)
In our extensive work in developing therapies in this area, Parexel has established best practices for patient enrollment and retention, site engagement, and minimizing lost-to-follow-up numbers. We have an established network of sites and investigators covering in the U.S., Europe, Asia, and Australia.
An example of a recent project is a Phase Ib trial to assess safety and tolerability of patients with obesity and hepatic steatosis. The complex study design included multiple dose groups and cohorts with mandatory 72-hour gaps between treatments, complicated by a demanding three-month schedule. Our team implemented innovative strategies and protocol amendments, along with a tool allowing a more flexible window for each study visit, which significantly improved study schedule compliance. We achieved the last patient/first visit milestone 76 days ahead of the contracted date.
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Frequently asked questions
Endocrine and metabolic disorders are a group of conditions that affect the body's endocrine system and metabolic processes. The endocrine system consists of glands that produce and secrete hormones, which are chemical messengers that regulate various bodily functions. These hormones control processes such as growth, development, metabolism, reproduction, and mood. When the endocrine system malfunctions, it can lead to disorders such as diabetes, thyroid disorders, growth hormone deficiencies, and adrenal gland disorders. These conditions can result from glands producing too much or too little of certain hormones, or the body's inability to respond properly to hormones.
Metabolic disorders, on the other hand, involve disruptions in the body's ability to break down, process, or utilize nutrients from food. These disorders can affect the metabolism of carbohydrates, proteins, or fats, leading to a range of health issues. Examples of metabolic disorders include phenylketonuria (PKU), which affects protein metabolism, and various lipid disorders that impact fat metabolism. Some endocrine disorders, such as diabetes, are also considered metabolic disorders because they affect the body's ability to process glucose. Both endocrine and metabolic disorders can have significant impacts on overall health and often require ongoing medical management to maintain proper bodily functions and prevent complications.
Contract Research Organizations (CROs) specializing in endocrine and metabolic disorders typically offer a wide range of services to support pharmaceutical and biotechnology companies in their drug development efforts. These services often include protocol development, patient recruitment, and clinical trial management for various endocrine and metabolic conditions such as diabetes, thyroid disorders, obesity, and rare metabolic diseases. CROs may also provide specialized laboratory services for hormone assays, metabolic profiling, and biomarker analysis. Additionally, they often offer regulatory affairs support, helping sponsors navigate the complex regulatory landscape for endocrine and metabolic therapies, including interactions with regulatory bodies like the FDA and EMA.
Another key area of CRO services in this field is data management and biostatistics, which are crucial for analyzing the complex datasets generated in endocrine and metabolic clinical trials. CROs may also provide medical writing services for regulatory submissions, clinical study reports, and publications. Many CROs have expertise in designing and conducting long-term safety studies, which are particularly important for chronic endocrine and metabolic conditions. Furthermore, some CROs offer specialized services such as pharmacokinetic/pharmacodynamic (PK/PD) modeling for hormonal therapies, real-world evidence generation, and health economics and outcomes research (HEOR) specific to endocrine and metabolic disorders.
Endocrine and metabolic Contract Research Organizations (CROs) typically focus on several key areas that are critical to advancing research and treatment in these fields. One primary area of focus is diabetes, encompassing both Type 1 and Type 2 diabetes, as well as gestational diabetes. This includes research into new insulin formulations, novel glucose-lowering agents, and technologies for diabetes management such as continuous glucose monitors and artificial pancreas systems. Another major focus is on obesity and related metabolic disorders, including research into weight loss medications, metabolic surgery outcomes, and treatments for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Thyroid disorders, including hypothyroidism, hyperthyroidism, and thyroid cancer, also represent a significant area of research for these CROs.
Additionally, endocrine and metabolic CROs often concentrate on rare diseases and genetic disorders affecting metabolism. This includes research into conditions such as lysosomal storage disorders, mitochondrial diseases, and inborn errors of metabolism. Hormonal disorders affecting growth and development, such as growth hormone deficiency and Turner syndrome, are also key areas of focus. Many CROs are increasingly involved in research related to the endocrine aspects of reproductive health, including polycystic ovary syndrome (PCOS) and male hypogonadism. Furthermore, there is growing interest in the metabolic components of other diseases, such as the relationship between diabetes and Alzheimer's disease, or the metabolic effects of various cancer treatments. As personalized medicine advances, CROs are also focusing on biomarker discovery and validation for endocrine and metabolic conditions to enable more targeted therapies.