Opportunity Within Clinical Trials
Examining how innovation within clinical trials can address the awareness, access and patient mistrust issues that limit the industry's true potential.
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Clinical trials are the gateway to unlocking insights and answers about the safety and effectiveness of novel drugs and other therapies.
For patients with chronic disease, participation in clinical trials can produce both immediate and long-term health benefits and lead to greater care plan adherence.
For healthcare organizations, clinical research studies and the development of novel therapeutics have the potential to drive some of the greatest shifts in healthcare cost structure and outcomes.
As we strive to open new doors to treatments and cures for disease through clinical trials, innovation across provider enablement, patient access, and study generalizability will serve as keys.
Within a clinical research study, patient participants receive specific therapeutic interventions according to a research protocol created by investigators. At the highest level, the goal of clinical studies is to augment the existing base of medical knowledge.
The following stakeholders drive the process of conducting clinical trials:
Study Participants - patient volunteers selected to participate in a respective clinical study.
Principal Investigator - often a medical doctor who leads the conduct of a clinical trial at a study site. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.
Sponsor - the entity that sponsors, or funds, a clinical study. Sponsors most commonly include pharmaceutical companies, academic medical centers, voluntary groups, or Federal organizations (e.g., VA).
Contract Research Organization (CRO) - a company that provides clinical trial management services. CROs are hired by sponsors to plan, coordinate, execute, and supervise the processes involved in the development of a clinical trial. CROs function as the central contact point between the sponsor and other trial actors (e.g. ethics committees, regulatory agencies, vendors, and hospitals).
Regulatory Authorities - the Food & Drug Administration (FDA) oversees clinical trials in the US to ensure they are designed, conducted, analyzed and reported according to federal law and good clinical practice (GCP) regulations.
IRB / IEC Ethics Committees - Organizations responsible for ensuring that the clinical trial in question is ethical and that participants’ rights and welfare are protected.
There are three phases to complete in the clinical trial process before a sponsor can submit their treatments to the FDA for consideration to be sold on the market, each containing its own purpose in ensuring that a treatment is safe and effective for use by the public.
After approval by the FDA and manufacturing of the drug on a large scale by the sponsor, the process enters Phase 4, which is also referred to as post approval surveillance. During this time, the main objective is to check the drug's performance in real life scenarios, to study the long-term risks and benefits of using the drug and to discover any rare side effects.
$195 billion is spent annually in biopharmaceutical R&D and $60 billion is spent annually in serviceable clinical trials.Due to well established clinical infrastructure and the preference of trial sponsors to conduct clinical trials in countries with a robust regulatory landscape, Europe and North America account for 80% of total spend.
Typically, 40% of a clinical research study’s total budget is allocated for recruitment, making it the biggest cost center for sponsors.In terms of studies by specialty, a recent JAMA analysis showed that oncology trials account for 21.8% of all trials and constitute the single largest clinical discipline, followed by mental health (9.0%), infectious disease (8.3%), diabetes (6.1%), and cardiology (5.7%).
Pain Points & Considerations
The need for efficient, fast-paced, and trustworthy clinical trials is expected to expand as demand for novel medications grows and as sponsors look to mitigate additional expenses from increasingly stringent study approval requirements.
However, before this need can be met, sponsors have to overcome long-standing pain points around enrollment and racial & ethnic inequities.
With the lack of patient awareness and access underlying these pain points, there is significant opportunity for innovation to help optimize the abysmally low participation and diversity rates observed across clinical trials today.
A staggering 86% of clinical trials are delayed and 66% close early because of poor recruitment. These delays affect not only study costs, but also subsequent sales, causing potential sponsor losses of $600,000 to as high as $8 million per day.
There are many aspects that drive deficient enrollment, including top-down issues such as lack of provider enablement or bottom-up issues such as lack of patient access.
Providers serve as gatekeepers to patient participation, yet both primary (i.e., clinical investigators) and referring physicians face strong headwinds to broadening enrollment.
As it relates to the former, less than 5% of physicians participate as clinical trial investigators. Many physicians cite the uncompensated costs of performing research and the lack of specialized operational support needed to actually run a trial in one’s practice as reasons for not opting in.
In terms of the latter, referring physicians have long suffered from having to navigate inefficient study discovery and patient matching tools - all on top of managing their normal physician workload. As a result, referring physicians often have little knowledge about available clinical trials and do not discuss this treatment option with patients.
In a recent medical study that examined the factors involved in patient decisions to enroll into cancer clinical trials (CCT), only ~10% of survivors reported that they were aware of the possibility of CCT participation at the time of their diagnosis and only a staggering 3% reported participation in a CCT.
And yet, of the 10% of patients that were made aware of CCT participation, 73% were made aware by either a primary, referral, or other physician. The study concluded that participation in a CCT was directly related to the level of physician involvement reported by the survivor.
In order to drive up the percentage of patients made aware of clinical trials by their provider (just 7.3% in the study above), operational inefficiencies need to be addressed first.
For clinical investigators, we’ve started to see wraparound services that provide staffing, operational support, regulatory training and tech to help boost the liquidity of independent clinicians and practices that engage in clinical research.
For referring physicians, provider networks and health systems need to be equipped with tech-enabled workflows that both streamline study discovery (in a much better way than navigating the archaic ClinicalTrials.gov) and incorporate contextually-relevant patient conditions for better matching. This can enable providers to more efficiently identify appropriate studies and properly educate patients on the pros and cons of participation, helping to increase the top-of-funnel pool and enrollment conversion in the process.
As it relates to patient access, potential participants are often stymied by the downward-propagating effects of geographical constraint, which lead to financial toxicity and insufficient study representability.
The traditional clinical trial model relies on a network of physical clinical research sites for trial execution, requiring patients to travel to a site for each visit. As such, only 8% of patients are approached to join a clinical trial because most do not live near a participating research site.
The fragmented nature of physical clinical research sites leads to prohibitive financial costs for potential participants. Specifically, the indirect out of pocket costs (e.g., travel, lodging) associated with participation have been shown to exacerbate barriers to accessing clinical trials for those who cannot afford them.
The prohibitive costs of participation ultimately play a huge part in the lack of broad inclusion among clinical participants. Ostracizing potential participants from rural areas or low income communities can severely compromise the ability to draw valid and generalizable conclusions regarding safety and efficacy.
In order to combat these systemic issues that result from the clinical trial model having been largely unchanged over the past 60 to 90 years, continued innovation from decentralized trial platforms will help to reduce the impact of geographic barriers and enable the recruitment of virtually any patient.
In an alternate approach, pharmacy retail giants such as Walgreens and CVS are expanding their reach into clinical trials by leveraging their vast network of retail locations to boost awareness of trials and to facilitate actual clinical trial visits. It’s a compelling strategy given that over 70% of Americans live within 5 miles of a Walgreens or CVS store and as the retail pharmacy giants look to broaden their push into consumer-centric, tech-enabled healthcare services.
As it relates to financial toxicity, novel financial reimbursement programs also ought to be considered to help drive greater patient access given that broad inclusion among clinical participants is essential for maximal value.
🚩 Racial & Ethnic Inequities
Similar to how excluding patients from rural areas or low income communities can negatively affect study generalizability, underrepresenting patients of color in studies has an even larger negative impact on the safety and efficacy of study conclusions and novel therapies.
Clinical trials have long suffered from inadequate patient diversity that is rooted in hereditary mistrust and lack of culturally competent research.
Fears of experimentation stem from historical government and health system atrocities (e.g., Tuskegee Experiment, VCU Organ Thieves), driving the mistrust of clinical trials amongst diverse populations.
For example, a recent survey of breast cancer patients conducted by the Metastatic Breast Cancer Alliance revealed that 71% of Black respondents said they trusted trials, compared to 91% of White respondents. One of the most salient results was that 60% of Black respondents were never offered a clinical trial or had a discussion about one with their providers.
Naturally, this systemic dynamic makes underrepresented populations distrustful of physicians and more likely to turn to online resources for information on the pros and cons of clinical trials participation or on a particular chronic condition.
While websites and social media could represent alternative channels to promote informed health care decision making to mitigate mistrust, the lack of diversity in online content about clinical trials and discussion around racial disparities is concerning.
A recent medical study on prostate cancer-related videos revealed that Black men, who are disproportionately affected by prostate cancer compared with non-Black men, are significantly underrepresented in YouTube videos from a search for ‘prostate cancer clinical trials’. Of the 128 videos with people examined, one video (<1%) exclusively featured a Black individual. Additionally, only two videos (1%) discussed racial/ethnic disparities in prostate cancer.
Moreover, a related medical study that examined prostate cancer-related content found that of 1,526 total people featured in the websites and videos analyzed, the perceived racial distribution was 9% Black, 8% Asian and 1% Latinx - versus 55% White (and 27% no consensus).
The conflation of negative patient perception and the lack of culturally competent information and research has contributed to the poor racial and ethnic diversity figures observed in clinical trials today.
In April of this year, the FDA issued draft guidance on how to develop a "Race and Ethnicity Diversity Plan" to ensure sponsors are enrolling representative numbers of participants from underrepresented racial and ethnic populations in clinical trials.
The draft guidance suggests that sponsors of drug trials should submit their diversity plans as soon as practicable, but no later than when a sponsor is seeking feedback for applicable pivotal trials (usually the End-of-Phase-2 meeting).
This motion comes at a time when major stakeholders are finally calling for more diversity and inclusion across the entire US healthcare system as the link between increased spending and health care disparities related to race, socioeconomic status, and sex/gender comes to light.
With more regulatory framework requiring participant diversity within clinical trials expected to come, there’s significant whitespace for human-centric companies and organizations to provide solutions that combine “tech + touch” to earn the trust of communities. For those interested in learning more, we’ve extensively detailed this approach in an earlier piece titled Hidden Heroes in Healthcare.
The uptick in clinical trials fundraising activity to start the year should come as no surprise despite being underfunded historically compared to sectors such as Mental Health, Telemedicine, Health IT and DTx.
Clinical trials represents a fascinating opportunity space - one that has the potential to drive significantly outsized value within healthcare, but that is still propped up by archaic infrastructure riddled with fragmentation, a lack of innovation and systemic racism.
We remain encouraged by the increasing number of tech-enabled companies taking on nascent aspects of the space and have strong conviction that human-centric innovation can significantly broaden study awareness, access and inclusion, ultimately helping drive participation closer to an act of social good.
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