July 2023 Rare Disease News

The Accelerating Kids Access to Care Act (AKACA) is a bi-partisan bill that would help reduce the time it currently takes children covered by Medicaid or the Children’s Health Insurance Program (CHIP) to access specialized care when providers in their home state cannot address their care needs.

This important and common-sense legislation will reduce burdens on health care providers, facilitate access to critical treatment, and reduce the risk of care disruption and associated negative outcomes. Last year, more than 100 rare disease patient organizations joined a similar letter of support for the AKACA, but unfortunately, the legislation did not move forward. Now, it is set to be re-introduced in the House and Senate the week of July 10, and we want to show Congress how important this legislation is to the rare disease community.

NORD urges your organization to sign on to this letter supporting the Accelerating Kids Access to Care Act (AKACA) that is being co-led by NORD and the Leukemia and Lymphoma Society. The text of the letter and sign-on form are linked here. The deadline to sign the letter is 5:00pm ET on Thursday, July 6, 2023. If you have any questions, please contact Mason Barrett at MBarrett@rarediseases.org.

This is an organization-level sign on letter but stay tuned for ways you and your community can get involved in supporting the AKACA individually!

Public comments are a great way to ensure the FDA hears the patient perspective and understands how changes to FDA policy will impact our community. Two current opportunities are particularly timely:

NORD plans on submitting comments to both the “Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act Guidance for Industry” and “Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations” draft guidances. These guidances are important to our patient population by providing additional guidelines to industry on developing therapies for pediatric populations. Comments on these guidances are due by Monday, July 17.
NORD also plans on submitting comments to the “Decentralized Clinical Trials for Drugs, Biological Products, and Devices” draft guidance. This guidance is important to our community by providing guidance to industry on expanding access to participation in clinical trials. Comments are due by Tuesday, August 1.

Please contact Hayley Mason at hmason@rarediseases.org if you have any questions on how this impacts the rare disease community or would like to share your organization’s perspective or comments with NORD.

Medicaid beneficiaries historically have needed to routinely re-establish their eligibility. When Congress enacted the Families First Coronavirus Response Act (FFCRA) at the outset of the COVID-19 Public Health Emergency, it required state Medicaid programs to keep beneficiaries continuously enrolled in the program throughout the duration of the COVID-19 Public Health Emergency. At the end of March 2023, the continuous enrollment requirements ended, and many states have begun the Medicaid eligibility redetermination process, which means they are contacting everyone currently covered by their state’s Medicaid program to determine if they remain eligible. Nine additional states will begin this process in July, but it could take a full year for programs to re-establish everyone’s eligibility.

Challenges such as difficulties reaching beneficiaries or providing the required documentation in a timely manner may result in the loss of coverage for millions of individuals who are still eligible. In fact, according to the Kaiser Family Foundation, as of June 20, 71% of the Medicaid disenrollments to date have been procedural, meaning cases where beneficiaries did not complete the enrollment process in time or returned incomplete information to the state Medicaid agency. Procedural disenrollment rates are particularly high in Kansas, (89%), Connecticut and West Virginia (87%), and Arkansas (82%).

If you or a family member are currently covered by a Medicaid plan, make sure your current address is on file with your state Medicaid program, and keep an eye out for correspondence from your state agency in the mail, which may be critical to keeping your coverage active.

Please help spread the word about this within your community! To learn how to update your information to support retaining your Medicaid benefits, click here. Further state specific information on Medicaid unwinding can be found here. NORD will be hosting a webinar on Medicaid unwinding at the end of July - more info to follow soon!

NORD's comprehensive Rare Disease Database® offers vital information on more than 1,300 rare diseases including their symptoms, causes, treatments, clinical trials, and sources of help such as patient advocacy organizations. The Rare Disease Database is utilized daily by patients, physicians, researchers, and regulators working to understand and treat rare disorders.

This month, NORD's Educational Initiatives team added four new reports on ASXL3-Related Disorder, Lafora Disease, TCF7L2-Related Neurodevelopmental Disorder, and HNRNPU-Related Disorder.

We also updated 16 reports in the Rare Disease Database, including: Beta Thalassemia, Binswanger Disease, Cohen Syndrome, Congenital Adrenal Hyperplasia, Congenital Lactic Acidosis, ESCO2 Spectrum Disorder, Factor X Deficiency, Functional Neurological Disorder, Glanzmann Thrombasthenia, Klippel-Trenaunay Syndrome, Pseudohypoparathyroidism, Recurrent Respiratory Papillomatosis, Staphylococcal Scalded Skin Syndrome, Tooth Agenesis, Tuberous Sclerosis, and Turner Syndrome.

This new registry is a natural history study that consists of electronic surveys to collect information from people diagnosed with EHE about their initial diagnosis, treatments and how those treatments worked, symptoms, and changes in their EHE over time. Patients, or their legal adult representative, can enter information from anywhere in the world. The data is confidential and stored securely in the IAMRARE® online patient registry.

To learn more about the EHE Global Patient Registry, go to EHERegistry.iamrare.org.
To learn more about The EHE Foundation, go to fightehe.org.

Drug development can be extremely challenging for rare diseases because of small patient populations and a limited understanding of how these diseases can manifest and progress.

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) brings together the experiences of patients and the clinical data of researchers to improve our shared understanding of rare diseases and accelerate clinical drug development.

You can learn more and access the platform itself below, and address any questions to rdcadap@cpath.org.

NHF is thrilled to announce that the 75th Annual Bleeding Disorders Conference (BDC) will take place from August 17 to 19, 2023, in National Harbor, Maryland, at the beautiful Gaylord National Resort & Convention Center! This opportunity is for anyone with any bleeding disorder, not just hemophilia.

Join NHF for three full days of educational sessions, networking opportunities, and access to our exhibit hall. Your registration includes entrance to the Opening Session, Awards Luncheon, and the exciting Final Night Event. We also have an Educational Kids Program available for children 12-years-old and younger. Learn more.

Don't miss the premier patient event, the Platelet Disorder Support Association (PDSA)’s ITP Conference 2023 from July 21-23 at the Swissôtel in Chicago. PDSA has designed a robust and interactive conference program that promises to help patients grow in their understanding of ITP, partner more effectively with their care team, and take control of their ITP and overall health-related quality of life. Conference speakers, some of the foremost experts on ITP in the world, will present general sessions and lead small-group breakouts to answer patient questions and present on the latest ITP research and advances in diagnosis, treatment, and management. Learn more.

Join CurePSP on July 20 for a free webinar on understanding brain donation for neurodegenerative diseases! Brain donation is key to advancing the understanding, treatments, and cure of progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and multiple system atrophy (MSA).

Dr. Gabor Kovacs, a neuropathologist at the University of Toronto, will shed light on how brain tissue is used to study neurodegenerative diseases. He will be joined by Diane Crompton, whose husband was diagnosed with PSP, and who will speak about her personal experience with the brain donation process and what it meant for her husband’s legacy. Learn more.

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Improving the health and well-being of people with rare diseases by driving advances in care, research and policy. Learn more at: rarediseases.org

NORD is a registered 501(c)(3) charity organization. Please note that NORD provides this information for the benefit of the rare disease community. NORD is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder.