This article was written by PharmaPhorum.
Clinical trials are designed to evaluate the safety and efficacy of new therapies or medical devices. During these trials, evidence is gathered to determine if the asset is safe for patients, providing the intended benefit at the appropriate dosage, as well as identifying and minimizing any potential side effects.
In recent years, advancements in statistical techniques, as well as more extensive use of advanced technologies, have helped garner more accurate results across clinical trials. However, these tools have also added some complexity to the process, requiring the need for more substantive pre-trial planning.
Therefore, prior to conducting a clinical trial, life sciences companies need to have a clear direction for the asset they are testing, and a deep understanding of the market size, dynamics, and clinical economics for the health condition they aim to treat. This means having clear answers to the following questions:
- What is the total number of patients with the health condition?
- Who is diagnosing and who is treating these patients?
- What is the diagnosis rate – and at what stage of care are patients being diagnosed?
- Are prevalence and diagnosis rates increasing or decreasing?
- Are certain societal groups or geographical regions more likely to be undiagnosed?
- Who are the main competitors in the space – and how have existing treatments been adopted within the healthcare systems?
- Which payers are covering existing treatments?
- What challenges are there for reimbursement / rejection by payers?
These insights not only inform trial design, but also help predict how the asset may shift existing market dynamics. A targeted market access analysis is essential to support rapid adoption.
This is where PurpleLab Managed Services come in. This solution empowers clinical development teams to reduce risks and optimize trial execution by leveraging comprehensive real-world data. To find out more, pharmaphorum spoke with Jason Gagner, MBA, MS, VP of product management at PurpleLab.
Laying the cornerstone of clinical trial development
Clinical development teams must anticipate and address any hurdles that are likely to arise throughout the lifecycle of their asset. This means closely monitoring trends and shifts across markets and competitive landscapes, from pre-phase 1 trials to post-launch and beyond.
“An approach that we have seen many clinical teams have success with is when they take the time to not only formulate an end-to-end action plan, but establishing KPIs early and following them throughout the project,” said Gagner.
“The Managed Services team at PurpleLab, supported by the robust data PurpleLab has brought together, allows for seamless transitions between trial phases and project milestones as the supporting data builds upon itself to better inform each step forward,” he continued. “We are long past the days of needing to start every phase of a clinical trial or a new product launch from scratch.”
PurpleLab is helping to shape the marketplace for future growth and challenges with their Managed Services solution, which offers answers to the questions of “Who? Where? Why?”
“Our HEOR team routinely employs advanced analytical and AI/ML techniques to identify key drivers of disease risk, treatment outcomes and, most importantly, healthcare resource utilization (HCRU) and expenditures,” Gagner explained. “This information, coupled with knowledge of patient-level variation in clinical, demographic, and social factors, can inform patient selection and recruitment, reduce the likelihood of unforeseen adverse events – culminating in a maximization of positive outcomes and trial success.”
The path to payers, and to patients
“New product launches are always an exciting time and also a stressful time for life science companies,” noted Gagner. “Companies have invested significantly to get to this point. At PurpleLab, we not only look at what the potential market is, but we also examine who early adopters of new medications are and prospectively monitor adoption, barriers to uptake, and market indicators that convey how a launch is trending in as close to real-time as possible.”
To do so, it is necessary to ask:
- Whose claims are being rejected and why?
- Whose claims are being approved?
- Who is receiving a prescription, but not filling them?
- Are there certain demographic trends? For example, is it an insurance issue, a healthcare system issue, or a patient-awareness issue?
PurpleLab’s Managed Services investigates these areas in order to anticipate challenges that may arise across different geographical regions or healthcare systems. This helps companies adapt their development and marketing strategies before they impact broader adoption.
“Even before you start your phase 1, we can monitor the clinical space and start identifying the biggest market concerns that might impede your new therapy,” explained Gagner. “That way, if there’s a major shift in the market, you can adjust as you go, instead of getting blindsided during a Phase III study. It becomes a risk mitigator.”
Real-world data, real-world results
Managed Services like PurpleLab’s have demonstrated tangible results. For example, in a haematologic disease trial, PurpleLab identified 15 high-performing sites that achieved 80% enrollment success, thereby reducing start-up time by several weeks. In another instance, the team conducted an analysis that focused on the utilization of PET scans across a variety of diagnoses, which revealed that PET scan utilization increased year-over-year, while the percentage of patients receiving PET scans went down within each diagnosis-specific sub-group.
“As a result of this work, PurpleLab was able to identify healthcare organizations (HCOs) that were more likely to refer patients out-of-system to receive PET scans, leading to substantial losses in revenue for those “leaking” HCOs,” explained Gagner. “The findings also indicated that the amount of leakage incurred by certain health systems would have exceeded the cost of a new PET scanner in less than 36 months.”
Risk mitigation and reimbursement
Regulatory bodies, especially the Centers for Medicare & Medicaid Services (CMS), play a major role in drug classification and reimbursement. CMS leverages certain standards (e.g., the USP Medicare Model Guidelines and the USP Drug Classification) as part of their process to determine outpatient formulary adequacy for certain plans.
PurpleLab actively monitors federal policy changes and provides public comments on Requests for Information (RFIs) and Requests for Comment (RFC) from agencies like the FDA.
“The FDA recently put out an RFC with a focus on leveraging HL7 Fast Health Interoperability Resources (FHIR) for RWD submissions – this could be a game-changer in terms of bridging the gap between data collected in clinical practice and data submitted as part of a study,” said Gagner.
“When you combine drug classification with other information, such as demand utilization, disease trends, and drug shortages, you get a very interesting picture that can inform opportunities for therapy development,” he continued. “Overall, the PurpleLab team has a unique understanding of the regulatory and standards landscape that can help our customers leverage current opportunities, and plan for future changes.”
“They’ve reclassified things at a higher level,” Gagner explained. “There’s specificity that’s lost in some medications and it’s drastically changing the reimbursement for those medications. We have a clinical team that’s able to go back through and take the way your treatment is supposed to be classified and provide the evidence back to CMS and help get that treatment classified properly.”
“This is so that you’re getting reimbursed the way that it should be or, I should say, this way that the healthcare systems are being reimbursed appropriately, so the healthcare systems are not losing money off of a misclassification for reimbursement,” he continued.
To this end, PurpleLab Managed Services has classified and standardized its terminologies. Reference data and vocabulary standards are the cornerstone of the company’s approach to defining patient populations (e.g., NDC codes for patients on a GLP-1, or ICD10CM codes for patients who have a diagnosis of cystic fibrosis) for analytics.
“We’ve mapped them across multiple different terminologies so that we can compare apples to apples and be consistent in our communication and in our evaluations,” expanded Gagner. “Using one terminology or one set of coding, you might get a certain procedure, but then be able to cross-reference that across all the others, so that we can be inclusive in how we look for things, how we describe them and are able to provide those views back to either the life sciences companies or to the healthcare systems on how things are being coded for reimbursement, because different codes have different reimbursement rates and sometimes the wrong code or a generalized code is used, opposed to the specific code.”
“PurpleLab’s HealthNexus platform solves for these industry-wide pain points – vocabulary acquisition and maintenance, and creation and maintenance of concept groups (aka value sets) within our platform,” he explained. “In the absence of this solution, life sciences and other customers are left trying to web scrape vocabularies and create / maintain value sets in excel spreadsheets, without any distributed authoring or data governance capabilities. For customers that don’t have the in-house resources to build and maintain these concept groups, PurpleLab’s Managed Services stands ready to provide clinical informatics expertise, tailored to meet clinical and commercial objectives.”
In this way, when it comes to clinical trial development, PurpleLab Managed Services focuses on supporting clients who are interested in managing project costs, timelines, and milestones, providing comprehensive support and insight generation that guides pre- and post-marketing strategic decision-making.
Reach out to learn more.