How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Furlong, Pat ^a   Bridges, John F P ^b   Charnas, Lawrence ^c   Fallon, Justin R ^d   Fischer, Ryan ^a   Flanigan, Kevin M ^e   Franson, Timothy R ^f   Gulati, Neera ^g   McDonald, Craig ^h   Peay, Holly ^a   Sweeney, H Lee ⁱ  

^a Parent Project Muscular Dystrophy   (United States)

^b JOHNS HOPKINS BLOOMBERG SCHOOL OF PUBLIC HEALTH   (United States)

^c SHIRE   (United States)

^d BROWN UNIVERSITY   (United States)

^e NATIONWIDE CHILDREN S HOSPITAL   (United States)

^f YourEncore   (United States)

^g Suneel's Light Foundation   (United States)

^h UNIVERSITY OF CALIFORNIA   (United States)

ⁱ UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Advocacy; Clinical trial; Drug development; Duchenne muscular dystrophy; FDA; Industry guidance; Patient engagement; Public policy; Rare disease

Indexed keywords

DYSTROPHIN;

ALZHEIMER DISEASE; BUDGET; CAREGIVER; CLINICAL TRIAL (TOPIC); COMMUNITY ACQUIRED PNEUMONIA; DISEASE COURSE; DRAFT INDUSTRY GUIDANCE; DRUG INDUSTRY; DUCHENNE MUSCULAR DYSTROPHY; FOOD AND DRUG ADMINISTRATION; HEALTH CARE INDUSTRY; HEALTH CARE ORGANIZATION; HISTORY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IRRITABLE COLON; LIFESPAN; LIMIT OF QUANTITATION; LUNG DISEASE; MUSCLE BIOPSY; MUSCLE WEAKNESS; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; OUTCOME ASSESSMENT; PARENT PROJECT MUSCULAR DYSTROPHY; PATIENT ADVOCACY; PATIENT GUIDANCE; PEER REVIEW; PUBLIC HEALTH; REVIEW; RISK BENEFIT ANALYSIS; SYSTEMIC LUPUS ERYTHEMATOSUS; TELECONFERENCE; UNITED STATES;

MUSCULAR DYSTROPHY, DUCHENNE; PATIENT ADVOCACY; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84948703784     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0281-2     Document Type: Review

References (12)

1. Muscular Dystrophy Association. Duchenne muscular dystrophy: overview [Internet]. Chicago, IL: MDA Inc; http://mda.org/disease/duchenne-muscular-dystrophy/overview Accessed 16 Dec 2014.
2. National Institute of Neurological Disorders and Stroke. Muscular dystrophy: hope through research [Internet]. Bethesda, MD: NINDS; 2014. http://www.ninds.nih.gov/disorders/md/detail-md.htm Accessed 16 Dec 2014.
3. Wicklund MP. The muscular dystrophies. Continuum (Minneap Minn). 2013;19:1535-70. 6 Muscle Disease.
4. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77-93.
5. Centers for Disease Control and Prevention. Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years-four states, 2007. MMWR Morb Mortal Wkly Rep. 2009;40:1119-22.
6. McNeil DE, Davis C, Jillapalli D, Targum S, Durmowicz A, Coté TR. Duchenne muscular dystrophy: drug development and regulatory considerations. Muscle Nerve. 2010;41(6):740-5.
7. European Medicines Agency. Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. 2013. http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2013/03/WC500139508.pdf Accessed 21 April 2015.
8. Parent Project Muscular Dystrophy. Putting patients first [Internet]. Hackensack, NJ: PPMD; 2013. http://www.parentprojectmd.org/site/DocServer/WhitePaper-2013FINAL.pdf?docID=13883 Accessed 21 April 2015.
9. Administrative Practices and Procedures; Good Guidance Practices, 21 CFR Parts 7, 10, 14, 19, 25, 101, 107, 110, 114, 170, 310, 312, 314, 316, 500, 514, 601, 803, 814, and 860 2011. http://www.fda.gov/downloads/AboutFDA/Transparency/TransparencyInitiative/UCM285124.pdf Accessed 22 April 2015.
10. Peay HL, Hollin I, Fischer R, Bridges JFP. A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for Duchenne muscular dystrophy. Clin Ther. 2014;36(5):624-37.
11. Duchenne Muscular Dystrophy Cardiac Recommendations for the FDA. 2014. http://api.ning.com/files/jPrLLXuRuu6o8BpzprF0lmYRjYsot4VdsGfAC9grIog1VccYaW3xVXOHCjRN∗pZ8gOtJaLVialKeInFtZnjc6tGHC5WD5lnt/CardiacrecommendationsfortheFDAFINAL.pdf Accessed 21 April 2015.
12. Working Group/Workshop Executive Summary. Contemporary cardiac issues in Duchenne muscular dystrophy. 2014. http://www.nhlbi.nih.gov/research/reports/2014-duchenne-muscular-dystrophy Accessed 21 April 2015.