Survey: The Top 5 Innovations, Strategies and Technologies in Pharmaceutical R&D

According to our research, 98% of drug producers report they have embraced new innovations, strategies and technologies by implementing them into their trials today. Here are the ones leading the pack.

Clinical trials have evolved over the past few years, especially since the start of COVID-19. The pandemic compelled drug developers and sponsors to adapt, adjust and innovate quickly to improve the pharmaceuticals research and development process and hasten the speed to market.

As such, developers and sponsors are adopting methods like decentralized trials, or working with partners that can do so, and identifying areas for clinical trial process improvements to accelerate phase trials for drugs.

The PPD clinical research business of Thermo Fisher Scientific recently surveyed more than 150 leaders at biopharmaceutical and biotechnology companies across the globe to gather their insights on trends in pharmaceuticals research and development.

Close to half of the respondents (48%) shared that the average pharmaceutical pipeline timeline moves more slowly than it did two years ago. As a result, there has been widespread adoption of new innovations, strategies and technologies within drug and pharmaceutical research companies.

Over half of the survey respondents reported their organization now utilizes the following innovations, strategies and technologies:

1. Adaptive trial design (64%)
2. Digitalization (e.g., cloud computing, APIs, digital platforms) (62%)
3. Greater use of real-world data (RWD)/real-world evidence (RWE) (59%)
4. Big data and analytics (data science) (53%)
5. Decentralized, digital and remote trials (50%)

Keep reading to learn more about the top approaches to clinical trial innovation that drug developers are using and other approaches on the horizon.

1. Adaptive Trial Design

Adaptive trial design references a clinical trial design that “allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial.” Nearly two-thirds of respondents reported that their organizations are currently using adaptive trial design (64%) and digitalization (62%).

Adaptive designs are “smart.” They allow drug developers to use resources and time much more efficiently, while increasing scientific precision. Effective adaptive designs lead to improved learning, more efficient, better treatment for trial participants, and better information for regulators and the medical community overall.

In our experts’ experience, sponsors that adopt adaptive trial designs see reduced costs and streamlined development processes. This leads to greater efficiency and success rates for their clinical programs.

To gain an edge with this innovative approach, drug developers should look for a partner that delivers:

• Extensive experience in designing, defining, planning and executing adaptive trials
• Biostatistical, medical and regulatory expertise
• Diverse therapeutic experience, including oncology, general medicine, nervous system disorders, metabolic disorders, infectious diseases and autoimmune disorders

A solid partner with expertise, resources, infrastructure and seasoned leadership helps developers create comprehensive plans using adaptive clinical trials — and execute them successfully.

2. Digitalization

Nearly two-thirds of respondents (62%) reported that their companies are embracing digitalization, such as cloud computing, application planning interfaces (APIs) and digital platforms. This involves “leveraging digital technology to improve participant access, engagement, trial-related measurements, and/or interventions, [enabling] concealed randomized intervention allocation, and [providing] the potential to transform clinical trials and lower their cost.”

Drug developers should seek partners who have a demonstrated commitment to best-in-class technologies and combine new and innovative solutions with simplified processes and real-time analytics.

3. Greater Use of Real-World Data (RWD) and Real-World Evidence (RWE)

More than half of respondents (59%) shared that their organizations are using real-world data (RWD) and real-world evidence (RWE) more often. RWD is “data that relates to patient health status and/or the delivery of health care routinely collected from a variety of sources.” RWE is “clinical evidence regarding the usage and potential benefits, or risks of a medical product derived from analysis of RWD.”

Because clinical trial diversity remains one of drug developers’ biggest challenges, RWD is particularly important in helping to compensate for limitations in clinical trial representation. It is impossible to execute a global clinical study that fully includes all patient, lifestyle, environmental and genetic types. In order to get as close to causation as possible to evaluate drug efficacy, it is critical to supplement trial results with RWD.

Use of RWE (clinical evidence derived from RWD analysis) continues to evolve. With RWE’s growing importance in demonstrating value for market access and commercialization, it’s imperative that drug developers navigate myriad data sources, extract the most relevant and valuable insights from that data, and analyze the insights that drive decision-making.

A key to success is to partner with a team of experts in evidence-based solutions that has a deep understanding of and experience in extracting valuable insights from RWD. This team will design and execute prospective data collection, direct-to-EMR, direct-to-patient or hybrid study approaches that result in tailored, fit-for-purpose data for your study.

4. Big Data and Analytics (Data Science)

Medical decision-making is becoming more dependent on data analysis, particularly large and complex collections of data sets. As drug developers acknowledge that trials are becoming increasingly complex, a little over half of respondents to our survey (53%) shared that they are using big data and analytics (data science).

Working with a partner that uses a strategic approach to big data enables teams to sharpen the focus on direct-to-patient engagement campaigns and community outreach, get trials up and running quickly, and meet diverse enrollment goals in record time.

5. Decentralized, Digital and Remote Trials

Decentralized, digital and remote trials are becoming the new norm: 50% of survey respondents shared that they are currently using these approaches. Decentralization involves “bringing an increased proportion of a trial’s activities to the patients rather than bringing patients to a trial site.”

PPD’s 2021-2022 decentralized clinical trials industry survey revealed that COVID-19 had a substantial impact on ongoing clinical trials, and more than half of respondents shared that they expect the impacts to last well beyond the pandemic. As a result, there was an uptick in implementation of decentralized mitigation strategies, including remote site visits/monitoring, a virtual pre-site selection visit, replacing paper with digital clinical outcome assessments, and ensuring availability of remote consent/eConsent.

As digital and decentralized trials become an expected aspect of studies, drug developers should partner with contract research organizations (CROs) that have expertise in executing decentralized clinical trials, and utilize a variety of capabilities and solutions — including telemedicine, direct to/from patient models, remote consent/eConsent, home health care and nursing, devices and wearables, and remote monitoring.

Beyond these top five approaches, drug developers and sponsors are exploring and pursuing additional methods and strategies to ensure clinical trial success.

Among these additional new innovations, strategies and technologies are:

• Personalized/precision medicine: A way in which health care providers can offer and plan specific care for their patients, based on the patient’s genes.
• Data collection through wearables/connected health devices: Devices that remotely collect data from decentralized clinical trials, allowing for more frequent data collection and enabling long-term, real-time patient monitoring.
• Genetic medicine: Using cell and gene therapy, RNA therapy and other cellular therapies to understand the cause of disease and how patients respond to individual therapies.
• Artificial intelligence: An emerging solution for various health care management problems.
• At-home nursing for drug administration, routine patient monitoring and reporting: A type of home telehealth enabling monitoring and transfer of patient health data to a health care provider.
• DNA sequencing for enrollment criteria: A method to analyze the human genome to detect diseases caused by gene mutations.
• Innovative contracting: New ways to streamline the contracting process.
• Investing in biomarker measurement in blood and tissues: The ability to leverage biological markers (molecules, enzymes, genes, gene products, hormones or specific cells) in understanding cancer through emerging multi-omics technologies.
• Non-viral gene delivery: A promising, yet challenging, delivery system that is low in cytotoxicity, immunogenicity and mutagenesis, and may move the gene therapy field forward.

Drug developers and sponsors should be quick to embrace innovative approaches to clinical trials.

Over the past few years, drug developers have been forced to shift their clinical trial process, due to the impacts of the COVID-19 pandemic, a stronger focus on patient centricity and an increase in the number of therapies under production.

These shifts have required them to embrace new innovations, strategies, technologies and non-traditional methods — with great success. Going forward, drug developers and sponsors should be quick to adapt and apply these new clinical trial approaches as the future foundations of clinical trial success.

Note: Of the 152 drug developers surveyed, 133 answered the question about the innovations, strategies and technologies currently being used by their organization; percentages above reflect a respondent pool of 133.