Rare-disease and compassionate-use medicines in Spain

Key rules

• Who applies: Only your hospital doctor can make the request to the AEMPS, not you.
• No self-import: You cannot import a foreign medicine yourself; it comes in through the hospital pharmacy or an authorised importer.
• EU approval: EU approval of an orphan medicine does not mean Spain's public system funds it automatically.
• Cost: In a public hospital you do not pay for a medicine supplied through compassionate use.

The short answer

You cannot apply for these medicines yourself. Your hospital doctor requests them for you from the AEMPS, through routes the AEMPS regulates. Your job is to start the conversation with your specialist and to keep the paperwork moving.

Which route fits your medicine

Compassionate use. This is for a medicine not authorised in Spain. It applies when you have a serious, chronic or life-threatening disease and no satisfactory alternative. Your doctor asks for it in your name. The hospital pharmacy service and management support the request, and you give informed consent.

The request goes to the AEMPS, usually through its online application. It includes:

• a clinical report that justifies the need
• the manufacturer's agreement to supply the medicine
• the hospital director's authorisation

The AEMPS then issues an authorisation for you personally. If the medicine is in a clinical trial, the trial sponsor or a trial extension may be used instead. Your regional health service may also have to approve.

Foreign medicine (medicamento extranjero). This is for a medicine authorised only abroad, when Spain has no authorised equivalent. Your doctor and the hospital pharmacy request it for you through the AEMPS. The request needs a clinical justification, the dose and duration, and your consent. After the AEMPS authorises the import, the hospital pharmacy or an authorised importer brings it in. Hospitals usually handle the paperwork. You cannot import the medicine yourself.

Medicines already authorised in the EU. An EU-approved orphan medicine is not automatically covered in Spain. Each one needs a national decision on funding and price, made drug by drug and indication by indication. Once it is funded, you get it through the hospital pharmacy, free or with the usual co-payment. This is often limited to set indications and clinical protocols. Before it is funded, your hospital may be able to get it case by case through a hospital commission, compassionate use or a regional exceptional approval. Access depends on the gap between EU approval and the funding decision.

Who pays

In a public hospital you do not pay for a medicine supplied through compassionate use. The manufacturer usually provides it free, and the hospital dispenses it. For an imported foreign medicine, the hospital or the regional health service usually pays. Arrangements depend on the hospital, region and manufacturer. In a private hospital, billing may differ, so ask who pays before you start.

What to do first

1. Ask your specialist whether the medicine is already authorised and funded in Spain for your condition.
2. If it is not, ask the hospital pharmacy which route applies.
3. Agree with your doctor on the report, dose and duration.

The checklist below walks through these steps, and the questions below answer the common doubts.

Checklist

• Ask your hospital specialist to request the medicine for you, because only the doctor can apply to the AEMPS.

• Check whether the medicine is already authorised and funded in Spain for your condition before choosing a route.

• Ask the hospital pharmacy whether the medicine needs the foreign-medicine route or compassionate use.

• Agree with your doctor on the clinical report, dose and duration that justify the request.

• Give your informed consent to the treatment.

• Ask whether the manufacturer has agreed to supply the medicine and whether your regional health service must also approve.

• Ask who pays for the medicine before you start, especially in a private hospital.

Frequently asked questions

Can I ask the AEMPS for compassionate use myself?

No, the request must come from your treating doctor, not from you. The doctor files it for you as a named patient, with a clinical report, the manufacturer's agreement to supply and the hospital director's authorisation. You give informed consent.

Can I import a medicine from abroad myself?

No, patients cannot import a foreign-only medicine for personal use. Your Spanish doctor and the hospital pharmacy request it for you from the AEMPS, and the hospital pharmacy or an authorised importer brings it in. This is only possible when no authorised equivalent exists in Spain.

Does Spain's public system pay for an EU-approved orphan medicine automatically?

No, each orphan medicine needs its own national pricing and funding decision before the public system covers it. Funding is often limited to certain uses and clinical protocols. While a decision is pending, ask your hospital doctor about compassionate use or a regional exceptional approval.

Do I pay for a medicine supplied through compassionate use?

No, you do not pay for the medicine itself in a public hospital. The manufacturer usually supplies it free, and the hospital pharmacy dispenses it.