Right-to-Try Laws: Hope, Hype, and Unintended Consequences

Bateman-House, Alison; Kimberly, Laura L.; Redman, Barbara K.; Dubler, Nancy Neveloff; Caplan, Arthur L.

doi:10.7326/m15-0148

Cited by 33 publications

(22 citation statements)

References 2 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…There is no guarantee that the product sought will be effective and/or safe, much less that it will be effective and/or safe for the particular patient, and these individual INDs are not purposed to collect data on the drug. [7][8][9][10] The FDA's consideration of an expanded-access application generally comes only after the commercial sponsor has agreed to provide the investigational drug. The FDA cannot require the commercial sponsor to provide its investigational drug for expanded access use.…”

Section: Discussionmentioning

confidence: 99%

How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

McKee

Markon

Chan-Tack

et al. 2017

The Journal of Clinical Pharma

View full text Add to dashboard Cite

In this review of individual patient expanded-access requests to the Center for Drug Evaluation and Research for the period Fiscal Year 2010 to Fiscal Year 2014, we evaluated the number of applications received and the number allowed to proceed. We also evaluated whether drugs and certain biologics obtained under expanded access went on to be approved by the Food and Drug Administration. Finally, we considered concerns that adverse events occurring during expanded access might place sponsors at risk for legal liability. Overall, 98% of individual patient expanded-access requests were allowed to proceed. During the study period, among drugs without a previous approval for any indication or dosage form, 24% of unique drugs (ie, multiple applications for access to the same drug were considered to relate to 1 unique drug), and 20% of expanded-access applications received marketing approval by 1 year after initial submission; 43% and 33%, respectively, were approved by 5 years after initial submission. A search of 3 legal databases and a database of news articles did not appear to identify any product liability cases arising from the use of a product in expanded access. Our analyses seek to give physicians and patients a realistic perspective on the likelihood of a drug's approval as well as certain information regarding the product liability risks for commercial sponsors when providing expanded access to investigational drugs. The US Food and Drug Administration (FDA)'s expanded-access program maintains a careful balance between authorizing patient access to potentially beneficial drugs and protecting them from drugs that may have unknown risks. At the same time, the agency wishes to maintain the integrity of the clinical trials process, ultimately the best way to get safe and effective drugs to patients.

show abstract

Section: Discussionmentioning

confidence: 99%

How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

McKee

Markon

Chan-Tack

et al. 2017

The Journal of Clinical Pharma

View full text Add to dashboard Cite

show abstract

“…Although there are credible arguments in favor of increasing patient access to medical interventions that are still undergoing testing, ethicists and policy makers have warned that unscrupulous businesses may take advantage of such deregulation to market their products directly to consumers under the guise of a 'right-to-try'. Even well-intentioned companies and researchers may have a difficult time conveying to interested patients the likely efficacy of these products [25,42]. As our exploration of crowdfunding campaigns for unproven stem cell interventions shows, those seeking these products are influenced by scientific-and research-based markers of legitimacy, prone to overstating the proven efficacy of these products, and likely to misunderstand their role in the process of legitimate scientific research.…”

Section: Future Perspectivementioning

confidence: 99%

Selling Stem Cell ‘Treatments’ as Research: Prospective Customer Perspectives from Crowdfunding Campaigns

Snyder

Turner

2018

Regen. Med.

View full text Add to dashboard Cite

show abstract

“…Other ethical issues surround compassionate access to investigational products that CompAC does not, and was not created to, address, including whether it is ever ethical to provide agents with a very small chance of benefit to very sick patients, 20 and the likelihood of therapeutic misestimation, in which patients with life-threatening illnesses may be more inclined to believe in the potential benefit of an agent than meager evidence supports. 21…”

Section: Problems With the Compac Modelmentioning

confidence: 99%

Ensuring Justice in Access to Investigational Neurological Drugs

Kearns¹,

Bateman-House²,

Caplan³

2018

Semin Neurol

Self Cite

View full text Add to dashboard Cite

Patients who suffer from life-threatening illnesses or are stricken with conditions that could result in serious morbidity who have exhausted all appropriate treatments may choose to try, through the Food and Drug Administration's expanded access program, an investigational drug or device in development. The program has succeeded for decades in allowing patients to access potentially helpful but still experimental agents. Nevertheless, the administration of investigational drugs outside of clinical trials raises several ethical issues. Of particular concern are the validity of informed consent and the absence of a framework to ensure that experimental drugs are allocated justly and transparently. Although there are some safeguards to help protect the soundness of consent, little work to date has been done to guarantee that investigational medical products are allocated justly and transparently. We introduce a novel pilot project that seeks to address this issue.

show abstract

Right-to-Try Laws: Hope, Hype, and Unintended Consequences

Cited by 33 publications

References 2 publications

How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

How Often Are Drugs Made Available Under the Food and Drug Administration's Expanded Access Process Approved?

Selling Stem Cell ‘Treatments’ as Research: Prospective Customer Perspectives from Crowdfunding Campaigns

Ensuring Justice in Access to Investigational Neurological Drugs

Contact Info

Product

Resources

About