The year 2022 reflected a transformative path for the drug development industry. It is without a doubt that 2022 predicted change and opportunity in biopharma and biotech clinical trials in 2023. Every year, the FDA’s Center for Drug Evaluation and Research (CDER) clarifies to drug developers required study design elements, as well as other data needed on the drug application to support a full and comprehensive assessment. To perform this, CDER is required to understand the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat.
The US Food and Drug Administration (FDA) approved around 26 novel drugs in 2022. Approval processes were slow in comparison to 2020 and 2021, where 53 and 50 approvals were achieved respectively. Going into the third year of the pandemic, we know that the virus still impacts industry operations. Several companies have reported that the pandemic altered their drug development strategy with new priorities and collaborative approaches, which shape how organizations proceed today. Together, top vaccine development companies can join efforts, being transparent with one another to finally stop the spread of the virus around the world.
Please join me for an exploration of this interesting topic, as the biopharma and biotech industries adapt to new advancements in drug development processes, and we anticipate a year full of changes and growth in the life sciences field. Thanks for connecting with us, and we invite you to continue checking in to the EG Life Sciences Content Library for additional interesting blogs, white papers, and more.
2023, A YEAR OF GROWTH AND CHANGES
Decentralized Clinical Trials
Based on a PPD clinical research business of Thermo-Fisher Scientific performed last spring 2022, we surveyed more than 150 drug developers around the globe to collect insights on trends in pharma, research, and development. These findings reflect a significant shift after the pandemic, plus a growing importance of patient diversity and increasing complexity of trials. Survey respondents emphasize the challenges, opportunities, and goal innovations, as well as the impact of COVID-19 over the past two years. Most of them faced challenges with patient recruitment in clinical trials as a top pain point within their organization. In addition, the survey reveals that 27% of their trials would be decentralized by 2024.
Decentralized strategies are crucial, and biopharma and biotech organizations should be focused on these strategies and/or create partnerships with those with digital-based trial strategies to improve patient experience, engagement, and retention. Partnering with digital-based operations will facilitate speed, deep regulatory expertise, and a technology-agnostic approach, which will allow that works accurately and efficiently without knowing the basic details.
Novel Therapeutic Platforms
Novel therapeutics will dominate the development pipeline as developers become more familiar with clinical trials and regulatory challenges. Approval of the mRNA-based vaccines for COVID-19 has been the key to opening the path of a mainstream future for novel therapeutic platforms, including other applications such as cell-gene therapies and acid therapeutics treatments. Leveraging new technologies has been one of the greatest opportunity areas in clinical trials. There are many big companies merging efforts with dedicated CROs (Contract Research Organizations) to expand their capabilities and applications of mRNA technologies, such as Merck, Pfizer, BioNTech, and others have had long-term strategic collaborations, like Thermo-Fisher and Moderna.
Digitalization
Patient data is critical, and the life sciences industry is accelerating the path to introduce new methods for collecting data through BYOD (Bring Your Own Device). The use of personal devices such as mobile phones, tablets, and other wearables in a clinical setting offer a unique opportunity while improving patient engagement, significantly reducing costs. Enabling patients to use their own devices reduces costs and time, in addition to protecting patient information. This policy can also accelerate study start-up time, simplify the tracking of patient progress, allow sponsors to collect data, and support the safety and efficacy of a treatment and its impact on a patient’s quality of life. Beyond any costs, the BYOD has also been proven to increase compliance.
Most of the biopharma and biotech organizations are using digital tools in their trial operations. Some of these are cloud computing, artificial intelligence, and IoT (internet of things).
New Trial Methods
In 2023, a significant focus will be not only on data collection, but on the innovation of trial designs. This will also help to fast-track decision-making processes, study progress, trends, and risk management through digitalization approaches such as the use of AI (Artificial Intelligence), ML (Machine Learning) for data analysis.
Biopharma and biotech organizations are using the RWD (Real World Data) and RWE (Real World Evidence) for trial design, and advances in data sciences are enabling developers to use these tools if organizations follow important FDA advice to ensure plan effectiveness.
The number of Phase IV clinical trials significantly increased in 2022 in comparison to 2020, and this will increase in 2023 as experience collecting and analyzing RWD grows. Another interesting design is Adaptive design. This can make clinical trials more flexible by using results accumulated during the trial to modify the trial course, in accordance with pre-specified requirements. Adaptive design can be adapted across all clinical phases in a way that is often more efficient, informative, and ethical than trials with a traditional design, since they often make better use of time and money, and they might require fewer participants.
Traditionally, clinical trials include three steps:
- The trial is designed.
- The trial is conducted as prescribed by the design.
- Once the data is ready, it is analyzed according to a pre-specific analysis plan.
Adaptive design includes pre-planned changes, including but not limited to:
- Refining the sample size
- Removing treatments or doses
- Changing the allocation ratio of patients to trial arms
- Identifying patients most likely to benefit and focusing recruitment efforts on them
- Stopping the whole trial at an early stage for success or lack of efficacy
Vendors
Significant challenges have been highlighted associated with the need for expertise and the increase of outsourcing in clinical trial activities. Drug developers need experience, priorities, and capabilities. Most of the outsourced services are clinical diagnostic laboratories, clinical management, and biostatistical analysis resources, respectively. Partnership with a dedicated CRO, along with highly skilled experts, is critical as digitalization becomes a significant tool in clinical development and cell-gene therapy trials. Drug developers have been facing challenges with development timelines, increasing trial complexity and challenges in recruitment and retention of staff. This justifies the fact that most of these organizations are outsourcing highly skilled experts and dedicated CROs to advance in their trial and drug development goals.
Regulatory Compliance
Achieving safe and effective drugs depends on a patient population that imitates the real world. Recently, the FDA mandate called for a Race & Ethnicity Diversity Plan for every study. This requires not only patient organizations and site communities but sponsors and CROs to adapt the way they approach trial feasibility, design, and execution. Drug developers will face challenges to minimize roadblocks and provide training, support to sites, and patients to effectively retain diverse populations.
Summary
As I like to say, the future is now and there is more to come in the life sciences world for a better way of living. Interact and share your thoughts, or send your comments about this interesting post with us on social media!
EG Life Sciences continuously provides expertise and education to the life science community. Connect with us and find out more at https://www.eglifesciences.com/resources.
References
- "Biological Approvals by Year." U.S. Food and Drug Administration. Accessed 13 February 2023. https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/biological-approvals-year
- "2022 Biological Approvals." U.S. Food and Drug Administration. Accessed 13 February 2023. https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2022-biological-approvals
- "Advancing Health Through Innovation: New Drug Therapy Approvals 2022." U.S. Food and Drug Administration. Accessed 13 February 2023. https://www.fda.gov/media/164429/download
- "Data Report: The State of the Drug Development Industry." PPD. Accessed 13 February 2023. https://www.ppd.com/pharmaceuticals-research-and-development/
- "Getting Ahead in mRNA Vaccine Development with an Experienced CRO Partner." PPD. Accessed 13 February 2023. https://www.ppd.com/blog/mrna-vaccine-development-with-experienced-cro-partner/
- "BYOD: The Future of Clinical Trials." Technology Networks. Accessed 13 February 2023. https://www.technologynetworks.com/drug-discovery/blog/byod-the-future-of-clinical-trials-365712
- "FDA Offers Advice on Using Real-World Evidence from Registries." Technology Networks. Accessed 13 February 2023. https://www.centerwatch.com/articles/25880-fda-offers-advice-on-using-real-world-evidence-from-registries
- "Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials Guidance for Industry." U.S. Food and Drug Administration. Accessed 13 February 2023. https://www.fda.gov/media/157635/download
