Endocrine Society and NORD Spotlight Rare Diseases in New Fellows Program

By Galen Gammino, Program Manager of Development & Funded Programs

Endocrinologists are the go-to specialists for treating diabetes, one of the world’s most common diseases affecting roughly 37.3 million people in the U.S. and 537 million people worldwide, according to the Cleveland Clinic.

But endocrinologists also are experts in treating diseases that affect small populations.

This is the rationale behind our new Rare Endocrine Disease (RED) Fellows Program, developed by the Society with support from the National Organization for Rare Disorders (NORD).

The program aims to address critical gaps in awareness, diagnosis, and care of rare endocrine diseases. It also aims to equip fellows with the knowledge and practical skills needed to improve patient outcomes.

A rare disease, as defined by NORD, is a disease, disorder, illness, or condition that affects fewer than 200,000 Americans. In the United States, more than 10,000 rare diseases affect more than 30 million Americans.

Of these, roughly 440 affect the endocrine system.

Given the sheer number of rare diseases overall, the goal of the RED Fellows Program isn’t to cover each and every one, but rather to help fellows develop their ability to identify and tackle the kinds of rare conditions they may find in their practice. 

Rare diseases to be addressed in the program include bone and mineral disorders, the hypothalamic-pituitary-adrenal (HPA) axis, metabolic and lipid conditions, reproductive disorders, and neuroendocrine tumors (NETs).

We are delighted by the enthusiastic response so far to the program. We have received far more applications than slots available for the fellowship.

Fellows Program Fills a Gap

A total of 50 U.S. fellows will participate in the in-person component, April 17-18, at the Society headquarters in downtown Washington, D.C. Fellows will engage with leading experts in rare endocrine diseases and participate in interactive, case-based discussions.

Fellows also will gain access to a NORD rare-disease patient panel and may continue their learning from  on-demand, multi-module courses that feature micro-lessons covering individual rare endocrine diseases.

Upon completion of the program, participants should be better able to:

• Recognize the complex symptom patterns and telltale signs of various REDs.
• Implement appropriate, evidence-based, and emerging screening strategies (i.e., genetic testing) for rare diseases to mitigate testing-associated patient costs, diagnostic delay, and achieve differential diagnosis.
• Assess risk factors for disease or treatment complications in individuals with REDs, particularly in cases of comorbidities.
• Devise individualized treatment strategies that account for patient history, transitions in care, and socioeconomic factors to manage those with REDs.
• Apply evidence-based approaches in the treatment and symptom management of rare diseases when approved therapies are unavailable to improve long-term outcomes of individuals with REDs.

A unique feature of the in-person program is its focus on the patient’s perspective. Fellows will have the opportunity to hear from real patients, and learn how these diseases impact them personally. They also will learn how these patients navigate the health system and understand how supportive or unsupportive their health care providers have been.

NORD was especially involved in helping develop the patient and advocacy components of the program.

Another unique element of the in-person workshop will be sessions featuring a geneticist and genetic counselors who will explain the importance of genetic testing and interpreting results.

Society’s Growing List of Fellows Programs

The RED Fellows program joins other fellowship programs offered by the Society, including the Type 1 Diabetes Fellows Series, the Obesity Fellows Program, and our overall Fellows Training Series.

No other U.S. organization offers a fellowship program such as this to address rare endocrine diseases. 
The idea for the RED Fellows Program came about when industry partners at ENDO heard about the Type 1 Diabetes Fellows Series.

They asked the Society about the potential for creating a fellows program centered on rare endocrine disease. The proposal then entered the concept phase, where it was considered by both the Special Programs Committee and Clinical Endocrine Education Committee (CEEC). Both widely supported the idea.

From CEEC's perspective, endocrinologists might see questions on rare endocrine diseases, say on a board exam, but not experience a case until one day it pops up in their office down the line.

This program will fill this gap and help fellows develop their ability to both diagnose and treat rare disease in practice.

The RED Fellows program is supported by educational grants from Alexion Pharmaceuticals, Inc.; Neurocrine Biosciences, Inc.; BridgeBio Pharma, Inc.; Chiesi USA,Inc.; Corcept Therapeutics, Inc.; and Crinetics Pharmaceuticals, Inc.