Evidence-Based Strategies to Facilitate Access to Therapies for Patients with Spinal Muscular Atrophy

Faculty

Crystal M. Proud, MD
Director, Neuromuscular Clinic and Spinal Muscular Atrophy Center, Children's Hospital of the King's Daughters
Medical Director, CHKD Novel Therapeutics and Gene Therapy Center, Norfolk, VA
Crystal M. Proud, MD

. Proud is the director of the Neuromuscular Clinic and Spinal Muscular Atrophy Center at Children’s Hospital of The King’s Daughters (CHKD), working collaboratively to provide a comprehensive approach to patients with neuromuscular diseases. She attended college at the University of Virginia where she majored in Neuroscience, and then studied at Eastern Virginia Medical School where she received her Doctorate of Medicine. Dr. Proud pursued general Pediatrics training in Atlanta, Georgia at Emory University and then trained in Neurology to receive special qualifications in Child Neurology at Stanford University. She remained at Stanford for her Neuromuscular Medicine fellowship and then returned home to Virginia to join her current practice as part of Children?s Specialty Group, serving the patients at CHKD in Norfolk, Virginia. During her subspecialty training at Stanford University, Dr. Proud participated in landmark clinical trials including one that supported FDA approval of the first treatment for spinal muscular atrophy (SMA). She has continued this interest at CHKD where she now serves as primary investigator for several clinical trials evaluating novel treatments for patients with various neuromuscular conditions. Her research focus is in the development and conduct of therapeutic trials for SMA and Duchenne muscular dystrophy. Dr. Proud serves as the medical director for the CHKD Novel Therapeutics and Gene Therapy Center, with a commitment to optimizing clinical care and offering participation in clinical research to patients and families with rare disease.

Erin Neil, DO
Clinical Assistant Professor of Pediatrics, Director, Pediatric Neuromuscular Program
Co-Director, Muscular Dystrophy Association Clinic, Michigan Medicine, University of Michigan Medical School
Medical Director, State of Michigan's SMA Newborn Screening Program, Ann Arbor, MI
Erin Neil, DO

Dr. Erin Neil is a Clinical Assistant Professor of Pediatrics at the University of Michigan, where she is the Director of the Pediatric Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Clinic. She serves as the Medical Director for the State of Michigan’s SMA Newborn Screening program, which includes administrative and clinical oversight for care of newborns diagnosed with SMA.

Dr. Neil completed medical training at the Kansas City University, College of Osteopathic Medicine; pediatric neurology training at the Detroit Medical Center/Wayne State University/Children’s Hospital of Michigan; and Neuromuscular fellowship at University of Texas Southwestern, Dallas.

Statement of Need

Spinal muscular atrophy (SMA) is a rare disease but is also the most common fatal genetic disease of infancy. While the approval of the first drug treatment for SMA in 2016 and the FDA approval of the first gene therapy and oral small molecule for SMA have revolutionized SMA management, significant delays in diagnosis and treatment initiation have been observed. This is due to clinicians having limited knowledge regarding SMA diagnostic tests and assessments as well as the latest clinical evidence on approved and emerging therapies, which often contributes to poor outcomes.

This enduring material focuses on implementing evidence-based strategies for diagnosing SMA, applying the latest clinical evidence of approved and emerging therapies, and recognizing individual patient- and parent-/caregiver-related factors that guide clinical decision-making, in order to facilitate access to therapies for SMA management.

Learning Objectives

At the end of this CME/CE activity, participants should be able to:

  • Determine the tests and clinical assessments that can aid in diagnosing SMA as well as monitoring disease activity and treatment response.
  • Apply the latest clinical evidence to evaluate the cost-effectiveness of approved and emerging therapies for SMA.
  • Recognize the individual patient- and parent-/caregiver-related factors that guide clinical decision-making in SMA management.

The following learning objectives pertain only to those requesting CNE or CPE credit:

  • Determine the tests and clinical assessments that can aid in diagnosing SMA as well as monitoring disease activity and treatment response.
  • Summarize the latest clinical evidence to evaluate the cost-effectiveness of approved and emerging therapies for SMA.
  • Recognize the individual patient- and parent-/caregiver-related factors that guide clinical decision-making in SMA management.

Financial Support

Supported by an educational grant from Biogen MA, Inc.

Target Audience

Managed care medical directors/physicians and specialty and managed care pharmacists.

Credit Information

Physicians (ACCME) 1.0

CME Outfitters, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Note to Nurse Practitioners

Nurse Practitioners can apply for AMA PRA Category 1 Credit through the American Academy of Nurse Practitioners (AANP). AANP will accept AMA PRA Category 1 Credit  from organizations accredited by the Accreditation Council for Continuing Medical Education. Nurse practitioners can also apply for credit through their state boards.

Pharmacists/Pharmacy Tech (ACPE) 1.0

This application-based activity is approved for 1.0 contact hour (0.10 CEUs) of continuing pharmacy credit.

ABIM MOC 1.0

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 medical knowledge MOC point in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

ABPN MOC

ABPN Diplomates may select any CME activity relevant to their practice to count towards ABPN MOC requirements.

Pediatricians (ABP MOC) 1.5

Successful completion of this CME activity, which includes participation in the activity and individual assessment of and feedback to the learner, enables the learner to earn up to 1.5 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program. It is the CME activity provider’s responsibility to submit learner completion information to ACCME for the purpose of granting ABP MOC credit.

Royal College MOC

Through an agreement between the Accreditation Council for Continuing Medical Education and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

MIPS Improvement Activity

Completion of this accredited CME activity meets the expectations of an Accredited Safety or Quality Improvement Program (IA_PSPA_28) for the Merit-based Incentive Payment Program (MIPS). Clinicians should submit their improvement activities by attestation via the CMS Quality Payment Program website.

Disclosure Declaration

It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and attempted to resolve any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process. The following information is for participant information only. It is not assumed that these relationships will have a negative impact on the presentations.


Dr. Proud reports that she receives research support from AveXis/Novartis Pharmaceuticals Corporation and Biogen. She is on the speakers bureau for AveXis/Novartis Pharmaceuticals Corporation and Biogen. She is a consultant for AveXis/Novartis Pharmaceuticals Corporation; Biogen; and Genentech, Inc./Roche.


Dr. Neil has no disclosures to report.


  • Tony Graham, MD (peer reviewer) has no disclosures to report.
  • Mae Ochoa, RPh (peer reviewer) has no disclosures to report.
  • Susan Perry (planning committee) has no disclosures to report.
  • Jan Perez (planning committee) has no disclosures to report.
  • Sharon Tordoff (planning committee) has no disclosures to report.
  • Disclosures were obtained from the CME Outfitters, LLC staff: No disclosures to report.

Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.

Obtaining Credit

Post-tests, credit request forms, and activity evaluations must be completed online (requires free account activation), and participants can print their certificate or statement of credit immediately (75% pass rate required). This website supports all browsers except Internet Explorer for Mac. For complete technical requirements and privacy policy, visit our Privacy & Confidentiality page.

 

NOTE: Pharmacist CE Universal Activity Number, Enduring: 0376-0000-21-061-H01-P

Questions about this activity?

Call us at (877) CME-PROS or (877) 263-7767.

 

MMV-113-041621-29

Evidence-Based Strategies to Facilitate Access to Therapies for Patients with Spinal Muscular Atrophy
Event Date: 04/16/2021 at 8:00 am EST