Widening patient engagement for orphan drugs trials

背景孤儿药是指用于诊断、预防或治疗危及生命或进行性疾病的罕见药物。在一般人群中，罕见（或“罕见”）疾病的发病率不到1/2000。例如特发性肺纤维化（IPF），这是一种缩短寿命的肺部疾病，随着时间的推移会恶化，使患者呼吸困难。制药行业最近强调了开发孤儿药的重要性，无论是出于医疗还是经济原因。随着新的疾病亚型的发现，罕见疾病的数量每年都在增加，用于治疗这些疾病的新药的资金也在增加。英国在药物开发的早期阶段是欧洲的领导者，但在评估药物安全性或与其他治疗方法相比的后期阶段福尔斯于其他国家。如果英国想成为药物开发的世界领导者，这一点很重要。为应对英国脱欧和COVID-19大流行，英国罕见病战略和英国制药工业协会都呼吁将更广泛的患者纳入临床药物试验，但研究表明，参与和拒绝或退出孤儿药试验的患者的需求和经历往往被忽视。我们对孤儿药患者经验的了解是基于对参与试验和/或治疗有积极经验的患者、提供这些治疗的医生以及在药物/试验开发中发挥作用的患者和公众代表的调查答复。调查答复大多是描述性的，缺乏深入、丰富的细节，无法为改进或创新提供信息。对于孤儿药临床试验中未接受活性药物治疗（安慰剂组或对照组）的患者和退出试验的患者的经验，存在特别的知识差距。我们该怎么办？一名经验丰富的定性研究人员将被借调到一家欧洲制药公司（加拉帕戈斯），以促进生物医学行业和学术界之间的知识交流，为英国制药行业和孤儿患者寻找深度学习的机会。我们的目标是：-了解更多关于孤儿药开发和深入的IPF患者/家庭经验。- 在药学实践中嵌入定性研究（例如深入访谈和焦点小组），使更广泛的患者/家庭群体有机会参与孤儿药研究。- 通过以下研究活动，扩大孤儿药的参与范围，扩大孤儿药的覆盖面和受益范围，改善临床试验和药物使用，使孤儿患者和制药行业受益：-与IPF患者、其医疗保健专业人员以及招募患者参加药物试验的组织的人员进行深入访谈。与工作人员的访谈和对加拉帕戈斯价值观和做法的观察。- 培训加拉帕戈斯的工作人员与病人进行访谈和焦点小组，并评估其有效性。患者和公众参与（PPI）的代表将定期审查研究结果，并为患者和招募患者参加孤儿药试验的第三方组织共同设计可访问的资源。这些信息将用于全面了解加拉帕戈斯的转型计划，以扩大患者参与。将制定和评估使用定性方法的培训和指导，以将患者参与知识交流纳入宿主活动。最终产出将解决ABPI目标，包括面向患者的资源，用于招募孤儿药试验，科学家和学者研讨会以及同行评审的出版物。

BackgroundOrphan drugs are medicines intended for diagnosis, prevention or treatment of life-threatening or progressive disorders that are rare. A rare (or 'orphan') disease affects less than 1 in 2,000 people within the general population. An example is Idiopathic Pulmonary Fibrosis (IPF), a life shortening lung disease that gets worse over time making it hard for the person who has it to breathe.The pharmaceutical industry has recently highlighted the importance of the development of orphan drugs for both medical and financial reasons. The number of rare diseases increases each year as new sub-types of disease are identified, as does the amount of money spent on creating new medicines to treat them. The UK is a European leader in the early stages of drug development, but falls behind other counties in the later phases which assess the safety of a drug or how it might compare with other treatments. This matters if the UK wants to become a world-leader in drug development. In response to Brexit and the COVID-19 pandemic, the UK Strategy for Rare Diseases and the Association of the British Pharmaceutical Industry have both called for the inclusion of a wider range of patients in clinical drug trials, but research suggests that the needs and experiences of patients who take part and decline or withdraw from orphan drug trials are often overlooked. What we know about patient experience of orphan drugs is based on survey responses from patients who have had a positive experience of trial participation and/or treatment, doctors who provide these treatments, and patient and public representatives who have played a role in drug/trial development. Survey responses are largely descriptive, and lack in-depth, rich detail needed to inform improvement or innovation. There are particular gaps in knowledge about the experience of patients in clinical trials of orphan drugs who do not receive the active treatment (placebo or comparator groups), and those who drop out of a trial. What will we do?An experienced qualitative researcher will be seconded to a European-wide pharmaceutical company (Galapagos) to facilitate knowledge exchange between the biomedical industry and academia, to identify opportunities for deep learning for the UK pharmaceutical industry and patients with orphan conditions. We aim to:- Learn more about orphan drug development and in-depth patient/families experience of IPF. - Embed qualitative research (e.g. in-depth interviews and focus groups) in pharmaceutical practice to enable a wider group of patients/families to have the opportunity to be involved in orphan drug research. - Expand orphan drug engagement to widen their reach and benefit, improve clinical trials and medicine use, to benefit both people with orphan conditions and the pharmaceutical industry.This will be possible through the following research activities:- In-depth interviews with IPF patients, their healthcare professionals, and people from organisations that recruit patients to drug trials.- Interviews with staff and observations of Galapagos' values and practice. - Training Galapagos staff to conduct interviews and focus groups with patients, and assess their effectiveness.- Patient and Public Involvement (PPI) representatives will regularly review and input into the research findings and co-design accessible resources for patients, and third party organisations that recruit patients to orphan drug trials.This information will be used to produce a comprehensive understanding of the Galapagos' transformation planning for widening patient engagement. Training and guidance for using qualitative methods to embed patient engagement for knowledge exchange in host's activities will be developed and assessed. Final outputs will address the ABPI objectives and include patient-facing resources for recruitment to trials of orphan drugs, seminars for scientists and academics, and peer reviewed publications.