Improving Patient Outcomes in the Treatment and Management of Cystic Fibrosis: What's New in CFTR Modulator Therapy

A continuing medical education activity sponsored by NAMCP and AAMCN.

This activity is an archive from the live session from the 2019 Spring Managed Care Forum. If you participated in the live session, you are not eligible for continuing education credits from this archive.

This activity is valid from June 1, 2019 to August 1, 2020

Instructions for CME/CNE: Complete the pre-test, listen to the audio and view the slides, complete the post test, complete the evaluation form and hit submit. You will be asked to enter your name and email address on the pre-test, evaluation and post-test. If you close your internet browser without completing the post test, you will have ONE more opportunity to complete. A score of 70% must be achieved on the post test to receive continuing education credits. If you do not pass the post test after two attempts, you will not be eligible to try again. Once you complete the evaluation form and score 70% or higher on your post test, you will automatically be given your certificate.

To print or save your certificate, you will need to click on the “download” button and either print or save.


Audience: This activity is intended for healthcare professionals practicing in managed care environments.

This presentation is supported by an educational grant from
Vertex Pharmaceuticals

Cystic fibrosis (CF), also known as mucoviscidosis, is an autosomal recessive, genetic disorder that affects mostly the lungs but also the pancreas, liver, kidneys, and intestine. caused by the presence of mutations in both copies of the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). Those with a single working copy are carriers and otherwise mostly normal. CFTR is involved in production of sweat, digestive fluids, and mucus. When CFTR is not functional, secretions which are usually thin instead become thick. According to the Cystic Fibrosis Foundation, about 30,000 people are currently living with cystic fibrosis, and approximately 1,000 new cases of CF are diagnosed each year. There is no cure for cystic fibrosis, but recent treatment breakthroughs have the ability to provide a greater quality of life and improved outcomes for patients with CF. The condition is diagnosed by a sweat test and genetic testing and screening of infants at birth takes place in some areas of the world.

Long-term issues include difficulty breathing and coughing up sputum as a result of frequent lung infections. Other symptoms include sinus infections, poor growth, fatty stool, clubbing of the finger and toes, and infertility in males among others. The degrees of symptoms vary among patients and fortunately, for about half of CF patients, new treatments have become available for those who have two copies of the F508del mutation in their CFTR gene. This newer CFTR modulation therapeutic focuses on counteracting the damage caused by the disease, enabling patients to breath more easily and stay out of the hospital, improving outcomes and quality of life. This option is a major breakthrough in CF management, because it is the first therapy that specifically targets an important genetic mutation. Additionally, There has been new data presented in recent months in the cystic fibrosis arena, especially as it relates to combination therapies.

Upon completion of this activity, participants will be able to:

  • Compare and contrast current and emerging CFTR-based therapeutic options, including combination therapies, in terms of efficacy, safety, and clinical potential to improve pulmonary and non-pulmonary outcomes of patients with cystic fibrosis

  • Examine the role of newer combination CFTR modulator therapies and discuss ways to integrate them into the evolving treatment paradigm

  • Describe challenges and opportunities in CF disease management, including quality of life and total cost of care

  • Explore recent clinical trial data on emerging triple combination therapies and where they may fit in cystic fibrosis management

  • Analyze the importance of newborn screening, accurate diagnosis, genomic evaluation, and early pharmacologic intervention in the management of cystic fibrosis

  • Assess the managed care considerations of CFTR modulator therapies by exploring where these agents fit into an evolving cystic fibrosis management paradigm

Faculty: Gregory Sawicki, MD, MPH
Director, Cystic Fibrosis Center
Boston Children's Hospital
Assistant Professor of Pediatrics
Harvard Medical School


Dr. Sawicki serves on an advisory board for Gilead and Vertex. His presentation has been peer reviewed for any bias.
  Planning Committee:
Bill Williams, MD has no relevant financial relationships to disclose.
Jeremy Williams has no relevant financial relationships to disclose.
Will Williams has no relevant financial relationships to disclose.
Jacqueline Cole, RN, MS, CMCN has no relevant financial relationships to disclose.

NAMCP and/or the presenter has copyright or has received permissions for use of materials provided in this activity.

Accreditation & Designation
The National Association of Managed Care Physicians (NAMCP) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

NAMCP designates this enduring material for a maximum of 1 AMA PRA Category I creditsTM.

The American Association of Managed Care Nurses (AAMCN) is accredited as a provider of continuing nursing education by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC).

Nurses who complete this activity and achieve a passing score will receive 1 hour in continuing
nursing credit.

This activity has been approved by the American Board of Managed Care Nursing for 1.0 contact hour toward CMCN recertification requirements.

This presentation is supported by an educational grant from
Vertex Pharmaceuticals

NAMCP and/or this website does not provide medical advice, diagnosis or treatment. NAMCP does not endorse or imply endorsement of the content on any linked website. This website is to be used as an informational resource. With any health related concern, consult with your physician or healthcare professional.

Click Here To Continue