February 14 2023 Andrew Park, Pharm D.

Over the past 15 – 20 years, projected peak sales and return on investment (ROI) of new drugs has been shrinking (source: Statista and Deloitte Centre for Health Solutions). To offset this, pharmaceutical companies are pushing to get more drugs to market and multiple indications for each agent. There has also been a proliferation of smaller pharmaceutical companies, often start-ups who are dependent on venture capital and have only 1-2 candidates in their pipeline; as of 2020, pharmaceutical companies outside of the 50 largest hold 63% of agents actively in development (source: Pharmaprojects; Tufts CSDD).

The nature of drugs in development has shifted as well.  Since 2000, the percentage of approvals targeting rare and orphan diseases has grown from 11% to 58%; the low incidence rate of these diseases present challenges for clinical testing. The share of oncology drugs in clinical testing has also grown steadily, and now account for approximately a quarter of drugs in clinical testing. Clinical trials for both rare diseases and oncology often require sites across a greater number of countries, longer enrollment timelines, and more cycles of treatment while enrolling fewer patients per site as compared to non-oncology and non-rare disease studies. Adding to this complexity, the percentage of drugs that rely on genetic or other biomarker testing to select or stratify patients has grown substantially and, as of 2021, comprise 64% of drugs in development industry-wide (source: Pharmaprojects; Tufts CSDD).

Given the current drug development environment and above challenges, anything that will reduce development costs, streamline clinical trials, and speed up time to market is an advantage. As noted in Part 1 of this blog series, decentralized clinical trials (DCT) strategies have existed for years and hold promise to accomplish these goals. DCT strategies include, but are not limited to, telemedicine, wearables, home delivery of study drug/supplies and remote monitoring. Studies can incorporate DCT strategies on a continuum from fully virtual (e.g., no investigational site) to mostly traditional. These strategies must be implemented based on the study protocol, since not all will be appropriate. For example, home delivery of an early phase study drug that requires infusion and patient monitoring may not be feasible.

Despite their promise, the pharmaceutical industry has been somewhat slow to adopt DCT, but the COVID-19 pandemic greatly accelerated this transition. A few DCT solutions that saw large increases in use in clinical trials compared to just prior to the pandemic include telemedicine visits (22% to 57%), home delivery of study drug (20% to 64%), and remote monitoring (43% to 70%) among others that saw less prominent increases (source: Tufts CSDD May 2022).

Studies can incorporate DCT strategies on a continuum from fully virtual (e.g., no investigational site) to mostly traditional.

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Through the pandemic, PROMETRIKA helped our sponsors pivot to remote monitoring and other DCT strategies as needed for their studies, but this rapid shift has revealed potential downsides. The industry has been reassessing deployed DCT strategies and their associated challenges (e.g., start-up cost and time, unclear ROI, compliance and legal challenges, and lack of site level training and resources). Many pharmaceutical companies and CROs expect to REDUCE the routine use of some DCT strategies including telemedicine, home shipment of study supplies and home visits (source: the Avoca Group, 2021).

On the investigator side of things, while DCT has broadened options and improved the experience for patients, many sites have reported challenges related to implementation of DCT including (source: WCG survey of 3,500 investigative sites, April 2021 and Tufts CSDD, 2022 based on three virtual focus groups):

  • barriers to patient enrollment using DCT tools
  • insufficient staffing / additional workload
  • longer contracting and start-up timelines
  • insufficient training
  • delays in study supplies and site payments.

Despite the challenges, there is reason to look forward to how DCT can be implemented well in current and future clinical trials. Based on our experience, PROMETRIKA identified rare disease studies as good candidates for implementation of DCT strategies. In one of our rare disease studies, patients were geographically distant from study centers which made patient visits and retention difficult. Implementing telemedicine, home visits, and direct home shipment of study drug led to decreased costs to both patient and sponsor, decreased travel burden for the patient, and improved patient retention.

Every study is unique and requires a different approach for implementation of DCT strategies. PROMETRIKA’s knowledge and experience help us guide our sponsors to what will work best for their studies and plans.

PROMETRIKA hosted a live webcast about “Exploring the Impact of Decentralized Clinical Trials”. To learn more, we invite you to watch the video here.