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Registry -patient data

  • 1.  Registry -patient data

    Posted 20-Jan-2019 01:58
    Hi,
     
    Does anybody know about patient data registries and can such registries be cloud based? Does this require regulatory approval? Do the regulations differ for the various geographies?
     
    This is a new subject for me and any guidance in this regard will be helpful.
     
    Thanks & Regards,
     
    Rashmi Pillay
    Regulatory Affairs Associate

    Ellex 
    3-4 Second Avenue
    Mawson Lakes SA, 5095
     
    T + 61 8 7074 8105
    rpillay@ellex.com
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  • 2.  RE: Registry -patient data

    Posted 21-Jan-2019 09:11
    Most patient registries are web-based whether they are structured for health care provider input only, patient provider input only, or a combination. The regulatory and informed consent requirements can be complicated, depending on the type of data being collected and the purpose. The most important consideration for these registries is data encryption, security, and access control. In my opinion, I would not think of them any different than what I want from my online banking information.

    I can recommend the following online book if you would like to dig deeply into the question of registries. I believe all chapters are accessible online.

    Registries for Evaluating Patient Outcomes
    NCBI Bookshelf remove preview
    Registries for Evaluating Patient Outcomes
    This User's Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is 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 one or more predetermined scientific, clinical, or policy purposes.
    View this on NCBI Bookshelf >


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    Glen Park
    Jersey City NJ
    United States
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  • 3.  RE: Registry -patient data

    Posted 21-Jan-2019 10:26
    Another issue that I don't think FDA has gotten into yet is that, historically, patient registries have been used to serve the needs of healthcare providers and payors, not regulators.  For these reason, they have been designed to assess patient outcomes, rather than products.  The existing knowledge base is tilted toward (perhaps exclusively drawn from) this experience.  If anyone has yet dug deep into how well this knowledge base will serve to inform the establishment and maintenance of more product-focused registries intended for regulatory use, I've missed it.

    ------------------------------
    Julie Omohundro, ex-RAC (US, GS), still an MBA
    Principal Consultant
    Class Three, LLC
    Durham, North Carolina, USA
    919-544-3366 (T)
    434-964-1614 (C)
    julie@class3devices.com
    ------------------------------



  • 4.  RE: Registry -patient data

    Posted 21-Jan-2019 15:02
    Julie,
    While I agree with you that there are many challenges to using real world evidence in regulatory decision-making, it seems quite pessimistic to say that we are years away from the use of registry data for regulatory purposes.  If we accept the definition of real world evidence as data collected using standard research methods that are not part of controlled clinical trials, there is abundant evidence that FDA will make regulatory decisions using such data. In 2017 FDA approved a new indication (transcatheter aortic valve replacement) for a medical device based on product registry data without requiring traditional, randomized clinical trials.

    Probably the most widely accepted use of such data is natural history data. In 2017, FDA approved Brineura (cerliponase alfa) as a treatment for a form of Batten disease, following a "single-arm" study which used a natural history "control." FDA used real world data generated on studies of related diseases to approve Vimizin (Elosulfase alfa) in 2014, an enzyme replacement therapy (ERT), as the first approved treatment for Morquio syndrome. FDA approved Avelumab (a progammed death ligand blocking human IgG1 lambda monoclonal antibody) as a therapy for metastatic Merkel cell carcinoma.  Real world data was used as benchmarks to characterize the natural history of the disease. FDA approved Myozyme (alglucosidase alfa), an enzyme replacement therapy (ERT), in 2006 for treatment of Pompe disease, a rare inherited lysosomal storage disorder, following a clinical study which used a historical cohort of untreated individuals as a benchmark.

    Other approvals have been based on expanded access programs.  FDA approved Lutathera (lutetium Lu 177 dotatate), a radioactive drug (or radiopharmaceuical) for treatment of somastatin receptor-positive instances of a type of cancer that affects the pancreas or gastrointestinal tract known as gastroenteropancreatic neuroendocrine tumors (GEP-NETs), based in part on data generated through the expanded access program. 

    While the 21st Century Cures Act forced FDA to develop a specific program for real world evidence, the regulatory basis has always been there. The medical product world is just beginning to understand the potential power of this approach for rare diseases and difficult to treat conditions where randomized controlled trials cannot be performed to obtain the evidence that result in regulatory decisions. The FDA website on Real World Evidence provides a really good start to providing a clear path for using such data for decision-making.

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    Glen Park
    Jersey City NJ
    United States
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  • 5.  RE: Registry -patient data

    Posted 21-Jan-2019 10:17
    ​To the best of my knowledge, no regulatory approval is required to set up a registry.  The question is whether any given regulatory agency will accept the data in it to support regulatory approval or other regulatory requirements (eg, postmarket surveillance). 

    It's my impression that FDA is in the very early stages of struggling with the potential use of registries for regulatory something, under the fuzzy umbrellas of "real-world evidence" and "patient engagement." As of the second meeting of its Patient Engagement Advisory Committee last fall, it seemed to have progressed to the point of identifying these key (and interrelated) issues:  data integrity, data security, patient privacy, and patient consent.  All of these issues are significantly complicated by cloud storage, I think.

    If I were in dire need of a registry to address some type of FDA requirement, I personally would not put it in the cloud at this time.  I think these issues are challenging enough already.  If I were not in dire need of a registry to address some type of FDA requirement, I think I might wait this one out a bit, until FDA has gotten farther along on a road that may be a long and challenging one, and it's too soon to tell if it is likely to lead to a happy ending.  I think two years from now, things will be much clearer.

    ------------------------------
    Julie Omohundro, ex-RAC (US, GS), still an MBA
    Principal Consultant
    Class Three, LLC
    Durham, North Carolina, USA
    919-544-3366 (T)
    434-964-1614 (C)
    julie@class3devices.com
    ------------------------------



  • 6.  RE: Registry -patient data

    This message was posted by a user wishing to remain anonymous
    Posted 22-Jan-2019 08:15
    This message was posted by a user wishing to remain anonymous

    I agree with Julie, the registry data or Real world evidence for initial regulatory approvals is still in the early stage. Perhaps there are examples for use of registry data as post approval commitments but not for initial or original NDA/BLA approvals.  The examples cited by Glen are not approved based on "Real World evidence". I have been dealing with oncology regulatory for more than 20 years and I would not call an approval based on single arm trial as Real World Evidence. The single arm trial approvals in the oncology and other rare diseases Vs historical data are in existence since the accelerated approvals were initiated in the early 90s by the FDA, so this is nothing new!!


  • 7.  RE: Registry -patient data

    Posted 22-Jan-2019 09:46
    Perhaps I did not make clear that I agree that it would be difficult to obtain approval for an original NDA/BLA solely on the basis of Real World Evidence. As I stated at the outset, I am using the definition of Real World Evidence as information obtained using research methods other than a randomized, controlled clinical trial. Demonstration of penicillin efficacy did not require a randomized, controlled clinical trial since there was abundant real world experience showing that patients died without it. It is clear that many products have not required a randomized, controlled clinical trial for approval because historical data was sufficient to demonstrate outcome without the product. So, I think we are in agreement that the regulatory basis for using Real World Evidence has always been part of regulatory decision making, and well before the early 90s. 

    It is my hope that we don't come across as pessimistic about what Real World Evidence can provide and discourage our product development colleagues from finding creative uses of registries and other sources of clinical information (such as electronic health records) in developing supportive evidence of efficacy and safety just because we don't think the regulatory authorities have clearly laid out the regulatory pathway for us yet. As we have experienced, we should not wait around for FDA to create regulatory strategy, hoping that they will tell us how to innovate.

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    Glen Park
    Jersey City NJ
    United States
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  • 8.  RE: Registry -patient data

    Posted 25-Jan-2019 09:18
    And I should make it clear that my comments were driven by discussions at the Patient Engagement Advisory Committee, which addressed registries containing "patient-generated health data," a swamp if there ever was one.

    As with everything else in this space, no one seems to be entirely clear on "patient-generated health data" means. For the purposes of the meeting, CDRH defined it as "health-related data...that is created, recorded, gathered, or inferred by or from patients or their designees...to help address a health concern."  I think this definition leaves a lot of windows open, but that seems to be the current state of this art.

    I'd be only too happy to discourage my product development colleagues from seeking creative uses for these registries, but so far they haven't seemed to need any discouragement.

    ------------------------------
    Julie Omohundro, ex-RAC (US, GS), still an MBA
    Principal Consultant
    Class Three, LLC
    Durham, North Carolina, USA
    919-544-3366 (T)
    434-964-1614 (C)
    julie@class3devices.com
    ------------------------------



  • 9.  RE: Registry -patient data

    Posted 21-Jan-2019 15:04
    Hello,

    When it comes to Patient Information, the Office of Civil Rights regulates this in the US. HIPAA laws apply and a Business Associate's agreement is required by the cloud provider.

    Europe has just this year started to enforce their General Data Protection Regulation. This needs to be payed attention to as it covers European citizens, not just European geographies. A European citizen treated in the US is covered by this law. A processing agreement with the cloud provider is also required.

    These do not require regulatory approval but will carry large fines if an organization is caught out of compliance.


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    Michael Reents
    Bradenton FL
    United States
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  • 10.  RE: Registry -patient data

    Posted 24-Jan-2019 23:02
    Edited by Rashmi Pillay 24-Jan-2019 23:33
    ​Hi ,

    Continuing on the same topic , as per the FDA Real World Evidence Guidelines  '

    ' if data are being gathered to determine the safety and effectiveness of the device, and the process for gathering the data would influence treatment decisions, such administration would likely not be within the normal course of medical practice, and an IDE may be required.
    So what kind of data is allowed to be shared on the Registry ? Are chat room discussions allowed ( which may involve off -label claims discussions) ?

    Are the EU regulations in line with the FDA regarding registry requirements ?

    Can we have disclaimers in the registry , whereby we mention the approved uses and that treatment of off-label indications is at the discretion of the physicians ?

    Regarding data anonymisation / pseudonymisation and compliance to GDPR , HIPAA and other privacy laws , if patient data is to shared on registry , what data is allowed to be shared ? Because even some images have the biometric identification of the individual so then even that's traceable?

    Regards,

    Rashmi



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    RashmiAdelaideAustralia
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  • 11.  RE: Registry -patient data

    Posted 25-Jan-2019 08:09

    Chat rooms were discussed at the last Patient Engagement Advisory Committee meeting. 

    The Committee did not seem to be made up of individuals who believe what they read on the internet.  There was an awareness that the internet has no shortage of posters who aren't who they present themselves to be (eg, as patients with a particular disease or condition, or as users of a product), and that there are those who use the internet to spin stories rather than to share information. 

    This is the issue of data integrity, and the "data" posted in most chat rooms have none.  Alternatively, if you proactively set up a chat room with measures to assure data integrity, you collide head on with patient protection issues.



    ------------------------------
    Julie Omohundro, ex-RAC (US, GS), still an MBA
    Principal Consultant
    Class Three, LLC
    Durham, North Carolina, USA
    919-544-3366 (T)
    434-964-1614 (C)
    julie@class3devices.com
    ------------------------------



  • 12.  RE: Registry -patient data

    Posted 25-Jan-2019 09:24
    Registries are structured databases, not chatrooms.  They have the same security and controls that electronic data collection systems used in clinical trials have. A patient in the registry database is known only by a code, and are therefore considered pseudonymized.

    ------------------------------
    Glen Park
    Jersey City NJ
    United States
    ------------------------------



  • 13.  RE: Registry -patient data

    Posted 25-Jan-2019 10:13
    That's part of what makes this such a swamp.  Lots of different constituencies bringing their own understanding of what constitutes, RWE, a registry, etc, to the table, and not realizing that their understanding is not the same as everyone else's.  One understanding of a "patient registry" is a registry populated with patients, not health data, which can be used for market research.

    ------------------------------
    Julie Omohundro, ex-RAC (US, GS), still an MBA
    Principal Consultant
    Class Three, LLC
    Durham, North Carolina, USA
    919-544-3366 (T)
    434-964-1614 (C)
    julie@class3devices.com
    ------------------------------