Communicating Uncertainties Associated With the Benefits and Risks of New Cancer Drugs

London School of Economics and Political Science

Status: Enrolling

Conditions: Cancer

Treatments: Other: Statement communicating uncertainty with an unvalidated surrogate endpoint; Other: Statement communicating uncertainty with a single arm trial; Other: Statement communicating uncertainty with treatment effect size; Other: Statement communicating uncertainty with a limited study population; Other: Statement communicating uncertainty with limited study duration

Study type: Interventional

Funder types: Other

Identifiers

NCT06997185

540788

Details and patient eligibility

About

This nationally representative randomized survey of US adults will evaluate the effect of using brief statements to communicate different sources of uncertainty about the benefits and harms of new cancer drugs on participants' decisions and understanding.

Full description

Many newer cancer drugs are approved before uncertainties with their underlying clinical trial evidence have been adequately studied, in turn making it difficult to accurately determine the drug's benefits and harms. Prescription drug information rarely communicates these uncertainties. In a nationally representative sample of US adults, this study will evaluate the effect of using brief statements to communicate different sources of uncertainty about the benefits and harms of new cancer drugs on participants' decisions and understanding.

In the pre-intervention phase, participants will be given information about a hypothetical new drug approved for the treatment of non-small cell lung cancer. Participants will be asked how likely they would be to take the drug, and how certain they are that the drug will work. Participants will then be randomized to 1 of 5 statements about a source of uncertainty with the drug's evidence that were based on the most common sources of uncertainties with new cancer drugs that are cited in FDA approval decisions: (1) single-arm trial designs, (2) limited study populations (i.e., generalizability of clinical trial evidence), and (3) limited study durations (i.e., long-term benefits and harms). Two additional uncertainties were also included that are frequently mentioned in the scientific literature: (4) the use of unvalidated surrogate endpoints to support new cancer drug approvals, and (5) uncertain treatment effect size (i.e., the magnitude of therapeutic benefit). The post-intervention questions will re-assess participants' decision making and perceptions of uncertainty, as well as their understanding of the uncertainty communicated in the statement.

Enrollment: 3,000 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Adults 18 years of age and older
• Adults fluent in English
• Adults residing in the United States

Exclusion criteria

• Participants who do not meet each of the 3 inclusion criteria

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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3,000 participants in 5 patient groups

Single arm trial

Experimental group

Treatment:

Other: Statement communicating uncertainty with a single arm trial

Limited study duration (long-term benefits and harms)

Experimental group

Treatment:

Other: Statement communicating uncertainty with limited study duration

Limited study population (generalizability)

Experimental group

Treatment:

Other: Statement communicating uncertainty with a limited study population

Unvalidated surrogate endpoint

Experimental group

Treatment:

Other: Statement communicating uncertainty with an unvalidated surrogate endpoint

Treatment effect size (magnitude of benefit)

Experimental group

Treatment:

Other: Statement communicating uncertainty with treatment effect size