Patient Registry: The Power In Numbers

Patient Registry: The Power In Numbers

I used to think that as a patient I had no power to effect change for my family and others with Alport syndrome – that we had to endure the recurring cycle of disease, generation after generation. I now know that we, as patients, play a pivotal role in changing the course of the disease and our actions will have a tremendous impact on our future.

One of the key ways we can have an impact is by participating in the patient registry.

ASF President Sharon Lagas explains why the patient registry is so important to the Alport syndrome community.

What is the Alport syndrome Patient Registry?

The US registry is called ASTOR (Alport syndrome Treatments and Outcomes Registry) and is located at the University of Minnesota.  There are some other Alport syndrome registries in other countries. The patient registry is a database of people affected by Alport syndrome that stores confidential data such as name, contact information and medical information.  The registry has safeguards to protect patient’s privacy and will not disclose the patient’s identity or location.

Researchers need access to enough people with the disease who can participate in research studies. For a rare disease like Alport syndrome, one medical center will not have enough patients for researchers to carry out valid research studies.  The patient registry assists researchers in quickly identifying and contacting eligible individuals willing to participate in research. Having a strong patient registry will facilitate trials on additional treatments.

Why you and your family members should join the Alport syndrome Patient Registry  

I recently attended a webinar about the drug development process, trying to learn as much as I can to advocate for the Alport community. The speaker started by saying that drug development is a very high risk endeavor and then proceeded to give these statistics:

  • 30% of drugs fail in Phase I (testing safety in healthy people)
  • 60% of drugs fail in Phase II (testing on patient population)
  • 30% of drugs fail during the application process to the FDA (US Food and Drug Administration)
  • 14% of drug applications fail to receive FDA approval.

You can do the math – it amounts to very few drug approvals!

I understand that failure is part of the research and scientific process, but it is very disheartening to a rare disease patient to see these statistics.  As you know, there are very few studies or trials for a rare disease like Alport syndrome and even more upsetting is the fact that many of these failures can be attributed to lack of patient recruitment. There simply were not enough patients enrolled in the study. This does not mean that there are not enough patients for a study or trial on Alport syndrome. It is estimated that there are 30,000 to 60,000 Alport syndrome patients in the USA. The problem is that eligible patients were either not contacted or not aware of the study or unwilling to participate.

I am constantly asking the question, what can we as patients or as a patient organization do to increase the odds for more treatments for Alport syndrome? As a patient organization, ASF supports the patient registry, is funding research to generate more ideas for new therapies, getting information to the patient and medical community on studies/trials, and collaborating with many stakeholders, such as international patient organizations, clinicians, geneticists, researchers, and pharma/biotech industry, for improved diagnosis, treatments, and awareness.

But how can you or I, as a patient, have an impact?  No drug or treatment can be approved without us. Clinicians, researchers, and pharma all need patients to test their therapies or participate in studies. They need patients to report outcomes and inform them of the changes to their quality of life and effects of their disease.

The best way for you as a patient to make a difference is to join the registry. As a member in the patient registry you are notified immediately of any study/trial for which you are eligible to participate.  When you and your family members join the patient registry you are increasing the Alport syndrome community’s ability to impact their own future.

There is power in numbers even for a rare disease like Alport syndrome. Let’s do what we can to change the statistics. Join the Alport syndrome patient registry now!


Additional Resources:
Alport syndrome Treatments and Outcomes Registry  US patient registry
Current Clinical Studies/Trials for Alport syndrome

Translate »