Orphan and rare disease medicines
Patients with rare diseases can wait years for a medicine to be registered in their country. Where the regulator allows it, we source orphan and specialty medicines for named patients and hospitals, with cold chain where needed.
In short
Orphan medicines treat rare diseases and are often registered in only a few markets. MedsAcross helps hospitals and physicians obtain them through the destination's named-patient or special-access route, sourcing from licensed supply chains, and ships each order with its batch documents and, for biologics, a qualified cold-chain lane.
What it is
Orphan designation and approval come from regulators such as the US FDA and the European Union. A medicine approved in one region can stay unavailable elsewhere for years, because its owner has not applied or the market is small.
We check where the medicine is approved, which presentations exist and whether a manufacturer's access programme already covers your patient. If a programme exists, we tell you, because it may be the faster route.
Many orphan medicines are biologics stored at 2 to 8 °C or frozen, so cold-chain planning starts at the quote.
Who it is for
- Hospitals treating patients with rare diseases
- Physicians and specialist centres
- Patient organisations, working through the treating physician
- Importers serving rare disease centres
Who uses this service
How it works, step by step
Typical times from our process. Your quote shows the lead time for your medicine and your country.
- 1
Request
Same daySend the medicine, the patient's country, the quantity and the date needed.
- 2
Availability and access check
24 to 48 hoursWe check approvals, presentations and any access programme for the patient's country.
- 3
Documents and review
Within the quote targetA pharmacist reviews the prescription, the clinical justification and the import approval.
- 4
Quote
With the reviewQuoted to the hospital or licensed consignee with the cold-chain lane and lead time.
- 5
Payment and import approval
Set by your regulatorAdvance payment by wire. Dispatch follows the import approval.
- 6
Cold-chain dispatch
3 to 15 days transitA qualified shipper with a data logger, sent by medical courier or air cargo.
Documents needed
Your quote comes with a checklist that shows who provides each document and its status.
Prescription and clinical justification
From the treating physician, explaining why registered alternatives are not suitable.
Import approval
The special-access permit or named-patient approval in your country.
Hospital licence
Of the hospital or licensed consignee receiving the medicine.
Certificate of analysis
For the supplied batch.
Temperature logger file
For refrigerated and frozen products, linked to the lot.
Patient consent
Where the route requires informed consent to an unlicensed medicine.
Legal route notes
Rules differ by country and change over time. Each note cites a regulator or a published legal analysis. Your local regulatory adviser confirms the route for your case.
Physicians applying to a manufacturer's managed access programme are often asked for their licence number, registering council and a CV.
United Kingdom. The importer notifies the MHRA, and the import must not proceed if the MHRA objects within 28 days. Cost is not an accepted reason.
Source: GOV.UK, notification of intent to import an unlicensed medicine
Russia. An unregistered medicine for a specific patient needs Ministry of Health permission, decided within 5 working days.
Source: Brace Law Firm, import of medicinal products into Russia
Brazil. Health establishments may import unregistered products for their own care under RDC 488/2021.
The European Medicines Agency publishes data tables on authorised medicines, which we use to check where a medicine is approved.
Check your country
Each import guide lists the regulator, the legal routes, the documents and typical approval times.
Questions and answers
Is an access programme better than a named-patient import?
Sometimes. A manufacturer's programme may supply eligible patients free or at a set price. We tell you when we find one for your country, and you choose the route.
Can you supply biologics that need cold chain?
Yes, in qualified shippers with a data logger. Some advanced therapies need specialist handling that only the manufacturer provides, and we say so at the quote stage.
What if the medicine is not approved anywhere?
Then it is an investigational product, available only through a clinical trial or the developer's own expanded access programme, and we cannot supply it.
Ask us to find an orphan medicine
Tell us the medicine and the patient's country. We check approvals, access programmes and supply options and reply with the route.