Glossary: medicine access terms in plain language
Short, sourced definitions of the words you will see on regulator forms and hospital letters.
Reviewed by the Reserve Meds clinical operations team on 17 October 2026. Sources accessed 17 October 2026.
The short answer
These are the terms that come up most when a family tries to get a medicine that is not registered at home. A named-patient or special access request is made in your own country; expanded access and Right to Try are US routes for unapproved drugs; accelerated approval, Breakthrough Therapy and orphan designation describe how a drug was developed or approved.
- Local routes: Named patient, special access, personal-use import.
- US routes for unapproved drugs: Expanded access, Right to Try.
- Approval types: Accelerated approval, conditional authorisation, exceptional circumstances.
- Supply terms: Cold chain, DSCSA, companion diagnostic.
Routes to a medicine
- Named patient: supply of a medicine for one identified patient, usually requested by the treating doctor or hospital. EMA describes it as a doctor obtaining a medicine from the manufacturer for an individual patient under the doctor's responsibility.[1]
- Special Access Program (Saudi Arabia): the SFDA route for a patient with a serious or immediately life-threatening condition to receive an unregistered medicine when no comparable alternative exists.[2]
- Personal-use import: clearing a medicine for your own use within national limits, such as up to three months of non-controlled medicine in Saudi Arabia.[3]
- Expanded access (compassionate use, US): FDA's pathway for a patient with a serious or immediately life-threatening condition to use an investigational product outside a trial, with the manufacturer's agreement and FDA authorization.[4]
- Right to Try (US): a federal pathway for eligible patients to seek certain investigational drugs that have completed Phase 1, without FDA reviewing each request. Manufacturers are not required to supply.[5]
- Compassionate use programme (EU): a programme run by an EU member state under Article 83 of Regulation (EC) No 726/2004 for a group of patients.[1]
How drugs are approved
- Accelerated approval (US): approval for a serious condition based on a surrogate endpoint reasonably likely to predict benefit, with confirmatory trials required.[6]
- Breakthrough Therapy (US): a designation that speeds development of a drug that may substantially improve on available therapy. It is not an approval.[7]
- Orphan drug designation (US): a status for drugs for rare diseases that brings incentives such as tax credits, user fee exemption and potential seven years of exclusivity.[8]
- Conditional marketing authorisation (EU): an approval on less complete data for serious diseases, valid for one year and renewable, with obligations to complete studies.[9]
- Exceptional circumstances (EU): an approval when comprehensive data cannot be provided because the condition is rare or full data collection is impossible or unethical.[10]
- PRIME (EU): EMA's support scheme for promising medicines under development for unmet needs.[11]
Supply and safety
- Companion diagnostic: a test that gives information essential for the safe and effective use of a specific medicine.[12]
- Cold chain: storage and transport within a medicine's labelled temperature range, monitored and recorded throughout.[13]
- DSCSA: the US Drug Supply Chain Security Act, which builds package-level tracing of certain prescription drugs.[14]
- Falsified medicine: a product that deliberately misrepresents its identity, composition or source.[15]
- Compounded drug: a medicine prepared for a specific patient's need; compounded drugs are not FDA-approved.[16]
US review terms
- Fast Track: a US process for drugs that treat serious conditions and fill an unmet need, with more frequent FDA meetings and possible rolling review.[17]
- Priority Review: FDA's goal of acting on an application within 6 months instead of 10 months under standard review.[18]
- Rare pediatric disease priority review voucher: a transferable voucher FDA may award on approval of a qualifying drug; no vouchers may be awarded after 30 September 2029.[19]
- Form FDA 3926: the form a physician uses for an individual patient expanded access request.[20]
How Reserve Meds helps
Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help families and treating doctors understand the route, prepare the documents the regulator or hospital asks for, arrange supply from the US once approval is in place, and keep everyone updated. We do not diagnose, prescribe or decide what a regulator approves.
We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them. Your coordination fee is quoted in writing for your case before you commit. No hidden charges. We give you a written timeline estimate for your case before you commit.
Ask us about a medicine
Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.
Frequently asked questions
What is the difference between named patient and expanded access?
Named-patient supply is arranged in the patient's own country for a medicine not registered there. Expanded access is a US FDA pathway for investigational drugs not yet approved.
Is compassionate use the same everywhere?
No. In the US it usually means FDA expanded access; in the EU it means a member-state programme under Article 83; other countries have their own routes.
Does orphan designation mean a drug is approved?
No. It is a status that gives the developer incentives. The drug still needs full approval.
What does Reserve Meds coordinate?
Access to FDA-approved medicines only, through a US-licensed wholesale partner, once the local regulator has approved the import.
Sources for these answers: [1, 4, 8]
Sources
All sources were opened and checked on 17 October 2026. Rules and labels change, so the treating hospital or regulator confirms the current position before anything is filed.
- European Medicines Agency: Compassionate use (accessed 17 October 2026)
- SFDA: Special Access Program (SAP) guidance, version 1.0 (accessed 17 October 2026)
- SFDA: Conditions and Requirements for the Clearance or Exportation of Individuals for Personal Use, version 1.1 (accessed 17 October 2026)
- US FDA: Expanded Access (accessed 17 October 2026)
- US FDA: Right to Try (accessed 17 October 2026)
- US FDA: Accelerated Approval Program (accessed 17 October 2026)
- US FDA: Breakthrough Therapy (accessed 17 October 2026)
- US FDA: Designating an Orphan Product, Drugs and Biological Products (accessed 17 October 2026)
- European Medicines Agency: Conditional marketing authorisation (accessed 17 October 2026)
- European Medicines Agency: Exceptional circumstances (glossary) (accessed 17 October 2026)
- European Medicines Agency: PRIME, priority medicines (accessed 17 October 2026)
- US FDA: Companion Diagnostics (accessed 17 October 2026)
- World Health Organization: TRS 1025, Annex 7, Good storage and distribution practices for medical products (accessed 17 October 2026)
- US FDA: Drug Supply Chain Security Act (DSCSA) (accessed 17 October 2026)
- World Health Organization: Substandard and falsified medical products (fact sheet) (accessed 17 October 2026)
- US FDA: Compounding and the FDA, Questions and Answers (accessed 17 October 2026)
- US FDA: Fast Track (information for patients) (accessed 17 October 2026)
- US FDA: Priority Review (information for patients) (accessed 17 October 2026)
- US FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs (2026 update) (accessed 17 October 2026)
- US FDA: Expanded Access, How to Submit a Request (Forms), including Form FDA 3926 and eRequest (accessed 17 October 2026)
Related
- All access pathways
- Answers to common access questions
- Named-patient access, explained
- US expanded access (compassionate use)
This page is general information, not medical or legal advice. It does not replace advice from your own doctor or the regulator.