Rettig RA, Jacobson PD, Farquhar CM, Aubry WM; AcademyHealth. Meeting (2004 : San Diego, Calif.).
Abstr AcademyHealth Meet. 2004; 21: abstract no. 1621.
RAND, DC/RAND Health, 1200 South Hayes Street, Arlington, VA 22202-5050 Tel. (703) 413-1100 x5299 Fax (703) 414-4717
RESEARCH OBJECTIVE: To tell the story of high-dose chemotherapy with autologous bone marrow transplantation (HDC/ABMT) for treating metastatic and high-risk breast cancer; and to draw lessons for the future. STUDY DESIGN: Multiple methodologies of interviews, legal case analysis, analysis of HCUP inpatient data, document analysis. POPULATION STUDIED: the participants in the HDC/ABMT story women patients, physicians, patient advocates, lawyers, judges, juries, insurers, state legislatures, technology assessors, clinical trialists, and the press. PRINCIPAL FINDINGS: HDC/ABMT, as a medical procedure, was not required to be evaluated by a Phase 3 randomized clinical trial (RCT), as FDA requires of new drugs. Some in the medical profession, some insurers, and the National Cancer Institute, supported RCTs. Others in oncology legitimated the procedures use on the basis of Phase 2 studies. Physician legitimation, patient demands, patient advocacy, litigation, state legislative mandates, drove utilization of the procedure concurrent with its evaluation by RCTs. Accrual of patients to clinical trials was severely hampered by the availability of the procedure. CONCLUSIONS: The basic conflict in the HDC/ABMT experience is between the need to listen and respond to women with a terminal diagnosis who wish access to an experimental treatment and, on the other hand, the need to protect and maintain the integrity of the evidence-based evaluation process. Some institutions (courts, plaintiffs attorneys, the press, patient advocates) respond well to individual patients; others (insurers, technology assessors, clinical trialists) respond well to the need for population-based data on effectiveness; a few (independent medical review, providers of information for patients, and some patient advocates) attempt to respond to both. IMPLICATIONS FOR POLICY, DELIVERY OR PRACTICE: Effective policy for dealing with new medical procedures must devise transparent ways to respond to both of the conflicting values of patient demands for access to experimental treatment AND to the need to protect & maintain the evaluation process. At the emergence of a new procedure, NCI, the medical profession, health insurers, and patient advocates should provide clear information about known risks and benefits to prospective patients for the treatment in question on a far more collaborative basis than in the past. Financing of Phase 3 trials of procedures should receive sustained attention. Educational efforts about clinical trials should be directed to patients, advocates, judges, and the media. Technology assessments should be more available to the public. The response to demands for early access to experimental procedures must be active and evidence-based, coupled with independent medical review.
Publication Types:
Keywords:
- Bone Marrow Transplantation
- Breast Neoplasms
- Carcinoma
- Clinical Trials as Topic
- Clinical Trials, Phase III as Topic
- Delivery, Obstetric
- Female
- Forecasting
- Humans
- Insurance Carriers
- Longitudinal Studies
- Technology Assessment, Biomedical
- Transplantation, Autologous
- drug therapy
- economics
- methods
- surgery
- therapy
- transplantation
- hsrmtgs
UI: 103624655
From Meeting Abstracts