wanghaisheng commented 6 years ago

wanghaisheng commented 6 years ago

https://mp.weixin.qq.com/s?src=11&timestamp=1524930900&ver=844&signature=XVm3tL2NexqKxM-G1kqcZeTaoPhAzgglyuEwnxzO6P1vE1IjgGlaCNbvsns-kh6juLcx3s5t0ceAoVKMXOQS5ejQNfiBzCQVHHfF3KRqY81SHe9zqDWKtqIjbv-eNjlL&new=1 殷咏梅教授：CSCO BC于3年前在江泽飞教授牵头下组织了全国的11家中心，迄今已收集3万余例乳腺癌患者的数据资料，并从不同层面对这些资料进行了回顾分析。我们3人将对各自所在中心不同的数据分析内容进行介绍。首先，请王海波教授对您中心的“中国乳腺癌病例特征”的研究设计思路、主要研究结果及临床实践意义进行一下介绍。王海波教授：当时创建CSCO BC数据库的初衷是通过真实世界数据的研究来弥补临床试验的不足，我的任务是对中国女性乳腺癌患者按不同年龄段进行分组，来观察临床病理学及治疗模式的不同。通过最近的研究发现了许多有意义、有价值的结果。过去的统计没有临床数据，这次是将数据库中近3万例不同年龄段的女性乳腺癌患者以10岁进行分段，观察不同年龄段患者临床病理学和治疗模式的不同。从目前研究来看，整体而言，国内女性乳腺癌患者实际发病年龄与流行病学调查结果基本相似，高发年龄段为40~50岁，和国外略有不同，美国高发年龄段是在60岁左右，相较之下我国的患者高发年龄约年轻10岁。我们的研究还发现，小于40岁的女性患者整体临床病理分期偏晚，3期、4期占的比例较高，T3患者比例较高。另外，在过去国外报道中三阴性分子分型在年轻患者的比例较高，但这次有意思的是，从现有分析来看各年龄段分子分型没有太大差异，只是在>70岁的Luminal A型较高。对于我们关注的三阴性，不同年龄段并没有太大差异，这可能是中国人的特点，下一步需要对此进行进一步的探讨。

治疗模式方面，对于Luminal A型淋巴结阴性患者，CSCO BC指南中推荐要慎重选择化疗，但我们在临床实际工作中发现，对于<40岁的女性患者大多选择了新辅助治疗或化疗，即临床中对于这些年轻女性稍有些“矫枉过正”的治疗，加大了治疗或略强于指南的推荐，中国特色很明显。还有OFS，指南推荐的是用于绝经前具有中高危复发因素的患者，在实际调查中，我们也确实发现绝经前具有中高危复发因素的患者应用的比例较高，这与指南相符。

另外，对于保乳手术，欧美国家60岁以上患者采用的比例较高，而中国患者在40岁以下，特别是30岁以下的患者采用保乳术的比例较高，这也体现了“中国特色”，说明中国年轻女性对身体的美观度要求更高。整体而言，此次研究就像淘金矿一样淘到了一些金子，发现了一些对临床具有指导意义的数据。

wanghaisheng commented 6 years ago

大数据时代肿瘤心脏病学的机遇和挑战 https://mp.weixin.qq.com/s?src=11&timestamp=1524933921&ver=844&signature=fZZpi1HH43jeD*SrD6V2-b0gLzfaOTGUm6OD3EvEYXJCIkcMiEqp45gXcsqwAu-TqeoRWGg*YFhwK5qZWO3qgqMQT*ML2B7MZG9mufrCbY8XM7brzzihX4qwzQA1l4Rj&new=1

wanghaisheng commented 6 years ago

https://www.ema.europa.eu/en/human-regulatory/post-authorisation/patient-registries

wanghaisheng commented 6 years ago

an inventory of patient registries http://www.encepp.eu/encepp/resourcesDatabase.jsp

wanghaisheng commented 6 years ago

https://www.ema.europa.eu/documents/presentation/presentation-registry-initiative-april-2018_en.pdf

Patient registries are organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time. Patient registries can play an important role in monitoring the safety of medicines. The European Medicines Agency (EMA) has set up an initiative to make better use of existing registries and facilitate the establishment of high-quality new registries if none provide an adequate source of post-authorisation data for regulatory decision-making.

The initiative for patient registries, launched in September 2015, explores ways of expanding the use of patient registries by introducing and supporting a more systematic and standardised approach to their contribution to the benefit-risk evaluation of medicines within the European Economic Area:

wanghaisheng commented 6 years ago

http://rethinkingclinicaltrials.org/chapters/conduct/startup/startup-introduction/

wanghaisheng commented 5 years ago

https://espn-online.org/images/sunumlar/S.%20Parker%20-%20Global%20registries%20challenges%20and%20solutions-Moscow%20CME-%202013.pdf Global registries for rare diseases:challenges and solutions S. Parker - Global registries challenges and solutions-Moscow CME- 2013.pdf

wanghaisheng commented 5 years ago

https://www.ncbi.nlm.nih.gov/books/NBK164514/

Patient Registries

Patient registries have been defined as “an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves a predetermined scientific, clinical, or policy purpose(s).”6 In brief, a patient registry is a collection—for one or more purposes—of standardized information about a group of patients who share a condition or experience. The use of “patient” in patient registries is often used to distinguish the focus of the data set on health information. Currently, there is no consistent definition of the term “patient registry” used in the health research field. Terms such as clinical registries, clinical data registries, disease registries, and outcomes registries are also used to describe the same data collection method.7 Examples of individual researcher-generated registries can be found at the AHRQ Registry of Patient Registries, available at https://patientregistry.ahrq.gov.

Traditional Patient Registries

Patient registries have traditionally been researcher-generated. Research institutions, academic clinical institutions, or individual research teams establish a registry, using private or Federal funds, for the purpose of observational data collection that can be used for a specific research agenda. These registries may be organized and operated in a variety of forms and formats. They may be operated by a single institution or by a collaborative of multiple institutions or clinics. Researcher-generated patient registries currently exist for a wide range of chronic or rare conditions, including many forms of cancer, diabetes, cystic fibrosis, acute coronary syndrome, and arthritis.

The purposes for patient registries can range widely. According to the National Institutes of Health, “Registries can be used to recruit patients for clinical trials to learn about a particular disease or condition; to develop therapeutics or to learn about population behavior patterns and their association with disease development; developing research hypotheses; or for improving and monitoring the quality of health care.”8 Patient registries can also be used to monitor outcomes and study best practices in care or treatment.9 They may pursue a specific, focused research agenda, collecting data for a limited time to answer a specific research question (or questions), or may collect data on an indefinite basis to answer a variety of existing and emerging research questions. Patient registries may also include the collection of tissue or blood samples collected in a variety of ways.

The creation and use of researcher-generated patient registries has grown steadily for several decades, although the actual number of existing registries in the United States is unknown. In 2012, the Agency for Healthcare Research and Quality launched an online registry of patient registries to provide a searchable database of patient registries in the United States.10

Patient-Powered Patient Registries (PPRs)

PPRs are similar in many ways to researcher-generated patient registries in definition, purpose, and features. At times, these registries are somewhat indistinguishable from traditional registries, with one exception: In patient-powered patient registries, patients and family members “power” the registry by managing or controlling the collection of the data, the research agenda for the data, and/or the translation and dissemination of the research from the data.

Experts in the field, however, differ in their individual conceptualizations of what constitutes a valid PPR. In the view of some, only registries that are created, maintained, and controlled by patients or patient advocacy organizations can be considered “patient-powered,” while others focus on the specific contributions of patients—that is, their involvement and contribution to all aspects of the registry—as the critical factor, regardless of the registry’s ownership or involvement by commercial or professional interests. These differences in conceptualization have added to the challenges of classifying PPRs for the broader research field. Terms such as “patient-generated,” “patient-run,” “patient-powered,” and “participant-controlled” can be found among various users.

Patient-powered patient registries are also organized and operated in a variety of forms and formats. They may be operated by a single organization or by a collaborative of multiple organizations. Like researcher-generated registries, patient-powered patient registries exist for a wide range of conditions.

PPRs may also pursue a specific research question or conduct ongoing data collection to answer a variety of existing and emerging research questions. Several PPRs have biobanks, or repositories, where patients can provide samples of blood or tissue to be used in research. Other patient advocacy organizations, such as the TMJ Association, use their registry as a recruitment vehicle for existing clinical trials, inviting members of the TMJ community whose profile matches a trial protocol.

Although the genesis of PPRs has not been studied or documented, most seem to originate from a patient support or advocacy organization, either as the direct intention of the organization or as an added component. The goal of these registries is to enhance translational research by providing data that could better characterize the disease, discover biomarkers, or provide information to assist patient and family decisionmaking.11 In several cases, the creation of a PPR followed the formation of a support and information-sharing network of patients or families who shared a set of experiences with a disease or condition. The earliest documented PPR is the Hereditary Disease Foundation, created in 1983. While the support and advocacy network met a variety of patient and family needs, the foundation’s co-founder, Nancy Wexler, also collected samples from people who were affected by Huntington Disease for the purpose of advancing research.4 An example of the typical genesis of a PPR can be found in the story of Pat Furlong, a registered nurse and the mother of two sons, Patrick and Christopher, who were both diagnosed with Duchenne muscular dystrophy when little was known about the condition. Refusing to accept that answers could not be found, she reached out to other families of the rare disease, founded the Parent Project Muscular Dystrophy, and eventually created a patient and family registry, DuchenneConnect (www.duchenneconnect.org), which is both an information sharing network and registry.12 An additional examples of patient-powered individual condition registries is the Life Raft Group Patient Registry and Tissue Bank (http://liferaftgroup.org/patient-registry), which collects data from patients affected by Gastrointestinal Stromal Tumor (GIST), a rare family of cancers.

Like researcher-generated patient registries, there is no single complete listing or documented number of PPRs in the United States. An effort to document patient-powered patient registries is being undertaken by the American Association for the Advancement of Science through funding by the Agency for Healthcare Research and Quality.13 One study of 201 disease advocacy organizations found that forty-five percent had supported a research registry or biobank.11

Concerns About Patient Registries

Patient registries have been promoted and praised in both chronic14,15 and rare16,17 disease practice communities, and debated among comparative effectiveness researchers as to whether they provide valid data to compare treatments.2,18–20 Patient-generated patient registries in particular have been criticized on several levels, including the concern that only a small minority of patients with sufficient education and ability are able to participate, and that data may be biased for a variety of reasons.21 Experts have noted the lack of standardization in data collection and potential competition for registered patients across registries, which could create a fractured set of patient data. For example, if multiple patient registries exist for a single condition, there is a greater likelihood that competition for patients may limit any given data set. This is a particular concern for rare diseases, where an affected population may be very small, resulting in small data sets for each registry that are less able to draw valid conclusions for the population. Meta-analysis across registries may be challenging or impossible should each competing registry collect different data from the same patients, or collect the data under different timeframes.16 More specific concerns have been expressed regarding issues of patient consent and rights violations relative to tissue samples submitted to biobanks, in particular the concern about whether patients are fully aware of the possible subsequent uses of their specimens.22 Despite these concerns, patient registries have yielded a significant amount of research that meets the needs of patients and families. PPRs have also added to disease and treatment knowledge. In addition to self-published monographs, several PPRs have used their data or had their data used in published research, although no formal list of publications across PPRs exists.4,11,23

Research Networks

Some patient registries are part of broader research collaboratives that connect individual registries into a larger network of registries that collect data on one or more conditions. The network provides a shared infrastructure and standardized data collection across registries. Collected data from each registry may be combined for analysis, although participation in a research network does not eliminate the ability of any individual patient registry from analyzing only the data from the registry alone. Research networks may also have existing and emerging research agendas that are realized through an established collection of researchers who study the collective or registry-specific data based on the wishes of the patient network or each registry. In some cases, the network may make data available to researchers who request use for purposes that are unrelated to the agenda of those operating the registries.

Both researcher-generated and patient-powered research networks exist. An example of a researcher-generated research network is the SEER registries (seer.cancer.gov/registries), a collaboration of 19 registries created and managed by the National Cancer Institute. An example of a patient-generated research network is the Genetic Alliance Registry and BioBank (www.biobank.org), a collaboration of more than 1,200 individual disease advocacy organizations.

There is little published literature to date on PPRNs, although several large networks have emerged in the past five years. A report created for the Patient-Centered Outcomes Research Institute (PCORI), developed a taxonomy of research networks in an effort to create an inventory of existing networks. The taxonomy classified three distinct categories: Clinical Data Research Networks (CDRN), Patient-Powered Research Networks (PPRN), and Patient Registries based on a set of distinguishing characteristics.24 Although the taxonomy and characteristics vary somewhat from other descriptions and opinions, the taxonomy serves as a critical starting point for future research on the use of patient-powered networks and patient registries as data collection tools.

The Genetic Alliance Registry and BioBank (GARB) serves as an example of the evolution that can occur from an individual patient-powered disease registry to a PPRN. In 1995, PXE International, identifying itself as a “research advocacy organization,” created a patient-powered registry and biobank to accelerate translational research in pseudoxanthoma elasticum, a rare genetic metabolic disorder. The organization, led by founder Sharon Terry, became a mentor to a number of other organizations that also wished to create PPRs. This soon led to the formation of GARB in 2003 with eight disease advocacy organizations, using their infrastructure, model, and methods to create the broader network.4 In a recent expansion of GARB, the registry platform has become disease-agnostic and now includes more than 2,000 diseases.

Other networks have had similar beginnings25. In 1998, Stephen Haywood was diagnosed with ALS (Lou Gehrig’s Disease). The Heywood family began searching across the globe for information that might extend or improve Stephen’s life. In 2004, frustrated by the lack of open and accessible information, Stephen’s two brothers, Benjamin and James Heywood, and a lifelong friend Jeff Cole, all MIT graduates, founded PatientsLikeMe with 12 disease communities.26 In 2011, PatientsLikeMe opened to all conditions and now includes approximately 1,200 individual disease registries. PatientsLikeMe is a for-profit corporation.25 A new not-for-profit network entitled Registries For All (www.reg4all.org) provides a single platform available to all patient-powered disease registries. The network was formed through a partnership of organizations including GARB, CFIDS Association of America, National Psoriasis Foundation, and the Inflammatory Breast Cancer Research Foundation. The team was awarded $300,000 in early 2013 from the Partners for Patient Health Innovation Challenge, funded by Sanofi US, an international pharmaceutical company.

It is important to note that PPRNs differ in their structure and operation. Some PPRNs such as GARB enable individual organizations and registries who are part of the network to maintain their autonomy and identity. Others, such as PatientsLikeMe, organize the network by disease communities that may be supported by multiple disease-advocacy organizations.

wanghaisheng commented 5 years ago

Patient Registries - Registries for Evaluating Patient Outcomes - NCBI Bookshelf.pdf

wanghaisheng commented 5 years ago

https://www.nih.gov/health-information/nih-clinical-research-trials-you/list-registries

List of Registries

var addthis_config = { ui_508_compliant: true }

Frequently Asked Questions

What is a registry?

A registry is a collection of information about individuals, usually focused around a specific diagnosis or condition. Many registries collect information about people who have a specific disease or condition, while others seek participants of varying health status who may be willing to participate in research about a particular disease. Individuals provide information about themselves to these registries on a voluntary basis. Registries can be sponsored by a government agency, nonprofit organization, health care facility, or private company. It’s always good to check first to know who sponsors the registry – or – look for information on a registry’s site to know about their sponsor(s).

Why are registries needed?

Registries can provide health care professionals and researchers with first-hand information about people with certain conditions, both individually and as a group, and over time, to increase our understanding of that condition. Some registries collect information that can be used to track trends about the number of people with diseases, treatments, and more. Other registries invite people to sign up to be contacted about participating in clinical research. These ask very basic questions about health history that would help determine whether someone is possibly eligible to join a research study.

It sounds like these registries collect personal health information. Is there a risk that such information could be disclosed?

Government agencies have strict privacy requirements set by law such as the Federal Information Security Management Act (FISMA), and the Health Insurance Portability and Accountability Act (HIPAA). If registries have followed all of these rules, the likelihood of identifiable personal information being shared is very small.

What benefits will someone receive from participating in a registry?

Participation in a registry is likely to increase what we know about a specific condition, help health care professionals improve treatment, and allow researchers to design better studies on a particular condition, including development and testing of new treatments. Being part of a clinical trials registry can help people interested in participating in research connect with clinical investigators. However, individuals (and their families) who choose to participate in a registry should understand that participation will not guarantee a treatment or cure for their condition or that they will be eligible to join a study.

Who has access to the information in a registry?

Usually, a federally-funded registry has a very limited list of individuals (registry coordinator) who may have access to participants’ personal, identifying information. ;Those individuals must be specially trained and certified regarding information security requirements.

Who owns the data from a registry? Who makes decisions about how these data will be used?

The data collected in a disease registry is stripped of personal information. It belongs to the sponsor of the registry, and depending on how the registry is set up, may be shared with the participants and their families, and approved health care professionals and researchers. However, personal, identifying information is kept private. Usually, a registry has a governing committee that makes decisions about how the data can be used or shared.

Can a participant withdraw from the registry?

Yes. Registries are free and voluntary; there is no penalty for choosing to withdraw at any point.

Who should the participant contact with additional questions or concerns?

For any questions about participation or any issues that may arise, registries provide a contact, usually the registry coordinator.

How is a registry different from a clinical trial?

Registries focused on specific diseases or conditions collect information voluntarily from people with those conditions. Clinical trials registries collect basic health information from people who agree to be contacted about participating in future clinical trials or studies.

A clinical trial is the study of new ways to prevent, detect or treat diseases or conditions. Volunteering for a registry does not mean a person has signed up for a clinical trial. Participation in a disease registry can sometimes become a first step toward participation in a clinical trial, but registries and specific trials are not directly linked.