Ezekiel J. Emanuel

We analyzed data from Medicare and the American Hospital Association Annual Survey to compare characteristics and baseline performance among hospitals in Medicare’s voluntary (Bundled Payments for Care Improvement initiative, or BPCI) and mandatory (Comprehensive Care for Joint Replacement Model, or CJR) joint replacement bundled payment programs. BPCI hospitals had higher mean patient volume and were larger and more teaching intensive than were CJR hospitals, but the two groups had similar risk exposure and baseline episode quality and cost. BPCI hospitals also had higher cost attributable to institutional postacute care, largely driven by inpatient rehabilitation facility cost. These findings suggest that while both voluntary and mandatory approaches can play a role in engaging hospitals in bundled payment, mandatory programs can produce more robust, generalizable evidence. Either mandatory or additional targeted voluntary programs may be required to engage more hospitals in bundled payment programs.

BACKGROUND/AIMS:

Healthy volunteers in phase 1 clinical trials contribute to the development of safe drugs and other biologics and accept risks and burdens without anticipated health benefits from participation. Although emerging data have shown that healthy volunteers are influenced by risk, some still worry that financial incentives lead them to take on unreasonable risk. Yet little is known about healthy volunteers' preferences and how they make choices about enrolling in research studies.

METHODS:

We surveyed 654 healthy volunteers at the end of their participation in a phase 1 Pfizer trial in the United States, Belgium, and Singapore to examine their reported willingness to enroll in studies of different types, with various procedures, and with possible side-effects.

RESULTS:

The majority of respondents were willing to join many kinds of studies, but fewer were willing to participate in first-in-human vaccine studies or studies of psychiatric drugs than in other study types. With regard to procedures, a substantial proportion were unwilling to participate in studies that involved invasive procedures, such as a lumbar puncture (45.4%) and bone marrow biopsy (42.3%), but willing to participate in studies with less invasive procedures such as a computed tomography scan of the heart (86.8%), magnetic resonance imaging (87.4%), and skin allergy testing (86.8%). Although there was some variation by gender and region, the majority were willing to participate in studies with side-effects like pain (80%) or nausea and vomiting (64%), but only a minority were willing to join if the research drug would result in their having a one in a million chance of death (34.4%), a small chance of kidney damage (16.7%), or influence how their mind works (23.2%; Figure 4).

CONCLUSION:

Our results suggest that healthy volunteers are willing to participate in a wide range of types of phase 1 clinical trials, and express preferences for low risk and familiar studies and study procedures, preferences which are partially affected by offers of payment.

When Dr. Hortense screens her patients in Chicago for cervical dysplasia and cancer, she conducts a pelvic exam, takes a sample of cervical cells, and sends them for Pap cytology and human papilloma virus DNA co-testing. But when she conducts cervical cancer screening in Botswana, she employs a much simpler diagnostic strategy. She applies acetic acid to highlight precancerous lesions and visually inspects the cervix-a technique known as the VIA (visual inspection with acetic acid) method. She treats suspicious lesions with cryotherapy. There are multiple reasons that Dr. Hortense uses VIA in developing countries. It requires no specialized laboratory facilities or highly trained personnel. With immediate results, there is no delay in diagnosis and treatment, ensuring that patients are not lost to follow-up. Most importantly, VIA is considerably cheaper than Pap and HPV co-testing. This difference in care between Chicago and Botswana presents an ethical dilemma in global health: is it ethically acceptable to provide some patients cheaper treatments that are less effective or more toxic than the treatments other patients receive? We argue that it is ethical to consider local resource constraints when deciding what interventions to provide. The provision of cheaper, less effective health care is frequently the most effective way of promoting health and realizing the ethical values of utility, equality, and priority to the worst off.