Assuntos
Tratamento Farmacológico da COVID-19 , COVID-19 , Ensaios de Uso Compassivo , Reposicionamento de Medicamentos/métodos , Controle de Medicamentos e Entorpecentes , Drogas em Investigação , Acessibilidade aos Serviços de Saúde , COVID-19/epidemiologia , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/métodos , Ensaios de Uso Compassivo/normas , Aprovação de Drogas/métodos , Controle de Medicamentos e Entorpecentes/organização & administração , Controle de Medicamentos e Entorpecentes/tendências , Drogas em Investigação/provisão & distribuição , Drogas em Investigação/uso terapêutico , Serviços Médicos de Emergência/ética , Serviços Médicos de Emergência/métodos , Acessibilidade aos Serviços de Saúde/organização & administração , Acessibilidade aos Serviços de Saúde/tendências , Humanos , Internacionalidade , Papel do Médico , Medição de Risco , SARS-CoV-2RESUMO
BACKGROUND: Nowadays one of the most critical aspects of innovative cell-based therapies is the unregulated industry, as it is becoming a competitor of the regulated system. Many private clinics, worldwide, advertise and offer cell-based interventions treatments directly to the consumer and this poses a risk to both vulnerable patients and health systems. Several countries have implemented Compassionate Use Programmes (CUP) that provide patients with medicines that have not yet completed the approval pathway, in the event that no reasonable alternative exists. Recently, in the public discourse, compassionate use has been increasingly associated with a patient's right to try. Thus, the aim of this study was to assess public knowledge of the clinical trials process with specific reference to innovative stem cell treatments, and trust in the institutions responsible for regulatory activities. We also asked people about their "right" to use unregulated therapies. METHODS: We developed an ad hoc questionnaire on three main areas of concern and administered it to 300 people in the patient waiting room at an Italian university hospital. RESULTS: Our findings suggest that people have a good knowledge of the clinical trials process and trust in healthcare institutions. Nonetheless, one person in two believes it is a right to use unregulated therapies. CONCLUSIONS: We stress the need, in the age of cellular therapies, for a commitment to support vulnerable patients and to strengthen awareness among the public about the substantial boundary that differentiates experimental therapies from unproven therapies. There should not be a "right to try" something that is unsafe but rather approved treatments and in line with good clinical practice. The trend, which emerged on this issue from our study, is quite different, confirming the urgent need to improve health information so that it is as complete as possible.
Assuntos
Ensaios de Uso Compassivo , Direitos do Paciente , Direito à Saúde , Transplante de Células-Tronco , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Ensaios Clínicos como Assunto , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/legislação & jurisprudência , Cultura , União Europeia , Feminino , Humanos , Itália , Masculino , Turismo Médico , Pessoa de Meia-Idade , Direitos do Paciente/ética , Direitos do Paciente/legislação & jurisprudência , Segurança do Paciente , Direito à Saúde/ética , Direito à Saúde/legislação & jurisprudência , Risco , Transplante de Células-Tronco/ética , Transplante de Células-Tronco/legislação & jurisprudência , Terapias em Estudo/ética , Confiança , Estados Unidos , Adulto JovemAssuntos
Ensaios de Uso Compassivo , Terapia Genética , Ensaios de Uso Compassivo/economia , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/legislação & jurisprudência , Terapia Genética/economia , Terapia Genética/ética , Terapia Genética/legislação & jurisprudência , Humanos , Estados Unidos , United States Food and Drug AdministrationRESUMO
Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or 'preapproval', access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients' best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice.
Assuntos
Tomada de Decisão Clínica/ética , Ensaios de Uso Compassivo/ética , Indústria Farmacêutica/ética , Drogas em Investigação/uso terapêutico , Relações Interprofissionais , Centros Médicos Acadêmicos , Anticorpos Monoclonais/uso terapêutico , Antineoplásicos/uso terapêutico , Ensaios Clínicos como Assunto/ética , Indústria Farmacêutica/organização & administração , Drogas em Investigação/provisão & distribuição , Comitês de Ética em Pesquisa/organização & administração , Ética Médica , Ética Farmacêutica , Humanos , Mieloma Múltiplo/tratamento farmacológico , Projetos PilotoRESUMO
There are increasing demands on regulators and insurers internationally to provide access to medicines more quickly, and often on the basis of less robust evidence of safety, efficacy or cost-effectiveness than have traditionally been required. These demands arise from a number of sources, including those advocating for access to medicines for patients with life-threatening diseases, rare diseases, or subsets of common diseases and where entire populations are threatened in the context of public health emergencies. In response to these demands, policymakers have instituted a number of initiatives aimed at speeding up access to medicines, which we refer to collectively as "accelerated access" programs. While there are strong arguments for accelerated access programs, these programs also raise a number of socio-political, epistemic and moral issues. Some of these issues are common to all types of accelerated access programs, while others are specific to particular types of accelerated access. Here, we offer a conceptual framework that highlights ethically relevant similarities and differences among different kinds of accelerated access processes for the purpose of enabling ethically and politically-informed policy making.
Assuntos
Ensaios de Uso Compassivo/ética , Drogas em Investigação/uso terapêutico , Ensaios de Uso Compassivo/economia , Drogas em Investigação/economia , Equidade em Saúde , Política de Saúde , Acessibilidade aos Serviços de Saúde/ética , Consentimento Livre e EsclarecidoRESUMO
BACKGROUND: Today, public and private bodies around the world are trying to facilitate and increase expanded access to unapproved, investigational drugs for patients with unmet medical needs. METHODS: This paper discusses three major shifts in the field of expanded access and presents an argumentative account of ethical issues connected with those shifts, based on a literature study and unstructured interviews with 35 stakeholders in the Netherlands. RESULTS AND DISCUSSION: Traditionally, expanded access has been based on three key principles: 1) it is exceptional, 2) it is done 'out of compassion', and 3) it has a therapeutic aim. Current efforts to facilitate expanded access affect these key principles, rendering expanded access a default option, allowing companies to charge for investigational drugs and gather data on its outcomes. These shifts may generate new ethical issues, including false hope, safety concerns and funding issues, which must be anticipated by physicians, pharmaceutical companies, payers and policymakers. CONCLUSION: Healthcare systems allow for the use of promising unapproved drugs in exceptional circumstances, but do not always assist patients with unmet medical needs in getting access. It is time to replace the current patchwork of practices with systems for expanded access in which criteria are clearly described, responsibilities are assigned and arrangements are made, so that patients will know what (not) to expect from expanded access.
Assuntos
Drogas em Investigação/uso terapêutico , Acessibilidade aos Serviços de Saúde/ética , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/legislação & jurisprudência , Drogas em Investigação/economia , Acessibilidade aos Serviços de Saúde/legislação & jurisprudência , Humanos , Países BaixosAssuntos
Ensaios de Uso Compassivo/ética , Alocação de Recursos para a Atenção à Saúde/ética , Oligonucleotídeos/uso terapêutico , Seleção de Pacientes/ética , Atrofias Musculares Espinais da Infância/tratamento farmacológico , Ética Médica , Humanos , Lactente , Terapias em Estudo/ética , Estados UnidosAssuntos
Antituberculosos/uso terapêutico , Ensaios de Uso Compassivo/ética , Diarilquinolinas/uso terapêutico , Mycobacterium tuberculosis/efeitos dos fármacos , Tuberculose Pulmonar/tratamento farmacológico , Adolescente , Ensaios de Uso Compassivo/legislação & jurisprudência , Farmacorresistência Bacteriana Múltipla , Feminino , Humanos , Índia , Jurisprudência , Mycobacterium tuberculosis/crescimento & desenvolvimento , Tuberculose Pulmonar/microbiologiaRESUMO
BACKGROUND: When a new intervention or drug is developed, this has to pass through various phases of clinical testing before it achieves market approval, which can take many years. This raises an issue for drugs which could benefit terminally ill patients. These patients might set their hopes on the experimental drug but are unable to wait since they are likely to pass away before the drug is available. As a means of nevertheless getting access to experimental drug, many seriously ill and terminally ill patients are therefore very willing to participate in randomised controlled trials. However, only very few terminally ill patients are able to actually participate, and those that do participate are at risk of participating solely as a way of getting experimental drugs. Currently, there are, however, ways of getting access to drugs that have not (yet) gained market approval. One such mean is via expanded access or compassionate use programs where terminally ill patients receive experimental new drugs that are not yet market approved. In this paper, I examine some of the common justifications for such programs. MAIN BODY: The most frequently voiced justifications for compassionate use or expanded access programs could be put in one of three categories. First, there are justifications of justice, where compassionate use programs could be seen as a just or fair way to distribute experimental new drugs to patients who are denied access to RCT's through no fault of their own. Second, such programs could be justified by reference to the ethical principle of beneficence where it could be claimed that terminally ill patients stand to benefit greatly at very little risk (as they are already dying). Third, there are considerations of autonomy where, it is claimed, patients should be able to exercise their autonomy and have access to such drugs if that is there free choice and they are fully aware of the risks associated with that choice. SHORT CONCLUSION: In this paper, I argue currently all justifications are potentially problematic. If they truly form the basis for justification, compassionate use programs should be designed to maximize justice, beneficence and autonomy.
Assuntos
Pesquisa Biomédica/ética , Ensaios de Uso Compassivo/ética , Dissidências e Disputas , Análise Ética , Acessibilidade aos Serviços de Saúde/ética , Terapias em Estudo/ética , Beneficência , Humanos , Autonomia Pessoal , Preparações Farmacêuticas , Justiça Social , Assistência TerminalRESUMO
Texas' right-to-try law makes experimental drugs more accessible to terminally ill patients when all other FDA-approved treatment options are unavailable or are unlikely to prolong life.
Assuntos
Ensaios de Uso Compassivo/legislação & jurisprudência , Drogas em Investigação/uso terapêutico , Acessibilidade aos Serviços de Saúde/legislação & jurisprudência , Neoplasias Ovarianas/tratamento farmacológico , Inibidores de Poli(ADP-Ribose) Polimerases/uso terapêutico , Doente Terminal , Ensaios de Uso Compassivo/ética , Feminino , Humanos , Imunoterapia , Direitos do Paciente , TexasAssuntos
Anticorpos Monoclonais/uso terapêutico , Ensaios de Uso Compassivo/métodos , Indústria Farmacêutica/métodos , Mieloma Múltiplo/tratamento farmacológico , Seleção de Pacientes/ética , Criança , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/legislação & jurisprudência , Ensaios Clínicos Controlados como Assunto/métodos , Citosina/análogos & derivados , Citosina/uso terapêutico , Aprovação de Drogas/legislação & jurisprudência , Indústria Farmacêutica/ética , Indústria Farmacêutica/legislação & jurisprudência , Humanos , Masculino , Organofosfonatos/uso terapêutico , Defesa do Paciente , Mídias Sociais/estatística & dados numéricos , Estados Unidos , United States Food and Drug Administration/legislação & jurisprudênciaAssuntos
Comitês Consultivos/ética , Ensaios de Uso Compassivo/ética , Tomada de Decisões/ética , Indústria Farmacêutica/ética , Comitês Consultivos/organização & administração , Anticorpos Monoclonais/uso terapêutico , Antineoplásicos/uso terapêutico , Temas Bioéticos , Ensaios de Uso Compassivo/estatística & dados numéricos , Aprovação de Drogas , Indústria Farmacêutica/estatística & dados numéricos , Guias como Assunto , Humanos , Mieloma Múltiplo/tratamento farmacológico , Defesa do Paciente , Estados Unidos , United States Food and Drug AdministrationAssuntos
Ensaios de Uso Compassivo/legislação & jurisprudência , Aprovação de Drogas , Indústria Farmacêutica/legislação & jurisprudência , Drogas em Investigação , Ensaios Clínicos como Assunto , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/tendências , Aprovação de Drogas/legislação & jurisprudência , Aprovação de Drogas/métodos , Aprovação de Drogas/organização & administração , Indústria Farmacêutica/ética , Drogas em Investigação/uso terapêutico , Humanos , Estados Unidos , United States Food and Drug AdministrationAssuntos
Esclerose Lateral Amiotrófica/terapia , Ensaios de Uso Compassivo/legislação & jurisprudência , Direitos do Paciente/legislação & jurisprudência , Temas Bioéticos , Colorado , Ensaios de Uso Compassivo/ética , Aprovação de Drogas , Drogas em Investigação/provisão & distribuição , Drogas em Investigação/uso terapêutico , Humanos , Seleção de Pacientes/ética , Transplante de Células-Tronco/efeitos adversos , Transplante de Células-Tronco/economia , Estados Unidos , United States Food and Drug AdministrationRESUMO
Patient access to experimental drugs outside of clinical trials is called compassionate use or expanded access. Compassionate use/expanded access presents a powerful ethical dilemma in that it involves competing claims that both have moral weight: specifically, an individual patient's very understandable desire to try to extend his or her life versus the orderly and efficient functioning of a drug development and clinical trial system that benefits much larger numbers of patients. Patient advocates, the FDA, pharmaceutical trade groups, and state and national legislators in the US are all currently weighing in on patient access to experimental drugs, and new guidelines and rules are being introduced. In this editorial, we discuss the impulse to rescue individual patients facing dire diseases and underscore the ethical questions that such rescue efforts raise.
Assuntos
Ensaios de Uso Compassivo/tendências , Drogas em Investigação/uso terapêutico , Aplicação de Novas Drogas em Teste , Ensaios de Uso Compassivo/ética , Ensaios de Uso Compassivo/legislação & jurisprudência , Política de Saúde , Humanos , Aplicação de Novas Drogas em Teste/legislação & jurisprudênciaRESUMO
The recent Ebola outbreak in West Africa began in the spring of 2014 and has since caused the deaths of over 6,000 people. Since there are no approved treatments or prevention modalities specifically targeted at Ebola Virus Disease (EVD), debate has focused on whether unproven interventions should be offered to Ebola patients outside of clinical trials. Those engaged in the debate have responded rapidly to a complex and evolving crisis, however, and this debate has not provided much opportunity for in-depth analysis. Additionally, the existing literature on access to unproven therapies has focused on contexts like HIV/AIDS and oncology, which are very different than the Ebola epidemic. In this paper, we examine the ethical issues surrounding access to unproven therapies in the context of the recent Ebola outbreak to yield new insights about this controversial and unsettled issue. We argue first that, in this context, the interests of patients in obtaining access to unproven therapies are not fully aligned with the interests of their providers and drug developers. Second, we focus on the resource constraints facing providers, funders, and patients and conclude that they often counsel against the use of unproven interventions against EVD.