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

Burns, Ted M.; Smith, G.A.; Allen, Jeffrey A.; Amato, Anthony A.; Arnold, W. David; Barohn, Richard J.; Benatar, Michael; Bird, Shawn J.; Bromberg, Mark B.; Chahin, Nizar; Ciafaloni, Emma; Cohen, Jeffrey A.; Corse, Andrea M.; Crum, Brian A.; David, William S.; Dimberg, Elliot L.; Sousa, Eduardo A. De; Donofrio, Peter D.; Dyck, Peter James; Engel, Andrew G.; Ensrud, Erik; Ferrante, Mark A.; Freimer, Miriam; Gable, Karissa; Gibson, Summer; Gilchrist, James M.; Goldstein, Jonathan; Gooch, Clifton L.; Goodman, Brent P.; Gorelov, Dmitri; Gospe, Sídney M.; Goyal, Namita; Guidon, Amanda C.; Guptill, Jeffrey T.; Gutmann, Laurie; Gutmann, Ludwig; Gwathmey, Kelly; Harati, Yadollah; Harper, C. Michel; Hehir, Michael K.; Hobson‐Webb, Lisa D.; Howard, James F.; Jackson, Carlayne E.; Johnson, Nicholas E.; Jones, Sarah; Juel, Vern C.; Kaminski, Henry J.; Karam, Chafic; Kennelly, Kathleen D.; Khella, Sami; Khoury, Julie; Kincaid, John C.; Kissel, John T.; Kolb, Noah; Lacomis, David; Ladha, Shafeeq; Larriviere, Dan; Lewis, Richard A.; Li, Yuebing; Litchy, William J.; Logigian, Eric L.; Lou, Jau Shin; Macgowen, Daniel J L; Maselli, Ricardo A.; Massey, Janice M.; Mauermann, Michelle; Mathews, Katherine D.; Meriggioli, Matthew N.; Miller, Robert G.; Moon, Jean; Mozaffar, Tahseen; Nations, Sharon P.; Nowak, Richard J.; Ostrow, Lyle W.; Pascuzzi, Robert M.; Peltier, Amanda; Ruzhansky, Katherine; Richman, David P.; Ross, Mark A.; Rubin, Devon I.; Russell, James A.; Sachs, George; Salajegheh, Mohammad; Saperstein, David; Scelsa, Stephen N.; Selcen, Duygu; Shaibani, Aziz; Shieh, Perry B.; Silvestri, Nicholas J.; Singleton, Jessica; Smith, Benn E.; So, Yuen T.; Solórzano, G; Sorenson, Eric J.; Srinivasen, Jayashri; Tavee, Jinny; Tawil, Rabi; Thaisetthawatkul, Pariwat; Thornton, Charles A.; Trivedi, Jaya; Vernino, Steven; Wang, Annabel K.; Webb, Tyler A.; Weiss, Michael D.; Windebank, Anthony J.; Wolfe, Gil I.

doi:10.1002/mus.25009

Cited by 23 publications

(12 citation statements)

References 11 publications

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“…In their letter, they expressed their concerns about the European Union legislation applicable to orphan drug licensing. Similarly, in 2015, neuromuscular doctors collectively published their concern about the potential for the price of the drug to be dramatically increased should the drug company Catalyst obtain FDA approval …”

Section: Challengesmentioning

confidence: 99%

Therapeutic strategies for congenital myasthenic syndromes

Lee

Beeson

Palace

2018

Annals of the New York Academy of Sciences

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To date, more than 25 genes have been implicated in the etiology of the congenital myasthenic syndromes (CMS), and an ever-growing phenotypic landscape is now encountered in the CMS clinic. Unlike the autoimmune form of myasthenia, there is no role for immunomodulatory agents in the treatment of CMS. The present-day drug repertoire comprises acetylcholinesterase inhibitors (mainly pyridostigmine), 3,4-diaminopyridine (3,4-DAP), ephedrine, salbutamol/albuterol, open-channel blockers (fluoxetine, quinidine), or a combination of these. These are prescribed by the specialist in an off-label manner, as there is no drug currently licensed for the treatment of these rare diseases. The effective pharmacological agent varies according to the genetic form of CMS, and it is important to realize that an agent that provides benefit in one CMS subtype can be harmful in another. In addition, the time to treatment response is variable and tends to be commensurate with the drug used. Here, we summarize for the clinician the therapeutic strategies employed in this ever-evolving disease spectrum. We also address the barriers to treatment and discuss the treatment of CMS in pregnancy.

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Section: Challengesmentioning

confidence: 99%

Therapeutic strategies for congenital myasthenic syndromes

Lee

Beeson

Palace

2018

Annals of the New York Academy of Sciences

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“…In the UK, for example, pricing has led to the previous framework rising from approx. £600 per year per patient to £60,000; the situation seems the same in Germany as well as recently in the United States [22].…”

Section: Lambert-eaton Myasthenic Syndrome (Lems)mentioning

confidence: 89%

“…22.3) can be prepared by a pharmacy; it can also be used to treat Lambert-Eaton myasthenic syndrome (LEMS) as a guideline-compliant therapy and has been approved by the EMA as an orphan drug (amifampridine). The high costs of this procedure as well as the procedure itself has been critically discussed [22].…”

Section: Rare Disease (Orphan) Designationmentioning

confidence: 99%

Off-Label Use in Rare Diseases: Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes

Janzen¹

2018

Neurology International Open

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AbstR Ac tThe off-label use of licensed drugs is widespread, and the risk of adverse events is elevated, especially if the scientific evidence is low. The treatment of rare diseases ( < 5/10,000) therefore may increase the risk of off-label use. The optimized standard treatment is primarily based on the regulatory decisions of the German Federal Joint Committee. The valid guidelines and the regulations of the German health system are discussed in this article. The criteria for indication and monitoring of off-label use are shown, especially focused on the problem of refractory myasthenia gravis. Escalation of treatment results regularly in off-label use. The arguments within the applicable guidelines should be followed. Some new aspects of the European regulations are likewise discussed.

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“…As Catalyst Pharmaceuticals prepares to launch its second Phase 3 clinical trial of Firdapse [our version of 3,4‐diaminopyridine (3,4‐DAP)] in patients with Lambert–Eaton myasthenic syndrome (LEMS), we feel it necessary to dispel misconceptions in the recently published editorial by Burns and colleagues …”

mentioning

confidence: 99%

“…Currently, we estimate that a small fraction of LEMS patients receive 3,4‐DAP and only through the cumbersome process of compassionate use programs. Many physicians believe that 3,4‐DAP is the most effective treatment for patients with LEMS . Catalyst believes that perpetuating a system that provides access to so few patients is morally wrong, because it leaves most LEMS patients without treatment.…”

mentioning

confidence: 99%

A response to a recent editorial by concerned physicians on 3,4‐diaminopyridine

McEnany

2016

Muscle and Nerve

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

Cited by 23 publications

References 11 publications

Therapeutic strategies for congenital myasthenic syndromes

Therapeutic strategies for congenital myasthenic syndromes

Off-Label Use in Rare Diseases: Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes

A response to a recent editorial by concerned physicians on 3,4‐diaminopyridine

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