BACKGROUND Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. METHODS In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients' views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. RESULTS Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. CONCLUSION When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.

中文翻译：

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未满足医疗需求的患者需要什么？对患者的观点和经验进行定性研究，以扩大在荷兰的未经批准的研究治疗方法的使用范围。

背景技术有未满足的医疗需求的患者有时会诉诸非标准的治疗选择，包括在临床试验，富有同情心的使用或指定患者的方案中使用未经批准的研究药物。对于尚未批准的研究用药物，未满足医疗需求的患者的观点和经验尚未经过经验检验。方法在这项定性研究中，对患有慢性或危及生命的疾病（n = 39）的患者进行了探索性访谈和焦点小组讨论，涉及与非标准治疗方案有关的主题，例如寻找非标准治疗方案，患者对医生的道德义务以及他们愿意或不希望使用非标准治疗方案的条件的看法，包括扩大获得未经批准的治疗的机会，研究药物。结果尽管一些受访者正在积极寻找非标准的治疗选择，但受访者对扩大获取研究药物的现有机会的知识和/或经验很少。他们对治疗医生抱有很高的期望，假设他们知道非标准的治疗选择，包括其他地方的临床试验和扩大的访问计划，并假设他们会告知患者有关这种选择的信息。受访者谨慎地权衡了寻求扩大获取途径的风险和潜在利益，理由是与药物安全性和有效性，副作用，药物相互作用以及维持良好生活质量的科学证据有关。受访者强调了教育和自信对于获得优质医疗保健的重要性，并愿意为研究用药自掏腰包。患者对平等获得新的和/或非标准的治疗选择表示担忧。结论当标准治疗方案的终点出现时，患者可能更喜欢主治医生与他们讨论非标准治疗方案，包括扩大获得未经批准的研究性药物的机会。尽管我们的受访者对扩展获取计划的理解程度不同，但他们似乎能够就非标准治疗方案做出充分考虑的选择，并对此类选择的安全性和有效性抱有现实的期望。