Expanded Access Programme: looking for a common definition

Iudicello, Antonella; Alberghini, Lucia; Benini, Giulia; Mosconi, Paola

doi:10.1186/s13063-015-1108-0

Cited by 24 publications

(26 citation statements)

References 4 publications

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“…11 18 22 An expanded access program (EAP; sometimes called a compassionate use program) provides an investigational treatment to patients who have a serious or terminal disease outside of a clinical trial, although definitions vary by country. 23 After data became available from the avelumab registrational clinical trial in mMCC, the EAP was initiated to provide avelumab for compassionate use to patients with mMCC who had limited treatment options. The EAP was an ad hoc program that incorporated treatment guidelines but was not protocol-driven.…”

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confidence: 99%

Efficacy and safety of avelumab treatment in patients with metastatic Merkel cell carcinoma: experience from a global expanded access program

Walker

Lebbe

Grignani

et al. 2020

J Immunother Cancer

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BackgroundAvelumab, a human anti–programmed death-ligand 1 immunoglobulin G1 monoclonal antibody, showed favorable efficacy and safety in patients with metastatic Merkel cell carcinoma (mMCC) in the phase II JAVELIN Merkel 200 trial, leading to approval in multiple countries. We describe real-world experience with avelumab in patients with mMCC from an expanded access program.MethodsEligible patients had mMCC and progressive disease during or after chemotherapy or were ineligible for chemotherapy or clinical trial participation. Patients received an initial 3-month supply of avelumab (administered as 10 mg/kg intravenously every 2 weeks until progressive disease or unacceptable toxicity); resupply was allowed following complete response, partial response, stable disease, or clinical benefit per physician assessment.ResultsBetween December 15, 2015, and March 4, 2019, 558 of 620 requests from 38 countries were medically approved, and 494 patients received avelumab. Among 240 evaluable patients, the objective response rate was 46.7% (complete response in 22.9%, including 3 of 16 potentially immunocompromised patients), and the disease control rate was 71.2%. The median duration of treatment in evaluable patients with response was 7.9 months (range, 1.0–41.7) overall and 5.2 months (range, 3.0–13.9) in immunocompromised patients. No new safety signals were identified. The expanded access program closed for new requests on December 31, 2018, as required after regulatory approval; benefitting patients continued to receive avelumab.ConclusionsThe avelumab expanded access program for patients with mMCC demonstrated efficacy and safety in a real-world setting, consistent with the results from JAVELIN Merkel 200, and provided a treatment for patients with limited options.

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confidence: 99%

Efficacy and safety of avelumab treatment in patients with metastatic Merkel cell carcinoma: experience from a global expanded access program

Walker

Lebbe

Grignani

et al. 2020

J Immunother Cancer

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“…Moreover, in principle, expanded access programs are dedicated to patients ineligible for enrollment in clinical trials. Studies whose primary objectives include the evaluation of the safety and/or efficacy of IDs should not be classified as expanded access programs, but as clinical trials [ 41 ].…”

Section: Main Textmentioning

confidence: 99%

Ethics framework for treatment use of investigational drugs

Borysowski

Górski

2020

BMC Med Ethics

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Background Expanded access is the use of investigational drugs (IDs) outside of clinical trials. Generally it is performed in patients with serious and life-threatening diseases who cannot be treated satisfactorily with authorized drugs. Legal regulations of expanded access to IDs have been introduced among others in the USA, the European Union (EU), Canada and Australia. In addition, in the USA an alternative to expanded access is treatment under the Right-to-Try law. However, the treatment use of IDs is inherently associated with a number of ethically relevant problems. Main text The objective of this article is to present a coherent framework made up of eight requirements which have to be met for any treatment use of an ID to be ethical. These include a justified need for the use of an ID, no threat to clinical development of the ID, adequate scientific evidence to support the treatment, patient’s benefit as the primary goal of the use of an ID, informed decision of a patient, fair access of patients to IDs, independent review, as well as the dissemination of treatment results. Conclusions While this framework is essentially consistent with the legal regulations of expanded access of the USA, the EU, Canada and Australia, it is substantially wider in scope because it addresses some important issues that are not covered by the regulations. Overall, the framework that we developed minimizes the risks and threats, and maximizes potential benefits to each of the four key stakeholders involved in the treatment use of IDs including patients, doctors, drug manufacturers, and society at large.

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“…There is great variation in the terminology used to denote policies and programs dealing with the issue of patient access to these unapproved drugs that are still in development. 3 Even within individual countries, terms are often used interchangeably or in ways that can be misleading. Based on several years of conversations with patient advocates, health care providers, regulatory staff, and members of the pharmaceutical industry, it is clear to us that this terminological inconsistency results in confusion and ambiguity, possibly with deleterious effects on patient health.…”

Section: Introductionmentioning

confidence: 99%