Patient Resources · Treatment Access
Regulatory agencies, official program links, application requirements, and patient-facing navigators for compassionate use and early access programs around the world.
Last updated May 2026
If you are reading this page, you have likely already read our companion article on Right to Try, Compassionate Use, and Early Access. This page is the practical extension of that article. It gives you the names of the agencies, the working links to their official pages, what your physician will need to submit, and where to find help navigating the process.
Every country handles early access differently. The agency names change. The forms change. The links break. We update this page as programs change, but always confirm the current procedure with your oncology team and the regulatory agency itself before relying on it.
One reminder that matters more than any link below. A physician must submit these applications on the patient’s behalf. Patients cannot apply directly. The role of this page is to help you bring the right names and the right forms to the right conversation.
Jump to a country (these are the countries represented on the private Appendicure Facebook Group. As people from new countries join, I’ll add them here. Click on your country to go straight there:
🇺🇸 United States
Regulator: U.S. Food and Drug Administration (FDA) | Programs: Expanded Access (Compassionate Use), Right to Try
Official Pages
FDA Expanded Access (main hub)
FDA Expanded Access: Information for Patients
FDA Expanded Access: How to Submit a Request (Forms)
For Oncology Patients (Use This First)
The FDA’s Project Facilitate is a single-point-of-contact call center built specifically for oncology Expanded Access requests. Your oncologist (not you) calls or emails them, and FDA oncology staff walk through the request with the physician.
Phone: (240) 402-0004 (Monday to Friday, 8:00 am to 4:30 pm ET)
Email: OncProjectFacilitate@fda.hhs.gov
After-hours emergency: FDA Emergency Call Center 866-300-4374
What Your Physician Will Need
- A serious or life-threatening diagnosis with no comparable approved alternative
- The patient is not eligible for a relevant clinical trial
- Manufacturer agreement to provide the investigational drug
- FDA Form 3926 (single-patient Expanded Access)
- IRB review (often satisfied through the Form 3926 waiver)
- Informed consent from the patient
Patient-Facing Navigator
Reagan-Udall Foundation Expanded Access Navigator. This is the most useful single resource for U.S. patients and oncologists. It includes a directory of pharmaceutical company Expanded Access policies, an eRequest tool for physicians, and downloadable forms. It was built specifically to make the process less of a maze for families.
🇬🇧 United Kingdom
Regulator: Medicines and Healthcare products Regulatory Agency (MHRA) | Programs: Early Access to Medicines Scheme (EAMS), “Specials” (unlicensed medicines)
Official Pages
EAMS Overview (GOV.UK)
EAMS: Information for Applicants
NHS Accelerated Access Collaborative: EAMS
How It Works
EAMS is a two-step process. The manufacturer first obtains a Promising Innovative Medicine (PIM) designation based on early clinical data. The MHRA then issues a Scientific Opinion supporting prescribing of the medicine. Once a positive Scientific Opinion is issued, your specialist can request access for you. The EAMS medicine is supplied free of charge by the company to the NHS during the EAMS period, typically 12 to 18 months ahead of full marketing authorisation.
Contact
MHRA EAMS coordinator: eams@mhra.gov.uk
Patient-Facing Navigator
Cancer Research UK: Early Access to Medicines Scheme. A patient-facing explainer in plain English with guidance on talking to your specialist about EAMS eligibility.
🇨🇦 Canada
Regulator: Health Canada | Program: Special Access Program (SAP)
Official Pages
Special Access Program: Request a Drug
SAP: Information for Patients
SAP Overview and FAQs
What Your Physician Will Need
- A serious or life-threatening condition where conventional therapies have failed, are unsuitable, or unavailable
- A completed Special Access Request (SAR) form
- Clinical justification supporting the request
- Manufacturer willingness to supply the drug
Authorizations are typically for up to six months of chronic treatment. Repeat orders go through SAP again. SAP processes urgent requests as quickly as possible.
Note for Patients
Only health care practitioners can apply. Patients cannot apply directly. Costs of the drug and any associated care are not covered by SAP itself, and funding may need to be arranged through provincial health authorities or private insurance.
🇦🇺 Australia
Regulator: Therapeutic Goods Administration (TGA) | Programs: Special Access Scheme (SAS), Authorised Prescriber Scheme
Official Pages
TGA: Access to Unapproved Therapeutic Goods for Individual Patients (SAS)
SAS: Guidance for Health Practitioners
SAS and AP Online System
SAS Categories
Category A: For patients who are seriously ill with a condition from which death is reasonably likely within months. Notification-based, used after supply.
Category B: For patients who do not fit A or C. Requires TGA pre-approval before supply.
Category C: For specified products with established history of use. Notification within 28 days of supply.
All applications now go through the online SAS and AP system. From July 2024, paper submissions are no longer accepted.
Authorised Prescriber Pathway
If a medical practitioner expects to prescribe an unapproved therapeutic good to multiple patients with the same condition on an ongoing basis, the Authorised Prescriber pathway may be more practical than repeated SAS applications.
🇩🇪 Germany
Regulators: Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM); Paul-Ehrlich-Institut (PEI) for antibody drugs, blood products, and vaccines | Program: Compassionate Use (Härtefallprogramm)
Official Pages
BfArM: Compassionate Use (in German)
BfArM: Current Active Compassionate Use Programs
How It Works
Compassionate use in Germany is structured around programs (Härtefallprogramme) for groups of patients with the same diagnosis, not individual one-off requests. The manufacturer registers the program with BfArM (or PEI, for the product categories above). The list of currently active programs is published on the BfArM and PEI websites. Each program entry includes the manufacturer contact for requesting access.
For individual patients who do not fit any active program, the treating physician may pursue an “individueller Heilversuch” (individual treatment attempt) under their own clinical authority, which is not regulated by the compassionate use ordinance.
What Your Physician Will Need
- Confirmation that the diagnosis matches the program’s defined patient group
- Documentation that no authorised therapy is satisfactorily working
- Direct contact with the manufacturer listed in the BfArM program entry
🇳🇿 New Zealand
Regulator: Medsafe (part of Health New Zealand / Te Whatu Ora) | Programs: Section 29 of the Medicines Act 1981 (unapproved medicines, named-patient supply); Section 25 (authorised prescriber procurement)
Official Pages
Medsafe: Supplying Unapproved Medicines
Medsafe: Use of Unapproved Medicines and Approved Medicines for Unapproved Purposes
How It Works
Section 29 permits New Zealand suppliers to provide an unapproved medicine to a medical practitioner for the treatment of a named patient under their care. The supplier reports the supply to Medsafe (prescriber name, patient name, place of supply). The medical practitioner takes responsibility for safety, quality, and efficacy, since Medsafe has not evaluated the product.
Pharmac (the public funding agency) may fund some unapproved medicines on a named-patient basis under exceptions schemes, but funding is separate from access and not guaranteed.
Patient-Facing Resource
Healthify: Unapproved Medicines. Plain-language explainer of Section 29, off-label use, and informed consent for patients.
🇮🇪 Ireland
Regulator: Health Products Regulatory Authority (HPRA) | Programs: Exempt Medicinal Products (EMP), Compassionate Use Programmes, Early Access Programmes
Official Pages
HPRA: Early Access to Medicines (patient page)
HPRA: Exempt Medicinal Products
How It Works
Ireland operates under two main exemptions. Clinical trial enrollment is the first route. Where no authorised medicine is available and no trial fits, a prescriber may request supply of an unauthorised medicine (an Exempt Medicinal Product) for a patient under their care. Compassionate Use Programmes coordinated through the EU framework also operate for patients with serious unmet need, often free of charge from the manufacturer.
Contact
HPRA general enquiries: hpra.ie/contact-us
🇮🇳 India
Regulator: Central Drugs Standard Control Organisation (CDSCO) | Programs: Compassionate Use under the New Drugs and Clinical Trials Rules 2019; Named Patient Program access; personal-use import under Rule 36 of the Drugs and Cosmetics Rules 1945
Official Page
Central Drugs Standard Control Organisation (CDSCO)
How It Works
A medical practitioner or government medical organisation may apply to CDSCO to import an unapproved new drug for compassionate use. The patient must have a life-threatening condition, a condition causing serious permanent disability, or an unmet medical need. The drug must be in at least Phase 3 clinical trials in India or in another country. CDSCO is generally expected to review within 30 days.
For drugs approved in another country but not yet in India, the Named Patient Program pathway permits import of limited quantities for personal use under physician prescription, regulated through Form 12B import permits.
Note for Patients
Access in India varies significantly by institution, manufacturer participation, and patient resources. Major academic cancer centers and large private hospitals tend to be most experienced with these pathways. Costs are typically borne by the patient.
🇸🇪 Sweden
Regulator: Läkemedelsverket (Swedish Medical Products Agency) | Programs: Compassionate Use Programme (CUP), licensförskrivning (license prescribing for individual patients)
Official Pages
Läkemedelsverket: Unauthorised Medicinal Products through a Compassionate Use Programme (English)
Läkemedelsverket: License Prescribing (Swedish)
How It Works
Sweden runs two parallel pathways. A Compassionate Use Programme is set up by a manufacturer for a defined group of patients, typically in connection with a pending EU marketing authorisation. Once approved by Läkemedelsverket, prescribers can supply the medicine to patients meeting the program’s criteria without individual licence applications.
License prescribing (licensförskrivning) is separate and used for individual patients. The prescriber motivates the need, and a pharmacy applies for the licence through the KLAS e-service.
🇮🇱 Israel
Regulator: Israeli Ministry of Health, Pharmaceutical Division | Programs: Compassionate use under Regulation 29C of the Pharmacists Regulations (1986); Form 29 for individual unregistered preparations
Official Page
Ministry of Health: Application for District Pharmacy Approval for Private Use of Unapproved Drugs
How It Works
Regulation 29C permits import and supply of unregistered pharmaceuticals in specific circumstances, including compassionate use for life-threatening or seriously debilitating conditions where no satisfactory alternative is available. The treating physician completes a Ministry of Health Form 29, which functions as both prescription and access request, and submits supporting clinical justification. Form 29 is valid for six months.
Compassionate use applications can cover drugs not registered anywhere in the world, drugs registered abroad but not in Israel, and off-label use of registered drugs.
Additional resources worth knowing
Kids v Cancer: Compassionate Use Navigator
Although built for pediatric oncology, the Kids v Cancer Compassionate Use Navigator offers some of the clearest templates available for any U.S. Expanded Access request, including sample letters to drug companies, application checklists, and an application flowchart. Adult oncology teams can and do adapt these resources.
ClinicalTrials.gov: Expanded Access Listings
Many manufacturers list their active Expanded Access programs on ClinicalTrials.gov. Use the search filter “Expanded Access” combined with terms like the drug name, mechanism, or molecular target (such as the gene mutation in your tumor profile).
EURORDIS
For European patients, EURORDIS, the European Organisation for Rare Diseases, is a long-standing advocate for early access pathways across EU member states and a useful entry point to country-specific patient organisations.
How to use this page with your care team
A short script for the next appointment
“Before we close out today, I want to ask about a few specific pathways. Have we considered an Expanded Access or compassionate use request for any investigational therapy? Is there a tumor-agnostic trial that fits my molecular profile, even if the trial is not appendix cancer specific? Is there a manufacturer with an Expanded Access policy for [drug name] we could approach?”
Bring this page, or the official link for your country, to the appointment. Your physician is the one who applies. Your role is to make sure the conversation happens.
Found a broken link or outdated information?
Regulatory pages move and program names change. If you find something on this page that no longer works or that has been updated, please let us know through the Appendicure contact page so we can keep this resource accurate for the next family who needs it.
Medical and legal disclaimer. This page is for educational purposes only and does not constitute medical or legal advice. Eligibility for investigational-access programs varies by country, institution, manufacturer policy, and individual medical circumstances. Regulatory program names, links, eligibility criteria, and forms change over time. Always confirm the current procedure with your oncology team and the regulatory agency itself before relying on any information here. Patients should discuss all treatment decisions with their qualified healthcare professionals.