Value Based Clinical Trials

Promoting Value-Based Clinical Trials in Medical Affairs

The pharma industry, along with healthcare more generally, is pursuing value-based healthcare (VBHC). This is promising news for all stakeholders: Payers invest in treatments that provide better patient care; healthcare providers are no longer motivated by fees for service; and patients are at the centre of the clinical experience.


But VBHC requires careful coordination among stakeholders. Medical affairs teams are the leaders of these stakeholder networks, sharing standardized data to support trial designs and decisions.

What It Means to Take a Value-Based Approach

Value-based healthcare puts patient outcomes at the heart of the treatment. It’s best viewed as a four-prong approach, explain Kenny O’Neill and Sheryl Coughlin, Ph.D. at EY. The goals are to provide better patient care, better health for populations, reduce costs and help HCPs avoid burnout.

VBHC differs from fee-for-service (FFS) models in that the former is less about the number of patient visits and more about the quality of treatment they receive. O’Neill and Coughlin say that the patient experience is seldom prioritized in the FFS model and healthcare providers can suffer from stress of trying to treat patients and generate revenue through increased visits and patients through doors.

The result of the FFS model is tense relationships between payers and providers. Value-based healthcare changes this by putting patients first while providing treatment. The approach helps payers cut costs and doctors stay focused and safe from overwork.

It also gives patients a greater sense of value and fair play. Value-based healthcare, “by establishing value-generating networks among different healthcare stakeholders, can serve as the long sought-after redistributive mechanism that compensate patients for the industrial exploitation of their personal medical records,” write Federico Esposti and Giuseppe Banfi at BMC Health Services Research. VBHC success requires “data-permeable networks” to ensure procurement value by linking all stakeholders within the network.

Pharma’s Commercial Models Are Changing

Tied to the move towards VBHC, medical affairs teams need to be communicating the complexities of new therapies in accessible ways to healthcare professionals, payers and patients to drive this collaborative ecosystem, writes Jennifer Turcotte at Salesforce.

Optimization of data is integral to the success of pharma. Turcotte says that data should be unified for providers, patients and partners on a common platform. Doing this will grant access to all stakeholders as to why decisions are made.

Benefits include working collaboratively towards a patient-centric strategy with marketing, sales, and MA teams all working in unison. Data also enables MA teams to help commercial teams formulate better targeting strategies and personalized customer experiences.


Payers’ Attitudes to Value-Based Therapies

Value or outcomes-based pricing models may also be good for pharma’s reputation. For a start, it’s making pricing more transparent, writes Jeff Lagasse, associate editor at Healthcare Finance. This is particularly so for the orphan and cancer drug markets.

John Kerins, managing director at healthcare investment bank Cain Brothers, tells Lagasse that pharma is increasingly approaching payers to set up a value-based payment arrangement for new products. He says the message from pharma is: “If it doesn’t work, we’ll give you your money back.”

Linking payment to outcomes — showing the drug can treat patients the way the drug label says it can — is a positive move from pharma. Still, more needs to be done, writes Nitin Jain, managing principal at global sales and marketing consulting firm ZS Associates. Value-based partnerships (VBP) should be a priority for pharma because consumers expect what they buy to work and it can help companies deliver results to patients. Jain says pharma is capable of doing better than it currently does, and striving for VBP will help spur innovation.

He points to examples where pharma companies with value-based partnerships can make a difference. These include the one-half of patients with diabetes taking insulin who “don’t have their diabetes under control” and the “36 percent of deaths due to opioid overdose in 2017 were caused by prescription opioids,” he writes. These realities are opportunities for pharma “to create beyond-the-pill offerings” in which treatment is based on outcomes, Jain adds.

Value-Based Care and Real-World Evidence

Clinical researchers have long predicted that VBHC would become a reality. Tied to this is the use of real-world evidence, writes Alethea Wieland, founder and president at Clinical Research Strategies. RWE is needed to inform value-based payment decision-making. The cost of healthcare has been rising steeply yet efficiencies have not reflected this pricing. But now regulation supports the prioritization of RWE.

A 2019 Bipartisan Policy Center Report, “Expanding the Use of Real-World Evidence in Regulatory and Value-Based Payment Decision-Making for Drugs and Biologics,” recommends: “clearing barriers to the access and use of real-world data to provide an evidence base for regulatory evaluation and value-based payment programs, expanding opportunities to use new data sources and approaches, and advancing new models of collaboration among payers, manufacturers, regulators, clinicians, and – most importantly – patients.”

Data Drives Decision-Making

Artificial intelligence can help pharma design better trials. Researchers can use this technology to mine data to improve patient safety, trial population selection and speed up drug development, writes Matthew Clark, Ph.D., director of Scientific Services for R&D Solutions at Elsevier.

Part of the value of AI is its ability to collate and analyze data from clinical trials with RWE to predict how the effects of a drug could differ in the two settings. MA teams would be able to use this data as evidence for some of pharma’s decisions and safeguarding pharmacovigilance.

RWE from medical records and also from trial patients relying on wearables and remote-monitoring tech will increase the amount of data available to researchers, explain Tanisha Carino and Mark B. McClellan at Health Affairs. The data could help to facilitate the move towards VBHC.

Patient-generated data outside of trial settings will help researchers understand patients’ lived experience. This will inform how regulatory decisions are made but also how sponsors and trial managers design trials and deliver information to patients, providers and payers about how drugs and therapies should work. Carino and McClellan say this is also in line with a primary goal of the 21st Century Cures Act.

Pharma needs to improve its use of RWE. Doing so will bring many benefits, writes Alexandre Bréant and Marc-Olivier Bévierre at consulting firm Cepton Strategies. One such benefit is better clinical trials that understand the needs of patients and the medical community, blending RWE and data from individual trials and pooling data from several trials.

Real world evidence can also make investment more enticing for payers as it highlights the link between efficacy and effectiveness. However, pharma needs to be aware that RWE is seldom as clean as trial data and there are collection challenges associated with RWE. One such challenge is the need for personnel to collect and analyze data from RWE. They need data scientists with biostatistics knowledge and IT acumen.


MA Teams In a Digital Space

The interoperability of RWE and clinical trial data will present new possibilities to pharma as it will provide a more complete profile of patients’ health. Halloran Consulting Group’s Paola Murphy and Hannah Yee write that the interoperability of data will necessitate a change in clinical operations. Clinical staff will need to reassess their thoughts about data and its management outside of traditional trial settings.

It will also require multiple stakeholders to participate and collaborate in understanding what data is relevant and how it can provide maximum benefit to clinical research. It will also mean there is greater transparency in the efficacy of the data so it can be used to inform more accurate value-based decisions.

Digital engagement with stakeholders will also be necessary and MA teams will drive this. After all, HCPs prefer digital engagement to in-person meetings and many use social media for professional purposes to help deliver better patient care, says healthcare marketing consultant Hilary Armstrong, Ph.D.

MA teams can help deliver their messaging by connecting with digital opinion influencers —  medical professionals, industry analysts, reporters, patients, caregivers, or external experts. Indeed, they can help bring unique perspectives and improve outreach for the pharma company but from a non-market-driven angle, Armstrong explains.

The pharma industry understands the need to shift towards value-based healthcare from the fee-for-service model. Payers, healthcare professionals and pharma will benefit from therapies that produce better results. Certainly patients will thrive because of this shift in focus. MA teams, as always, are tasked with engaging various stakeholders to show them why VBHC decisions are always best. Analyzing RWE and other important data, and communicating it in a compelling narrative, will drive MA’s success.

Images by: stockbroker/©, tumsasedgars/©, pressmaster/©

Want to stay up to date with our news?

To top