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Safety Reporting for a Natural History study

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  • Anon
    07/01/2021 15:52
    A Natural History study being planned is considered interventional, due to blood draws, ECGs, ultrasounds, etc. that are performed to collect data. However, there is no investigational product or device. Are the same AE / SAE collection / reporting requirements applicable as for an interventional study with an investigational product?
  • Anon
    07/01/2021 17:40
    If it is interventional trial then I assume it will have to be submitted to an ethics Commiitte and to the regulatory authority. Being submitted to regulators, it will be governed by laws you can look up on their website. This will inside requirements for safety reporting. Your protocol will cover what safety reporting will be done. A risk based approach (see guidance) is recommended. Your protocol and submission will include this risk based approach to safety reporting.
  • Dominique Chesnais
    15/01/2021 15:18
    Your requirements should be more about IEC approval and data protection. EC should be informed of any issues occurring with subjects while participating in that project. As a natural history study, it could have some marketed drugs. Assuming that a medicine was prescribed and a subject had a (S) AE, it would be the responsibility of the prescriber to report the (S) AE as per national requirements. However, as you have mentioned that your study is interventional, i.e. beyond normal medical practice, without defining these additional features, it is impossible to comment further.
  • Anon
    18/01/2021 09:02
    Thanks, Dominique. IEC and data privacy are covered in the protocol. Good point about (S)AEs related to other eventual medications the patients may be taking. This would be required by standard practice, but the requirement could be reinforced in the protocol. To provide the missing clarification: This Natural History study is for a rare genetic disease. There is no recommended / required change to standard of care; all "interventions" are for diagnostic purposes (blood draws, ultrasound, physiotherapy tests / measurements, etc.), to develop a clearer picture of the disease progression. There is no treatment intervention, only physical intervention (as described above) and psychological intervention (completion of questionnaires, e.g. QoL). The regulatory definitions are unclear in this area - hence the study team's questions on requirements. The plan is to follow GCP as/where applicable/possible, but in the area of safety reporting, more guidance would be appreciated. Hence, my question
  • Dominique Chesnais
    19/01/2021 07:45
    Thanks for your additional information. As your question is about the difference in the AE / SAE collection / reporting requirements in a natural history study for a rare disease vs. an interventional study with an investigational product, you could refer to the compassionate use of medications with the EMA GVP Module VI – Collection, management and submission of reports of suspected adverse reactions to medicinal products (Rev 2) (https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-good-pharmacovigilance-practices-gvp-module-vi-collection-management-submission-reports_en.pdf), with some sub-sections in chapter VI.C. Operation of the EU network. You can also refer to an EMA page “Compassionate use” at https://www.ema.europa.eu/en/human-regulatory/research-development/compassionate-use and look at the cases exhibited, though they apply to a pharmaceutical sponsor with a single product. You could contact other groups proceeding with some rare diseases and even the EMA for seeking advices, in particular, if your project aims at using some registered medicines.
  • Juergen Kaetzler
    01/02/2021 06:50
    Dominique's post with the relevant guidances is very helpful and as he already stated reporting of adverse reactions etc. will fall under the "normal" national regulatory requirements. As far as I understand you do not have a trial falling under the directive 2001/20/EC (clinical trial on medicinal products) nor you have a clinical trial on a medical device (regulation 2017/745). I can only speak for Germany and Austria: in this case you do not have that "strict" SAE reporting requirements like you would have in a clinical trial on medicinal products. Nevertheless, some Ethics Committees require to be informed about SAEs during the study. But they leave it up to the sponsor how to define SAEs. >> We conducted a study where literature did not give specific treatment recommendations; patients were treated "in the opinion of the investigator" based on the descriptions in literature. We randomised patients to the treatment modalities mentioned in the guidelines. So we had an intervention (randomisation and treatment) but treatment was according to guidelines and to local practice. Treatment consisted of "regular" treatment (drugs, diagnostics, surgery where needed etc.) We restriced SAE criterion "hospitalisation" to "hospitalisation at the Intensive Care Unit" - so we received a SAE report only for ICU admissions and not for each "normal" hospital admission. This was approved by all IECs that were involved in the trial. >> As Dominique wrote you should have an eye on national/local requirements.
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