Cost strategies for medical device trials in the new era of digital health.
Clinical trials generate the evidence required to enable the marketing of safe and effective medical devices. The clinical trials market size has been growing despite the COVID-19 pandemic in 2019-2022, and the trend is expected to continue.
Industry and global regulators acknowledge the need to conduct clinical trials more efficiently and cost-effectively to bring innovative healthcare products to patients more quickly. Recently, FDA released several draft guidances related to decentralized clinical trials (DCTs) and digital health technologies (DHTs), encouraging industry sponsors, investigators, and other key stakeholders such as contract research organizations (CROs) as well as technology providers to increase adoption and implementation for faster trials.
The pandemic motivated DCT adoption, a key tool for implementing a faster trial.
The industry is expected to harness big data to optimize planning and predictability and proactively identify risks, to better understand costs per patient and impact of patient recruitment and retention. In 2023, DCTs have moved toward patient flexibility by utilizing digital tools.
Not all trials are suited for a decentralized approach. Studies requiring specialized physical assessments or a trial involving an implantable device need to utilize physical investigational sites. The active phase of these studies is conducted in a highly specialized environment. The possibility exists, however, of using a decentralized approach for long-term follow-up of patients conducted using new technologies that can transfer information to the investigator or sponsor via remote technologies. Trials such as blood pressure monitoring, post-surgical pain management, or wearable glucose monitoring are examples that are highly suitable for DCTs.
Clinical trials comprise an enormous percentage of any new medical product development budget.1 The new era of DHTs and DCTs allows for potential cost reductions. Consider the following cost drivers of potential cost savings:
Budget projections for medical device development-to-launch costs are expected to increase by 4.7% by 2028.2 Class III premarket approval (PMA) devices are expected to have the highest cost of 50% more than for Class II 510K products,6 and Class II home diagnostics products budgets are expected to grow by 5.1% by 2030.5
Similarly, pharmaceutical industry trial budgets are expected to increase by 5.6% until 2032, as noted in a report by Precedence Research.4
Visiongain reports that the DCT market is expected to grow 7.6% by 2033 due to increased use of DHT and home healthcare.7 The number of trials with virtual, decentralized, or some decentralized features continues to increase globally.3 In the US, by 2024, about 75% of trials will use remote patient monitoring and by 2025, the virtual market is expected to growth by 13%.4
With the rising number of clinical trials and increased costs for development-to-launch, the industry is moving to DCTs and DHTs, using registries and real-world evidence data to reduce the cost of trials.
The main advantages of DCTs are show in Figure 1 and include:
If the clinical trial design is suited for a DCT study, the challenges should also be given very careful consideration (see Figure 2 below).
To address the challenges, sponsors should be aware of associated costs of technologies employed in the trial by ensuring that the technologies chosen are validated and offer data security and privacy of personal health information. Partnering with technology and digital health companies may provide some future cost savings.
A virtual trial was conducted for a home use device intended to reduce anxiety.
Patient identification was led via social media platform study advertising, which allowed interested participants to sign in and respond to pre-screening trial-specific questions for eligibility. Eligible participants were enrolled by a study healthcare professional (HCP) during a telehealth visit.
The investigational products were shipped directly from the sponsor-designated person directly to the patients’ homes. Training was virtual via video platform.
The study was IRB-approved centrally and the CRO served as the designated single site, managing the study virtually by assessing the anxiety level at different visits by a HCP via a telehealth platform and a self-reported patient outcome questionnaire through an eDiary and ePRO platform.
The study visits were tracked by an automatic scheduler and emails reminders were sent automatically to the participants. Monitoring was reduced due to the very few assessments needed to be source verified. Safety was assessed by self-reported outcomes by participants and reviewed by the CRO safety officer.
For this virtual trial, the sponsor study cost savings included:
As with any trial, challenges arose. Challenges associated with the study’s virtual nature included:
A decentralized hybrid trial was conducted for a home use device intended for pain management of the extremities. The study was IRB-approved centrally and the sites accepted the central IRB.
The study was managed by a CRO in hybrid mode. Patient identification was conducted by each of the participant sites as determined by the HCP. The CRO determined group allocation to the device or the standard of care. The device group received the devices shipped directly to the patients’ homes. Device training was conducted by an online video platform.
Both participant groups reported pain scale daily by completing a questionnaire via an ePRO platform.
The study visits were tracked by sites and the CRO. Monitoring was reduced due to the limited number of assessments to be source verified from the sites. Safety was assessed by self-reported outcomes by participants and reviewed by the site staff.
The study cost savings were experienced in the following areas:
Challenges associated with this study included:
While there are many differences between pharmaceutical and medical device industry trials, one major difference surrounds whether or not the study product is billed to insurance.
While an investigational drug is provided for free and not billed to insurance, an investigational device may be reimbursable. The Medicare Prescription Drug, Improvement, and Modernization Act of 2003 (MMA) allowed Medicare payment of the routine costs of care furnished to Medicare beneficiaries in certain categories of investigational device exemption (IDE) studies.
The study sponsor should assess the device reimbursement and whether its Centers for Medicare & Medicaid Services (CMS) device category falls into:
CMS requires pre-authorization for covering the trial costs and billing, which should adhere to Medicare reporting requirements (i.e., “extended coverage” per the National Coverage Determination [NCD] 310.1 guide).
In any trial, billing issues may arise and the most common reasons include double-billing, deeming a clinical trial qualified or routine when it is not, and falsely billing non-covered items/services or when patients were not seen.
To mitigate these risks, sponsors should ensure they collect the key documents and perform a coverage analysis, which should include identifying every subject on every study for every visit, performing a full bill hold for all subjects, working through all institutional claims and invoices, and reviewing for correct billing and that coding is applied properly.
The number of DCTs is expected to increase due to the industry’s need for faster, more accessible, and cost-effective solutions to deliver medical innovations to patients. Sponsor companies conducting clinical trials should consider engaging in DCTs when possible and leverage various DHTs to reduce cost. When budgeting a trial, sponsors should pursue cost savings while addressing DCTs’ challenges with the following solutions:
Adelina Paunescu, PhD, VP of clinical affairs, MEDIcept, Inc.