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If you know the name of a drug manufacturer, you can easily search using the alphabetical menu below. Or you can search in free text – simply type the disease, drug, company name, or other detail into the Search bar below and click on the magnifying glass. For broadest results, type the terms without quotation marks; to narrow your search to an exact match, put your terms in quotation marks (i.e.; “breast cancer”). See Search Tips for tips and definitions to make your searches more effective.

The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision. Data contained in this directory is provided either directly by the company or by ClinicalTrials.gov for the companies listed in this directory.

The Expanded Access listings provided on this website are limited to those companies currently in the Company Directory and include listings for expanded access which are Available, Temporarily Not Available and Approved for Marketing, which in some cases includes treatments which have not yet reached the market. In addition, many companies are willing to consider expanded access requests even if they do not show active Expanded Access Listings in this directory. Contact the company to explore the possibility of individual access to an investigational therapy.

To learn more about clinical trials and expanded access, please visit our patient and physician guides on this website.

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Company Name
Phone Number & Email
Company Acknowledgement

Eisai

Phone Number & Email
Expected Application Timeframe
2 business days
Metastatic Breast Cancer

This expanded access program is an open-label, multi-center study, which will consist of a PreTreatment Phase and a Treatment Phase. Patients with locally advanced or metastatic breast cancer who fulfill the eligibility criteria will be treated. The program will continue in each country until eribulin is approved, reimbursed and launched in that country, or termination of the program by the Sponsor.

Partial Onset Seizures

The Extended Access Program (EAP) is a managed access programme for Perampanel. The main objective of this EAP is to ensure that patients participating in studies E2007-A001-207, E2007-G000-307, or E2007-G000-235 continue to have access to perampanel until such time perampanel tablets become commercially available for the treatment of Partial Onset Seizures (POS) in the country in which they reside. This EAP will consist of 2 phases: - Screening: The patient will start the program once the Screening assessments are completed and the patient is qualified for participation. - Treatment: Additional assessments, physical examinations, and dosage changes will be clinically determined by the treating physician. Patients will enter this program on the same dose of perampanel that they were receiving at the end of their participation in previous study. Doses of perampanel and of concomitant anti-epileptic drugs (AEDs) can be adjusted (i.e., added,removed, or changed in dose) based on clinical judgment. Treatment will be prescribed as long as clinically appropriate according to the judgement of the treating physician and the approved Summary of Product Characteristics (SmPC). The program will complete in a staggered fashion, country by country, as and when perampanel becomes commercially available for the treatment of POS in each country.

Differentiated Thyroid Cancer

This Expanded Access Program (EAP) consists of a Prerandomization Phase and a Randomization Phase. Only subjects with radioiodine-refractory DTC who fulfill the eligibility criteria will be treated. These subjects will be treated until progression of disease or unacceptable toxicity.

Primary Generalized Tonic-Clonic or Partial Onset Seizures

The main objective of this EAP is to ensure that participants participating in Study E2007-G000-332, Study 311 or EAP 401 continue to have access to perampanel until such time that the appropriate formulation of perampanel becomes commercially available in the country in which they reside or until no participants remain in the EAP.

Thyroid Cancer

This is an Expanded Access Program to make lenvatinib available to participants with radioiodine-refractory differentiated thyroid cancer in Brazil. Participants who have no other treatment options available, and who, in the opinion and clinical judgment of the treating physician, would benefit from treatment with lenvatinib will be enrolled. This is a multicenter, open-label program consisting of 2 phases: a 28-day pretreatment phase (including screening) and a treatment phase. Treatment will be provided as long as there is a clinical benefit based on tumor assessments performed according to the center's standard of care and the judgment of the participant's treating physician.

Lennox Gastaut Syndrome

This is an extended access study for participants who have completed Rufinamide Study E2080-G000-303 to continue to have access to rufinamide until it becomes commercially available in Poland or until no participants remain in the EAP.

Eli Lilly

Phone Number & Email
Expected Application Timeframe
5 business days
Mesothelioma

This study is to evaluate the effects (good and bad) of ALIMTA and Cisplatin or ALIMTA alone on you and your malignant pleural mesothelioma as well as make ALIMTA available to patients who qualify for treatment.

Metastatic Breast Cancer

The treating physician/investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

Cancer

This study is for patients who have participated in a previous study and who continue to receive benefit to have continued access to study drug and/or treatment.

Metastatic Breast Cancer

The treating physician/investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

Non Small Cell Lung Cancer
Medullary Thyroid Cancer
Colon Cancer
Breast Cancer
Pancreatic Cancer
Papillary Thyroid Cancer
Other Solid Tumors With Evidence of Activating RET Alteration

Expanded access for patients with cancer with RET activation who are ineligible for an ongoing selpercatinib (also known as LOXO-292) clinical trial or have other considerations that prevent access to selpercatinib through an existing clinical trial.

Soft Tissue Sarcoma

The purpose of this study is to continue to provide olaratumab to eligible patients who are currently receiving olaratumab commercially for the treatment of soft tissue sarcoma (STS).

EMD Serono

Phone Number & Email
Expected Application Timeframe
5 business days

Entasis Therapeutics

Expected Application Timeframe
3 business days

Epirium Bio Inc

Phone Number & Email
Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

Epirium Bio is committed to developing safe and effective therapies for intractable neuromuscular and neurodegenerative diseases associated with mitochondrial depletion, as well as primary mitochondrial disorders, and providing those therapies to the broadest group of patients as quickly as possible. We also recognize that there are many diverse conditions in which mitochondrial depletion or dysfunction are a key component of the disease process. As part of our commitment to the rare disease community, we will support compassionate use / expanded access programs* when we have substantial scientific evidence to support both the safety and the potential efficacy of an investigational medical product for a given indication, and when it is logistically practicable.

Epirium has a developed a process for determining whether the company will provide an experimental therapy under compassionate use.
In the first step, the company will evaluate whether:
• there is substantial scientific evidence to support both the safety and the efficacy of an investigational medical product for a particular indication;
• it has been established that access on a compassionate use basis will not compromise clinical trials or the regulatory pathway for an investigational medical product;
• there is adequate supply of the investigational medical product; and
• the investigational medical product can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.
If the company decides that, under the first step, availability of the investigational medical product on a compassionate use basis is possible, then the company will evaluate an individual’s request for access.
This second step uses the following criteria:
• the patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
• there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
• patient enrollment in a clinical trial is not possible;
• potential patient benefit justifies the potential risks of treatment;
• providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication, and;
• all necessary regulatory/institutional approvals have been obtained to allow the administration of the investigational medical product.
Requests for access to investigational medical products must be made by a qualified and licensed physician and will be evaluated by Epirium. Patients interested in seeking expanded access to an Epirium investigational medical product should talk to their physician. Qualified and licensed physicians may make compassionate use / expanded access requests by contacting Epirium by e-mail at info@epirium.com. Epirium anticipates it will acknowledge receipt of such requests within five business days of their receipt
* Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation.

Available Therapies via Single-Patient EA

EPM-01 ((+)-epicatechin)

Disease/Category-Specific EA Policies/Criteria

https://epirium.com/wp-content/uploads/2020/05/Epirium_Bio_Statement_On_Compassionate_Use.pdf

Erytech

Phone Number & Email
Expected Application Timeframe
48 hours
Single-Patient EA Policies/Criteria

Erytech’s Public Policy – Compassionate Use (Expanded Access)

Erytech has no marketing approvals for any of its products throughout the globe.

Erytech is a science-led organization researching and developing new medicines through its ERYCAPS® platform. We are currently operating in North America and Europe.

This policy sets out the general principles for Erytech providing compassionate use. It is intended as global guidance, although Erytech will act in strict accordance with all local country laws and regulations on providing compassionate use.

Erytech’s lead product, eryaspase, is supported by clinical evidence from late stage clinical development1.

Erytech is currently conducting late stage clinical trials in second-line pancreatic cancer (Trybeca-1) in Europe and the US, and in first-line Triple Negative Breast Cancer (Trybeca-2) in Europe. More details, including participating investigator centres, can be found on CT.gov or by sending an email to:

medaffairs@erytech.com

Erytech recognises that not all cancer patients will be suitable candidates for these studies. Where enrolment into a clinical trial is not an option for them, it may be in the interest of patients to have access to eryaspase. Under these circumstances, and where patients are in a life-threatening situation with no satisfactory alternative treatment options, Erytech may provide a treating healthcare professional (HCP) with an investigational medicine such as eryaspase.

As general guidance, Erytech will consider providing Compassionate Use according to the following:

• Erytech considers the appropriateness of compassionate use for all our investigational medicines early on in the planning of our research programmes. At an early stage in a medicine’s development there could be only limited understanding. For example:

  • Uncertainty on the best way to provide the medicine to patients, such as the exact dose to use and frequency.
  • Establishing the medicine’s efficacy and safety profile.

- Erytech provides compassionate use in two ways:

  • Formal programmes
    • Patient populations meeting specific criteria managed under a compassionate use protocol.
  • Named (or individual) patients where appropriate and subject to fulfillment of our criteria.

All requests for compassionate use, which must be made by an HCP, will be considered by Erytech and according to the following:
o Patients are in a life-threatening situation.
o There are no satisfactory alternative treatments (confirmed by the HCP).
o There is sufficient evidence to believe the potential benefit to the patient justifies the risk.
• Compassionate use decisions are made by Erytech’s Chief Medical Officer and are final.

Other key considerations include:

• Any concern that compassionate use of the investigational medicine might somehow compromise any related clinical trial or regulatory pathway.
• Restricted to countries where Erytech is able to provide its product. Currently, this is North America and Europe.
• That use in formal progammes will only take place in countries where Erytech intends to seek regulatory approval and to make the medicine available; the same limitation will not necessarily apply for named (or individual) patients.
• That the proposed compassionate use complies with local laws and regulations.

These criteria ensure Erytech has a consistent approach for our decisions on compassionate use.


To investigate further options for the appropriate access to Erytech’s medicines, any HCP can contact Erytech by sending an email to:

medaffairs@erytech.com

Erytech will acknowledge receipt of a compassionate use request submitted by an HCP within 48 hours.

Patients interested in accessing an Erytech medicine for compassionate use should talk to their doctors.

Erytech is committed to transparency in its interactions with HCPs and health care organizations/institutions consistent with applicable laws and/or codes of practice applicable to the pharmaceutical industry. Erytech will fulfil all regulatory requirements to make public information about our compassionate use activities.



1. Hammel P, Fabienne P, Mineur L, et al. Erythrocyte-encapsulated asparaginase (eryaspase) combined with chemotherapy in second-line treatment of advanced pancreatic cancer: An open-label, randomized Phase IIb trial. Eur J Cancer. 2019;124:91- 101.

Available Therapies via Single-Patient EA

Eryaspase

Currently under clinical study in:

- Pancreatic cancer
- Triple Negative Breast Cancer
- Acute Lymphoblastic Leukaemia (ALL)

Disease/Category-Specific EA Policies/Criteria

https://erytech.com/contact/compassionate-use/

Esperare

Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

ER-004 COMPASSIONATE USE/EXPANDED ACCESS POLICY in XLHED

Expanded access/Compassionate use refers to the use of an investigational therapy, that have not yet been approved by government regulatory agencies, outside a clinical trial, for patients with serious or life- threatening diseases or conditions who lack therapeutic alternatives.

Esperare’s mission is to advance treatments for rare diseases and to promote treatment accessibility through a patient-centered approach.
Accordingly, a Patient Advisory Council dedicated to the XLHED program has been established and provides the opportunity to Esperare and patient groups representatives to work in close collaboration. In particular, clinical development plans are discussed in order to refine development and generate robust and necessary data for an application and to contribute to the common goal of giving the greatest chance of success to this program, with the ultimate objective of maximizing the benefits and minimizing the risks for XLHED patients and their families.

Requests for compassionate use/expanded access will be considered on a case by case basis , in accordance with applicable regulations and in alignment with our mission and values and with the safety of the patient and childbearing mother as a priority. Of note, ER-004 is delivered to the patient before birth by injection into the amniotic fluid, an untried route of administration that requires careful consideration in each individual case.

Inquiries regarding the expanded access may be sent to info.er004@esperare.org.
Esperare will endeavor to acknowledge receipt of any expanded access questions or requests within 5 business days.

Available Therapies via Single-Patient EA

ER-004 (also known as EDI200) is a protein replacement therapy designed as a substitute for endogenous EDA, a protein missing in XLHED.