Dec
19
To generate evidence, we need to understand the types of data that are important and available within the patient’s “real world”. The generation of useful disease specific data requires an understanding of the relevant information surrounding the patient and their illness to gain a well-rounded view of the cause of...
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Dec
07
When setting up a disease registry, there are several things to consider, such as choosing your registry type (for example, patient registry, natural history or clinical), engaging participants, and financing the project. However, you should not overlook how you plan to use the data collected to ensure that your registry...
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Nov
12
Historically, research into rare diseases has been siloed and lacking in standardization. This has resulted in meaning overlaps, wasted time, and wasted resources, all of which delay potential breakthroughs for patients. You can ensure the data in your registry actively supports new research, rather than hinders it, by employing a...
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Oct
29
When you invest in a patient registry, you want it to remain viable for many years and ensure that your data can contribute to research and treatment development. Here are three key questions to consider that will help you secure the future of your investment, your patients, and your registry....
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Oct
13
When exploring the idea of establishing a registry for your organization, the prospect can be daunting and the options, seemingly endless. There are a range of options available, costing from next to nothing up to millions of dollars, with each having pros and cons, making deciding which option to choose...
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Oct
05
With individual rare diseases affecting such a relatively small proportion of patients globally, capturing effective data to aid with research into treatments is incredibly important. Relying on data from clinical trials alone is no longer sufficient to ensure safety, efficacy, and discovery of biomarkers for new drug development. Increasingly regulators...
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Sep
01
The need for real-world evidence to inform therapies can no longer be denied. Gathering standardized feedback from patients where they live can give researchers richer details into the effectiveness of treatments. However, sharing real-world experience with researchers and the general public is also the foundation of patient advocacy. We spoke with Alan...
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Aug
05
https://www.youtube.com/watch?v=jRfPtBnur1s Building on the success of our most recent webinar on Patient-Reported Outcome Measures (PROMs), our August webinar will expand on the topic of PROMs, delving deeper into the realities of implementation. Featuring Dr Femida Gwadry-Sridhar, a pharmacist, epidemiologist, methodologist and founder of medical technology company Pulse Infoframe, Julien Wiggins,...
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Aug
05
https://www.youtube.com/watch?v=Wg-Bx8H1-j4& Daniel Lewi (CATS Foundation), Ellen Haf Davies (Aparito), and Femida Gwadry-Sridhar (Pulse Infoframe) discuss their experiences using rare disease patient data to drive research forward. Streamed on 29 July 2020.
Jul
20
July 20, 2020 — Robert Iannone, Executive Vice President, Research and Development and Chief Medical Officer at Jazz Pharmaceuticals Narcolepsy is still a widely misunderstood and under diagnosed disorder. Sadly, in children and adolescents, narcolepsy can often be mistaken for laziness or lack of motivation. This means many children living...
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