Patients' Decision-Making Process Regarding Participation in Phase I Oncology Research

Agrawal, Manish; Grady, Christine; Fairclough, Diane L.; Meropol, Neal J.; Maynard, Kim; Emanuel, Ezekiel J.

doi:10.1200/jco.2006.06.0269

Cited by 151 publications

(181 citation statements)

References 30 publications

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“…The biggest motivators for our patients considering early‐stage clinical trials were the chance of tumor shrinkage and the paucity of alternative treatments; these findings were similar to those of a prior report 8. This highlights the fact that patients view phase 1 studies as an extension of previous conventional anticancer treatment 17.…”

Section: Discussionsupporting

confidence: 81%

“…An alternative explanation is that this subset of patients, despite having been exposed to this information, have chosen to maintain unrealistic hope. The concept of therapeutic optimism has been reported before,8, 14, 17 and phase 1 physicians have to walk a fine line between reiterating a patient's poor prognosis, which may be seen as patronizing, and not disregarding the importance of human hope 10, 11, 14…”

Section: Discussionmentioning

confidence: 99%

“…Entry into a phase 1 study requires the judgment of the relative likelihood of benefit versus toxicity and is essentially an individual patient's decision 5, 6. This encompasses a patient's motivations, expectations, and ability to comprehend the complexities of clinical trial research 7, 8. Ethically, healthcare professionals must ensure that patients are well informed about the likelihood of risks and benefits, have a good understanding of the trial, and know the alternatives to study participation to aid the decision‐making process 9, 10, 11…”

Section: Introductionmentioning

confidence: 99%

“…Research into patients' perceptions has generally focused on participants who have already enrolled or who are considering entry into early‐phase clinical trials, that is, after discussions have already taken place 8, 12, 13, 14, 15. Patients have demonstrated unrealistically high expectations of personal benefit or even a cure from these investigational agents 15, 16, 17.…”

Section: Introductionmentioning

confidence: 99%

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A study of motivations and expectations of patients seen in phase 1 oncology clinics

Dolly

Kalaitzaki

Puglisi

et al. 2016

Cancer

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BACKGROUNDTo better inform clinical practice, this study was aimed at capturing patients' motivations for enrolling in phase 1 trials and at quantifying their expectations of the benefits, risks, and commitment associated with clinical trials and the impact of the initial consultation on their expectations.METHODSThis was a single‐center, prospective, quantitative study of newly referred adult patients considering their first phase 1 oncology trial. Participants completed questionnaires before they were seen and an abbreviated follow‐up version after their consultation.RESULTSQuestionnaires were completed by 396 (99%) and 301 (76%) before and after the clinic, respectively. Participants ranked the possibility of tumor shrinkage (84%) as the most important motivation for considering a phase 1 trial; this was followed by no alternative treatments (56%), their physician's recommendation (44%), and the fact that the research might benefit others (38%). When they were asked about the potential personal benefit, 43% predicted tumor shrinkage initially. After the consultation, this increased to 47%. Fourteen percent of patients expected a cure. When asked about risks, 71% of the participants expected moderate side effects. When asked about expectations of time commitments, a majority of patients did not anticipate weekly visits, although this was understood by 93% of patients after the consultation. Overall, patients were keen to consider trials and when asked before and after the consultation 72% and 84% were willing to enroll in studies, respectively.CONCLUSIONSThis study reports that more than 80% of patients enroll in early‐phase clinical oncology trials motivated by the potential of a clinical benefit, with approximately half expecting tumor shrinkage and approximately a tenth anticipating a cure. The typical phase 1 response rate is 4% to 20%, and this discrepancy exemplifies the challenges faced by patients and healthcare professionals during their interactions for phase 1 studies. Cancer 2016;122:3501–3508. © 2016 American Cancer Society

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Section: Discussionsupporting

confidence: 81%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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A study of motivations and expectations of patients seen in phase 1 oncology clinics

Dolly

Kalaitzaki

Puglisi

et al. 2016

Cancer

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“…Previous studies have addressed patient perceptions and expectations of treatment outcomes, potential benefits and side effects, informed consent, 6,7 and ethical aspects of phase 1 oncology trials. 8 To our knowledge, no study has prospectively assessed patients' willingness to undergo PD/PK tests, such as skin biopsy or hair follicle collection; has asked about patients' willingness to participate in frequent imaging studies, including functional imaging; or has assessed the number of tests/studies that patients would tolerate in a single trial within the context of evaluating biomarkers and the biologic effects of new agents.…”

mentioning

confidence: 99%

Patient willingness to undergo pharmacodynamic and pharmacokinetic tests in early phase oncology trials

Tibes

Piper

Smith

et al. 2011

Cancer

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BACKGROUND: Increasingly, early phase clinical trials involve pharmacodynamic (PD) and pharmacokinetic (PK) assays as well as frequent imaging studies. The authors conducted a prospective study examining patients' willingness to undergo such tests and the number of tests the patients would tolerate. METHODS: A prospective, correlative study was conducted using a self-reported questionnaire to measure patients' willingness on a scale from 1 (not willing) to 10 (very willing) to undergo various procedures (eg, tumor and skin biopsies, blood tests) and imaging studies (eg, magnetic resonance imaging, echocardiogram). In addition, correlations were assessed between the number and type of tests and demographics, previous test experience, inconvenience, and insurance coverage. Sixty-one patients (22 women and 39 men) with advanced malignancies were enrolled. Descriptive, nonparametric, and parametric inferential statistics were used. RESULTS: Overall willingness to undergo study-required tests was very high. Patients were most willing to undergo urine, blood, ultrasound, x-rays, echocardiogram, and computed tomography studies and were least willing to undergo tumor and skin biopsies and magnetic resonance imaging (all P .01). Significant inverse relations were observed between the frequency of a particular test and patient's willingness to undergo such tests. Inconvenience and prior negative experiences for more invasive tests (eg, skin biopsies) modestly affected willingness to undergo these tests again. College education, insurance coverage, and the requirement of tests for enrollment were correlated positively with willingness to undergo tests. CONCLUSIONS: The current findings provide the first prospectively collected data on patients' willingness to undergo PK/PD tests and imaging studies associated with early stage oncology drug trials and can serve as basis for further exploration toward the design of patientfriendly, biomarker-driven clinical studies in oncology. Cancer 2011;117:3276-

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Participants in Phase 1 Oncology Research Trials<subtitle>Are They Vulnerable?</subtitle>

Seidenfeld

Horstmann²,

Emanuel³

et al. 2008

Arch Intern Med

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Phase 1 oncology trials involve risk and offer a relatively low prospect of benefit to participants. Some claim that participants constitute a vulnerable population requiring special protections. We undertook this study to determine whether phase 1 oncology trial participants have demographic and health status characteristics of a vulnerable population. We reviewed participant demographic and health status data from phase 1 trials sponsored by the Cancer Therapy Evaluation Program at the National Cancer Institute that began between 1991 and 2002 and from 11 previously published studies. Main outcome measures were median age, sex, race/ethnicity, performance status, previous therapy, educational achievement level, and health insurance coverage. Almost 10 000 participants in trials sponsored by the Cancer Therapy Evaluation Program had a median age of 57 years, 90% self-identified as white, 93% had near-normal performance status, 85% had some form of health insurance, and 92% had been previously treated for cancer; 20 000 individuals from published studies had comparable profiles. The demographic and health status characteristics of phase 1 oncology trial participants are not those of a conventional vulnerable population and suggest little reason to assume that, as a group, they have a compromised ability to understand information or to make informed and voluntary decisions.

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Patients' Decision-Making Process Regarding Participation in Phase I Oncology Research

Cited by 151 publications

References 30 publications

A study of motivations and expectations of patients seen in phase 1 oncology clinics

A study of motivations and expectations of patients seen in phase 1 oncology clinics

Patient willingness to undergo pharmacodynamic and pharmacokinetic tests in early phase oncology trials

Participants in Phase 1 Oncology Research Trials<subtitle>Are They Vulnerable?</subtitle>

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