Drug & vaccine development

01Overview and value chain

Markers: [EC: EU pharmaceutical legislation reform (2023-2024) | OECD: Bio-pharmaceutical R&D systems | Regulator: FDA (US), EMA (EU), NMPA (China)]

Drug and vaccine development is the multi-stage pipeline — target identification, hit-to-lead screening, lead optimization, preclinical testing, then Phase I-III clinical trials and regulatory submission — that converts a validated biological target into an approved medicine, at a cost of $0.5-3.5 billion and 7-15 years per product with a success probability of just 10-30% depending on product type. Moderna and Merck’s personalized mRNA cancer vaccine, intismeran autogene (formerly mRNA-4157/V940), encodes up to 34 patient-specific tumor neoantigens delivered via lipid nanoparticles; June 2026 data showed a 49% lower melanoma recurrence risk when combined with checkpoint inhibitor therapy, positioning it for regulatory submission. BioNTech, having pivoted its mRNA platform from infectious disease toward oncology, presented late-stage pipeline data at the 2026 ASCO and ELCC conferences for candidates including pumitamig and gotistobart, alongside multiple lung-cancer trial-in-progress programs. In China, CanSino Biologics continues advancing its proprietary mRNA vaccine platform, reporting Phase II safety and immunogenicity data for its CS-2034 mRNA vaccine in Chinese adults. Eli Lilly has parlayed its GLP-1 dual-agonist tirzepatide (Mounjaro/Zepbound) into a portfolio strategy spanning diabetes, obesity and expanded cardiometabolic indications, reinforcing its position at the January 2026 JPMorgan Healthcare Conference even amid looming patent challenges. The regulatory landscape is itself shifting fast: the EU’s 2023-2024 pharmaceutical legislation reform proposes cutting standard data-exclusivity periods from 8 to 6 years while offering the 2 years back only to companies launching simultaneously across all 27 member states, a plan the EFPIA industry federation has sharply criticized as weakening European competitiveness against the US; China’s 2015 IND reform shifted from a permission-based to a notification-based system, driving a fivefold increase in clinical trials and pushing the country’s active trial count (4,700+) to second globally behind the US (12,000+).

The key directions of drug and vaccine development are:

1. Target identification and hit-to-lead screening: AI-assisted molecular library screening to identify and validate biological targets, then discover initial lead compounds.
2. Phase-based clinical development: sequential Phase I (20-100 volunteers, safety), Phase II (100-500 patients, efficacy) and Phase III (1,000-5,000 patients, pivotal) trials building the evidence base for regulatory approval.
3. Personalized mRNA vaccine platforms: patient-specific neoantigen-encoding mRNA vaccines manufactured individually from each patient’s tumor biopsy, extending mRNA technology from infectious disease into oncology.
4. Regulatory-acceleration pathways: FDA Breakthrough Therapy/Fast Track/RTOR, EMA’s proposed exclusivity reform, and NMPA’s Priority Review (cutting review time from 180 to 60 days) all competing to shorten time-to-market for qualifying candidates.

Sectoral value chain

[Target identification & validation] ──> [Hit-to-lead screening (AI-assisted)] ──> [Lead optimization]
                                                                                              │
                                                                                 (Preclinical in vitro/in vivo testing)
                                                                                              │
     [Approval & market launch → Phase IV surveillance] <──── [Regulatory submission (NDA/BLA/MA)] <─── [Phase I → II → III trials]

Value chain levels

Cross-cutting technologies of the sector:

• AI-assisted target and molecule design: generative design algorithms applied to both small-molecule drug candidates and mRNA vaccine sequence/neoantigen selection, compressing early-stage discovery timelines.
• Personalized mRNA cancer vaccine manufacturing: synthesizing an individual mRNA vaccine encoding a specific patient’s tumor neoantigens from biopsy sequencing data, requiring per-patient rather than per-batch production logistics.
• Lipid nanoparticle (LNP) delivery: the delivery vehicle that made mRNA vaccines and personalized cancer vaccines clinically viable, protecting mRNA payloads through cellular uptake and endosomal escape.

02US

The United States remains the world’s largest drug-development ecosystem by R&D spending and active trial volume, anchored by FDA acceleration pathways and blockbuster platform economics.

Moderna/Merck’s personalized cancer vaccine data, Eli Lilly’s GLP-1 portfolio strategy, FDA acceleration pathways

• Moderna and Merck: their personalized mRNA cancer vaccine intismeran autogene showed a 49% lower melanoma recurrence risk in June 2026 data when combined with checkpoint inhibitor therapy, encoding up to 34 patient-specific tumor neoantigens per treatment.
• Eli Lilly: has parlayed its GLP-1 dual-agonist tirzepatide (Mounjaro/Zepbound) into a broader portfolio strategy spanning diabetes, obesity and expanded cardiometabolic indications, reinforcing its position at the January 2026 JPMorgan Healthcare Conference even amid looming patent challenges.
• FDA acceleration pathways: Breakthrough Therapy, Fast Track and Real-Time Oncology Review (RTOR) designations continue to compress review timelines for qualifying candidates, part of why the US maintains the world’s largest active clinical trial count (12,000+) and pharmaceutical R&D investment (~$220 billion annually).

03CN

China has become the world’s second-largest clinical trial ecosystem following its 2015 IND reform, with domestic mRNA vaccine platforms now competing internationally.

CanSino’s mRNA vaccine platform, 2015 IND reform impact, NMPA Priority Review acceleration

• CanSino Biologics: continues advancing its proprietary mRNA vaccine platform, reporting Phase II safety and immunogenicity data for its CS-2034 mRNA vaccine in Chinese adults, building on its earlier position as a global pioneer of adenoviral-vector vaccines.
• 2015 IND reform impact: China’s shift from a permission-based to a notification-based investigational new drug system drove a fivefold increase in clinical trial volume, pushing the country’s active trial count (4,700+) to second globally behind the US.
• NMPA Priority Review: an accelerated regulatory pathway for oncology and rare-disease candidates cuts standard review time from 180 to 60 days, part of a broader post-2015 reform package that has pushed China’s annual innovative-drug approvals up roughly 40% since 2020.

04EU

The European Union hosts a world-class pharmaceutical R&D base now navigating its most significant legislative reform in two decades, alongside a genuine mRNA-oncology pivot.

BioNTech’s oncology pipeline pivot, EU pharmaceutical legislation reform, EFPIA industry pushback

• BioNTech: having built its reputation on COVID-19 mRNA vaccines, presented late-stage oncology pipeline data at the 2026 ASCO and ELCC conferences for candidates including pumitamig and gotistobart, alongside multiple lung-cancer trial-in-progress programs, reflecting the platform’s pivot from infectious disease toward cancer immunotherapy.
• EU pharmaceutical legislation reform: the European Commission’s 2023-2024 proposal would cut standard data-exclusivity periods from 8 to 6 years, restoring the 2 years only to companies launching a product simultaneously across all 27 EU member states rather than staggering rollout by market size.
• EFPIA pushback: the European pharmaceutical industry federation has sharply criticized the reform as weakening European competitiveness relative to the more patent-holder-friendly US legal environment, a live policy dispute shaping where global drug developers prioritize their earliest-market launches.

05Leading companies and research institutes

06Tech stack and innovations

The drug and vaccine development stack combines computational discovery tools with the clinical and regulatory infrastructure needed to prove safety and efficacy at scale:

1. AI-assisted hit-to-lead screening:
   • Generative and predictive machine-learning models screen virtual compound libraries and mRNA sequence/neoantigen combinations far faster than traditional wet-lab screening, compressing the discovery phase that historically consumed years of a drug’s development timeline.
2. Personalized mRNA cancer vaccine manufacturing:
   • Each patient’s tumor biopsy is sequenced to identify tumor-specific neoantigens, which are then encoded into an individually synthesized mRNA vaccine formulated in lipid nanoparticles — a fundamentally different manufacturing logistics model than standard batch-produced pharmaceuticals.
3. Phase-based clinical trial design:
   • The sequential Phase I (safety, 20-100 volunteers), Phase II (efficacy, 100-500 patients) and Phase III (pivotal confirmation, 1,000-5,000 patients) structure remains the core evidentiary framework regulators require, even as accelerated pathways compress timelines between phases for qualifying candidates.

07Value chains and production pipelines

Industrial pipeline for developing and launching a personalized mRNA cancer vaccine

┌───────────────────────────┐      ┌───────────────────────────┐
│ 1. Tumor biopsy &          │ ───> │ 2. Neoantigen prediction   │
│    sequencing                   │      │    (AI-assisted)                │
└───────────────────────────┘      └───────────────────────────┘
                                                 │
                                                 ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 4. LNP formulation &        │ <─── │ 3. Individual mRNA         │
│    fill-finish                   │      │    sequence synthesis           │
└───────────────────────────┘      └───────────────────────────┘
              │
              ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 5. Combination-therapy     │ ───> │ 6. Regulatory submission &  │
│    clinical administration      │      │    market launch                │
└───────────────────────────┘      └───────────────────────────┘

Stage 1: Tumor biopsy and sequencing

A sample of the patient’s tumor is biopsied and subjected to next-generation sequencing to identify the specific genetic mutations present in that individual’s cancer.

Stage 2: Neoantigen prediction (AI-assisted)

Machine-learning algorithms analyze the sequencing data to predict which mutated proteins (neoantigens) are most likely to trigger a strong immune response, selecting up to several dozen candidate targets unique to that patient’s tumor.

Stage 3: Individual mRNA sequence synthesis

An mRNA sequence encoding the selected patient-specific neoantigens is synthesized on a per-patient basis, a manufacturing model fundamentally different from standard batch production of identical doses.

Stage 4: LNP formulation and fill-finish

The synthesized mRNA is encapsulated in lipid nanoparticles that protect it during cellular uptake and enable endosomal escape, then filled into patient-specific dose vials under GMP conditions.

Stage 5: Combination-therapy clinical administration

The personalized vaccine is administered alongside checkpoint-inhibitor immunotherapy, training the patient’s immune system to recognize and attack cells bearing the identified tumor neoantigens.

Stage 6: Regulatory submission and market launch

Following successful Phase III trial results demonstrating a clinically meaningful benefit (such as reduced recurrence risk), the sponsor compiles and files a regulatory submission before market launch and Phase IV post-marketing surveillance.