The Oncologist, Vol. 3, No. 5, 354-356,
October 1998
© 1998 AlphaMed Press
Research and Quality Medicine in the Community: The Paradigm of Marrow Transplantation
Roy B. Jones
University of Colorado Health Sciences Center, Bone Marrow Transplant Program, Denver, Colorado, USA
Correspondence:
Roy B. Jones, Ph.D., M.D., University of Colorado Health Sciences Center, Bone Marrow Transplant Program, Campus Box B190, 4200 East Ninth Avenue, Denver, Colorado 80262, USA. Telephone: 303-372-9000; Fax: 303-372-9003; e-mail: bmtucu{at}entente.uhcolorado.edu
Key Words. Marrow transplant • Peripheral blood progenitor cells • High-dose chemotherapy • Autologous transplant
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Introduction
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Bone marrow transplantation (BMT), including the use of peripheral blood progenitor cells (PBPC), is a promising new technology for cancer treatment. BMT is defined here as the use of hematopoietic progenitor cells to support intensive cancer treatment. When obtained from the peripheral blood, these cells are called PBPC. Randomized trials suggest superiority of autologous BMT compared to conventional chemotherapy for patients with relapsed, responsive intermediate grade lymphoma or responding myeloma. Large phase II studies suggest benefit for selected patients with metastatic and primary breast cancer. These and additional data suggest an emerging role for BMT in cancer treatment. Counterbalancing these encouraging data, however, are the high cost and morbidity of BMT treatments. Rapid changes in technology also make it likely that major alterations in treatment delivery and cost improvements will occur within the next few years. How should society decide when this and other expensive and risky high-technology treatment should be performed in the community?
The critical concepts which underlie this decision are quality and research.
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Quality
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BMT is a complex and risky procedure. Expected mortality rates vary from <5% (autotransplant for primary breast cancer in a good risk patient) to 10%-25% (phase I trials of new combination therapy). Incremental benefit compared to standard therapy covers the same range based upon both patient and disease variables. The heterogeneity of these procedures exceeds by orders of magnitude that of typical solid organ transplantation. Even the progenitor cell source is variable and includes marrow, peripheral blood stem cells, and umbilical cord blood.
Only recently has nationwide accreditation of BMT programs begun after a several-year process of standards development. This accreditation process of necessity includes not only marrow-PBPC procurement standards and laboratory processing standards, but clinical performance and outcome standards, as well. The accrediting agency, the Foundation for the Accreditation of Hematopoietic Cell Therapy (FAHCT) through its publication, Standards for Hematopoietic Progenitor Cell Collection, Processing, and Transplantation (1996), recognize that BMT procedures vary widely in complexity and that certain centers will only be competent to perform a limited subset of these procedures. The FAHCT accreditation program was designed to have inspections performed by experts in the field in order to be sensitive to the requirements of quality assurance in an area undergoing rapid evolution. The FDA has clearly announced their intention to regulate this field. If FAHCT is unsuccessful in establishing a system of self-policing to assure quality, it is clear that the FDA will take primary responsibility for the inspection process, thus placing innovation and research in this field at risk.
FAHCT approval, however, is in reality limited to confirmation of an ability to perform standard BMT procedures. While a definition of these procedures is controversial, they will be identified in this review as reproducing treatments verified by randomized trial to produce superior relapse-free survival to conventional treatments, and to involve minimal progenitor cell manipulation.
It will require perhaps two years to complete the initial inspections of applicant programs. Centers which are accredited will have demonstrated that the personnel, resources, and knowledge base required to perform the procedure exist at the center.
While it has been suggested that certain more complex treatment regimens should be reserved for academic centers, it is likely that complex patient treatment programs will be easier for sophisticated community centers to manage than complex graft manipulation. In any case, the definition of a method as research-based is a more appropriate indicator of whether a method should be reserved for approved research centers.
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Research
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Non-standard BMT procedures should always be the subject of research. It is critical that these treatments not only offer the patient benefit but also offer benefit to society through the acquisition and reporting of data about their use. In a substantial majority of BMT procedures, non-standard treatments offer the patient the maximal hope of benefit. This is because most standard BMT procedures do not offer a probability (>50% likelihood) of cure. It is thus clear that the majority of BMT procedures are best performed in the research environment.
Historically, medical research has been conducted almost exclusively at academic centers. In recent years, sophisticated community centers have participated in government and industry-sponsored research. While these efforts indicate that well-conducted research is possible at community centers, a demonstrated track record of individual center research productivity and data submission/publication is critical. For example, membership in a cancer cooperative group or an ad hoc industrial research consortium without evidence of ongoing patient enrollment and quality data submission to a central reviewing authority is insufficient to demonstrate research competence. The same principles apply to academic centers as well.
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Financial Considerations
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Historically, reimbursement of facilities and physicians for procedure-oriented services has been superior to that paid for cognitive services. BMT is an example. Thus, many hospitals have developed BMT programs to enhance their revenue. This trend, similar to high-technology cardiac procedures which preceded BMT, creates artificial incentives to move non-standard BMT procedures from the research arena to routine patient care before cost-benefit is clearly demonstrated.
Any solution which addresses the problems outlined above must:- Increase research productivity.
- Improve access of patients to maximally beneficial treatments.
- Ensure quality.
- Speed the development of maximally cost-effective BMT treatments.
- Remove any prohibitions on insurance reimbursement for ethically conducted clinical BMT research.
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How Can These Objectives Be Accomplished?- Limit any reimbursement for non-standard BMT procedures to institutions and physicians who participate in approved research programs, and require periodic audits of institutional quality (FAHCT accreditation) and research participation. Non-participation or non-compliance would void approval to receive reimbursement of any type for non-standard BMT procedures. Research participation explicitly includes reporting of outcome data to a central authority. Develop uniform informatics and electronic database reporting methods whose design would be provided to participating institutions.
- A federally established advisory panel would accredit research programs, which must demonstrate both quality and research productivity. The advisory panel will be composed of representative interested parties including patients, physicians expert in BMT, personnel from the National Institutes of Health (NIH), insurers, biotechnology company representatives, and others as appropriate.
- Require insurance reimbursement for appropriate research studies. Institutions should bill the insurer at a rate no greater than 70% of standard patient care charges. Physician professional fees would be paid in the form of a yearly grant by an insurer consortium. Payment would be at a level of effort compatible with the number and complexity of patients studied. This would be done in the same manner as NIH grants, using a nationwide salary cap as the basis for physician payments.
- Guarantee access to disadvantaged patients (geographically isolated, handicapped, etc.) by providing stipends and travel support to ensure access to research studies.
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Conclusion
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For BMT to provide maximal benefit to patients in the future, research participation must be markedly enhanced, information retrieval made more comprehensive, costs of research decreased, and the quality of treatment and research improved. Only through participation of all involved parties and the development of a comprehensive solution will these goals be realized. The United States is unique amongst all worldwide health systems in its lack of planned utilization of regional "centers of excellence" to guarantee access to non-standard treatments while nurturing research. It is clear that healthcare funding constraints require that we adopt a more rational process for BMT research in the future. Community centers which demonstrate by FAHCT accreditation that they provide quality BMT services should be allowed to perform standard BMT procedures. Those few community centers with demonstrated research expertise could and should participate in non-standard BMT research programs.
accepted for publication August 24, 1998.
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Introduction
Quality
