Named-patient programs & compassionate use

01Overview and value chain

Markers: [EC: 21 CFR 312 Subpart I / EU Reg 536/2014 Art 83 / PRC Drug Administration Law Art 23 | OECD: bio-pharma | Regulator: FDA (US), EMA (EU), NMPA (CN)]

Named-patient programs (NPP) and compassionate use — also called expanded access (US), managed access or early access — are regulated pathways that let patients with a serious or immediately life-threatening condition receive an investigational biopharmaceutical before marketing approval, when they cannot enrol in a clinical trial and have no comparable or satisfactory alternative. The need is structural: rare disease alone affects roughly 350 million people worldwide, 95% of whom have no approved treatment, and carries an estimated US$1.25 trillion annual burden across the US and Europe; orphan drugs are projected to reach about 21% of global prescription sales (~US$409 billion) by 2032. Access friction is severe even after approval — in 2025 only 12% of new-to-brand specialty prescriptions were filled on the first try and nearly 80% were rejected upfront on prior-authorisation or step-therapy grounds — which makes pre-approval pathways, and the patient-support infrastructure around them, a distinct commercial service layer. Sponsors retain product responsibility while specialist operators (Clinigen, IQVIA, Syneos Health, Parexel, Premier Research, Tigermed) run the day-to-day program: eligibility adjudication, FDA/EMA/NMPA submission, GMP batch sourcing, import-clearance and named-patient distribution, and safety surveillance, typically delivering a decision on a compassionate-use request within a few business days of a complete physician submission.

The key directions of named-patient programs and compassionate use are:

1. Regulatory submission and eligibility adjudication: assembling the FDA Form 3926 single-patient IND (or intermediate/population IND), the EMA CHMP compassionate-use opinion under Article 83 of Regulation 536/2014, or the NMPA expanded-clinical-trial pathway under Article 23 of the PRC Drug Administration Law, each requiring proof of serious disease, exhaustion of alternatives and ineligibility for ongoing trials.
2. Physician-initiated access and medical review: the treating physician attests to medical necessity and there is no satisfactory alternative; the sponsor’s medical team independently confirms eligibility, establishes a dosing rationale and confirms informed consent and ethics-committee (IRB/IEC) approval before any drug ships.
3. GMP drug sourcing and global distribution: operators such as Clinigen release a GMP-compliant batch under named-patient labeling, secure export/import licences and QP certification, and route temperature-controlled product direct-to-patient or via a specialty pharmacy, frequently for therapies approved in only one jurisdiction (for example IZCARGO, approved solely in Japan, supplied worldwide via Clinigen’s Named Patient Supply Program).
4. Pharmacovigilance and real-world evidence (RWE): every treated patient enters a safety database with expedited serious-adverse-event reporting to FAERS/EUDRAVIGILANCE, and the accumulated data increasingly informs regulatory filings and payer value dossiers — turning compassionate use into a structured RWE-generation engine rather than a one-off favour.

Sectoral value chain

[Eligibility & medical justification] ──> [Regulatory & ethics submission] ──> [Sponsor medical review]
                                                                                       │
                                                                  (FDA IND / EMA CU / NMPA Art 23)
                                                                                       │
   [Patient supply & PV reporting] <─── [QP release & named-patient distribution] <── [GMP batch sourcing]

Value chain levels

Cross-cutting technologies of the sector:

• Expanded-access regulatory pathway: the harmonised set of FDA Form 3926 / EMA CTIS compassionate-use / NMPA Art 23 instruments that legalise pre-approval treatment.
• Patient support program (PSP) platforms: IQVIA’s Longitudinal Access and Adherence Data (LAAD) and direct-to-patient hubs that integrate benefits verification, adherence tracking and affordability support.
• Real-world evidence (RWE) & AI analytics: Syneos Health’s Kinetic engine and Thelansis-style RWE frameworks that convert compassionate-use caseloads into regulator- and payer-grade evidence.

02US

The United States operates the most permissive and highest-volume expanded-access regime in the world, codified in 21 CFR Part 312 Subpart I and operationalised through the FDA’s Expanded Access Program and the Oncology Center of Excellence’s Project Facilitate, with a mature commercial-cRO ecosystem handling the day-to-day program execution.

FDA expanded access (21 CFR 312), Project Facilitate, IQVIA/Syneos commercialization, Kexing biosimilar collaboration

• FDA Expanded Access Program & Project Facilitate: 21 CFR Part 312 Subpart I defines individual/intermediate/population INDs; the FDA Oncology Center of Excellence’s Project Facilitate shepherds single-patient oncology requests and in 2026 underpinned EAPs such as the CytoDyn leronlimab program in triple-negative breast cancer (first patient dosed 27 April 2026) and the Revolution Medicines daraxonrasib (RMC-6236) EAP in metastatic pancreatic adenocarcinoma (posted 7 May 2026).
• IQVIA (NYSE: IQV, Research Triangle Park): the largest commercialization partner for named-patient and patient-support programs, drawing on data from more than 5,000 supported launches and the LAAD dataset; on 13 May 2026 it expanded its AI-enabled biosimilar collaboration with Kexing Biopharm (688136.SH) spanning clinical, regulatory and commercialization services.
• Syneos Health (Morrisville, NC): pairs early-access pathway design for CNS and oncology assets with its Kinetic AI commercial-intelligence engine, whose KAI Conversations conversation-intelligence module is already used by 10 of the top 20 global pharmaceutical companies.

03CN

China operationalised compassionate use comparatively recently, through Article 23 of the 2019 Drug Administration Law and the 2020 GCP framework, which permit expanded clinical use (“拓展性临床试验”) of an investigational drug in a serious/life-threatening disease with no viable alternative, with domestic CROs such as Tigermed providing the regulatory scaffolding.

NMPA expanded-access pathway (Art 23), Tigermed CRO services, BeiGene/HUTCHMED early access, domestic biopharma outreach

• NMPA compassionate-use framework: Article 23 of the PRC Drug Administration Law and NMPA expanded-clinical-trial guidance allow pre-approval treatment when no satisfactory alternative exists, with risk-benefit adjudication by NMPA and the ethics committee; the framework balances humanitarian access against corporate drug-development risk.
• Tigermed (SZSE: 300347): the leading Chinese CRO providing the regulatory and operational scaffolding for domestic and cross-border expanded-access requests, including eligibility adjudication, NMPA submission and safety reporting for sponsors running Chinese compassionate-use programs.
• Domestic biopharma early access: companies such as BeiGene and HUTCHMED use expanded-access and named-patient mechanisms to give late-line oncology patients access to investigational assets while their China filings mature, frequently paired with Tigermed- or IQVIA-supported global program design.

04EU

The European Union frames compassionate use at two levels — a centralised CHMP opinion under Article 83 of Regulation (EU) 536/2014 for EU-wide serious cross-border threats, and national early-access programs run by individual member states — anchored by Clinigen (UK) as the dominant Managed Access operator and by CROs such as Parexel and Premier Research.

EU Art 83 compassionate use, Clinigen Managed Access, EMA CTIS, JCR IZCARGO NPS

• Clinigen (Ashbourne, UK): the global Managed Access and Named Patient Supply leader, operating end-to-end EAPs that include regulatory oversight, logistics and access management; in 2026 it ran the Ionis zilganersen EAP for Alexander disease (NCT07487389, posted 23 March 2026), the Karyopharm selinexor EAP for cancer (with Caligor Coghlan) and — under agreement with JCR Pharmaceuticals — the IZCARGO (pabinafusp alfa) Named Patient Supply Program for MPS II, opening a Japan-only-approved enzyme therapy to global early access.
• EMA compassionate-use instrument: Article 83 of Regulation 536/2014 lets CHMP issue a compassionate-use opinion for cohorts of patients with a serious condition and no satisfactory alternative, logged in EMA’s CTIS, while member states retain their own national early-access schemes (French ATU, UK EAMS, German Hardship Program).
• Parexel (Raleigh, NC, EU heritage) & Premier Research: Parexel brings 40 years and 22,000+ staff to early-access and oncology development (560+ oncology studies, 104,000 patients, 26,000 sites, 50+ countries over five years), while Premier Research’s Virtual Research Group supports late-phase and named-patient programs with remote and hybrid trial delivery.

05Leading companies and research institutes

06Tech stack and innovations

The named-patient service stack is a digital-regulatory compound: a patient-support and data platform layered onto a GMP drug-supply and pharmacovigilance backbone, increasingly instrumented with AI.

1. Patient support program (PSP) and direct-to-patient (DTP) platforms:
   • IQVIA’s Longitudinal Access and Adherence Data (LAAD) links de-identified patient-support records to broader healthcare datasets, letting first-in-class sponsors benchmark abandonment and persistence against analog launches; the platform supports benefits verification, co-pay assistance and adherence outreach across more than 5,000 historical launches.
   • Direct-to-patient (DTP) models embed distribution in commercialization strategy from day one, with hub services cutting the friction behind the stark 2025 finding that only 12% of new-to-brand specialty prescriptions filled on the first try while ~80% were rejected upfront on prior-authorisation grounds.
2. Real-world evidence (RWE) and AI analytics:
   • Syneos Health’s Kinetic commercial-intelligence engine (launched 2020) fuses proprietary and partnership data; its KAI Conversations module turns field interactions with healthcare providers into structured intelligence and is used by 10 of the top 20 global pharmaceutical companies.
   • Asset-specific early-access design (as in Thelansis’s CNS case study) uses longitudinal claims and real-world data to size the eligible population, quantify untreated-disease intervals and weigh named-patient, managed-access and compassionate-use options against payer expectations and pricing risk.
3. Regulatory submission and pharmacovigilance systems:
   • EMA’s CTIS logs compassionate-use opinions and cohort treatment plans (e.g. the Novartis pelacarsen/TQJ230 Managed Access Program for atherosclerotic cardiovascular disease, posted 22 May 2025), while FDA Form 3926 and the single-patient IND pipeline track US expanded access.
   • Safety surveillance flows into FAERS (US) and EUDRAVIGILANCE (EU), with Parexel-grade pharmacovigilance frameworks turning expedited SAE reports into the evidence packages that support later marketing applications and payer value dossiers.

07Value chains and production pipelines

Operational pipeline of a single-patient compassionate-use request — from treating-physician attestation through FDA Form 3926 / EMA Art 83 / NMPA Art 23 submission, Clinigen GMP sourcing and QP release to named-patient delivery and pharmacovigilance (GCP / GMP / cGDP)

┌───────────────────────────┐      ┌───────────────────────────┐
│ 1. Patient identification │ ───> │ 2. Regulatory & ethics    │
│    & eligibility screening│      │    submission             │
└───────────────────────────┘      └───────────────────────────┘
                                                  │
                                                  ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 4. GMP batch sourcing &   │ <─── │ 3. Sponsor medical review │
│    QP release             │      │    & treatment-plan sign-off│
└───────────────────────────┘      └───────────────────────────┘
               │
               ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 5. Import clearance &     │ ───> │ 6. Treatment, SAE         │
│    named-patient delivery │      │    monitoring & RWE capture│
└───────────────────────────┘      └───────────────────────────┘

Stage 1: patient identification and eligibility screening

The treating physician documents a serious or life-threatening diagnosis, confirms that approved therapies have failed or are intolerable and establishes that the patient cannot enrol in an ongoing clinical trial; the sponsor’s medical team validates this against the investigator brochure and prior-therapy history to produce a confirmed eligibility dossier.

Stage 2: regulatory and ethics submission

A complete request is filed — FDA Form 3926 single-patient IND in the US (21 CFR 312 Subpart I), a CHMP compassionate-use opinion under Article 83 of EU Regulation 536/2014, or an NMPA expanded-clinical-trial request under Article 23 of the PRC Drug Administration Law — alongside IRB/IEC approval and informed consent, with regulators typically returning a decision within 30 days (often far faster for emergency single-patient requests).

Stage 3: sponsor medical review and treatment-plan sign-off

The sponsor’s medical lead independently verifies medical necessity, confirms the benefit-risk justification against the cumulative safety database, fixes the dosing regimen and registers the patient with the program operator (e.g. Clinigen), producing a signed named-patient treatment plan.

Stage 4: GMP batch sourcing and QP release

A GMP-compliant investigational batch is allocated, labeled for named-patient use, released by a Qualified Person (EU) or equivalent, and packaged with the required export and import licences — the step that lets a therapy approved in only one country, such as JCR’s IZCARGO in Japan, reach an eligible patient abroad.

Stage 5: import clearance and named-patient delivery

Temperature-controlled logistics route the product direct-to-patient, to the treating site or via a specialty-pharmacy hub; import customs and national early-access notifications are cleared and hub services handle benefits verification and reimbursement so that treatment can start without administrative delay.

Stage 6: treatment, SAE monitoring and real-world evidence capture

The patient is treated under the named-patient protocol while serious adverse events are reported on an expedited basis to FAERS/EUDRAVIGILANCE; dosing, outcomes and long-term follow-up are captured in a safety database and increasingly packaged as real-world evidence that informs later marketing applications, payer value dossiers and label expansions.