Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4‐diaminopyridine

@article{Burns2016EditorialBC,
  title={Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4‐diaminopyridine},
  author={Ted M. Burns and Gordon A. Smith and Jeffrey A. Allen and Anthony A. Amato and W. David Arnold and Richard J. Barohn and Michael Benatar and Shawn J. Bird and Mark Bromberg and Nizar Chahin and Emma Ciafaloni and Jeffrey A. Cohen and Andrea M. Corse and Brian A Crum and William S. David and Elliot L. Dimberg and Eduardo A. De Sousa and Peter D. Donofrio and P. James B. Dyck and Andrew G. Engel and Erik R. Ensrud and Mark A. Ferrante and Miriam Freimer and Karissa L. Gable and Summer Bell Gibson and James M. Gilchrist and Jonathan M. Goldstein and Clifton L. Gooch and Brent P. Goodman and Dmitri Gorelov and Sidney M. Gospe and Namita Goyal and Amanda C. Guidon and Jeffrey T Guptill and Laurie Gutmann and Ludwig Gutmann and Kelly Graham Gwathmey and Yadollah Harati and Charles M. Harper and Michael K Hehir and Lisa D. Hobson‐Webb and James F. Howard and Carlayne E. Jackson and Nicholas E Johnson and Sarah Jones and Vern C. Juel and Henry J Kaminski and Chafic Karam and Kathleen D. Kennelly and Sami L. Khella and Julie A Khoury and John C. Kincaid and John T. Kissel and Noah A Kolb and David Lacomis and Shafeeq Ladha and Dan Larriviere and Richard Alan Lewis and Yuebing Li and William J. Litchy and Eric L Logigian and Jau Shin Lou and Daniel J L MacGowen and Ricardo A Maselli and Janice M. Massey and Michelle L Mauermann and Katherine Mathews and Matthew N. Meriggioli and Robert G. Miller and Joon-shik Moon and Tahseen Mozaffar and Sharon P. Nations and Richard J. Nowak and Lyle W. Ostrow and Robert M. Pascuzzi and Amanda Peltier and Katherine Ruzhansky and David P Richman and Mark A. Ross and Devon I. Rubin and James A. Russell and George M. Sachs and Mohammad Kian Salajegheh and David S. Saperstein and Stephen N. Scelsa and Duygu Selcen and Aziz I. Shaibani and Perry B. Shieh and Nicholas J Silvestri and J Rob Singleton and Benn Smith and Yuen T. So and Guillermo E. Solorzano and Eric J. Sorenson and Jayashri Srinivasen and Jinny O. Tavee and Rabi Tawil and Pariwat Thaisetthawatkul and Charles A. Thornton and Jaya R. Trivedi and Steven Vernino and Annabel K. Wang and Tyler A Webb and Michael D. Weiss and Anthony J Windebank and Gil I. Wolfe},
  journal={Muscle \& Nerve},
  year={2016},
  volume={53}
}
TED M. BURNS, MD, GORDON A. SMITH, MD, JEFFREY A. ALLEN, MD, ANTHONY A. AMATO, MD, W. DAVID ARNOLD, MD, RICHARD BAROHN, MD, MICHAEL BENATAR, MD, PhD, SHAWN J. BIRD, MD, MARK BROMBERG, MD, NIZAR CHAHIN, MD, EMMA CIAFALONI, MD, JEFFREY A. COHEN, MD, ANDREA CORSE, MD, BRIAN A. CRUM, MD, WILLIAM S. DAVID, MD, ELLIOT DIMBERG, MD, EDUARDO A. DE SOUSA, MD, PETER D. DONOFRIO, MD, P. JAMES B. DYCK, MD, ANDREW G. ENGEL, MD, ERIK R. ENSRUD, MD, MARK FERRANTE, MD, MIRIAM FREIMER, MD, KARISSA L. GABLE, MD… 
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References

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A randomized trial of 3,4-diaminopyridine in Lambert-Eaton myasthenic syndrome
TLDR
This study corroborates previous studies and many years of clinical experience showing that DAP is an effective and safe treatment for LEMS and indicates that patients who received DAP had a significantly greater improvement in the QMG score and in the summated amplitude of compound muscle action potentials recorded from three sentinel limb muscles.
RESULTS FROM THE DAPPER STUDY: INPATIENT DOUBLE-BLIND PLACEBO-CONTROLLED WITHDRAWAL STUDY OF 3,4-DIAMINOPYRIDINE BASE (3,4-DAP) IN SUBJECTS WITH LAMBERT-EATON MYASTHENIC SYNDROME (LEMS).
TLDR
Results from the 3,4-DIAMINOPYRIDINE BASE study of 3, 4-DAP in SUBJECTS with LAMBERT-EATON MYASTHENIC SYNDROME (LEMS) show that dAP acts as a “spatially aggregating agent” to cause dystonia in mice.
Unintended effects of orphan product designation for rare neurological diseases
TLDR
It is suggested that a paradoxical effect of orphan product exclusivity can be reduced patient access to existing drugs, and the use of tetrabenazine for chorea in Huntington disease, adrenocorticotropic hormone for infantile spasms, and enzyme replacement therapy with alglucosidase alpha for Pompe disease are highlighted.
3,4‐Diaminopyridine is more effective than placebo in a randomized, double‐blind, cross‐over drug study in LEMS
TLDR
A randomized, double‐blind, cross‐over drug trial of 3,4‐diaminopyridine showed significant efficacy over placebo in patients with Lambert–Eaton myasthenic syndrome, however, not all patients preferred this drug.
3,4-diaminopyridine for the treatment of Lambert–Eaton myasthenic syndrome
TLDR
Four randomized placebo-controlled trials of 3,4-DAP in patients with LEMS demonstrated a significant effect on muscle strength and compound muscle action potential amplitude and this drug is the mainstay for symptomatic treatment of LEMS.
Novel drug of choice in Eaton-Lambert syndrome.
TLDR
Symptomatic treatment with an immediately acting drug would be of value to the cancer patient with Eaton-Lambert syndrome, and if such a drug is proved safe it could also be used in the cryptogenetic form of the disease.
Efficacy of 3,4‐Diaminopyridine and Pyridostigmine in the Treatment of Lambert–Eaton Myasthenic Syndrome: A Randomized, Double‐Blind, Placebo‐Controlled, Crossover Study
TLDR
3,4‐Diaminopyridine and pyridostigmine are widely used to treat Lambert–Eaton myasthenic syndrome, either alone or in combination, and this could lead to a synergistic effect on neuromuscular transmission.
Open letter to prime minister David Cameron and health secretary Andrew Lansley
Neurologists and paediatricians call for action on “massive” rises in the prices of orphan drugs
A Delicate Balance--Pharmaceutical Innovation and Access.
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Even with many new therapeutic options, the proportion of health care spending devoted to retail prescription medications remains about the same as it was in 1960. Relying on competitive markets to
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