The aHUS Registry Explained (Part 1)

From its beginning the aHUS alliance’s website has featured articles about the aHUS Registry and the work it does (click here to see the first of those articles).
In a series of blogs planned to appear on the website over the next few months , Donna Ames Senior Manager, Clinical Project Lead , Global Registry Operations

Donna Ames of Alexion

is  going to answer questions about Registries to help get a greater understanding of what Registries are all about, and specifically, how the aHUS Registry operates and what is has achieved , as well as what it intends to achieve.
A Registry has been described 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 one or more predetermined scientific, clinical, or policy purposes.“ What does that mean?”
Registries are data collection studies to better understand the course of a given disease, as well as the impact of available treatment. These generally are observational, which means that the physician is entering information from  patients’ medical record  into a database after patient has signed informed consent. What medications are being taken, health information, disease symptoms and treatment effects. This information is collected through the hospital and medical records of the patient without the patient needing to make a visit The information collected is specific to the disease being studied and the information or data is compiled to provide greater understanding of the disease, the effect disease has on the patients’ lives and the outcome of available treatment.
So what can a Registry do?
Registries can be used to describe the natural history of the disease and/or to observe the impact of treatment on the course of the disease and patients’ lives. It helps us to understand differences in the way patients are treated and what happens after they are treated. The information captured in a registry can help examine factors that influence how physicians diagnose and predict a patient’s health in the future. Sometimes it looks at the quality of a patient’s life with the specific disease – is the treatment making a positive impact or does the patient continue to suffer? Registries also help to assess the quality of care patients receive.
Each registry is different so it could be any combination of these areas that are being looked at.
The data can be shared with health authorities around the world to enhance their understanding of the disease and treatment as well as the effects on the patients. It is also used by physicians to write publications to help educate other physicians.
Registries also help to describe care patterns of physicians and how effectiveness is rated. Data collected can help monitor the risk or benefit of the treatments.
Those are quite a challenge in themselves and we can explore them later, but does it make a difference in any way when the patients‘ disease is very, very rare?
Yes, rare disease research does pose several challenges. First, there is usually a small population of patients that are scattered around the globe. Also, rare diseases are often chronic, progressive, serious, and life-limiting or life-threatening. The natural history of the disease is often not well understood. There are issues with disease awareness and diagnosis from doctors not experienced with these rare diseases. And since we are learning there are times when instruments, tools, and information that are just lacking.
So aHUS needs a Registry?
Yes. For rare or ultra-rare diseases, an international, long-term disease registry may be the best, or only feasible way to increase knowledge about the characteristics and natural history of the disease. It also allows for the assessment of long-term effects (good or bad) of treatments.
But why cannot a trial do that?
Randomized clinical trials are designed to investigate new therapies prior to approval by regulatory authorities:
“Is this drug safe and efficacious?”
“How do changes in medication dosing (timing, frequency) impact safety and efficacy?”
Observational or registry studies can expand and extend the insights gained in clinical trials:
“What are the long-term effects of treatment at 5, 10, 15 years of treatment?”
“How effective and safe is the treatment in the more complex context of daily life compared to the more controlled context of clinical trials?”
“Is the treatment safe in patients with other serious illnesses?”
“What happens over the course of this disease?”
Could you sum up then what are the key advantages of having a Global aHUS Registry?
 Registries can be larger potentially include patients from more countries and go on for a longer period of time.
Registries allow for long-term follow-up based upon current clinical practice including changes in treatment or drugs approved for that disease, and in the more complex context of daily life.
Registries can also be sought after by regulatory authorities as basis for additional effectiveness and safety assessments after a treatment is approved for the specific disease.
Modifications to the database and information to be collected can be changed as clinical practice change. This keeps the data contemporary.
So, in essence, a global aHUS Registry can, over a long period time, include more and varied aHUS patients from what is a very small patient population and use up to date data about them to meet regulatory demands and help clinical understanding and practice.
But just how does this data get collected and recorded? That will be explained in the next blog in the series.
Note: the series was interrupted by a change in Alexion’s policy towards the Registry as a result of a cost cutting programme which impacted on recruitment. The effects of the policy change are reported elsewhere on the website click here.

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