Developing an Innovation Agenda Amid Cost-Savings Pressures
Establishing an Innovation Agenda Amid Cost-Savings Pressures
As sustaining rapid revenue growth has become more challenging, many medical device manufacturers have introduced sweeping corporate cost-savings programmes, often requiring across-the-board cuts in the order of 15 to 30 percent. Simultaneously, companies are under pressure to move more products to market faster. Yet, escalating requirements from regulators and payers for post-marketing research can not only slow down reimbursement timelines, but also require sustained investments in expertise and infrastructure.
Given the extent of these challenges in the medical device and diagnostics marketplace, it can be difficult to identify a pragmatic strategy to meet cost savings and productivity mandates. It can be unclear which initiatives, innovations, and investments should be pursued first.
Based on a history with most of the top 20 medical device manufacturers, ICON has identified six critical focal areas for innovation that can enable meeting and likely exceeding cost savings and productivity mandates.
We will review these six areas briefly in this blog. Further detail is available in ICON’s white paper, “Transforming Medical Device Development: Achieving Challenging Mandates for Cost Savings and Pipeline Productivity.”
(1 )Electronic Health Records (EHRs)
Within EHRs are patients’ treatment histories, lab results, demographics, and location.
Manufacturers that can access and query live EHR data can create a transformative new standard for developing and executing clinical research programmes.
Select organizations have already curated hundreds of millions of live EHRs from health systems and structured the data for real-time queries. This includes ICON’s partnerships with EHR4CR and TriNetX, which provide manufacturers an opportunity to immediately begin integrating EHR analytics into the development process. Transformative opportunities include applications for:
Recruitment — Analysis of EHR data enables identification of eligible patients at every site. By notifying investigators of relevant patients, manufacturers can accelerate enrolment without purchasing advertisements to push potential patients into a local clinic.
Site selection — Optimistic enrolment estimates from sites often draw out study timelines. EHR data provides an evidence-based approach for verifying estimates, granting manufacturers a definitive view of all relevant patients who match inclusion/exclusion criteria, those patients’ locations, and the sites that can most efficiently fulfil a trial's enrolment needs.
Protocol feasibility review — By using EHRs to validate a protocol’s inclusion and exclusion criteria against the actual patient population, manufacturers can assess how iterative modifications to those criteria may optimise recruitment and trial performance.
Observational studies and registries — Collecting pre-existing or accruing data through EHRs requires far fewer resources than does building and maintaining a registry, making it more practical to observe patients for longer periods of time.
(2) Adaptive Design
FDA and EMA regulators have encouraged manufacturers to adopt adaptive designs, which minimise the risk of failure or investment in futile trials,
Adaptive trials allow identification of (and reaction to) potentially regrettable design parameters. The CDRH called this concept “anticipated regret” in its guidance supporting adaptive design for medical device development. An adaptive design specifies timepoints for interim analyses, at which pre-approved changes to the trial protocol can address those potential “regrets.”
An interim analysis may identify, for example, that a study is underpowered to capture the investigational device’s positive effect on patients. The design would pre-specify an increase in the sample size (in a blinded fashion), so that a manufacturer would not need to invest in a second, appropriately powered study. In another case, an interim analysis may show that a device not effective at all or that an effective device has already met its endpoint, allowing for early termination and the redirection of limited funds to other development programs.
Furthermore, adaptive designs can help accelerate trials by preventing oversubscription or by combining typical separate studies into a seamless trial. For example, the same patient population can continue in a combined non-inferiority and superiority study or a combined pivotal and post-market study.
Adoption of adaptive design, while beneficial for de-risking an individual trial, is most valuable when applied across an entire portfolio. This ensures that limited resources are directed to the most productive products.
For deeper analysis of the types of adaptive designs relevant for medical device studies, including detailed case studies, download ICON's white paper.
(3) Patient Centricity
Patients are increasingly active, informed consumers entering clinics armed with research and opinions about their healthcare options, which is creating a demand on developers to take a more patient-centric approach to clinical trials.
While patient centricity, itself, is a philosophy of active listening and continuous improvement, several problematic aspects of clinical development stand to immediately benefit from considering the patient’s point of view. To create a competitive edge in bringing new devices to market faster and more cost-efficiently, three components of clinical development should be assessed:
Firstly, wearable devices and apps can improve the trial experience for patients. These tools can monitor a patient’s physical activity, adherence to medication, or response to a treatment, without requiring that he or she even steps foot into a hospital — with the end goal of reducing attrition and improving the appeal of participating in the study.
Secondly, patient attrition can often be traced to misunderstanding trial requirements during the consent process. Reengineering consent materials around patient expectations can improve patient engagement and trust, according to a recent study by Carnegie Mellon University that was funded by ICON plc. The researchers evaluated in their study a systematic methodology for reengineering consent materials. Furthermore, video and multimedia-based educational materials that are accessible outside of the trial site can help improve patient’s understanding and comfort with trial protocols and reduce the risk for protocol violations.
Finally, an EHR-driven feasibility review process can help refine protocols that are easier for patients to follow. When EHR data is also used to verify site enrolment estimates, the geographic mix of sites can be optimised to maximise convenience for patients.
(4) Real-World Data (RWD)
Given globally rising evidentiary demands from payers, as well as increased post-marketing surveillance requirements from regulators, the collection and utilisation of RWD has become central to bringing devices to market. Most recently, in the U.S., the passage of the 21st Century Cures Act emphasizes the rising importance of RWD for the entire development cycle. As a result, priorities should include both strategies to collect RWD more efficiently so as to contain costs and organisation-wide pilot programs to maximize the utility of RWD.
Manufacturers who proactively bridge the silos of the clinical and commercial groups during the product lifecycle can better align development efforts with unmet patient needs and accelerate time to market with the right evidence package.
This is an advance ICON calls “Real World Intelligence™”. By incorporating analysis of RWD into R&D decision-making, insights can reach R&D leaders that would ordinarily take years. For example, beta-blockers found vastly expanded use for controlling hypertension only after a retrospective analysis of four decades of their real-world use in heart attack patients.
(5) Healthcare Alliances
The advent of healthcare alliances, which are integrated networks of hundreds of investigators across multiple sites that cover millions of patient lives, afford manufacturers a transformative opportunity to enhance a study’s efficiency. Healthcare alliances mitigate the inefficiencies that occur when conducting trials with unaffiliated clinical sites. At the simplest level, healthcare alliances use standardised contracts and IT infrastructure to reduce start-up timelines and costs. Additional operational efficiencies are possible because sites can cross-utilise research staff as necessary and streamline project management for all sites in a trial to a single contact for the duration of a trial.
More importantly, healthcare alliances have established more rigorous study feasibility processes to ensure accurate enrolment forecasts, often drawing upon EHRs to pre-identify participants. Alliances also make further investments in investigator and patient relationships such that clinical research is more regularly included as a care option, thus expanding the accessible patient population. As a result, sites in healthcare alliances, such as within ICON’s PMG Research, are typically a study’s highest performing sites.
(6) Smarter Monitoring
Deploying risk-based monitoring (RBM) can mitigate escalating monitoring costs, which often consume a near majority of a study’s budget, while also enhancing a trial’s data quality. RBM represents a significant change from traditional monitoring approaches, which verify 100 percent of site data, but still do not catch all errors.
A greater opportunity to reduce errors that affect trial quality, however, exists in adopting root cause analysis techniques. Current monitoring approaches do not assist CRAs in discriminating why an error occurred, which may be a result of complex underlying causes in equipment, culture, workload, training, or other factors. Consequently, many corrective actions that monitors deploy do not prevent future errors that may emerge from the same original source. In fact, a new requirement in the ICH Good Clinical Practice (R2) 2016 guidance now requires that manufacturers address significant non-compliance through root cause analysis and then deploy bespoke corrective actions that prevent recurrence.
Patient Centric Monitoring, ICON's risk-based, patient-focused methodology for the design and execution of an adaptive monitoring strategy, has been deployed in recent trials to deliver 25% potential reduction in monitoring costs. This approach uses a root case analysis method known as human factors classification, which has been
adopted by NASA, Ford, and major airlines and militaries, to systematically identify the underlying behaviours and reasons for error.
Implementing an Innovation Agenda
In conclusion, today’s medical device manufacturers are faced with the seemingly contradictory need to reduce costs while simultaneously adhering to client and regulatory demands for additional post-market research. To resolve this conflict, it is crucial that developers establish an agenda that focuses on the aforementioned areas for innovation to accelerate timelines and reduce associated expenses.
Given the breadth of innovation required, it is suggested that developers establish pilot programs that are limited in scope and have clear objectives, and most importantly, to collaborate with one or more outside partners to ensure that their trials run smoothly and efficiently.
For deeper insights into these core areas for innovation, as well as in-depth analysis of opportunities to improve the efficiency of clinical outsourcing practices, read the full white paper: “Transforming Medical Device Development Achieving Challenging Mandates for Cost Savings and Pipeline Productivity”.