Biotechnology companies face new as well as long-established challenges – from securing sufficient funding to regulatory and policy uncertainty to manufacturing readiness to aligning global development strategy.
By collaborating and sharing experiences, the biotech industry has found ways to overcome many of these challenges and advance their innovations. The premier venue for such discussions has long been the BIO International Convention. This year, BIO was held in Boston, where more than 17,000 attendees from over 2,300 companies, representing 68 countries, came together to network, partner, and share insights across the full lifecycle of product development[1].
A manual review and analysis of the delegate list shows that attendees came from a vibrant mix of developers, with roughly 18 percent from early development – meaning preclinical to phase 2; 40 percent from commercial stage, and 37 percent unclassified, likely representing stealth-mode or niche life science firms.
Sessions exploring many industry trends took place over the four-day event, which was held from 16 through 19 June. One panel session, which I moderated, delved into the early development challenges facing biotechnology innovators. While speed to development, and ultimately to market, is a priority, of equal importance are smart sequencing, strategic clarity, and scientific credibility under tight timelines and rising scrutiny.
The discussion explored several key themes that innovators must confront. This article delves into some key questions and takeaways from the panel discussion.
Tackling common oversight with early-stage cell and gene therapy development
We have seen developers struggle with challenges related to donor selection and, notably, with CMC when shifting from research-grade to clinical-grade material, particularly around quality agreements with vendors.
One panelist identified a tendency for startups to overlook foundational cell and gene therapy documentation, donor eligibility, and comparability planning. To mitigate potential issues, there is a critical need for early partnership with experienced vendors and proactive quality agreements.
Challenges with building a global development strategy for neurodegenerative diseases
Any global development program requires a clear strategy, but there are unique regulatory and operational challenges that can occur with programs for neurodegenerative diseases. For small and mid-sized companies, there is growing pressure to effectively position early clinical strategies for global partnerships and in preparation for later-stage development.
One panelist noted the importance of finding alignment between the major health authorities when managing clinical programs for neurodegenerative disease, which can be challenging given such programs need region-specific endpoints, biomarker validation, and scalable trial designs. A key takeaway is to interact with health authorities early on to build both regulatory and investor confidence.
Connecting the dots between regulatory and corporate strategy
Alongside the need to manage global regulatory requirements is the importance of aligning those requirements with corporate strategy from early on.
Unfortunately, some development decisions that are underestimated early on can have serious ramifications later. Key among these, one panelist noted, are investigational new drug (IND) strategy, pre-IND meeting timing, and global protocol design. Seamless transitions between preclinical and clinical require tight alignment between program leads and regulatory teams.
Navigating the European Union’s unique policy and regulatory environment
The EU offers both an important market and a complex one, and early-stage biotech companies are advised to prepare for policy shifts as soon as possible.
One of the most notable is the Joint Clinical Assessment (JCA), which is now required under the Health Technology Assessment (HTA) regulation for all new advanced therapy medicinal products (ATMPs) and cancer treatments. The European Medicines Agency (EMA) is supportive of the new HTA framework and will provide relevant information from its own regulatory assessments[2]. EMA will also collaborate with the HTA Coordination Group in parallel joint scientific consultations (JSCs), demonstrating heightened partnership between the HTA process and the regulatory process.
However, as one panelist noted, the evolving JCA and real-world evidence frameworks demand earlier planning for endpoints and comparators and EMA/HTA alignment will influence trial locations, design, and readiness for go-to-market.
Minding the gap between early scientific development and commercial readiness
A key issue that is emerging is how innovations will translate into products that can be commercialized in global markets. Pricing pressures from policies such as the Inflation Reduction Act (IRA) and Most Favored Nation (MFN) are pushing developers to integrate access and reimbursement strategy earlier, one panelist pointed out. Commercial signaling is becoming as critical as clinical design.
A meeting of minds across early development
The panel discussion offered a lot of food for thought with one key message: Early development wins come not just from innovation, but from integration — of strategy, science, and foresight.
With the biotech industry undergoing further transformation – evidenced by the increased number of early development-stage companies attending this year’s BIO – the importance of being prepared can not be overemphasized. One consistent message across the panel was that biotech companies are seeking more than regulatory execution—they are looking for strategic partners. They are searching for an integration of regulatory foresight, technical CMC guidance, and global health authority engagement early in development to de-risk programs and improve both regulatory and investor confidence.
Early-stage companies no longer prepare regulatory filings just to satisfy agencies — they prepare them to inspire investor confidence. Proactive, credible engagement with global health authorities signals that a biotech is not only developmentally sound but also commercially viable.
About the author:
Michael Day, Ph.D., is Senior Director of Regulatory Strategy and CMC at PharmaLex. With more than 25 years of experience in pharmaceutical development and regulatory affairs, Mike specializes in U.S. FDA strategy, regulatory due diligence, and CMC lifecycle management for both early- and late-stage assets. He has led global development programs across a wide range of therapeutic areas, including biologics, biosimilars, and complex combination products. In his current role, Mike advises private equity firms, NewCos, and established biopharma companies on regulatory risk assessment, IND/BLA planning, and integrated regulatory-CMC strategies that support global commercialization.
[1] BIO International Convention. https://convention.bio.org/bio-2025
[2] New EU rules for health technology assessments become effective, EMA. https://www.ema.europa.eu/en/news/new-eu-rules-health-technology-assessments-become-effective
