Introduction
Companies take on significant risk to bring life-saving medicines to market. Consider these statistics:
- The rate of success for developing a new therapy is just 12%.
- The cost of developing a new drug has escalated from $802 million in 2003 to $2.6 billion, with ROI dwindling to about 3%.
Regardless of the promise of a phase II trial, it is ultimately the read-outs from the phase III trial that determine whether a therapy earns FDA approval and finds its place in the market. These phase III trials are conducted across multiple sites, with several hundred to several thousand participants. The complexity of this evidence-generating step cannot be understated, and every action shapes the financial and operational strategies behind trial success.
This article expands on a previous article on why many phase III trials operate at a loss and champions a shift toward innovative business models — models that integrate technologies for next-generation clinical trials, promising higher rates of success.
New Models
Incorporating clinical research as a therapeutic option
Today, just over 2 million patients participate in clinical trials; and while this may seem like more than enough, an estimated 58 million participants are actually needed. Low trial participation is a public health issue. Helping healthcare systems bring innovative therapies to their patients will be a potentially transformative business model. This would require technologies and infrastructure that enable specialty and primary care practices to enter the research ecosystem.
The Clint platform is one such technology that supports healthcare systems that are committed to clinical trial access for their patient populations to identify all patients within their electronic health records (EHR) system that are eligible (today) for actively recruiting phase III trials. This effectively opens up clinical trials as a treatment option.
This model can (and should) be aligned with unmet medical needs in the patient population of that healthcare system. By including clinical trials as a care option, healthcare systems may stand to reduce their cost of care, while improving their patients’ engagement in their own healthcare management - and ultimately, improving health outcomes. This applies to both large patient populations, as well as any therapeutic or disease category that fits the unmet medical needs for smaller populations (e.g., for rare diseases, chronic conditions, or rural populations).
Enabling academic medical centers to compete with smaller, private research entities
Relatively recent changes within the healthcare market have forced academic medical centers (AMCs) to manage their clinical research portfolio as if it were a self-sustaining business. This requires these institutions to identify the true cost of research and proactively plan for and manage it independently. Technologies, such as the Clint platform, integrate directly with EHR systems and provide the necessary insights for AMCs to make strategic and financial decisions that not only meet their bottom line but improve cash flow to sustain and scale their clinical research infrastructure. Stanford Health Care is among a handful of AMCs, including Duke and Vanderbilt, that are leading the charge in terms of process standardization for clinical research. Clint aims to help them get the word out about how cost-effective and impactful clinical research can be, so that other AMCs can also future-proof their clinical trials infrastructure and compete with smaller, private, often specialized research entities.
Hybrid fee-for-performance and fee-for-service models
While it is common for sites to operate on a fee-per-performance basis, they also have fixed operational costs — leaving many sites with no choice but to operate in the red. Sites and sponsors should restructure their contracts in a way that makes clinical trials more viable. For example, during contract negotiations, research sites include the Clint platform as a single, pass-through expense to trial sponsors. This yields a substantial return on investment — 92% — with one site saving $48,000 on a single trial.
Expanding the value proposition through collaborative site networks
Collaborative site networks expand the capabilities of single sites into a unified service ecosystem. This is an interesting opportunity in clinical development — such networks offer more than the sum of their parts since they are able to establish standardized processes and integrate population-level data and analytics into shared decision-making, thereby improving the overall efficiency and effectiveness of clinical research. Site networks can support development as early as phase II, by providing the real-world data (RWD) needed to design endpoints and protocols going into phase III. Each site can run feasibility assessments on the availability of eligible participants and coordinate with other sites in the network to meet recruitment goals with a new degree of specificity. Collectively, high-performing research site networks may prove to be a more reliable model for sponsors who, together with healthcare systems, are looking to improve enrollment and bring new therapies to-market sooner.
Sponsors are also interested in getting closer to patients and working with more high-performing and patient-centric sites. The Clint platform includes a clinician-facing dashboard which expands trial awareness and access to more clinicians and patients — effectively bringing trials directly to qualified patients. This includes expanded reach across participating research sites, as well as the opportunity to reach private practices and other referral sources.
For example, the Clint platform identified 939 participants within a single healthcare system that met the full inclusion criteria for an active cardiovascular trial. This is a significant portion of the approximately 16,000 participants needed for the study. By leveraging relationships and resources across site networks through the scaled application of the Clint platform, it is possible to expand the pool of potential participants. This makes clinical research more accessible and aligns the interests of sponsors, healthcare systems, research sites, and patients, ultimately leading to a more efficient and patient-centered research ecosystem.
The network effect
For Sites
- Capitalize on the networks’ relationships with sponsors to bring-in the right trials, aligned with financial and strategic objectives, including trials that meet the needs of their patient population
- Jointly conduct feasibility assessments to determine whether there are an adequate number of eligible patients across the site network — thus positioning sites favorably for the award of trials
- Data-driven negotiations and contracting set the stage for more pragmatic performance metrics, resulting in more successful performance and repeat award of trials
For Sponsors
- Informed clinical trial design, including protocol development and endpoint selection based on RWD; added capability of running simulations of different cohorts and endpoints in the data to optimize the trial design
- Site selection based on the availability of eligible participants in the healthcare system(s) of each site
- Expanded reach to all potential referral sites in the healthcare system(s)
Plan for market access when designing the clinical program
Life sciences companies can design clinical programs that incorporate market access plans from the outset. This involves making use of RWD to understand the market, pinpoint unmet medical need, and ultimately develop pricing and distribution models based on the potential reach of a new therapy. This necessitates an end-to-end model to bridge the gap from clinical development to product launch with a focus on value communication. The Clint platform provides the necessary insights each step of the way, and includes a point-of-care feature that empowers clinicians to receive timely recommendations for newly available products, ensuring that patients who stand to benefit from those treatments are not overlooked.
Industry collaboration
We advocate for partnerships between pharmaceutical companies, technology providers, healthcare systems, individual research sites and research networks to integrate innovation solutions and collaborative models. By fostering partnerships, we can share risks, reduce costs, and enhance trial outcomes, benefiting the entire healthcare ecosystem, and improving access to life-saving therapies. It will take a cultural shift as well as new business models to accomplish quality research. This ethos, coupled with strategic innovation, will not only streamline clinical trials but also change the way drug development works.
Authored by
Cassandra Broadwin, MPH, Jasmine LaCoursiere, MS, Malcolm Bohm, Rajesh Dash, MD
