Reviewed by Sharyn Rossi, PhD
When the U.S. Food and Drug Administration granted full approval of Leqembi, a new Alzheimer’s medication that treats patients in the early stages of the disease, Medicare coverage for it began immediately. But there was a condition: Doctors who prescribe Leqembi must participate in patient registries for the drug to be covered by Medicare.
This decision was significant because of the hefty price tag of the drug. For individuals paying out of pocket, the cost of Leqembi is $26,500 a year, in addition to the cost of the medical tests needed to determine if amyloid is present in the brain, such as PET scans or spinal taps. Most Americans with Alzheimer's get their health coverage through Medicare.
What are patient registries, and how will your health information be kept safe?
Here are six facts to know about patient registries, critical tools that help researchers develop new treatments and cures for diseases.
1. Patient registries are a collection of information about a group of people who share a diagnosis or a certain characteristic. By pooling information, researchers can learn more about a disease or condition and how it’s treated. They also can monitor outcomes, side effects, and other data for research purposes. Patient registries may also be referred to as clinical registries, disease registries, and outcomes registries.
The Leqembi patient registry, for example, is for people diagnosed with early cognitive impairment or mild dementia caused by Alzheimer’s disease, with evidence of amyloid-beta plaque in the brain. The data collected will help answer questions such as the extent to which Leqembi slows the decline of cognition and function for a broad range of people who take the drug.
2. Doctors, not patients, submit information to the registry. Doctors submit patient data through a dedicated online portal. The registry collects information such as a patient’s demographics, diagnosis, and medical history.
For people taking Leqembi, the nationwide portal is maintained by the Centers for Medicare & Medicaid Services (CMS). Doctors must report whether their patients are taking medication for blood clots, and they must include any side effects that patients may experience. Reporting other medications that patients may be taking is not required. Doctors also must submit the results of brain scans and other tests that assess amyloid levels, an Alzheimer's protein that is the target of Leqembi.
3. Registries are common tools that have successfully gathered information on patient outcomes for decades. Although the specifics of the Lequembi registry are not yet known, in general, patients are de-identified, which means their names, addresses, Medicare numbers, and other personal information are not shared. The goal is to further the knowledge of how certain drugs can potentially help others.
“Patient registries are an essential foundation for real-world data platforms that will ultimately be used to inform personalized medicine and better guide who receives treatment and when they receive it,” explained Sharyn Rossi, PhD, director of neuroscience research at BrightFocus Foundation.
4. Patient registries adhere to robust privacy protections. They follow applicable federal laws and regulations, including the Health Insurance Portability and Accountability Act, a federal law known as HIPAA that protects sensitive patient health information from being disclosed without the patient's consent or knowledge.
With the Leqembi registry, CMS and researchers have access to the information collected to conduct future studies, furthering the knowledge of how the drug can potentially help other people with Alzheimer’s disease.
5. Patient registries are among several means by which health and medical information is collected and pooled for research purposes. A different type of collection is a patient database, which contains a person’s private medical records. Also called a chart or electronic health record, it is information shared by providers who are caring for the patient. Databases are governed by HIPAA to protect sensitive health information.
Another type of data collection is a biobank, or a large collection of specimens linked to relevant personal and health information such as family history, lifestyle, and genetic information that are used for medical research. Biobanks store samples such as whole blood, plasma, urine, and DNA. The personal information contained in biobanks is confidential.
Yet another type is a brain bank, which collects brain and tissue samples from deceased donors and distributes them to investigators all over the world to be used in research. A brain bank donation represents a gift of knowledge and is of critical importance for the understanding of brain disorders.
6. You can help inform future medical advances and discoveries. Through an initiative called All of Us, the National Institutes of Health (NIH) is building one of the largest and most diverse health databases of its kind. The goal is to provide information from large numbers of people who reflect the diversity of the United States. Researchers use this data to learn more about why people get sick or stay healthy and to find better ways to prevent and treat illnesses. Anyone can participate at no cost, other than a small amount of time.
People who choose to join the NIH’s All of Us are asked to answer health surveys and to connect their electronic health records and wearable devices like an activity tracker or smartwatch. They also may choose to share biosamples such as their blood, urine, and saliva.
The information collected through All of Us helps researchers understand how health history, genetics, environment, and life experiences can impact health, providing an altruistic way to help further medical advances and discoveries to benefit society.
The bottom line
Patient registries help increase the understanding of a disease or condition and contribute to the development of treatment guidelines, including what therapies would be most effective. Because they adhere to strict privacy and security protections, patients’ personal health information is safe. The end result is a valuable tool for researchers, providers, and, ultimately, patients.
“Disease-specific registries help take the burden off affected individuals,” Dr. Rossi said. “They facilitate interactions among health care providers and provide comprehensive data to help assess the efficacy and tolerability of new treatments.”
Learn more about how to receive Medicare coverage of Leqembi.
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