News
survey study: Patient experiences with fludrocortisone
Survey on Fludrocortisone
Background: Teva Pharmaceuticals has discontinued their production of fludrocortisone. Their formulation was well liked because it was a distinct color and shape, and also because it was easy to divide into a 0.05 mg dose that many patients need. The remaining manufacturers' configurations lack those characteristics. This results in a potential patient safety issue with both dosing correctly using pills that are not configured to split, and also a safety issue distinguishing fludrocortisone from hydrocortisone pills.
Further issues were raised about some patients feeling that their symptoms were altered when they used one of the other manufacturers products. A number of patients (31%) raised issues regarding new symptoms they experienced when taking the other manufacturers’ fludrocortisone, although they could not definitively say that these symptoms resulted from the change in medication. The symptoms described were common to AI symptoms in general.
We had a strong response from members regarding their experiences in the survey. There were 434 overall responses to the survey. Of those responses 193 had changed their fludrocortisone source, and of those who had changed, 60 reported that there was a difference in their symptoms.
Given the commonality of AI symptoms to the symptoms experienced due to changes in drug manufacturer, it is very difficult to assess causation beyond simply recognizing that some individuals experience this issue. Further, the survey did not reveal any significant patterns in relationship to a particular manufacturer.
NADF’s action plan is to draft a letter to the FDA describing the patient impacts of these changes - particularly the issues around patient safety - and ask for a response from them.
Background: Teva Pharmaceuticals has discontinued their production of fludrocortisone. Their formulation was well liked because it was a distinct color and shape, and also because it was easy to divide into a 0.05 mg dose that many patients need. The remaining manufacturers' configurations lack those characteristics. This results in a potential patient safety issue with both dosing correctly using pills that are not configured to split, and also a safety issue distinguishing fludrocortisone from hydrocortisone pills.
Further issues were raised about some patients feeling that their symptoms were altered when they used one of the other manufacturers products. A number of patients (31%) raised issues regarding new symptoms they experienced when taking the other manufacturers’ fludrocortisone, although they could not definitively say that these symptoms resulted from the change in medication. The symptoms described were common to AI symptoms in general.
We had a strong response from members regarding their experiences in the survey. There were 434 overall responses to the survey. Of those responses 193 had changed their fludrocortisone source, and of those who had changed, 60 reported that there was a difference in their symptoms.
Given the commonality of AI symptoms to the symptoms experienced due to changes in drug manufacturer, it is very difficult to assess causation beyond simply recognizing that some individuals experience this issue. Further, the survey did not reveal any significant patterns in relationship to a particular manufacturer.
NADF’s action plan is to draft a letter to the FDA describing the patient impacts of these changes - particularly the issues around patient safety - and ask for a response from them.
Study sponsored by nadf: "People with Adrenal Insufficiency who are in Adrenal Crisis are Frequently Unable to Self-Administer Rescue Injections"
From AACE Endrocrine Practice, published March 3, 2025:
"Individuals with adrenal insufficiency (AI) are at risk of acute adrenal crisis and death, particularly during illness or trauma, and may require rapid treatment with parenteral glucocorticoid such as hydrocortisone (HC) to manage a crisis. Current guidelines recommend timely self-injection in an evolving crisis. Little is known about the patient experience with emergency injections. We surveyed people with adrenal insufficiency regarding success with emergency injections.
... Effective, timely, management of an impending adrenal crisis can prevent progression to hospitalization, multisystem failure requiring ICU care, and death. Reliance solely on patient self-injection may result in worse outcomes. Treating physicians should include patient education about injections and specific practical instruction in the technique, as well as the potential need for assistance in a crisis. FDA approval of a glucocorticoid auto injector, greater engagement with EMS clinicians, hospital emergency staff, and other healthcare professionals, are key for future success in managing adrenal crises."
Read the full article here.
"Individuals with adrenal insufficiency (AI) are at risk of acute adrenal crisis and death, particularly during illness or trauma, and may require rapid treatment with parenteral glucocorticoid such as hydrocortisone (HC) to manage a crisis. Current guidelines recommend timely self-injection in an evolving crisis. Little is known about the patient experience with emergency injections. We surveyed people with adrenal insufficiency regarding success with emergency injections.
... Effective, timely, management of an impending adrenal crisis can prevent progression to hospitalization, multisystem failure requiring ICU care, and death. Reliance solely on patient self-injection may result in worse outcomes. Treating physicians should include patient education about injections and specific practical instruction in the technique, as well as the potential need for assistance in a crisis. FDA approval of a glucocorticoid auto injector, greater engagement with EMS clinicians, hospital emergency staff, and other healthcare professionals, are key for future success in managing adrenal crises."
Read the full article here.
NADF's statement on norovirus outbreak
Norovirus is the most common type of viral gastroenteritis that is always present. It often spreads quickly in nursing homes, prisons, and facilities where food preparation and inadequate sanitation coexist. It is very contagious. The usual source is contaminated food. It can be spread by eating the food, touching contaminated dishes and utensils, and touching contaminated surfaces.
Symptoms are nausea, vomiting, diarrhea, abdominal pain, headache, and sometimes fever. The symptoms occur 12 to 48 hours after exposure, and typically last 1 to 3 days. Victims of the virus may remain contagious for several days after symptoms are gone. There is no specific drug for norovirus. Antibiotics do not help. Treatment is general support: replacement fluids, antinausea and anti-diarrhea medications, and treatment of any fever.
The best way to avoid norovirus is frequent hand washing. Masks are not effective against norovirus because it is not a respiratory disease. A mask might help if it reminds you not to touch your face. To protect yourself, wash hands thoroughly, disinfect surfaces, avoid touching your face.
Since individuals with adrenal insufficiency have a diminished tolerance for gastroenteritis, they need to be vigilant if they are aware of an outbreak in their neighborhood. Be prepared to treat the symptoms, replacing fluids aggressively, and take extra glucocorticoids according to the usual illness protocols. As usual, go to an emergency department if fluid loss from vomiting and diarrhea cannot be controlled.
Prepared by NADF Medical Director, Dr. Paul Margulies
For more information see:
Symptoms are nausea, vomiting, diarrhea, abdominal pain, headache, and sometimes fever. The symptoms occur 12 to 48 hours after exposure, and typically last 1 to 3 days. Victims of the virus may remain contagious for several days after symptoms are gone. There is no specific drug for norovirus. Antibiotics do not help. Treatment is general support: replacement fluids, antinausea and anti-diarrhea medications, and treatment of any fever.
The best way to avoid norovirus is frequent hand washing. Masks are not effective against norovirus because it is not a respiratory disease. A mask might help if it reminds you not to touch your face. To protect yourself, wash hands thoroughly, disinfect surfaces, avoid touching your face.
Since individuals with adrenal insufficiency have a diminished tolerance for gastroenteritis, they need to be vigilant if they are aware of an outbreak in their neighborhood. Be prepared to treat the symptoms, replacing fluids aggressively, and take extra glucocorticoids according to the usual illness protocols. As usual, go to an emergency department if fluid loss from vomiting and diarrhea cannot be controlled.
Prepared by NADF Medical Director, Dr. Paul Margulies
For more information see:
NADF and PRodigy ems announce free webinar
"Recognizing and Managing Adrenal Crisis in the Field with NADF"
Experts in endocrinology and emergency care will guide attendees through real-life case studies and practical strategies for managing adrenal crises in the field. Attendees will gain insights into the nuances of glucocorticoid and mineralocorticoid deficiencies, fluid resuscitation protocols, and the administration of life-saving steroids—whether in the form of carried medications or assisting with patient-provided solutions. Free CE for EMS Clinicians.
Speakers: Christopher Galton, MD; Mitchell Geffner, MD; Vanessa O’Rourke
Link to the webinar: http://link.prodigyems.com/nadf (free account required for viewing)
Experts in endocrinology and emergency care will guide attendees through real-life case studies and practical strategies for managing adrenal crises in the field. Attendees will gain insights into the nuances of glucocorticoid and mineralocorticoid deficiencies, fluid resuscitation protocols, and the administration of life-saving steroids—whether in the form of carried medications or assisting with patient-provided solutions. Free CE for EMS Clinicians.
Speakers: Christopher Galton, MD; Mitchell Geffner, MD; Vanessa O’Rourke
Link to the webinar: http://link.prodigyems.com/nadf (free account required for viewing)
FDA approves new cah medication crenessity
The FDA has approved CRENESSITY™ (crinecerfont) as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH).
CRENESSITY is a first-in-class treatment developed for individuals living with classic CAH. It is a breakthrough in the treatment landscape for this rare, serious, and lifelong genetic condition involving the adrenal glands. This approval marks a significant step forward in classic CAH treatment, and we're excited about its possibilities to improve patients’ lives.
More Information: To learn more, please find links to additional resources below:
CRENESSITY is a first-in-class treatment developed for individuals living with classic CAH. It is a breakthrough in the treatment landscape for this rare, serious, and lifelong genetic condition involving the adrenal glands. This approval marks a significant step forward in classic CAH treatment, and we're excited about its possibilities to improve patients’ lives.
More Information: To learn more, please find links to additional resources below:
- News Release: https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-fda-approval-crenessitytm
- CRENESSITY Product Website: Crenessity.com
- FDA-approved Prescribing Information (PI) here.
New nadf blog: "A day in the life" vignettes
NADF has added a new section to the quarterly newsletter, "A Day in the Life" vignettes. These vignettes will be collected on the NADF website as a blog series. Each entry details a day in the life of someone with adrenal insufficiency and shows them reacting to different stressors. The story is followed by a question section designed to engage the reader in critical thinking about proactive choices for living an adrenal insufficient life of the highest quality. Entries are written by Susan Majka, NADF Treasurer.
NAEMSP Blog: "Adrenal crisis and addison's disease: what ems needs to know"
The National Association of EMS Physicians (NAEMSP), in collaboration with NADF, has published an article on their blog titled, "Adrenal Crisis and Addison's Disease: What EMS Needs to Know". The article includes information on primary and secondary adrenal insufficiency, EMS treatment of adrenal crisis, and key considerations for EMS. This is part of NADF's ongoing EMS education efforts. NAEMSP.org/adrenal-crisis-and-addisons-disease-what-ems-needs-to-kno/
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EXCITING ANNOUNCEMENT FROM THE ADRENAL INSUFFICIENCY REGISTRY (MYAI) RESEARCH TEAM |
The MyAI Registry participants are invited to enroll into the DNA and Cortisol studies. There is no cost to participate in either study, and you may choose to participate in both! You will be provided with the sampling kits, mailing containers, and shipping labels. Click here for the QR code and more information on how to enroll:
NADF.us/dna-and-cortisol-studies.html
NADF.us/dna-and-cortisol-studies.html
cDC recommends updated covid-19 vaccine
CDC Recommends Updated COVID-19 Vaccine for Fall/Winter Virus Season: CDC.gov/ncird/whats-new/covid-vaccine-recommendations-9-12-2023.html
food outbreak investigations
Food poisoning poses a significant risk, especially for individuals with adrenal insufficiency, as it can lead to adrenal crisis. NADF strongly recommends staying informed about the latest food outbreak reports from the FDA. Ensure you receive timely updates by subscribing to the FDA's Email Updates on Food Recalls & Outbreaks:
FDA.gov/food/recalls-outbreaks-emergencies
For detailed information on current recalls of foods and dietary supplements, please visit the FDA's dedicated webpage: FDA.gov/food/recalls-outbreaks-emergencies/recalls-foods-dietary-supplements
To stay informed about investigations into foodborne illness outbreaks visit:
FDA.gov/food/outbreaks-foodborne-illness/investigations-foodborne-illness-outbreaks
Awareness can play a crucial role in ensuring the safety of your health and well-being!
FDA.gov/food/recalls-outbreaks-emergencies
For detailed information on current recalls of foods and dietary supplements, please visit the FDA's dedicated webpage: FDA.gov/food/recalls-outbreaks-emergencies/recalls-foods-dietary-supplements
To stay informed about investigations into foodborne illness outbreaks visit:
FDA.gov/food/outbreaks-foodborne-illness/investigations-foodborne-illness-outbreaks
Awareness can play a crucial role in ensuring the safety of your health and well-being!
announcement from neurocrine biosciences
September 12, 2023
Neurocrine Biosciences Announces Positive Top-Line Data from Phase 3 Study of Crinecerfont in Adults for the Treatment of Congenital Adrenal Hyperplasia (CAH). Follow link: neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-positive-top-line-data-phase-3
Neurocrine Biosciences Announces Positive Top-Line Data from Phase 3 Study of Crinecerfont in Adults for the Treatment of Congenital Adrenal Hyperplasia (CAH). Follow link: neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-positive-top-line-data-phase-3
recreating the adrenal gland in a petri dish
Read about this important study that could lead to new therapies for adrenal insufficiencies and a deeper understanding of the genetics of such disorders. penntoday.upenn.edu/news/penn-vet-recreating-adrenal-gland-petri-dish
nadf meeting with fda
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July 12, 2021
We are an adrenal disease community bonded by our shared experiences, and finally, the US Food and Drug Administration (FDA) had a chance to hear about it. Organized by Lori Engler, NADF Executive Director, the session described topics that are most important to the community. The presentation was conducted by several NADF leaders to educate and bring awareness to these topics, clearly explain how they affect our community and to suggest solutions.
Patient Listening Sessions help the FDA understand what is important to patients, caregivers, and advocates when medical products are being developed. The format was a one-hour discussion designed to help those involved in the drug approval process understand the obstacles and unmet needs of patients with adrenal insufficiency.
Here's a summary of topics discussed during NADF’s meeting with the FDA. For each topic, NADF discussed possible solutions, including immediate needs: Modified release hydrocortisone options, Autoinjector for hydrocortisone, and better protection of life-preserving medications to prevent shortages.
According to the FDA, “Patient Listening Sessions are a resource for the medical product Centers to quickly engage with patients for their advocates and are one of many ways the patient community can share their experience with a disease or condition by talking directly with FDA staff.” The FDA hopes to better understand the burdens that diseases and treatments have on our daily lives and to learn what is important for our community.
This was a strong starting point and a very important new direction for NADF. Just a couple of days after the meeting the FDA shortage database was updated…a positive sign that they really were listening.
We are an adrenal disease community bonded by our shared experiences, and finally, the US Food and Drug Administration (FDA) had a chance to hear about it. Organized by Lori Engler, NADF Executive Director, the session described topics that are most important to the community. The presentation was conducted by several NADF leaders to educate and bring awareness to these topics, clearly explain how they affect our community and to suggest solutions.
Patient Listening Sessions help the FDA understand what is important to patients, caregivers, and advocates when medical products are being developed. The format was a one-hour discussion designed to help those involved in the drug approval process understand the obstacles and unmet needs of patients with adrenal insufficiency.
Here's a summary of topics discussed during NADF’s meeting with the FDA. For each topic, NADF discussed possible solutions, including immediate needs: Modified release hydrocortisone options, Autoinjector for hydrocortisone, and better protection of life-preserving medications to prevent shortages.
- Topic I: How can the FDA help create significant drug innovation in the management of adrenal insufficiency? How can we place a higher priority on new adrenal insufficiency treatments given patients’ lower quality of life and higher mortality rates?
Novel modified-release hydrocortisone preparations are available in Europe and have been shown to improve quality of life, improve innate immunity responses, lead to weight reduction, and reduce hemoglobin. NADF leaders also emphasized the need for Solu-Cortef Emergency Administration Technology and explained adrenal crisis is recognizable by its typical symptoms of diarrhea, vomiting and nausea. Unfortunately, the delay between the start of these symptoms and the administration of emergency treatment is the principal cause of death. - Topic II: Drug shortages have been a significant problem especially given the lack of options for treatment of life-preserving medications. Drug shortages can be a matter of life and death.
NADF discussed the issues created by shortages including treatment interruption, transitions to less-effective treatments, and rationing of life-saving treatments.
According to the FDA, “Patient Listening Sessions are a resource for the medical product Centers to quickly engage with patients for their advocates and are one of many ways the patient community can share their experience with a disease or condition by talking directly with FDA staff.” The FDA hopes to better understand the burdens that diseases and treatments have on our daily lives and to learn what is important for our community.
This was a strong starting point and a very important new direction for NADF. Just a couple of days after the meeting the FDA shortage database was updated…a positive sign that they really were listening.
Eton Pharmaceuticals Announces Availability of Orphan Drug ALKINDI® SPRINKLE (hydrocortisone) in the United States
November 24, 2020
DEER PARK, Ill., (GLOBE NEWSWIRE): Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of ALKINDI® SPRINKLE for sale and distribution in the United States.
SEE FULL ANNOUNCEMENT: ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-availability-orphan-drug-alkindir
DEER PARK, Ill., (GLOBE NEWSWIRE): Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of ALKINDI® SPRINKLE for sale and distribution in the United States.
SEE FULL ANNOUNCEMENT: ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-availability-orphan-drug-alkindir
eton pharmaceuticals announces fda approval of orphan drug alkindi® sprinkle (hYDROCORTISONE) AS REPLACEMENT THERAPY IN PEDIATRIC PATIENTS WITH ADRENOCORTICAL INSUFFICIENCY
September 29, 2020
ALKINDI SPRINKLE is the first and only FDA-approved granular hydrocortisone formulation for adrenocortical insufficiency specifically designed for children. Eton expects ALKINDI SPRINKLE to be available in the fourth quarter of 2020.
SEE ARTICLE: globenewswire.com/news-release/2020/09/29/2101010/0/en/Eton-Pharmaceuticals-Announces-FDA-Approval-of-Orphan-Drug-ALKINDI-SPRINKLE-hydrocortisone-as-Replacement-Therapy-in-Pediatric-Patients-with-Adrenocortical-Insufficiency.html
ALKINDI SPRINKLE is the first and only FDA-approved granular hydrocortisone formulation for adrenocortical insufficiency specifically designed for children. Eton expects ALKINDI SPRINKLE to be available in the fourth quarter of 2020.
SEE ARTICLE: globenewswire.com/news-release/2020/09/29/2101010/0/en/Eton-Pharmaceuticals-Announces-FDA-Approval-of-Orphan-Drug-ALKINDI-SPRINKLE-hydrocortisone-as-Replacement-Therapy-in-Pediatric-Patients-with-Adrenocortical-Insufficiency.html
