Connexion Healthcare, 6 Terry Drive, Newtown, PA, 18940, USA.
International Fibrodysplasia Ossificans Progressiva Association (IFOPA), 1520 Clay Street, Suite H-2, North Kansas City, MO, 64116, USA.
Orphanet J Rare Dis. 2018 Jan 22;13(1):18. doi: 10.1186/s13023-018-0761-2.
Rare diseases are a global public health concern, affecting an estimated 350 million individuals. Only 5% of approximately 7000 known rare diseases have a treatment, and only about half have a patient advocacy organization. Biopharmaceutical companies face complex challenges in developing treatments for rare diseases. Patient advocacy organizations may play a major role by positively influencing research and development, clinical trials, and regulations. Thus, collaboration among patient advocacy organizations and industry is essential to bring new therapeutics to patients.
We identified an unmet need for guidelines on day-to-day decision-making by rare disease patient advocacy organizations when working with biopharmaceutical partners. We convened an Independent Expert Panel experienced in collaborations between patient advocacy organizations and biopharmaceutical companies (April 2017) to develop consensus guidelines for these relationships. The guidelines were based on an original version by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA). The Expert Panel reviewed and broadened these to be applicable to all patient advocacy organizations. Comments on the draft Guidelines were provided first by Panel participants and subsequently by six independent experts from patient advocacy organizations and industry.
The Panel comprised four experts from the rare disease community who lead patient advocacy organizations; three leaders who perform advocacy functions within biopharmaceutical companies; and two facilitators, both having leadership experience in rare diseases and industry. The finalized Guidelines consist of four main sections: Identification and Engagement With Companies, Patient Engagement and Patient Privacy, Financial Contributions, and Clinical Trial Communication and Support. The Guidelines address the daily considerations, choices, and consequences of patient advocacy organizations as they engage with biopharmaceutical companies, and offer recommendations for volunteer/paid leaders of the organizations on how to interact in a thoughtful, responsible, ethical way that engenders trust.
These Guidelines recommend best practices and standards for interactions between patient advocacy organizations and industry that will ultimately have a positive effect on the development of novel treatments. Patient advocacy organizations will be provided free access to these Guidelines to help bring clarification to day-to-day decision-making around their interactions, and for use as a living document with the potential for regular revisions and updates.
罕见病是全球公共卫生关注的一个问题，据估计影响了 3.5 亿人。在大约 7000 种已知的罕见病中，只有 5%有治疗方法，只有大约一半有患者倡导组织。生物制药公司在开发罕见病治疗方法方面面临着复杂的挑战。患者倡导组织可以通过积极影响研究与开发、临床试验和监管，在其中发挥重要作用。因此，患者倡导组织与行业之间的合作对于为患者带来新的治疗方法至关重要。
我们发现，当患者倡导组织与生物制药合作伙伴合作时，他们在日常决策方面需要指导，但目前缺乏相关指导。我们召集了一个在患者倡导组织与生物制药公司合作方面经验丰富的独立专家小组（2017 年 4 月），为这些关系制定共识指南。这些指南是基于国际纤维性骨发育不良进展协会（IFOPA）的原始版本制定的。专家小组对这些指南进行了审查和扩展，以使其适用于所有患者倡导组织。对指南草案的意见首先由小组成员提供，然后由来自患者倡导组织和行业的六名独立专家提供。
小组成员包括四位来自罕见病社区的专家，他们领导着患者倡导组织；三位在生物制药公司中从事倡导工作的领导者；以及两位协调人，他们在罕见病和行业都有领导经验。最终的指南由四个主要部分组成：与公司的识别和参与、患者参与和患者隐私、财务贡献以及临床试验沟通和支持。指南解决了患者倡导组织在与生物制药公司合作时的日常考虑、选择和后果问题，并为组织的志愿/付费领导人提供了如何以深思熟虑、负责任和合乎道德的方式互动的建议，从而建立信任。
这些指南为患者倡导组织与行业之间的互动提出了最佳实践和标准，最终将对新治疗方法的开发产生积极影响。将向患者倡导组织免费提供这些指南，以帮助澄清他们互动的日常决策，并将其作为一个具有潜在定期修订和更新的活文档使用。
