What Is Compassionate Use and How Does It Work in Latin America, Africa and Asia?

_{Key Takeaways}

• Compassionate use is a regulated route through which patients with serious or life-threatening conditions and no satisfactory approved alternative can receive unapproved or unregistered medicines outside a clinical trial.

• The same idea carries different names and legal machinery across markets: compassionate use (EU), expanded access (US), named patient programs, and special access schemes (Australia, Singapore).

• Asia has the most codified frameworks; Latin America leans on named-patient import authorizations, with Brazil the most developed; Africa mostly works through general unregistered-medicine provisions.

• The practical differences come down to three questions: one patient or a cohort, a drug still in trials or one approved elsewhere, and notification versus prior authorization.

• The rules are moving: Brazil's RDC 38/2013 has been under revision since 2023, and the African Medicines Agency treaty, in force since November 2021, targets regulatory harmonization across Africa.
  

_{What is compassionate use?}

Compassionate use is a regulated, ethical route through which a patient with a serious or life-threatening illness, and no satisfactory approved treatment left to try, can receive a promising medicine that has not yet been approved, or is not registered in their country, outside a clinical trial. For such a patient, the years a medicine spends in development and registration are years they may not have, and compassionate use exists to bridge that gap. The principle is close to universal, but the names, the legal basis and the mechanics vary widely from one market to the next, and that variation is where most of the confusion lies.

_{Why does compassionate use exist?}

New medicines take years to develop and register, and because regulators do not harmonize their requirements, a drug cleared by the FDA or EMA can remain unavailable in other markets long afterward. Compassionate use bridges that gap. Wherever it operates, the eligibility tests are strikingly consistent: a serious or life-threatening condition, no satisfactory approved alternative, a patient who cannot join an ongoing trial, enough evidence that the medicine is reasonably safe and effective, informed consent, and a physician who takes clinical responsibility.

_{What is the difference between compassionate use, expanded access and named patient programs?}

Even though the underlying idea is the same, regulators built it into different legal instruments, so the labels rarely line up across countries.

Compassionate use is the EMA's term, and in the EU it refers specifically to a cohort pathway: treatment for a group of patients with a medicine still in development, under Article 83 of Regulation (EC) No 726/2004. Its US counterpart is expanded access, which the FDA runs through three routes: individual patients (including emergencies), intermediate-size populations, and larger treatment protocols. A named patient program is narrower, covering a physician's request to import an unlicensed product, often one already approved abroad, for a single named individual. Special access scheme, or route, is the term of choice in Australia and Singapore.

_{What is early access, and how is it different?}

Early access is the broadest label and the one most often misused. France shows why the distinction matters: after replacing its old ATU system in July 2021, it now runs two separate tracks. Accès précoce (AAP) covers genuinely innovative medicines expected to reach the market, while accès compassionnel (AAC) covers named-patient use of products that meet an unmet need without necessarily being innovative. Across all these terms, the practical divide comes down to three questions: one patient or a cohort, a drug still in trials or one approved elsewhere, and notification versus prior authorization.

_{How does compassionate use work in Asia?}

Asia has the most codified frameworks, though they remain diverse. Australia's Special Access Scheme sorts requests into three categories: A and C are notification-only, while B requires prior TGA approval. Singapore's Special Access Route targets a 14-working-day decision. Japan channels compassionate use through expanded access clinical trials, a system live since 2016 that runs each request much like a small clinical trial. China codified compassionate use in Article 23 of its 2019 Drug Administration Law and adds regional import pilots such as Hainan and the Greater Bay Area. India published draft rules in June 2020 (GSR 354(E)) that would let hospitals import a drug in Phase 3 anywhere in the world within a 30-day CDSCO review, but the notification has not been finalized, so imports still rest on case-by-case CDSCO permissions.  

_{How does compassionate use work in Latin America?}

Latin America leans toward named-patient import authorizations, with Brazil the most developed. ANVISA's RDC 38/2013 runs three programs: expanded access, compassionate use and post-study supply, and obliges the sponsor to supply the medicine free of charge; a revision has been in public consultation since 2023. Mexico's COFEPRIS authorizes compassionate use under rules aligned with those for clinical trials, alongside a separate import route for individual patients. Argentina runs named-patient imports under ANMAT Disposition 4616/2019, with each import authorization valid for 90 days and any commercial promotion of the imported product prohibited. Colombia supplies “vital unavailable” medicines without a marketing authorization under Decree 481 of 2004.

_{How does compassionate use work in Africa?}

Africa is the least formalized of the three regions, and most authorities work through general unregistered-medicine provisions rather than a dedicated compassionate-use law. The regional reference model is South Africa's Section 21, under the Medicines and Related Substances Act of 1965, through which SAHPRA authorizes a named patient's access to an unregistered medicine once conventional therapies have failed or are unavailable. Harmonization is the region's defining theme: the African Medicines Agency, whose founding treaty entered into force in November 2021, aims to knit these fragmented national systems together over time.

_{What should companies check before running a program?}

The eligibility principles are consistent worldwide; the operating rules are not. Before supplying a medicine under any of these routes, a company needs four answers for each market: whether authorization covers a single named patient or a cohort; whether the pathway accepts a product still in trials or only one approved elsewhere; whether the regulator requires prior approval or simple notification; and what commercial terms apply, since some markets, such as Brazil, oblige the sponsor to supply free of charge, and others, such as Argentina, prohibit any promotion of the imported product. Asia and Latin America are formalizing these frameworks fastest, and Africa's harmonization agenda under the African Medicines Agency may narrow the gap over time.

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^FAQ

_{What is compassionate use, in simple terms?}

It's a regulated, ethical pathway that allows a patient with a serious or life-threatening illness, who has no satisfactory approved treatment, to receive a promising medicine that isn't yet approved or registered in their country, outside a clinical trial.

Does a company have to supply the medicine for free?

It depends on the market. Brazil's RDC 38/2013 obliges the sponsor to supply the medicine free of charge, while other markets, such as Argentina, instead prohibit any commercial promotion of the imported product rather than mandating free supply.

_{How can Speyside help pharmaceutical companies navigate compassionate use regulations across these regions?}

Speyside Group advises multinational pharmaceutical and life sciences companies on structuring compliant compassionate use, expanded access, and named patient programs across Latin America, Asia Pacific, and Africa. This includes assessing each target market's specific legal pathway, commercial terms, and approval timelines, and building the regulatory and stakeholder engagement strategy needed to bring a program online without triggering compliance or reputational risk.