Pharma's AI ROI gap isn't strategy or tech; it's the missing workforce qualification step every other regulated capability requires.

Pharma's AI ROI Problem Isn't a Tech Problem but a Qualification Problem

In this episode, we discuss AI drug discovery hype, agent security risks, and why pharma's AI workforce bets remain unproven.

Manufacturing Intelligence: Sizing Up Pharma's Biggest AI Deals, Part Two

Three regulatory actions are converging to move biologics, gene therapy, and the ATMP platform forward quickly

Regulatory Actions Impacting Biologics

Modernizing infrastructures, record drug approvals, and harmonization were all positive regulatory advancements in July 2026.

Regulatory Roundup—July 2026

This episode of Pharma Fundamentals provides an introduction to hold times in biopharmaceutical manufacturing and how they differ from hold times in small-molecule drug manufacturing.

Pharma Fundamentals: An Introduction to Hold Times in Biopharmaceuticals

The use of artificial intelligence is facing harder questions of governance and data quality

PharmTech Weekly Roundup—July 17, 2026

Three regulatory actions are converging to move biologics, gene therapy, and the ATMP platform forward quickly, say Susan J. Schniepp, distinguished fellow at Nelson Labs, and Siegfried Schmitt, PhD, vice president, technical, Parexel.

How evolving regulatory expectations are reshaping pharma development and manufacturing.

Drug Digest April 2026

, specializes in business development, new product planning, and early brand strategy. He helps companies lay the foundation for successful launches through data-backed strategies spanning indication assessment to portfolio strategy. With a strong background in pricing and market access, Nathan integrates both commercial and access insights into early asset development to maximize market opportunity.

 specializes in brand strategy, product launch, and advanced analytics. He helps companies navigate critical decisions across the asset lifecycle, from early strategy through launch execution, with a focus on maximizing patient access to treatment. Drawing on expertise in analytics, ML, and AI, Michael combines quantitative rigor with cross-functional insight to support more robust strategy and informed decision-making.

Rare Disease Day Background. Colorful awareness ribbon with group of people with rare diseases. | Image Credit: ©vetre-stock.adobe.com

As development costs rise and timelines extend in the United States and Europe, orphan drugs should increasingly be favored as vehicles for capital-efficient innovation.

 Their ability to generate clear clinical signal earlier, support flexible regulatory pathways, and require lower commercialization investment is driving buyers to engage earlier in development, often at Phase II proof-of-concept.

Together, these forces have fundamentally repositioned orphan drugs from defensive portfolio hedges to primary engines of biopharma deal-making. As development costs rise, evidentiary expectations intensify, and capital discipline tightens, orphan assets are now dictating when companies engage in transactions, how risk is allocated through deal structure, and which programs advance within increasingly competitive pipelines. In this environment, orphan drugs are not merely managing portfolio risk; they are actively reshaping the strategic logic that underpins modern biopharma business development.

Rare Disease Therapies: From Niche Experiment to Strategic Growth Engine

Rare Disease Treatments: Navigating the Economics of Global Innovation

Rare Disease Assets Hedge Against Portfolio Risk and Stabilize Long-Term Portfolio Value Potential

Orphan drugs and the evolution of business development and licensing (BD&L) strategy

Orphan drugs are no longer simply attractive options within business development portfolios. Increasingly, they should be viewed as the preferred vehicle for capital-efficient innovation and value creation.

 This shift reflects a growing alignment between orphan drug economics and the realities of modern biopharma development.

Rare disease programs are structurally advantaged across three dimensions that matter most to deal-makers: capital efficiency, speed to signal, and earlier value inflection. Smaller and more clearly defined patient populations enable reduced trial sizes, shorter enrollment timelines, and higher effect sizes that can be detected earlier in development. As a result, clinical development costs for orphan drugs are often materially lower than for broad-population assets, particularly in Phase I and II studies.

Commercialization economics further reinforce this advantage. Orphan drugs typically target concentrated prescriber bases, reducing the need for large-scale sales forces and broad promotional spend. Patient identification and engagement strategies are more targeted, and launch execution can be highly focused around centers of excellence and specialist networks. Taken together, these factors lower the total cost required to translate clinical success into commercial impact.

Regulatory environments have also proven more permissive for rare disease development.

 Regulators have demonstrated flexibility around endpoints, trial design, and post-approval evidence generation in settings of high unmet need. This adaptability shortens development timelines and increases confidence that early signal can translate into approval, supporting earlier transaction points.

Historically, however, deal activity has not reflected these advantages. Orphan drugs account for a disproportionate share of new approvals yet represent a smaller fraction of overall deal volume and aggregate deal value (

). This disconnect highlights how BD&L strategy has lagged behind the underlying economics of rare disease innovation.

 A) Analysis of pharma industry deal volume by year from 2004–2024, subdivided between deals involving a drug receiving an Orphan Drug Designation in the United States (n=3106) and deals that did not include any drugs receiving an Orphan Drug Designation in the US (n=18411). B) Analysis of Orphan Drug Designation prevalence in deals (e.g., M&A, partnerships) and approvals in the US, expressed as the percentage of overall volume. Orphan Deals are defined as any deal that involved a drug receiving an Orphan Drug Designation in the US (n=3009), expressed as a percentage of overall deals (n=20486). Orphan Approvals are defined as the initial US approval for a drug receiving an Orphan Drug Designation in any indication (n=608), expressed as a percentage of overall approvals (n=1095). C) Analysis of median pharma industry deal value by year from 2004–2024, subdivided between deals involving a drug receiving an Orphan Drug Designation in the US (n=968) and deals that did not include any drugs receiving an Orphan Drug Designation in the US. Figure courtesy of the author.

As capital discipline becomes paramount and portfolio volatility increasingly penalized, this misalignment is beginning to close. Orphan drugs increasingly represent not just attractive opportunities, but the most rational deployment of BD capital.

Deal structures reflect diverging orphan and non-orphan strategies

Disaggregating deal activity by transaction type highlights how business development strategies have diverged meaningfully between orphan and non-orphan assets in response to evolving scientific, financial, and macroeconomic constraints. Partnerships, licensing agreements, and acquisitions now play distinct and differentiated roles depending on orphan status.

Among orphan-involved deals, partnerships have emerged as the most dynamic transaction type. Partnership activity remained relatively stable from 2004 to 2014, expanded rapidly between 2015 and 2022, and then contracted sharply in 2023 and 2024, returning to pre-2014 levels (

). This cyclicality reflects the inherent sensitivity of partnerships to capital availability, as these structures rely on staged economics, long-dated milestones, and embedded optionality. Periods of low interest rates and abundant capital favor such arrangements, while rising rates significantly reduce the ability of therapeutic originators to find pre-market development capital, requiring capital-based deal structures.

Licensing activity in orphan deals declined more gradually over the same period, while M&A volumes showed modest growth with year-to-year variability. Notably, orphan M&A activity has proven more resilient to macroeconomic shifts, consistent with its role as a strategic tool reserved for late-stage or commercially validated assets that offer durable revenue and clear portfolio fit.

Non-orphan deal structures followed a different trajectory. Licensing volumes declined steadily by approximately 60% between 2004 and 2024, and partnerships fell by roughly 45%, while M&A activity remained broadly stable (

). These patterns suggest that external innovation sourcing outside rare disease has become structurally more selective, reflecting higher evidentiary thresholds, intensifying competition, and diminishing returns in large therapeutic areas.

Deal value further reinforces this divergence. For orphan assets, median licensing and partnership values generally remained below $200 million, reflecting earlier-stage access and deliberate risk-sharing (

). In contrast, orphan M&A values trended upward after 2022, consistent with growing scarcity of high-quality late-stage assets. Non-orphan deal values rose sharply across all structures after 2018, particularly for partnerships and licensing, signaling intensified competition for scale and platform-level advantage (

: A) Analysis of mean value of all deals by year of deal from 2004–2024, shown in millions US dollars. Orphan Deals are defined as any deal that involved a drug receiving an Orphan Drug Designation in the United States (n=3106); Non-orphan deals are defined as any deal that did not involve any drugs receiving an Orphan Drug Designation in the US (n=18,411). B) Analysis of mean value of M&A deals by year from 2004-2024, subdivided between deals involving a drug that received an Orphan Drug Designation in the US (n=545) and deals that did not involve any drugs receiving an Orphan Drug Designation in the US (n=4084). C) Analysis of mean value of partnership deals by year from 2004–2024, subdivided between deals involving a drug that received an Orphan Drug Designation in the US (n=1571) and deals that did not involve any drugs receiving an Orphan Drug Designation in the US (n=8918). Sample excludes data from deals finalized in 2023 given the outlier nature of the data. D) Analysis of mean value of licensing deals by year from 2004-2024, subdivided between deals involving a drug that received an Orphan Drug Designation in the US (n=990) and deals that did not involve any drugs receiving an Orphan Drug Designation in the US (n=5409). Figure courtesy of the author.

Why has Phase II become the orphan deal sweet spot?

Changes in deal structures have also been accompanied by changes in deal timing. One of the clearest manifestations of this shift is the trends in orphan drug transactions, specifically M&A and licensing deals toward Phase II (

). Buyers are engaging earlier, not despite scientific risk, but because risk in rare disease development manifests differently and is often resolved sooner.

Phase II studies in orphan indications frequently require smaller patient numbers and can deliver clear mechanistic readouts. Strong genotype-phenotype relationships and well-understood disease biology allow clinical relevance to be demonstrated earlier. This enables buyers to accept higher nominal risk in exchange for faster timelines and lower capital exposure.

Regulatory precedent reinforces this approach. In the past two years alone, FDA has approved six rare disease therapies supported primarily by single-arm trials. None of these programs included randomized controlled trials, and all received either traditional or accelerated approval 

. These approvals underscore the regulatory willingness to act on compelling early data in settings of high unmet need, further validating Phase II as a credible value inflection point.

Earlier engagement also carries strategic benefits beyond price. Acquiring or licensing around an orphan asset at Phase II allows buyers to shape pivotal trial design, align endpoints with commercial value drivers, and integrate access and evidence strategies earlier in development. This alignment reduces downstream friction between clinical development and commercialization.

In parallel, partnership activity is increasingly concentrating around marketed orphan assets (

). This trend reflects a preference for de-risked revenue streams in environments where late-stage development costs, access uncertainty, and post-approval evidence requirements continue to rise. For originator-manufacturers, partnering marketed assets offers immediate cash flow participation and commercial optionality while preserving capital discipline.

 A) Analysis of prevalence of M&A deals by year in 2009 (n=21), 2014 (n=33), 2019 (n=23), 2024 (n=34) wherein the lead asset was a drug receiving an Orphan Drug Designation in the US; sample is subdivided based on the development stage of the lead asset in the deal at the time the deal was completed, expressed as a percentage of overall M&A deals with an Orphan Drug Designation as the lead asset in the given year. B) Analysis of prevalence of partnership deals by year in 2009 (n=41), 2014 (n=61), 2019 (n=110), 2024 (n=50) wherein the lead asset was a drug receiving an Orphan Drug Designation in the US; sample is subdivided based on the development stage of the lead asset in the deal at the time the deal was completed, expressed as a percentage of overall partnership deals an Orphan Drug Designation as the lead asset in the given year. C) Analysis of prevalence of licensing deals by year in 2009 (n=54), 2014 (n=58), 2019 (n=40), 2024 (n=24) wherein the lead asset was a drug receiving an Orphan Drug Designation in the US; sample is subdivided based on the development stage of the lead asset in the deal at the time the deal was completed, expressed as a percentage of overall licensing deals an Orphan Drug Designation as the lead asset in the given year. Figure courtesy of the author.

Which therapeutic areas and modalities are driving orphan deals?

Orphan deal activity remains concentrated in therapeutic areas characterized by high unmet need and strong biological rationale, including oncology, neurology, metabolic, and genetic diseases (

). These same areas are often dominated by advanced modalities such as biologics, cell therapies, and gene therapies (

). This concentration matters because it mirrors the areas where development efficiency and early signal generation are most critical.

A) Analysis of volume of pharmaceutical industry deals completed from 2004–2024 that exclusively involved assets receiving an orphan drug designation in the US in any indication (n=4061), sub-divided based on therapeutic area of the lead asset in the deal. Sample excludes deals for which the therapeutic area of the lead asset was not available. B) Analysis of volume of pharmaceutical industry deals completed from 2004-2024 that included any asset receiving an orphan drug designation in the US in any indication (n=3811), sub-divided based on the molecule class of the lead asset in the deal. Sample excludes deals for which the molecule class of the lead asset was not available. Figure courtesy of the author.

Buy-side and sell-side perspectives on orphan value

Sell-side willingness to out-license or divest rare disease assets is shaped primarily by perceptions of market potential, but in a way that reflects disciplined portfolio management rather than reduced confidence in the science. Market research of industry experts revealed that there is greater readiness to transact assets with lower expected peak sales, consistent with rational capital allocation and prioritization decisions (

). This pattern reflects internal portfolio triage as organizations balance finite resources, development capacity, and strategic focus.

This effect intensifies with increasing company size. Larger organizations show lower tolerance for assets that fall below internal commercial thresholds, even when scientific rationale remains strong (

). As portfolios expand, internal competition for capital, management attention, and development infrastructure increases. In this context, out-licensing or divestment enable companies to redeploy resources toward higher-priority programs while retaining upside through milestones or royalties. Resource constraints, capability gaps, and financial risk consistently rank among the top drivers of sell-side willingness to transact, reinforcing that these decisions reflect portfolio optimization rather than opportunistic divestment 

Market Research analysis of industry experts (n=31). A) The average minimum peak sales of rare disease assets that experts would consider for a sale. B) Analysis of the minimum peak sales considerations by size of company
in which the expert worked. C) The top 5 factors that experts reported would make them want to sell an asset. Figure courtesy of the author.

On the buy side, industry experts reported lower appetite for assets with limited peak sales potential, indicating that minimum commercial thresholds remain relevant across all company sizes (

). Buy-side decisions are driven primarily by scientific validity, clinical differentiation, alignment with existing capabilities, and favorable access and pricing dynamics (

), while commercial and regulatory factors rank lower, reflecting uncertainty in early-stage development.

Market Research analysis of industry experts (n=31). A) Mean minimum peak sales threshold at which respondents would consider acquiring a rare disease asset. B) Minimum peak sales thresholds for asset acquisition stratified by respondent company size. C) Top five factors reported as drivers of buy-side willingness to pursue acquisition or in-licensing of a rare disease asset. Figure courtesy of the author.

Company size further differentiates behavior. Growth-stage biopharmaceutical companies demonstrate the greatest willingness to transact on both the buy and sell sides, regardless of asset value (

). Facing pressure to expand pipelines while managing capital and execution risk, these firms often act as intermediaries within the rare disease ecosystem. Overall, this shows that rare disease deal-making is shaped less by headline market size and more by the interaction of scientific confidence, portfolio fit, and organizational constraints.

What are some strategic implications for global dealmakers?

The deal-making patterns observed across orphan drug transactions point to a clear set of strategic implications for biopharma leaders. First, rare disease drugs should no longer be evaluated using the same timing, valuation, and ownership frameworks applied to non-rare disease assets. Their economic and regulatory characteristics favor earlier engagement, more flexible deal structures, and greater tolerance for scientific risk when early signal strength is high.

Second, Phase II has become a critical strategic inflection point. Buyers that engage at this stage may gain not only pricing advantage, but also meaningful influence over pivotal trial design, endpoint selection, and evidence-generation strategy. Earlier ownership or partnership enables tighter alignment between clinical development and downstream commercial requirements, reducing the risk that late-stage programs generate data that are scientifically compelling but commercially misaligned, especially in term of pricing, access and reimbursement.

Third, deal structure itself has become a strategic lever. The growing divergence between orphan and non-orphan transaction types underscores the importance of matching structure to asset profile. Rare disease Licensing and acquisitions models allow non-originating companies to manage uncertainty while preserving optionality, whereas partnerships are increasingly reserved for late-stage or marketed orphan assets with clear portfolio fit and commercial synergies. Capital discipline, rather than control at all costs, is emerging as the defining principle.

Finally, sourcing strategies must evolve. As development in the US and Europe becomes slower and more expensive, dealmakers will need to expand their aperture for where innovation originates. Comfort with non-traditional development geographies, alternative regulatory pathways, and earlier-stage clinical data will increasingly differentiate successful BD&L organizations from those constrained by legacy frameworks. China is quickly emerging as a complementary and increasingly important environment for early-stage orphan drug innovation due to a more permissive development and regulatory environment.

Orphan drugs have moved well beyond their historical role as niche or opportunistic assets. As demonstrated throughout this article, they are now actively reshaping how biopharma companies allocate capital, time transactions, structure deals, and source innovation globally.

The convergence of lower development and commercialization costs, regulatory flexibility, and earlier clinical signal has made orphan drugs particularly well suited to an era defined by capital discipline and portfolio risk management. These characteristics have driven a shift toward earlier-stage transactions, greater use of partnerships and licensing, and a more prominent role for growth-stage companies as intermediaries within the rare disease ecosystem.

At the same time, geographic sourcing patterns are changing. As traditional development centers face rising friction, more efficient early-stage environments are increasingly contributing to rare disease innovation. In this context, orphan drugs are no longer just portfolio hedges against volatility. They have become engines of deal-making that shape who transacts, when they transact, and where innovation originates.

For biopharma leaders, the implication is clear. Competitive advantage in rare disease will accrue not to those who pursue scale alone, but to those who adapt their BD&L strategies to the structural realities of orphan drug development and the evolving global innovation landscape.

Analyses was conducted using data from Global Data (December 2025), following the subsequent methodologies. A.) Deal volume per year is the total count of all pharmaceutical industry transactions (i.e., deals) that occurred globally in a given year from 2004–2024. Sample is subdivided between deals that involved any drug which received an orphan drug designation in the US in any indication (“Orphan”) and deals that did not involve any such drug (“Non-orphan”). B.) Orphan deal prevalence reflects the number of pharmaceutical industry transactions that involved a drug which received an orphan drug designation in the US in any indication, expressed as a percentage of all pharmaceutical industry transactions in the given year from 2005–2024. Orphan approval prevalence reflects the number of assets that received an orphan drug designation in the US in any indication, and were approved in that indication in the US, expressed as a percentage of all assets approved in the given year from 2005-2024. Approvals account for assets approved in their first indication in the US. C.) Median deal value by year is the median value in millions US dollars among all pharmaceutical transactions completed in the given year from 2004–2024. Sample is subdivided between deals that involved any drug which received an orphan drug designation in the US in any indication (“Orphan”) and deals that did not involve any such drug (“Non-orphan”).

Analyses were conducted using data from Global Data (December 2025), following the subsequent methodologies. A) Orphan deal volume by deal type reflects the total count of all pharmaceutical transactions globally involving an asset receiving an orphan drug designation in the US and completed in a given year from 2004–2024, subdivided based on the type of deal. B) Non-orphan deal volume by deal type reflects the total count of all pharmaceutical transactions globally that did not involve any assets receiving an orphan drug designation in the US and completed in a given year from 2004–2024, subdivided based on the type of deal. C) Orphan median deal value is the median value in millions US dollars of all pharmaceutical transactions globally involving an asset receiving an orphan drug designation in the US and completed in a given year from 2004–2024, subdivided based on the type of deal. D) Non-orphan median deal value is the median value in millions US dollars of all pharmaceutical transactions globally not involving an asset receiving an orphan drug designation in the US and completed in a given year from 2004–2024, subdivided based on the type of deal. Merger & acquisition deals are transactions where one company buys or combines with another to obtain ownership and control of assets or capabilities, strategic alliances (licensing) deals are agreements granting rights to use, develop, or commercialize an asset without transferring company ownership, and strategic alliances (partnership) deals are agreements where companies collaborate on development or commercialization of an asset while remaining independent.

Analyses were conducted using data from Global Data (December 2025), following the subsequent methodologies. Merger & acquisition deals are transactions where one company buys or combines with another to obtain ownership and control of assets or capabilities, strategic alliances (licensing) deals are agreements granting rights to use, develop, or commercialize an asset without transferring company ownership, and strategic alliances (partnership) deals are agreements where companies collaborate on development or commercialization of an asset while remaining independent. A) Merger & acquisition deals where the lead asset was an orphan drug represents the breakdown of all pharmaceutical M&A deals completed in the given year (2009, 2014, 2019, 2024) wherein the most valuable (i.e., lead) asset received an orphan drug designation in the US in any indication. Sample is sub-divided based on the development stage of the lead asset at the time the deal was completed, with each value expressed as a percentage of the total M&A deals where the lead asset was an orphan drug occurring in the given year. B) Licensing deals where the lead asset was an orphan drug represents the breakdown of all pharmaceutical licensing deals completed in the given year (2009, 2014, 2019, 2024) wherein the most valuable (i.e., lead) asset received an orphan drug designation in the US in any indication. Sample is sub-divided based on the development stage of the lead asset at the time the deal was completed, with each value expressed as a percentage of the total licensing deals where the lead asset was an orphan drug occurring in the given year. C) Partnership deals where the lead asset was an orphan drug represents the breakdown of all pharmaceutical partnership deals completed in the given year (2009, 2014, 2019, 2024) wherein the most valuable (i.e., lead) asset received an orphan drug designation in the US in any indication. Sample is sub-divided based on the development stage of the lead asset at the time the deal was completed, with each value expressed as a percentage of the total partnership deals where the lead asset was an orphan drug occurring in the given year.

 Analyses were conducted using data from Global Data (December 2025), following the subsequent methodologies. A) Volume of orphan deals by therapeutic area reflects the total number of pharmaceutical transactions globally completed from 2004–2024 wherein all assets involved in the transaction received an orphan drug designation in the US in any indication. Sample is sub-divided based on the primary therapeutic area addressed by the lead asset involved in the deal; for deals with multiple lead assets, the therapeutic area of the asset with the highest development stage at the time of the deal was used. Sample excludes any deals for which the therapeutic area of the lead asset was unavailable in the dataset. B) Volume of orphan deals by molecule class reflects the total number of pharmaceutical transactions globally completed from 2004–2024 wherein any asset involved received an orphan drug designation in the US in any indication. Sample is sub-divided based on the molecule class of the lead asset involved in the deal; for deals with multiple lead assets, the molecule class of the asset with the highest development stage at the time of the deal was used. Sample excludes any deals for which the molecule class of the lead asset was unavailable in the dataset.

 Data were collected through an online quantitative survey designed to capture senior industry perspectives on rare disease business development, including asset divestment, acquisition, and in-licensing decision-making. Respondents were required to meet predefined qualification criteria, including holding a senior-level role, having a minimum of five years of biopharmaceutical industry experience, and possessing demonstrated familiarity with clinical development, commercialization, and portfolio strategy in rare disease. The survey comprised 15 closed-ended questions using multiple-choice, matrix, and ranking formats. It was conducted between September 19 and Sept. 25, 2025, and yielded a final analytical sample of 31 qualified respondents (n=31). Results are presented as mean values across respondents and stratified by company size where applicable. Ranking analyses reflect the relative importance of decision drivers reported by respondents at the point of deal evaluation. Analyses focused on minimum peak sales thresholds considered acceptable for sell-side and buy-side transactions, respectively, as well as the primary factors influencing willingness to transact. All reported values represent aggregate respondent averages unless otherwise noted.

Edwards, N.; Spruce, J.; Bianco, M.; Kim, K.; Law, P.; and Khan, N. Beyond the Odds: The Rare Disease Winning Formula. ARYA Consulting Partners. 

Schmid, E. F. and Smith, D. A. Is Pharmaceutical R&D Just a Game of Chance or Can Strategy Make a Difference? 

Ringel M. S. What Is the Right Amount to Spend on Biopharma R&D? 

Kwisda, S.; Kremer, M.; Sievertsen, N.; Gassmann, O.; Hartl, D.; and Schuhmacher, A. (2025). Does pharma R&D need a strategic reset? Adapting to a changing US landscape. 

Schuhmacher A. Pharma Innovation: How Evolutionary Economics Is Shaping the Future of Pharma R&D. 

Edwards, N. and Bianco, M. The Orphan Advantage: How Rare Disease Assets are Redefining Biopharma Deal Strategy. ARYA Consulting Partners. 

Edwards, Bianco, M.; N.; Spruce, J.; Law, P.; and Khan, N. The Rare Disease Access Gap: Navigating the Economics of Global Innovation. ARYA Consulting Partners. 

 specializes in business development, new product planning, and early brand strategy. He helps companies lay the foundation for successful launches through data-backed strategies. Nathan integrates both commercial and access insights into early asset development to maximize market opportunity.

is a global, full-service boutique consulting firm that partners with biopharma companies to navigate complexity and support growth. We combine deep therapeutic expertise, real-world insights, and end-to-end commercialization capabilities with a people-first approach. Our mission is simple: deliver strategies that create measurable results and lasting impact for our clients, our people, and the communities we serve.

Articles

Orphan drugs are emerging as the most capital-efficient engines of biopharma innovation as US/EU development costs, evidentiary requirements, and timelines expand. Rare-disease programs deliver earlier, clearer clinical signal via smaller trials, concentrated prescriber bases, and regulatory flexibility, enabling earlier value inflection and lower commercialization spend. Deal behavior is diverging by orphan status: partnerships are more rate-sensitive, M&A is more resilient, and licensing/partnership values remain modest due to earlier-stage risk sharing. Phase II has become a preferred transaction point, while sourcing increasingly broadens to China.

In part four of this four-part series on rare diseases, the authors examine how orphan drug economics are reshaping biopharma deal-making behavior, accelerating earlier-stage transactions, and shifting the geography of innovation sourcing, with future rare disease innovation increasingly emerging outside traditional US and European development centers.

Rare Disease Therapies: How Orphan Drugs Are Reshaping Global Biopharma Sourcing

GSK Acquires 35Pharma | Created with Canva

On February 25, 2026, GSK announced an agreement to acquire 35Pharma Inc. for $950 million, signaling a strategic expansion into the specialized cardiopulmonary market, specifically targeting conditions with high mortality and limited therapeutic options.¹ The focus of this transaction is HS235, a protein-based therapeutic designed as an activin signaling inhibitor. This molecule targets the activin receptor signaling pathway, a validated therapeutic target for pulmonary hypertension (PH), a condition affecting approximately 82 million people globally with a five-year survival rate of only 50%. The global market for these therapies is projected to reach $18 billion by 2032, with activin signaling inhibitors expected to represent half of that value.

How Does the Design of HS235 Address Existing Clinical and Manufacturing Challenges?

A primary challenge in developing treatments for PH is managing the risk of adverse events, such as bleeding and telangiectasia, often caused by off-target binding to bone morphogenetic protein 9 (BMP9) and bone morphogenetic protein 10 (BMP10).¹ HS235 is engineered with enhanced selectivity to reduce binding to BMP9 and BMP10, which is particularly relevant for the significant patient population requiring concurrent anticoagulant or antiplatelet therapies. By improving the safety profile through molecular engineering, developers can potentially address a broader patient base while mitigating risks associated with existing ligands. Tony Wood, chief scientific officer, GSK, said in a press release,¹ “Pulmonary hypertension affects millions of people worldwide, yet patients are underserved. We're delighted to add HS235 to our pipeline, a potential best-in-class medicine with a differentiated profile to reduce risk of bleeding and provide potential metabolic benefits clinically relevant to PH patients. HS235's potential protective effects on vascular function, alongside potential benefits on fat-derived markers of metabolism and inflammation, also offer new development opportunities within our RI&I portfolio to achieve broader coverage across the metabolic, inflammatory, vascular and fibrotic drivers of multiple chronic diseases that affect the lung, liver and kidney.”

What Are the Developmental Prospects for HS235 Beyond Vascular Function?

The clinical development of HS235 has progressed through phase I trials in healthy volunteers, with upcoming studies planned for pulmonary arterial hypertension and PH due to heart failure with preserved ejection fraction.¹ These trials will take place in specialized academic centers in which PH programs are integrated into larger respiratory and critical care units. Beyond its vascular benefits, the underlying mechanism of the drug offers unique metabolic advantages, including fat-selective weight loss and improved insulin sensitivity. These secondary benefits are significant for the PH population, in which obesity and insulin resistance are prevalent comorbidities. Ilia Tikhomirov, CEO, 35Pharma, said in a press release,¹ “In recent years, we witnessed a revolution in our understanding of pulmonary hypertension and how this life-threatening disease could be reversed. We are pleased to be joining forces with GSK, a leader in respiratory and inflammatory drivers of disease, who shares our vision of HS235's potential to transform the treatment of this debilitating condition.” The integration of this asset into the respiratory, immunology, and inflammation portfolio provides scalable opportunities to treat chronic diseases affecting the lung, liver, and kidney by addressing metabolic and inflammatory drivers. Completion of the acquisition is subject to standard regulatory clearances in the US and Canada.

How Else Is GSK Advancing Its Drug Portfolio?

GSK recently announced the results of a study involving over 2.5 million adults that indicated Arexvy

, a recombinant respiratory syncytial virus (RSV) vaccine, was 75.6% effective in preventing related hospitalizations involving severe COPD and asthma flare-ups.² The data showed a 63.1% reduction in major adverse cardiovascular events during hospitalization. Significant reductions in severe flare-ups were also observed for patients with chronic obstructive pulmonary disease and asthma. On these findings. Christian Felter, global medical lead, RSV, GSK, said in a press release,² “These data underscore the value of our RSV vaccine in its observed association with helping to prevent RSV-related hospitalization and reducing the risk of acute events of certain chronic conditions associated with this potentially serious disease.” Ultimately, integrating these advancements into a single portfolio allows for broader clinical coverage across the vascular, metabolic, and inflammatory drivers that define modern pharmaceutical strategy.

GSK. GSK enters agreement to acquire 35Pharma Inc. London (UK): GSK plc; 2026. Available at: 

https://www.gsk.com/en-gb/media/press-releases/gsk-enters-agreement-to-acquire-35pharma-inc/

GSK. GSK’s Arexvy associated with reductions in certain RSV-related risks including heart attack, stroke and severe flare-ups of COPD and asthma, real world study shows. London (UK): GSK plc; 2026. Available at: 

https://www.gsk.com/en-gb/media/press-releases/gsk-s-arexvy-associated-with-reductions-in-certain-rsv-related-risks-including-heart-attack-stroke-and-severe-flare-ups-of-copd-and-asthma-real-world-study-shows/

GSK agreed to acquire 35Pharma for $950 million to expand into cardiopulmonary diseases with high mortality and limited options, centered on HS235, a selective activin signaling inhibitor for pulmonary hypertension. HS235 is engineered to minimize BMP9/BMP10 binding to reduce bleeding/telangiectasia risk, important for patients on anticoagulants or antiplatelets, while potentially improving vascular function and metabolic-inflammatory biomarkers. Phase I is complete, with planned studies in PAH and PH-HFpEF. Separately, real-world data linked Arexvy to fewer RSV-related hospitalizations and in-hospital MACE.

GSK acquires 35Pharma for $950M. The primary focus is HS235, a selective PH drug reducing bleeding risk with metabolic benefits for 82 million patients.

GSK in Agreement to Acquire 35Pharma for Nearly $1B

Univar Solutions News -  Created with Canva

Univar Solutions LLC announced on February 24, 2026 that its Ingredients + Specialties from Univar Solutions division has been appointed as the distributor for Ingredion Pharma Solutions, creating an exclusive distribution channel for pharmaceutical starches within the United States and Canada.

 This agreement integrates a specialized portfolio of functional excipients into an extensive distribution network to assist manufacturers in streamlining their supply chains. This transition provides a centralized source for critical materials, such as wet granulation binders, fillers, and lubricants. By centralizing procurement, development teams can reduce vendor complexity while gaining access to a broader range of high-quality ingredients.

"We are thrilled to enhance our product range while deepening our partnership with Ingredion," said Aaron Lee, global senior vice president of Health & Nutrition for Univar Solutions.

 "Through this agreement, we will expand access to pharmaceutical starches and other functional excipients in the United States and Canada, helping our pharmaceutical and nutraceutical customers address formulation challenges and grow their businesses. This partnership truly underscores our commitment connecting customers to high-quality ingredients and customized market solutions, and we are optimistic that the collaboration will help enhance tableting, softgel, and encapsulation applications."

How does this consolidation of supply impact product development?

The collaboration is intended to move beyond simple ingredient delivery by offering technical sales support and deep market knowledge to solve complex formulation hurdles.

 By leveraging facilities that adhere to current good manufacturing practices and providing regulatory assistance, the arrangement helps development teams navigate compliance requirements while maintaining product quality. This level of technical support is vital for professionals tasked with optimizing the stability and efficiency of modern delivery systems. By combining science and innovation with a leading portfolio of specialties, this partnership seeks to find the solutions needed to safely improve lives and communities.

What specific technical solutions are now accessible through this arrangement?

Manufacturers now have expanded access to a variety of specialized excipients, including gelatin replacements for vegetarian softgel designs and emulsions for encapsulation, according to the release from Univar Solutions.

 This includes specific starch-based products like UNI-PURE F, UNI-PURE FL, UNI-PURE GA, and UNI-PURE WG-220, which are engineered to improve formulation performance and overcome technical challenges. The availability of these materials through a single partner allows development teams to focus on ingredient innovation and the creation of high-value products.

"We are excited to expand our partnership with Ingredients + Specialties from Univar Solutions in the pharmaceutical starches, mannitol, and specialty excipients product space," said Joe Rogus, pharma senior sales manager – United States and Canada for Ingredion.

 "They are a world-class organization with an excellent track record in delivering high-value specialty products and services to pharmaceutical markets. We're confident that this collaboration will drive ingredient innovation and deliver exceptional value to our customers."

Integrating these resources allows for more creative approaches to tablet and capsule manufacturing by combining logistical strength with specialized chemical expertise. This strategic alignment ensures that developers have the necessary tools to improve the safety and effectiveness of their pharmaceutical products in a rapidly evolving market.

What other partnership has Univar Solutions made recently?

Earlier this month, the subsidiary Univar Solutions B.V. announced the expansion of its ingredient portfolio through an exclusive glycolic acid distribution agreement with the CABB Group in select European, Middle Eastern, and African markets.

 At the time, Nick Powell, CEO of Ingredients + Specialties from Univar Solutions said, "We are delighted to announce our partnership with the CABB Group in beauty and personal care and home care and industrial cleaning in EMEA, focusing jointly on efficacy and performance for customers and end consumers," said Nick Powell, CEO of Ingredients + Specialties from Univar Solutions. "This strategic alliance enhances our ability to deliver quality, innovative ingredients, and we are confident that together we can deliver greater value by offering glycolic acid solutions that meet the increasing demand for multi-purpose consumer products in the region."

Univar Solutions Selected as Ingredion Pharma Solutions Distributor in United States and Canada

Univar Solutions Expands Ingredient Portfolio with Exclusive Glycolic Acid Distribution Agreement in EMEA

Univar Solutions’ Ingredients + Specialties unit has been appointed exclusive US/Canada distributor for Ingredion Pharma Solutions’ pharmaceutical starches, consolidating access to functional excipients for drug and nutraceutical manufacturers. The model centralizes procurement of key materials used as binders, fillers, and lubricants while pairing distribution scale with technical sales, regulatory support, and cGMP-aligned supply. Expanded access includes starch-based UNI-PURE grades and additional solutions such as vegetarian softgel gelatin replacements and encapsulation emulsions, aiming to reduce vendor complexity and accelerate formulation problem-solving.

Univar Solutions' Ingredients + Specialties division has been appointed as the distributor for Ingredion Pharma Solutions, creating an exclusive distribution channel for pharmaceutical starches within the United States and Canada.

Univar Solutions Selected as Exclusive US and Canada Distributor for Ingredion Pharmaceutical Starches

Elisabeth Gardiner, PhD, is chief scientific officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With more than 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 IND filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Elisabeth held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Elisabeth gained experience in tRNA biology while working at aTyr Pharma and at Scripps Research Institute in the lab of Paul Schimmel. Elisabeth’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. Elisabeth earned her Ph.D. from the University of Wisconsin-Madison and holds a B.S. and an M.S. from Texas A&M University.

The Importance of Drug Development for Rare Diseases

FDA defines rare diseases as “a disease or condition that affects less than 200,000 people in the United States”. 

 The development of treatments for these diseases has unique challenges that can lead to a lack of options for patients. 

spoke with Elisabeth Gardiner, PhD, chief scientific officer of Tevard Biosciences, about these challenges and what makes treatments for these conditions so important.

Because rare disease treatments target a small number of people, there can be little return on investment for pharmaceutical companies. This can be set off by incentives given by regulators and governments,

 but it’s still can be a gamble for some companies. Gardinar suggests that a therapeutic approach that can be applied to multiple rare diseases may create the opportunities companies are looking for that can also help additional people.

“The ironic thing is that when drug hunters think about looking at targets for new drug discovery projects, they really look to rare diseases to get hints for targets and for this deep underlying biology that might be really interesting and exciting,” Gardiner says. “An example of this is PCSK9 inhibitors. They were originally found by looking at familial hypercholesterolemia that was actually quite rare. Homozygous familial hypercholesterolemia was discovered in the 1930s. This observation has yielded not only Nobel Prizes, but some of the most successful drugs that we have. Really understanding the work that was decoded by Brown and Goldstein, you know, that work earned them the Nobel Prize. That was on LDL [low-density lipoprotein] receptor mutations, and essentially, these genetic insights led to the development of PCSK9 inhibitors that can dramatically lower LDL. And in fact, there's even a genetic, medicine now, being brought forward by Verve. It's a CRISPR-based editor. It essentially, it can address familial hypercholesterolemia, but the way it's working in the clinic, it may be applicable to anybody with hypercholesterolemia. It's incredibly exciting.”

https://www.fda.gov/patients/rare-diseases-fda

FDA. Designating an Orphan Drug Product: Drugs and Biological Products. FDA.gov. August 12, 2025. 

https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

Elisabeth Gardiner, PhD, Chief Scientific Officer, Tevard Biosciences

Elisabeth is chief scientific officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With more than 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 IND filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Elisabeth held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Elisabeth gained experience in tRNA biology while working at aTyr Pharma and at Scripps Research Institute in the lab of Paul Schimmel. Elisabeth’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. Elisabeth earned her Ph.D. from the University of Wisconsin-Madison and holds a B.S. and an M.S. from Texas A&M University.

Editor's note: This transcript is a lightly edited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

Hi, I am Elisabeth Gardner. I'm CSO of Tevard Biosciences. There is a significant unmet medical need to address rare diseases. Given the challenges in developing treatments, especially disease-modifying treatments for rare disease, it's critical to be able to have an approach that can efficiently target multiple rare diseases.

I think that part of the problem is, you know, when you're going after a single rare disease, you don't actually get as much, bandwidth, much addressable population. So, if you have a therapeutic approach that can actually address multiple rare diseases, potentially rare diseases with the same sort of underlying genetic mechanism, then you really have an excellent opportunity to help a lot more people.

And I think rare diseases are very poorly named. It's only rare until it's your loved one that has the rare disease, and then all of a sudden you realize that you're part of this club that you really didn't want to be part of, and you're suddenly part of a really large community of people that are desperate for answers and desperate for cures.

The ironic thing is that when drug hunters think about looking at targets for new drug discovery projects, they really look to rare diseases to get hints for targets and for this deep underlying biology that might be really interesting and exciting.

An example of this is PCSK9 inhibitors. They were originally found by looking at familial hypercholesterolemia that was actually quite rare. Homozygous familial hypercholesterolemia was discovered in the 1930s.

This observation has yielded not only Nobel Prizes, but some of the most successful drugs that we have. Really understanding the work that was decoded by Brown and Goldstein, you know, that work earned them the Nobel Prize.

That was on LDL receptor mutations, and essentially, these genetic insights led to the development of PCSK9 inhibitors that can dramatically lower LDL.

And in fact, there's even a genetic, medicine now, being brought forward by Verve, Verve one oh one. It's a CRISPR-based editor. It essentially, it can address familial hypercholesterolemia, but the way it's working in the clinic, it may be applicable to anybody with hypercholesterolemia. It's incredibly exciting.

I think thinking about rare diseases, they provide insights for the larger population, but, I mean, there's just a huge unmet need. There's so many rare diseases out there.

There are three main challenges that I think everyone working on rare disease encounters. One is the lack of reliable models, either animal models or cellular models. Two is the complex biology of the disease, and three is the lack of funding. So many rare diseases do not have a cellular animal model that can permit drug screening or testing to be done in a way that's actually translational to the human condition.

It's tough, actually, to know if one cell line that is available or one mouse model that you might have access to is going to be representative of the human pathology that you're trying to treat.

It's a really black box. I mean, it's a lot of guessing. It's expensive and time-consuming to actually develop the models that are most relevant, and money and time are often in very short supply when you're trying to be innovative, and certainly, the patients can't wait that long.

One of the reasons why rare disease, medications take so long to get to the clinic is because we have very specific guidelines about how we bring drugs to the clinic, and part of that is, you know, we need a cellular model, we need an animal model, we need to do the toxicity testing, and then we can put it in humans.

I think all of that is, is really important, but when you talk about rare disease, you really need to think out of the box. I love actually what the FDA is doing right now. They're really trying to streamline the process for rare disease. They're well aware that the animal models may not be there.

They're focused on cell model development, and in fact, there's a whole coalition that's kind of working on not just thinking about how to move rare disease medications forward, but really thinking about how to build the cellular models, how to build the pediatric models, that, that might be key for, for getting things, into the clinic without having to build the animal models. And so, changing that timeframe, you know, changing that delay.

So, for rare disease research and development, the lack of funding is really a tricky thing. I think often this comes because of the complex biology of the disease, so investors may be loath to jump into investing in something that has such complex biology.

They look at it, and they think: "That's gonna take a long time for me to get return on my investment." And I think that's logical.

But when, when you think about it, you know, genetic diseases are not just one thing. It may be just one gene that is the causative gene that's making the pathology relevant to the patient, but often you're not dealing with a simple situation.

So that one genetic malformation may cause a whole cascade of biological problems, and so you're dealing with a potentially very fragile patient population.

And so that's another reason why people may have difficulty, actually getting into rare disease biology and thinking about it from a drug discovery perspective.

Often, patients are on another type of drug to manage symptomology, and this can actually mask the benefit of a potential new medication. It may actually exacerbate the condition, too.

So, for example, you may be on a genetic therapy that's very helpful to cure your disease by modifying the gene, but if that genetic therapy is causing liver damage, that could kill you. And so, you may have a patient that ultimately could be helped, but ends up having an exacerbation that leads to a lifetime of illness, unrelated to the original genetic cause.

So, I think that there's a lot of problems, you know, this sort of, this thought process that I have, you know, this one mutation that's caused me to have epilepsy.

But all of my organ systems are now disturbed because my epilepsy has actually damaged my brain, and so that could be GI issues, that could be verbal issues, that could be issues responding to other medication because brain chemicals are changed by the constant inflammation.

There's a lot of reasons why the complex biology of the disease just makes rare diseases very tricky, and that gets to the lack of funding. So, it really takes a lot of money and highly trained people to make a rare disease treatment work well.

One example, quite recent, that was highly successful was the Baby KJ story. This was highly publicized. It was truly a tremendous scientific effort for one baby, you know, with one rare disease. And stories like this catch the attention and, you know, really fill people with a lot of hope and excitement.

But the reality is that this took a tremendous amount of money and a tremendous amount of effort, and I would say altruism, on the part of a large number of people.

If you look at the paper, there's a lot of names, you know, on the author list, but everybody behind that, you know, this kid's parents, all the caregivers, all the nurses, therapists, rehab folks, everybody who worked with this kid, and, I mean, he didn't spend one night at the hospital.

He spent a long time at the hospital trying to make sure that the therapy was working and actually working through any, potential ramifications of the genetic, medication that he got.

I was so impressed when I read this because what a tour de force, right?

But a success story like this really hammers the point home that this is big business. I mean, it requires a lot of money to work efficiently. They took so little time to get that done, and honestly, the FDA worked with them in a tremendously efficient way.

But when you're an investor, you think, "Okay, that's just one patient. How am I going to get return if I throw millions of dollars at this? Will I be getting millions of dollars back? Will I be getting billions of dollars back? Is there that big a market?"

I think that the way that we can think about this is, you know, a lot of times you think, "Well, I can charge three million dollars for a single treatment," but when you're the patient, or the patient's family, and, or an insurer, and that's your option, you may not be very interested in that. I mean, that may be out of reach.

And so, I think that we need to be able to think long term and think about expanding out from the original patient population into the broader population, similar to the PCSK9 story. You know, from an investor standpoint, understanding how much return on investment you're going to get is a really sensible question. But I think it's important to be creative and having a real understanding about the complex biology so that you can expand and show the investors that we'll treat these 10 patients or maybe these 100 patients, but this potential therapy can be broad spectrum.

You know, we can really look across a lot of disease states if we can actually tackle this one disease state.

Videos

Elisabeth Gardiner, PhD, CSO of Tevard Biosciences, explains some of the challenges associated with the development of treatments for rare diseases.  

Lars Hahn is senior vice-president, Global Sales Organization, at Vetter.

In a world where complexity meets urgency, biopharmaceutical leaders are discovering that the true catalyst for innovation isn’t built on technology alone but driven from trust. Long-term partnerships with contract development manufacturing organizations (CDMOs) are emerging as the cornerstone of resilient, efficient, and patient-focused drug development and market supply. When biopharmaceutical and biotech companies come together with their fill/finish partners, products gain a competitive edge founded on reliability, resilience, and strategic insight.

During a time when the global biopharmaceutical landscape is evolving rapidly, there has been a major shift in the industry with new technologies, innovative molecules, and elevated expectations reshaping how we develop and deliver therapies to patients all around the world. This offers the industry a significant opportunity. The rise of 

, biologics, and the growing role of smaller biotech companies require everyone in the industry to rethink the need for strategic, trust-based partnerships.

There is an old African proverb, “If you want to go fast, go alone. If you want to go far, go together.” This perfectly reflects the 

 in biopharma. Success in developing, producing, and delivering medicines depends on strong collaboration. Companies that invest in and strengthen these partnerships will be better prepared for the changes and challenges ahead.

How are injectables reshaping the industry?

More than 60% of today’s R&D drug pipeline consists of injectables, and this share is still growing (1). This rise is connected to the fact that the most promising therapeutic areas like cell and gene therapies, biologics, or messenger RNA therapies mostly require injectable delivery systems.

Small biotech companies with one or two drug candidates are an important source of innovation with US biotech companies dominating this market internationally. Overall, 85% of all new drugs in 2024 came from these players, and 63% of them launched successfully (2,3). At the same time, financing these projects for those companies has become increasingly challenging.

To be able to keep up with the global bio/pharmaceutical industry’s growth, outsourcing is on the rise, and small biotech companies initiated 63% of clinical trials in 2024 compared to 26% by large pharma, down from 47% in 2015 (4). All these trends show one key takeaway, which is that, as injectables become more popular, CDMOs are becoming even more critical enablers of market acceleration. 

Small biotech companies with one or two drug candidates are an important source of innovation with US biotech companies dominating this market internationally.

The partnership imperative: from supplier to strategic ally

Long-term collaboration isn’t about transactions, it’s about trust, foresight, and shared growth. True partnership is based on six pillars:

Trust-based, long-term CDMO partnerships are becoming central to biopharmaceutical resilience as pipelines skew toward complex injectables and smaller biotechs drive innovation amid tighter financing. With injectables now comprising over 60% of R&D programs, outsourcing intensity is rising and elevating fill/finish providers from transactional suppliers to strategic allies. Effective partnerships rest on shared vision, expertise, risk anticipation, speed, quality, and transparency. Partner selection increasingly hinges on regulatory depth, technical flexibility, uncompromising quality, scalability, and cultural fit, aligning with a third biotech wave emphasizing deeptech, sustainability, and co-innovation.

Trust-based CDMO partnerships are becoming essential to accelerate injectable drug development, manage risk, and ensure resilient biopharma supply.

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