Beyond the Brink: Rescuing Clinical Trials for a Healthier Tomorrow
Clinical trials, the essential process by which new medical treatments are tested and brought to patients, are currently facing a profound crisis. Industry leaders are sounding the alarm, stating that the existing models are simply not sustainable and that without urgent, transformative change, the entire clinical trial system as we know it cannot continue. This critical juncture is characterized by several pressing issues: soaring costs, a shrinking and overworked workforce, difficulties in finding enough patients, and significant barriers to access for many who could benefit.
The Increasing Demand for Clinical Trials Collides with Limited Resources
One of the most significant challenges is the sheer volume of clinical trials now being conducted. While an increase in medical innovation might seem positive on the surface, the rapid growth in the number of potential new treatments, particularly in areas like oncology (cancer treatment), is creating an unsustainable demand. For instance, the number of new cancer treatments in development grew by an average of 13% annually between 2018 and 2022. This explosive growth is projected to more than triple the demand for patients to enroll in clinical trials by the year 2032. The current system is simply not equipped to handle this immense increase in demand, leading to concerns about a potential slowdown in the development of new drugs. To cope, we would need far more research sites to run trials, each site would need to handle more trials, and every site would have to become much better at enrolling patients.
A Dwindling and Overburdened Workforce
Compounding the issue of rising demand is a severe shortage of skilled professionals to conduct these trials. The people who manage and carry out clinical research are experiencing significant strain, leading to burnout and an exodus from the field. Data shows that over 80% of research sites in the United States have struggled with staffing shortages in cancer clinical research. This is largely due to overwhelming job expectations, inadequate pay, and limited opportunities for career advancement.
The numbers illustrate this crisis starkly: the global number of clinical trial investigators – the doctors and researchers who lead the studies – dropped by nearly 10%, from approximately 128,303 in 2017-18 to 116,948 in 2023-24. Even more concerning is the steep decline in trial site coordinators, who handle much of the day-to-day work, dropping from about 56,036 to 40,472 in the same period. Many healthcare and research workers left the field after the COVID-19 pandemic, creating a significant "exodus". This shortage means that it takes much longer to start new clinical studies because sponsors struggle to find investigators who have the time and capacity to take on new projects. If nothing is done to reduce the burden and inefficiencies currently affecting this workforce, this negative trend is expected to continue.
Significant Barriers to Patient Recruitment and Access
Even when there are enough staff, finding enough patients to participate in clinical trials is a persistent problem. The leading reason why clinical trials are stopped is not due to safety concerns or a lack of effectiveness, but because not enough patients enrolled. Industry data reveals that a staggering 60% to 70% of trial sites fail to enroll their initial target number of patients. This means that the money spent to set up and manage these non-enrolling sites, including contracts, training, and overhead, is essentially wasted.
Despite the growing need for participants, only about 5% to 8% of potentially eligible patients ever take part in a clinical trial, a figure that has remained stagnant for years. This low participation rate makes it impossible to meet the increasing demand for patients.
The problem is exacerbated by geographical inequalities in access. Most clinical trials, especially in the US, are conducted by large academic medical centers, which are typically located in major cities. In contrast, community hospitals and clinics, where the majority of patients receive their care, often lack the resources to participate in trials on a large scale. This creates a "stark picture" where vast areas of the country do not recruit for clinical trials at all, effectively shutting out patients living in more rural communities. For example, people in agricultural counties are likely to travel over 60 miles to participate in a clinical trial, compared to those in urban areas. This situation risks not only saturating or exhausting patient pools in states with smaller populations but also completely excluding rural communities from accessing potentially life-saving treatments.
Adding to these challenges, as treatments become more personalized (targeting specific genetic markers or small groups of patients), the pool of eligible patients shrinks even further. Research staff then have to spend an enormous amount of time sifting through medical records to find patients who meet the incredibly specific criteria for a trial. In common cancers like lung and breast cancer, numerous competing trials vie for the same limited pool of eligible patients. This often results in each trial enrolling too few participants, extending recruitment times, or even leading to trials being unable to meet their enrollment targets at all. While cancer research is at the forefront of this issue, other areas, such as chronic conditions like Alzheimer's disease and rare diseases, also face significant recruitment hurdles, often because patients are treated in rural settings unprepared for trials or are scattered globally. Relying on major academic centers and expecting rural patients to travel or disrupt their lives for a trial is simply not yielding sufficient results.
Furthermore, regulatory bodies like the US FDA require that trials submitted for drug approval include patients representative of the US population. While trials can be conducted globally to increase patient pools, the proportion of US patients in applications to the FDA has been declining. The FDA may accept data from outside the US, but the enrolled population must be relevant to the US population and medical practice. This was highlighted when an FDA advisory committee voted against approving a drug due to concerns that the non-US data was not applicable to the US population. This emphasizes the critical need to increase US participation in major trials to ensure novel treatments can actually reach patients.
The Escalating Cost of Clinical Trials
Clinical trials have always been expensive, but their costs are now reaching unprecedented heights. According to a November 2024 GlobalData report, the cost per trial has been steadily increasing since 2014, with single-country trials seeing a 2.9% annual rise and multinational studies experiencing a 4.9% annual increase over the past decade.
Oncology trials are particularly exorbitant, averaging around $30 million for a Phase 1 trial and nearly $60 million for a Phase 3 trial, with the largest trials exceeding $100 million in direct costs. These high and rising costs are a direct consequence of several factors:
Increased Complexity: Trials now involve more intricate treatment regimens, more adaptable designs, more extensive data collection, and frequent changes to protocols.
Declining Productivity: Fewer patients are being enrolled per site each month, leading to longer recruitment periods.
For the companies funding these trials (sponsors), longer trial timelines translate directly into higher expenses. Every single day a trial remains open without yielding results costs sponsors an additional $40,000 for site maintenance, monitoring, and administration.
Beyond these direct costs, delays in trial timelines also push back the launch of new therapies, resulting in substantial lost revenue for sponsors. For each day a drug launch is delayed, sponsors forfeit an average of $500,000 in lost revenue, a figure that can skyrocket to over $3 million per day for a blockbuster drug with annual sales exceeding $1 billion. This lost revenue is particularly painful now, as the periods during which a company has exclusive rights to sell a drug are shortening, especially for small molecule drugs in the US due to changes from the 2022 Inflation Reduction Act.
When factoring in these rising direct costs and the massive amounts of lost revenue due to delayed approvals, the total cost of bringing a new drug to market now stands at a staggering $2.3 billion. As Kent Thoelke, CEO of Paradigm Health, argues, "That is simply unsustainable". With mounting pressures on drug pricing in major markets, if trials continue to operate in this costly manner, fewer drugs will be economically feasible to develop. The financial return on investment is shrinking, potentially forcing sponsors to abandon promising therapies because the development costs are simply too high.
The Imperative for a Transformed Clinical Trial Model
The clear unsustainability of the current system, coupled with outdated patient enrollment methods, is driving an urgent search for transformative new models. Thoelke and other industry leaders believe that the solution lies in a fundamental re-engineering of the clinical research model.
The COVID-19 pandemic, though a global crisis, inadvertently provided a glimpse into this potential future. Out of sheer necessity, many rigid trial protocols were relaxed: patients could give consent electronically, provide samples at local labs, or participate from their homes via telemedicine. What the industry had resisted for years, a global crisis achieved in mere months: a significant shift toward expanding patient access to clinical trials. This flexibility allowed patients on life-saving therapies to continue participating in trials during the pandemic. However, Thoelke notes with concern that "most of those systems rolled back, we've gone back to the status quo".
To address these deep-seated inefficiencies, especially in identifying and recruiting eligible patients and reducing the data collection burden on research staff, new approaches are vital. Thoelke explains that "The process for running clinical trials has not dramatically improved in three decades". There is a critical need to re-engineer the research model using new technology and infrastructure that enables community and rural healthcare systems to participate in clinical trials at a much greater scale.
Companies like Paradigm Health are developing solutions, such as their AI-driven platform. This technology can interpret a patient's entire medical chart, accurately match the patient to suitable trials, and collect the necessary data with significantly less manual effort required from research staff. This platform can quickly process "massive volumes of records with high levels of precision".
This is not solely about technology; it is also fundamentally about increasing accessibility and promoting equity in healthcare. Without these advancements, many patients, particularly those outside major urban centers, cannot access cutting-edge trials, even when these trials represent their best or only treatment option. Paradigm Health has demonstrated that with the right support, community healthcare providers are fully capable of running complex oncology trials, thereby offering rural patients the same opportunities as those receiving care at large academic medical centers in cities.
Ultimately, to effectively serve both scientific progress and the patients who rely on it, the clinical trial industry must wholeheartedly embrace this transformation. If inefficiency continues to dominate, the promise of new medical advancements may simply remain out of reach.
Contract Research Organizations (CROs) specializing in Alzheimer's disease research:
Veristat: A Canadian CRO with expertise in neuroscience and a focus on developing therapies for CNS and neurological diseases, including Alzheimer's.
Ethica: Another full-service neurology CRO, based in Canada, with experience in Alzheimer's clinical trials.
TFS HealthScience: A global mid-size CRO with a significant presence in neuroscience, including Alzheimer's disease research, across multiple countries.
