Looking back, 2025 was a year of adjustment for biotech — instability shook an already fragile biopharma sector, AI gained significant momentum, the ability to merge open-source code with commercial software matured, and the use of real-world data (RWD) continued to increase. Most companies responded responsibly: pilots, internal capability builds, exploratory regulatory dialogue, and, unfortunately, many had significant layoffs.

2026 is poised to be about embracing complexity — it’s likely many of the trends begun in 2025 will continue, and in addition we’ll see more emphasis on women’s health, an increase in discussions on external control arms, and a spotlight on biostats/clinical pharmacology. From a regulatory perspective, both the US Food and Drug Administration and the European Medicines Agency are signaling the same thing: innovation is welcome, but only when it produces decision-grade evidence.

This has real implications for how drug development companies operate.

Women’s health has expanded beyond a niche category. Regulators are increasingly focused on sex-specific evidence and generalizability across all therapeutic areas. By 2026, this is no longer optional — it’s foundational. In December 2025, the FDA published guidance on studying sex differences, with recommendations to increase female enrollment and strengthen sex-specific analyses and reporting.

Smarter trial designs are now the default. Adaptive approaches, Bayesian methods, external control arms, and decentralized elements are acceptable — expected, even — but only when the assumptions are explicit and defensible. In addition, regulators are clarifying and further exploring the use of various datasets in regulatory submissions; there’s even been a few external control arms presented to regulators.

Behind all of this is a broader shift: biostatistics and clinical pharmacology are moving from support functions to strategic capabilities. Dose justification, estimands, missing data strategy, and model-informed drug development (MIDD) are now central to regulatory credibility.

For biopharma leaders, the message is clear:

For those companies that embrace complexity as the new normal, 2026 offers a powerful opportunity: faster development, clearer regulatory paths, and greater confidence — from regulators, investors, and, ultimately, patients.

Keep an eye out for these new trends in 2026:

Women’s health expands beyond a single therapeutic area

Women’s health is no longer confined to reproductive medicine or niche pipelines. Regulators are increasingly focused on sex-specific evidence and generalizability across all therapeutic areas.

In 2026, sponsors should expect:

• Greater scrutiny of female enrolment and retention
• Fewer waivers for sex-specific analyses
• Increased innovation in areas such as menopause, endometriosis, fertility, and autoimmune disease

Takeaway: Women’s health isn’t just a therapeutic area; it’s a generalizability requirement. If your statistical analysis plan can’t speak clearly about sex effects (or justify why not), expect pointed questions.

External control arms move from optional to strategic

External and synthetic control arms have crossed an important threshold. In 2026, they are no longer seen as novel add-ons but as intentional design choices, particularly in oncology, rare disease, and high unmet-need indications.

Regulatory acceptance is evolving and both the EMA and FDA are leaning in — carefully:

• EMA is actively developing a reflection paper on external controls (including RWD-derived arms) to shape consistent scientific expectations.1
• EMA is also doubling down on DARWIN EU, its federated RWE network, with plans to extend work beyond 2027 (tender activity flagged for the first half of 2026).2
• FDA continues to expand its RWE framing across programs (and sponsors are expected to demonstrate data relevance, reliability, and bias management — not just “we found a database”).3

Takeaway: External controls are not a shortcut; they’re a design choice that requires 1) pre-specified causal estimands, 2) transparent matching/adjustment strategy, and 3) sensitivity analyses that are realistic.

Biostatistics becomes a strategic capability

Clinical trial design is undergoing a quiet but fundamental upgrade. In 2026, efficiency is no longer achieved by cutting corners, but by thinking better upfront. Biostatistics is no longer a downstream function; it is becoming a strategic driver of development success.

Adaptive and Bayesian designs are becoming mainstream, particularly in early and mid-stage development. Sponsors are expected to define estimands clearly and adequately to address trial objectives, to integrate biomarkers earlier, and to design trials that answer regulatory questions — not just scientific ones. Smaller trials are acceptable; ambiguous trials are not.

In 2026:

• Estimands, including novel endpoints and strategies for handling intercurrent events, and missing data strategies are key for addressing primary and secondary trial objectives and are strategic topics for assessing drug product’s risk-benefit and totality of evidence.
• Bayesian methods are increasingly used not just in design, but in regulatory dialogue.
• Statistics, clinical pharmacology, and translational science should continue moving closer to towards seamless integration.

Takeaway: Bringing biostatisticians to the decision-making table early and leveraging quantitative decision-making frameworks will be important for managing overall pipeline decisions.

Clinical pharmacology takes center stage

Clinical pharmacology is having a moment — and for good reason. Regulators are increasingly unwilling to forgive poorly justified dose selection, study design optimization, population enrichment, extrapolation, and/or benefit risk assessment/labelling.

MIDD is becoming the norm:

• Since 2024, FDA is pushing Model-Informed Drug Development via ICH M15 draft guidance and its MIDD meeting program.4
• EMA is sharpening expectations for mechanistic models (PBPK/PBBM/QSP) used in MIDD, including how they should be assessed and reported.5 A guidance is expected to be completed in 2026.
• In December 2025, the FDA released a guidance that lists products that no longer need (or can reduce) 6-month non-human primate toxicity testing and, in April 2025, outlined a roadmap for reducing animal testing in preclinical safety studies.6,7

Takeaway: In 2026, there will be increased scrutiny in making sure sponsors show why the chosen dose is the best one and can justify the assumptions behind MIDD submitted, or why the approach wasn’t used.

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