ArticleOrphanet journal of rare diseases2015
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
Article in Orphanet journal of rare diseases, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
26 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Societal participation in the development of orphan drugs: a systematic review.Frontiers in medicine · 2025Pooled it
- Life with a Primary Immune Deficiency: a Systematic Synthesis of the Literature and Proposed Research Agenda.Journal of clinical immunology · 2016Pooled it
- Living with a rare disease: Why lived experience must shape research.PLoS medicine · 2026Article
- Patient organizations involvement in healthcare: a rapid review and conceptual framework.Globalization and health · 2026Review
- Patient engagement in clinical trial design for rare neuromuscular disorders: impact on the DELIVER and ACHIEVE clinical trials.Research involvement and engagement · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- Patients at the Heart of the Scientific Dialogue: An Industry Perspective.Oncology and therapy · 2023Article
- Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases.Orphanet journal of rare diseases · 2022Article
- A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy.The patient · 2022Article
- Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?Clinical pharmacology and therapeutics · 2022Review
- To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases.Orphanet journal of rare diseases · 2020Review
- Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity.JAMA network open · 2020Article
- A study protocol for quantifying patient preferences in neuromuscular disorders: a case study of the IMI PREFER Project.Wellcome open research · 2020Article
- Recognizing that Evidence is Made, not Born.Clinical pharmacology and therapeutics · 2019Article
- Article
- Developing an instrument to assess patient preferences for benefits and risks of treating acute myeloid leukemia to promote patient-focused drug development.Current medical research and opinion · 2018Article
- Symposium Title: Preference Evidence for Regulatory Decisions.The patient · 2018Article
- Research as an event: a novel approach to promote patient-focused drug development.Patient preference and adherence · 2018Article
- Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia.Patient preference and adherence · 2018Article
- Muscle MRI and functional outcome measures in Becker muscular dystrophy.Scientific reports · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors.
Funding
Abstract
Among the challenges confronting patients with rare diseases is a dearth of treatment options. The development of safe and effective new therapies is hampered by challenges associated with conducting clinical trials in small populations. In this article, we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. This unprecedented undertaking involved a broad coalition of more than 80 stakeholders collaborating across nine time zones to produce a document in only 6 months. We hope that other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.