Compassionate use programs in Italy: ethical guidelines

Ludovica De Panfilis,Roberto Satolli,Massimo Costantini

doi:10.1186/s12910-018-0263-8

Abstract

BackgroundThis article proposes a retrospective analysis of a compassionate use (CU), using a case study of request for Avelumab for a patient suffering from Merkel Cell Carcinoma. The study is the result of a discussion within a Provincial Ethics Committee (EC) following the finding of a high number of requests for CU program. The primary objective of the study is to illustrate the specific ethical and clinical profiles that emerge from the compassionate use program (CUP) issue. The secondary goals are: a) to promote a moral reflection among physicians who require approval for the CUP and b) provide the basis for recommendations on how to request CUP.Main bodyThe instruments for carrying out the analysis of the case study and the discussion are as follows:Analysis of the audio-recording of the EC meeting regarding the selected Case study.In-depth discussion of topics that emerged during the meeting by means of administration of 5 semi-structured interviews with 2 doctors involved in the case (proposing physician and palliative physician) and with 3 components of the EC who played a major role in the EC internal discussion.ConclusionsIn an exploration of emerging clinical and ethical issues, four primary themes arise: 1. efficacy, safety of the treatment and patient’s quality of life; 2. clear, realistic, adequate communication; 3. right to hope; 4. simultaneous Palliative Care approach. The results of ethical analysis carried out concern two areas: 1) ethical profiles relating to the use of CUP; 2) the role of the EC concerning the compassionate use of drugs and the need to provide recommendations on how to request CUP.With the aim of implementing these conclusions, the provincial EC of Reggio Emilia chose to steer the request for drugs for compassionate use through recommendations for good clinical and ethical practice based on the following assumptions: 1) the “simultaneous care” approach must be preferred. Secondly, 2) the EC’s assessment must be part of the decision-making process that the care team conducts before proposing compassionate use to the patient.

Highlights

This article proposes a retrospective analysis of a compassionate use (CU), using a case study of request for Avelumab for a patient suffering from Merkel Cell Carcinoma
The results of ethical analysis carried out concern two areas: 1) ethical profiles relating to the use of CUP; 2) the role of the Ethics Committee (EC) concerning the compassionate use of drugs and the need to provide recommendations on how to request CUP
With the aim of implementing these conclusions, the provincial EC of Reggio Emilia chose to steer the request for drugs for compassionate use through recommendations for good clinical and ethical practice based on the following assumptions: 1) the “simultaneous care” approach must be preferred

Summary

Introduction

This article proposes a retrospective analysis of a compassionate use (CU), using a case study of request for Avelumab for a patient suffering from Merkel Cell Carcinoma. In Italy, the use of drugs for nominal therapeutic purposes is regulated by the Decree of 8 May 2003 “Therapeutic use of medicinal products subjected to clinical trials” [3], which describes the methods for requesting the drug and the conditions in which authorization may be issued. According to this regulation, a drug can be requested for use outside clinical trial “when there is no valid therapeutic alternative to the treatment of serious illnesses, or rare diseases, or disease conditions that put the patient’s life at risk” [3]. The conditions under which authorization for use of the medicinal product may be issued are: 1) a phase 3 trial aimed at assessing the effectiveness of the medicinal product is ongoing or completed; 2) in special cases when illness puts the patient’s

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