Supporting Registry-based Studies for Medical Device and Biological Medical Products
Registry-based studies are an important type of observational study used in the pharmaceutical industry to collect data on the safety and efficacy of drugs and medical devices. These studies use data collected from product or disease registries to examine outcomes associated with specific medications or treatments. These types of studies help sponsors to collect data from a large number of patients over a long period of time. This can help to identify potential safety issues that may not have been observed in clinical trials, which often have limited follow-up periods and enroll a relatively small number of patients.
Our team of experts has years of experience in building various types of registries, including product registries, patient registries, and disease registries. We work closely with our clients to understand their specific needs and provide customized registry solutions to collect comprehensive, standardized data about a particular medical condition or treatment, with the aim of improving patient outcomes and advancing medical knowledge.
We have helped sponsors to create registries that can be used for:
> Short and long-term surveillance
> Fulfillment of postmarket observational study commitments for regulatory bodies, and
> Examine comparative safety and effectiveness assessments, including those in under-studied subpopulations.
How We Can Help
- Defining the research question and study objectives (primary & secondary)
- Finalizing study design (case series, cohort, case-control, or case-cohort)
- Drafting study protocols and seeking necessary IRB / IEC approvals
- Identification of core and optional data elements for the registry database
- Implementing data collection and data curation plan to ensure consistency and quality
- Performing registry data management and completing statistical analysis to present outcomes
- Generating insights on patient safety and product performance for informed decision making
Services We Offer
Registry studies typically involve the collection of data through a variety of methods, including patient interviews, surveys, and medical record review. Data may also be obtained through the use of device-specific data collection tools such as remote monitoring systems. The study design and methodology of registry-based studies can vary, with some studies being observational in nature and others being more of an interventional type.
With our proprietary Compier Registry platform, we can help sponsors in developing, curating and continously enhancing data quality for the following types of registry-based studies:
> Post-Market Surveillance to provide evidence in the post-authorisation phase
> Medical Device Long-term Safety & Performance Evaluation in selective populations
> Using Patient Registry data to complement the evidence generated in the pre-authorisation phase
> Use Product Registry database to evaluate the effects of medicinal products used during pregnancy and breast feeding
Common FAQs
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Natural history studies involve observing clinical practice and patient experiences without introducing any intervention. These studies help to better understand the natural course of a disease or condition and provide information on its progression over time. This information can be used to develop new treatments and to improve existing ones.
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Measures of clinical effectiveness, particularly related to compliance, are crucial for learning about what patients and practitioners actually do and how their actions affect real-world outcomes. For example, a treatment may have a high efficacy rate in clinical trials, but poor compliance in the real world can lead to reduced effectiveness. This is especially important for treatments that have poor compliance rates.
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Studies of effectiveness and safety that include clinician training and technique as part of the treatment study are critical to evaluating the treatment’s success. For example, a procedures such as placement of carotid stents, where proper technique is essential for good outcomes.
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Registries that include a heterogeneous patient population can provide greater generalizability (external validity) and allow for assessment of subgroup differences in treatment effects. Unlike randomized trials, registries generally have much broader inclusion criteria and fewer exclusion criteria.
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Follow-up studies for delayed or long-term benefits or harm are possible with registries, as they can extend over longer periods than most clinical trials due to their lower costs and lesser burden on participants. These studies are useful for understanding the long-term effects of a treatment or disease.
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Surveillance for rare events or rare diseases is another critical area where registries can be useful. Since these events or diseases are infrequent, large patient populations are necessary to study them effectively.
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In cases where randomization is unethical, such as intentional exposure to potential harm, safety studies of marketed products suspected of being harmful are necessary to evaluate the safety of the product.
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In cases where randomization is not necessary, such as when certain therapies are only available in certain places due to high cost or other restrictions, studies can still be conducted using a registry to evaluate the treatment’s effectiveness.
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In studies where blinding is challenging or unethical, such as studies of surgical interventions or acupuncture, alternative methods of evaluating effectiveness may be necessary.
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Studies of rapidly changing technology are essential to keep up with the pace of innovation and to evaluate the efficacy of new technologies as they become available.
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Studies of conditions with complex treatment patterns and treatment combinations can provide valuable insights into the best approaches for treating these conditions and improving patient outcomes.
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Studies of healthcare access and barriers to care can help identify areas where improvements can be made to ensure that all patients have access to high-quality care.
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Evaluations of actual standard medical practice are important for identifying areas where current practice may be falling short and for developing new and improved treatment approaches.
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During the process of designing a registry, it is important for registry planners to collaborate with stakeholders and relevant experts to make informed decisions. The selection of groups to be consulted during the design phase typically varies depending on the registry’s nature, funding source, funding mechanism, and the target audience for registry reporting. To get maximum value from the registry study, it is important to have the answers for following questions:
| Construct | Relevant Questions |
|---|---|
| Research question | What are the clinical and/or public health questions of interest? |
| Resources | What resources, in terms of funding, sites, clinicians, and patients, are available for the study? |
| Exposures and outcomes | How do the clinical questions of interest translate into measurable exposures and outcomes? |
| Data sources | Where can the necessary data elements be found? |
| Study design | What types of design can be used to answer the questions or fulfill the purpose? |
| Study population | What types of patients are needed for study? Is a comparison group needed? How should patients be selected for study? |
| Sampling | How should the study population be sampled, taking into account the target populations and study design? |
| Study size and duration | For how long should data be collected, and for how many patients? |
| Internal and external validity | What are the potential biases? What are the concerns about generalizability of the results (external validity)? |
Disease registries can provide a wealth of information for sponsors in the following areas:
> Assessing natural history, including estimating the magnitude of a problem; determining the underlying incidence or prevalence rate of a condition; examining trends of disease over time; conducting surveillance; assessing service delivery and identifying groups at high risk; documenting the types of patients served by a health provider; and describing and estimating survival.
> Determining clinical effectiveness, cost-effectiveness, or comparative effectiveness of a test or treatment, including for the purpose of determining reimbursement.
> Measuring or monitoring safety and harm associated with the use of specific products and treatments, including conducting comparative evaluation of safety and effectiveness.
> Measuring or improving quality of care, including conducting programs to measure and/or improve the practice of medicine and/or public health.