The data and insights highlighted in the article are drawn from the Biotech Playbook for Progress, a comprehensive resource built from a 2025 TriNetX survey of 150 biotech and pharma executives.
Biotech has always been a high-wire act: big science balanced on limited resources. But the wire is getting thinner. Development costs now often exceed $1 billion, timelines stretch over a decade, and trial success rates remain low, just 10–15% on average, and only 3.4% in oncology. For smaller, leaner organizations without the cushion of big pharma’s infrastructure, every decision, every dataset, and every patient counts.
To succeed, biotechs are rethinking how clinical research gets done. Real-world data (RWD), artificial intelligence (AI), and patient-centric strategies are no longer experimental; they’re emerging as the core toolkit for companies determined to accelerate progress while controlling risk.
RWD: From Volume to Value
The industry’s adoption of RWD has moved beyond pilot projects. Today, it’s fueling critical functions across the development lifecycle: from protocol design to patient identification and site outreach. For biotechs, this means faster trial initiation, reduced reliance on guesswork, and fewer costly amendments that can derail momentum.
But more data sources also bring more complexity. Many of the organizations participating in the TriNetX survey said they rely on five or more streams including lab results, genomics, registries, equity datasets, claims, and beyond. To unlock real value, these must be harmonized into a usable, unified picture. Without integration, the promise of RWD is easily lost to inefficiency.
Inclusion as a Strategic Lever
Trial inclusivity is shifting from regulatory checkbox to strategic advantage. With 99% of surveyed industry leaders advancing initiatives in this space, it’s clear that more representative trials are becoming the standard. Yet barriers remain: evolving regulations, dispersed patient populations, and narrow site strategies often stand in the way.
For biotechs operating in rare disease and oncology, inclusivity is more than good practice; it’s mission critical. Identifying small, scattered patient populations requires the broadest possible data lens. Inclusive trial design strengthens not just compliance, but also recruitment speed, study feasibility, and ultimately the odds of success.
AI and RWD: The Multiplier Effect
Pairing AI with RWD offers biotech companies the ability to accelerate insights, automate processes, and stretch limited resources further. More than half of industry leaders in the TriNetX survey are already deploying AI in this way, signaling the shift from theory to practice.
The benefits are tangible: faster analysis of patient eligibility, more efficient protocol design, and improved feasibility assessments. Yet challenges remain around data quality and security. For AI to serve as a multiplier, the underlying data must be robust, standardized, and responsibly managed otherwise small errors risk scaling quickly.
Centering on Patients
Biotech leaders increasingly recognize that patient-centricity is not just an ethical imperative but a business strategy. With the majority of survey respondents identifying RWD as a key enabler of patient-focused initiatives, the connection between evidence and empathy is tightening.
Understanding patient experiences, burdens, and access challenges allows companies to design trials that are more inclusive and less disruptive to participants. This not only improves recruitment and retention but also strengthens the credibility of outcomes with regulators and payers.
RWE as a Regulatory Asset
Real-world evidence (RWE) is gaining traction as a powerful complement to traditional trial data. Every executive who participated in the TriNetX survey agreed that RWE can strengthen regulatory submissions.
For biotechs working in rare disease, pediatrics, or urgent therapeutic areas, RWE can be especially valuable where randomized controlled trials are impractical or unethical. The key is ensuring that datasets are fit-for-purpose, i.e., designed and curated to address specific regulatory questions with clarity and rigor.
The Path Forward for Biotech
The blueprint for biotech progress is clear. Companies aiming to lead in this new landscape must:
