Intellectual Property and Access to Noninvasive Prenatal Genetic Testing

知识产权和无创产前基因检测的获取

• 批准号: 8421496
• 负责人: Subhashini Chandrasekharan
• 金额: $ 30.09万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-05-07 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8421496
• 关键词: Address; Adoption; Affect; Aneuploidy; Base Sequence; Bioethics; Bioethics Consultants; Biomedical Technology; Businesses; Cells; Clinical; Clinical Trials; Complex; DNA; Data; Decision Making; Development; Diagnostic; Diagnostic Procedure; Down Syndrome; Educational workshop; Ethics; Fostering; Genes; Genetic; Genetic screening method; Genomics; Goals; Guidelines; Health; Health Insurance Reimbursement; Health Policy; Healthcare; Hereditary Disease; Industry; Intellectual Property; Interview; Investments; Justice; Laboratories; Laws; Legal; Legal patent; Licensing; Light; Litigation; Maps; Medicine; Methods; Modeling; Neonatal; Outcome; Outcome Study; Outcomes Research; Paper; Patients; Policies; Policy Analysis; Policy Developments; Pregnancy; Principles of law and justice; Property Rights; Provider; Public Health; Qualifying; Qualitative Research; Quality Control; Regulation; Relative (related person); Research; Research Methodology; Research Personnel; Resources; Secure; Shadowing (Histology); Structure; Technology; Testing; Translations; Uncertainty; Universities; base; clinical practice; commercialization; cost; cost effectiveness; experience; fetal; fetal medicine; genetic analysis; health economics; improved; innovation; multidisciplinary; policy implication; prenatal; prenatal testing; public health relevance; reproductive; research and development; screening; theories

DESCRIPTION (provided by applicant): The purpose of this study is to provide empirical data on effects of intellectual property (IP) and commercialization on clinical translation of noninvasive prenatal genetic testing (NIPT) and identify potential barriers to clinical adoption and patient access. Advances in technologies for genetic analysis of cell-free fetal DNA could make NIPT routine. Early clinical trials indicate that sequencing-based NIPT tests for chromosomal aneuploidies are more accurate than currently used noninvasive screening tests. A commercial NIPT test for Down Syndrome recently became available and tests for common genetic conditions are in prospect. It is still too early to know the clinical utility and cost effectiveness of these tests. Nevertheless, NIPT could significantly change the paradigm of prenatal testing and screening and potentialy even lower costs. Intellectual property (IP) and commercialization promise to be important components in the emerging debate about when and how such technologies should enter clinical practice. IP could induce commercial investment in R&D, in regulatory approval, and in securing third-party payment. But exclusive IP rights could also hamper innovation, increase transaction costs for test developers and providers, and decrease patient access, especially if monopolies emerge. Indeed patents on foundational NIPT technologies have been exclusively licensed to companies, raising such concerns. The commercial landscape is quickly evolving and companies are already involved in patent litigation. The disposition of these patents could determine who can offer the tests and the business models that will prevail, which in turn can impact clinical adoption and patient access. The IP landscape for NIPT appears complex and is unclear. Few if any data are also available on stakeholders' views about effects of IP vs non-IP factors on clinical adoption, and patient access to NIPT.. This study will address these gaps with the following specific aims: 1) map IP relevant to NIPT and assess potential IP effects on development of new NIPT genetic tests; 2) identify and rank IP versus non-IP barriers to clinical adoption and patient access based on stakeholders' views; and 3) identify ethical and policy implications of potential barriers to patiet access. A multidisciplinary team of researchers with expertise in genetics, IP law, health policy, bioethics, health economics, maternal and fetal medicine and health law will use established qualitative research methods combined with legal, ethical, and policy analysis. One outcome of this study will be a careful empirical analysis of whether and how IP can affect patient access to NIPT genetic testing. This analysis will be enabled by a publicly available IP and commercialization landscape for NIPT technologies that we will create. Another expected outcome is a forecast of barriers to clinical adoption and patient access ranked by stakeholders. A workshop at the conclusion of the study will include stakeholder representatives groups and experts from relevant domains to identify approaches and policy priorities for reducing barriers to clinical translation and promoting patient access to NIPT.

描述（由申请人提供）：本研究的目的是提供关于知识产权（IP）和商业化对无创产前基因检测（NIPT）临床转化的影响的经验数据，并确定临床采用和患者获取的潜在障碍。无细胞胎儿DNA遗传分析技术的进步可以使NIPT成为常规。早期临床试验表明，基于测序的NIPT检测染色体非整倍体比目前使用的无创筛查测试更准确。一种用于唐氏综合症的商业NIPT测试最近可用，对常见遗传条件的测试也有望实现。现在要知道这些测试的临床效用和成本效益还为时过早。然而，NIPT可以显著改变产前检测和筛查的模式，甚至可能降低成本。知识产权（IP）和商业化有望成为有关这些技术何时以及如何进入临床实践的新兴辩论的重要组成部分。知识产权可以在研发、监管审批和确保第三方支付方面吸引商业投资。但是，专有知识产权也可能阻碍创新，增加测试开发人员和提供者的交易成本，并减少患者获取，特别是在出现垄断的情况下。事实上，基础NIPT技术的专利已经被专门授权给公司，这引起了人们的担忧。商业格局正在迅速演变，许多公司已经卷入了专利诉讼。这些专利的处置可以决定谁可以提供测试和将盛行的商业模式，这反过来又可以影响临床采用和患者获取。NIPT的知识产权前景似乎复杂且不明朗。利益相关者关于知识产权与非知识产权因素对临床采用和患者获得NIPT的影响的看法，即使有数据也很少。本研究将解决这些差距，具体目标如下：1)绘制与NIPT相关的IP图谱，评估IP对开发新的NIPT基因检测的潜在影响；2)根据利益相关者的观点，确定知识产权与非知识产权在临床应用和患者获取方面的障碍，并对其进行排名；3)确定患者获取的潜在障碍的伦理和政策影响。一个由遗传学、知识产权法、卫生政策、生物伦理学、卫生经济学、母胎医学和卫生法等专业知识的多学科研究人员组成的小组将利用已确立的定性研究方法，结合法律、伦理和政策分析。本研究的一个结果将是对知识产权是否以及如何影响患者获得NIPT基因检测进行仔细的实证分析。这种分析将通过我们将创建的NIPT技术的公开可用的IP和商业化景观来实现。另一个预期结果是对临床采用和患者获取障碍的预测，由利益相关者进行排名。研究结束时将举行研讨会，包括利益攸关方代表、团体和相关领域的专家，以确定减少临床转化障碍和促进患者获得NIPT的方法和政策重点。