Getting the Pulse on Biopharma and Biotech Companies: Differing Perspectives on Clinical Development

Our latest survey pinpoints the trends shaping the clinical R&D landscape and identifies variations between biopharma and biotech approaches.

The drug development industry is constantly adapting and evolving to bring novel therapeutics to market to improve the lives of patients across the globe.

While the drug development industry experienced setbacks during the COVID-19 pandemic, the field is again gaining momentum reminiscent of its pre-pandemic pace. That said, global turmoil has left an unmistakable mark on the industry. While it has resulted in challenges recruiting a diverse patient population, it has also inspired new tactics to accelerate the drug development process and improve efficiency.

To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. The results of this year’s survey reveal the latest trends in the clinical development industry and participants’ predictions for its future. Furthermore, the results bring to light differences in approach and perspective between respondents from small/mid-sized and large organizations.

Differences in size, resources and agility have led the two groups to take different approaches to clinical development. In many ways, these differences are shaping the industry, yielding new advancements to promote growth in drug development and pave the way for future success.

Clinical Development in the Biopharma and Biotech Arenas in 2023

Trial design approaches highlight differing priorities

Thanks to the rapid development of novel cell and gene therapy approaches to treat cancer, oncology continues to dominate drug development pipelines. Accordingly, oncology is the leading therapeutic area for drug development reported in our survey, regardless of sponsor size. Moreover, at 74%, more biopharma companies say oncology is a top priority compared to 56% of biotech companies.

Beyond cancer, metabolic, endocrine, cardiovascular and dermatology trials are also significant areas of development among larger sponsors that often focus on multiple disease indications at a time. Our survey indicates that smaller sponsors, which often work in only one therapeutic area, focus more heavily on immunology, rheumatology, neurology and rare disease indications.

Longer clinical development timelines were reported by 41% of participants, driven largely by respondents from small/mid-sized companies, with 51% saying they are experiencing longer trials. Conversely, large companies reported nearly the exact opposite, with 48% saying trial timelines are shortening. This discrepancy may be due, in part, to factors like protocol complexity and access to resources.

For example, the longer timelines seen by biotechs may be due to taking on more complicated protocols to assess treatments for orphan diseases. Additionally, biotechs have reduced staff and resources at their disposal, which could lead to their trials moving slower compared to their biopharma counterparts, who have the teams, technologies and resources to keep trials moving at a faster pace. Conversely, large biopharma companies are seeing accelerated patient recruitment, as reported in our survey, and are implementing approaches, such as using real-world data (RWD), that are speeding up their clinical trials and shortening timelines.

No matter the timeline, trials are being transformed by trends like artificial intelligence, patient-centricity and decentralized trials. In 2023, our survey found innovative trial design to be the top-reported trend driving transformation in clinical trials — a preference driven by small/mid-sized companies. Innovative trial design can include such practices as using flexible sample sizes, patient engagement, applicable historical information, multi-arm trials and novel endpoints. These trials require agility, the ability to customize, and the drive to think outside the box to address specific needs — qualities that biotechs are known to have in abundance. Biopharma companies show notable interest in emerging and established trends, including using RWD and leveraging new technology in drug development. These interests play to the strengths of larger companies, which have ample resources to explore new tools with the potential to streamline trial operations.

Innovative trial design is also a leading strategy for small/mid-sized companies during research and development, as is finding new approaches to investment and funding to address their focus on financial stability. Larger biopharma companies, in contrast, have more resources but tend to be less nimble. As a result, they may be less focused on finding new ways to structure clinical trials and more focused on improving practices already in place. This line of thinking is reflected by our survey data, which found they are prioritizing several strategies: improving patient diversity, focusing on patient centricity, pursuing personalized and precision medicine, and using RWD and big data.

Importantly, our examination of trial design highlights an increasingly popular concept: decentralized clinical trials (DCT). DCTs have been critical to drug development in the past few years, but continued benefits indicate that this trial modality is here to stay. Sponsors across the board have realized that this approach offers an opportunity to collect data more efficiently from a broader range of participants. While DCTs were a rarity prior to 2020, current survey respondents predict that in two years, 90% of their trials will include DCT elements.

Companies think differently about patient recruitment and engagement

Sponsors’ ability to recruit patients diminished in recent years while patients could not safely travel to trial sites, a factor driven by the recent pandemic. While this phenomenon is waning, 55% of survey participants report that patient recruitment remains a significant hurdle — the top reported challenge for both biotech and biopharma respondents, among 22 other concerns.

When ranking their top five challenges, small/mid-sized companies report focusing more on securing funding, whereas large companies were concerned with patient diversity. This difference results from biotechs running on a leaner budget, making funding a constant battle. In contrast, biopharma companies tend to have more funding, which allows their focus to shift to more high-level concerns.

The need to improve diversity in clinical trials has taken the spotlight in the past several years, and indeed, our survey identified a net positive change in the ability to achieve diverse patient enrollment. However, this is only partially reflective of the sponsors, as one in five respondents only recently started tracking patient diversity metrics and needs more time before they can conclude whether their efforts have improved year-over-year.

Furthermore, biopharma companies report being more optimistic about recruitment in general but say their ability to achieve diversity goals has worsened. This difficulty is likely because, despite extensive recruiting resources, companies are still facing patient mistrust in clinical research, lack of trial awareness and limited access, which discourage underrepresented populations from participating.

To attract trial participants and meet diversity goals, sponsors report employing an average of five patient participation strategies, including working with patient advocacy groups, building patient inclusion criteria into protocol designs, offering patient assistance resources, conducting remote monitoring and using patient-centric applications.

Making inclusion criteria part of protocol designs is a top strategy for small/mid-sized companies, as it minimizes the time and cost that goes into a clinical trial. On the other hand, large biopharma companies are driving patient relationship-building efforts by providing more patient education and conducting home visits — strategies they can pursue because they have more resources to partake in these beneficial but costly activities.

Vendor models and outsourcing practices differ between biopharma and biotech companies

In some capacity, outsourcing is common among all sponsors surveyed. When facing challenges such as longer timelines, increasing trial complexity and insufficient trial diversity, sponsors turn to vendors like contract research organizations (CROs) to help address these aspects of clinical development so companies can focus on their core competencies.

Vendors deliver services using a range of different models. Vendor models assessed in this study include:

• Strategic partners, who co-develop a drug and have risk-sharing agreements in place.
• Formal preferred partners, who have designation as a preferred vendor from procurement, purchasing, finance and legal departments, and may also offer pricing and rebate incentives.
• Informal preferred partners that are routinely used but have no formal designation or pricing, and rebate agreements are in place.
• Transactional, study-based vendors.

Our survey found that biopharma companies drive usage and preference for formal partners, but biotechs prefer strategic partners, followed by formal partners.

When it comes to outsourcing model preferences, our survey identified that functional service provider (FSP) and hybrid (FSP and FSO) models combined outpace full-service outsourcing (FSO) models in popularity. Respondents report the greatest outsourcing activity in patient recruitment and labs/diagnostics, at 39% and 42%, respectively. Other drug development activities likely to be outsourced include clinical trial management and managing RWD. While small/mid-sized organizations reported being more likely to outsource to supplement in-house resources, large organizations reported a greater likelihood to outsource medical writing, likely because this is a costly but highly specialized service for which in-house hiring can be a challenge.

Insights to Inspire Biopharma and Biotech Companies Moving Forward

Overall, our survey highlights fundamental differences in how biotech and biopharma companies approach clinical trials and the primary challenges they face. This phenomenon is due primarily to variations in funding, agility and disease indications of interest. No single approach is best. The differences between small/mid-sized and large companies ensure a constant stream of innovations to improve how clinical trials are conducted.

To accelerate clinical trials, sponsors can partner with a CRO to lighten the load and tap into decades of clinical trial expertise. Sponsors will find great value in selecting a partner who understands the unique needs of any client, large or small, and is poised to respond in ways that enable clients to overcome challenges and make the most of opportunities.

The PPD clinical research business of Thermo Fisher Scientific empowers drug developers by encouraging innovation in clinical development, offering expertise and access to diverse patient populations, and optimizing vendor models to deliver success.

Together, we can make the most of new trends and advancements in the industry to expertly advance your new therapeutics to the finish line.