Expanded access, compassionate use and named-patient supply compared

Three phrases come up whenever a patient needs a medicine that is not available through normal channels: expanded access, compassionate use and named-patient supply. They are often used as if they meant the same thing, and in everyday speech they overlap. In law they differ in the status of the product, who starts the request, which regulator is involved and where the medicine comes from.

This guide defines each term, compares them side by side, lists related national schemes and explains how a hospital can tell which route fits a request. For the step-by-step mechanics of the third route, see how named-patient supply works.

Three terms, three legal meanings

Expanded access in the United States

The FDA describes expanded access, sometimes called compassionate use, as a pathway for patients with serious or immediately life-threatening conditions to receive an investigational product outside clinical trials. All of these must apply: there is no comparable or satisfactory alternative, trial enrolment is not possible, the potential benefit justifies the risk and access will not interfere with development.

Requests fall into individual patient (including emergency), intermediate-size population and treatment categories. A physician can use Form FDA 3926 for an individual request, together with a letter of authorisation from the developer. In an emergency, an FDA official can authorise use by telephone, the physician submits the written application within 15 working days and notifies the IRB within 5 working days of starting treatment. Non-emergency requests have a 30-day waiting period unless the FDA responds sooner, and the FDA's request page states that on average it allows 99 percent of requests to proceed.

A separate US route, Right to Try, lets an eligible patient with a life-threatening condition seek an investigational drug that has completed a Phase 1 trial without FDA review of the request. The FDA notes that the law does not require the sponsor to supply the drug.

Compassionate use in the European Union

Article 83 of Regulation (EC) No 726/2004 sets the EU framework. EMA's compassionate use page describes programmes that give groups of patients access to an unauthorised medicine still in clinical trials or under marketing authorisation review, for life-threatening, long-lasting or seriously debilitating illnesses that cannot be treated satisfactorily with authorised medicines. The programmes are coordinated and implemented by Member States, which set their own rules. EMA's scientific committee, the CHMP, can give opinions on how such a medicine should be used, but EMA says these do not create a legal framework.

Named-patient supply

Named-patient supply is individual and national. In the EU, Article 5(1) of Directive 2001/83/EC lets Member States exempt a medicine supplied on a bona fide unsolicited order from a health care professional, for an individual patient under that professional's direct responsibility. EMA notes that on this basis doctors obtain medicines directly from manufacturers, under their own responsibility, without informing the Agency. Outside the EU the same idea appears as UK specials, Section 21 in South Africa and the Gulf special access routes. The product is often already approved in another country.

Side-by-side comparison

The key practical difference is the product. Expanded access and compassionate use exist because a medicine is not yet approved anywhere, so only its developer can supply it. Named-patient supply usually concerns a medicine that is approved somewhere but not licensed in the patient's country, so a licensed importer can source it from the manufacturer or an authorised distributor, including an exporter in another country.

Related national schemes

These schemes sit between the categories above. Canada's and Australia's programmes, for example, can cover both investigational products and medicines approved abroad.

How to tell which route fits a request

1. Check the product's approval status. If no regulator anywhere has approved it, the realistic options are a trial, the developer's expanded access or compassionate use programme, or an early access scheme.
2. If it is approved abroad, check the local exception route. Most countries have a named-patient or special access process requested by the treating physician or a licensed importer. The orphan medicine access guide lists common ones.
3. Identify the applicant. The physician, the hospital or a licensed importer applies, never the patient alone in the US, UK or EU, and never the overseas supplier.
4. Confirm controlled-drug status. Narcotic and psychotropic medicines need permits in both the importing and exporting countries, whatever the route.
5. Plan documents and time. Each route has its own evidence and clock, so use the document checklist and the lead-time estimator before promising a date.

Common misunderstandings

• There is one global compassionate use scheme. There is not. Each country, and in the EU each Member State, sets its own conditions, forms and timelines.
• Approval abroad is enough to import. A foreign approval is evidence, not permission. Saudi Arabia's Special Access Program, for example, relies on approval by a reference authority but still requires an SFDA no-objection letter and an import licence.
• The patient can apply directly. SAHPRA's Section 21 portal is completed by the treating practitioner, and UK unlicensed imports are notified by a licence holder. Patients and families should ask the treating physician or hospital to make the request.
• A lower price justifies a named-patient import. The Court of Justice of the European Union held in case C-185/10 that financial considerations cannot by themselves establish the special need that Article 5(1) requires.

Where a supplier in India fits

A supplier in India cannot provide an investigational medicine outside the developer's programme, and it cannot ship prescription medicines direct to patients in the US, UK or EU. In the US, the FDA's personal importation page states that a drug approved in another country but unapproved in the US is illegal to import.

The supplier's role is in named-patient and special access supply of products that are approved, for example in India or elsewhere, and in comparator sourcing for clinical trials. In those cases it provides product documents, clears Indian export controls such as the CDSCO export no objection certificate for medicines that are unapproved or new in India, and ships to the licensed consignee named on the approval. Hospitals and importers can start with a sourcing request once they know which route applies.