Right-to-Try Investigational Therapies for Incurable Disorders

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Patients with life-threatening disorders such as amyotrophic lateral sclerosis, for which only minimally effective medical therapies currently exist, often seek treatments not proven to be effective and not approved by regulatory agencies for use outside of experimental treatment trials. The expanded access (compassionate use) provisions of the US Food and Drug Administration (FDA) for access to such therapies are often perceived as being inadequate. In response, states have passed right-to-try laws designed to improve access to experimental therapies for patients willing to assume the risks associated with such treatments. This situation has resulted in conflicts between those who perceive access to such treatments as their right as autonomous individuals and those who believe that the principles of beneficence and nonmaleficence justify actions of physicians and regulators in controlling access to such treatments. A variety of factors also contribute to the inequitable distribution of such treatments. Better systems are needed to improve access to promising new treatments while protecting these vulnerable patients from the abuses associated with human research in the preregulatory era.

Original languageEnglish (US)
Pages (from-to)1451-1457
Number of pages7
JournalCONTINUUM Lifelong Learning in Neurology
Volume23
Issue number5, Peripheral Nerve and Motor Neuron Disorders
DOIs
StatePublished - Oct 1 2017

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Investigational Therapies
Beneficence
Therapeutics
Compassionate Use Trials
Amyotrophic Lateral Sclerosis
United States Food and Drug Administration
Physicians

All Science Journal Classification (ASJC) codes

  • Clinical Neurology
  • Genetics(clinical)

Cite this

Simmons, Zachary. / Right-to-Try Investigational Therapies for Incurable Disorders. In: CONTINUUM Lifelong Learning in Neurology. 2017 ; Vol. 23, No. 5, Peripheral Nerve and Motor Neuron Disorders. pp. 1451-1457.
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Right-to-Try Investigational Therapies for Incurable Disorders. / Simmons, Zachary.

In: CONTINUUM Lifelong Learning in Neurology, Vol. 23, No. 5, Peripheral Nerve and Motor Neuron Disorders, 01.10.2017, p. 1451-1457.

Research output: Contribution to journalArticle

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