Appendix A
Background and Conduct of the Workshop

STRUCTURE OF THE WORKSHOP

This appendix describes the breast cancer workshop project and documents the materials developed or used as background for the committee discussions. The main elements of the project were background reading, information on Health Care Financing Administration (HCFA) data sets and analyses done for the workshop, and a committee homework exercise that yielded a “first round” set of patient management topics.

The workshop was conducted in three public sessions and an executive session, in addition to the previous evening’s reception and background session. The first public session featured presentations by Regina McPhillips and Henry J.Krakauer of HCFA, a brief review by Kenneth I.Shine (chair of the study committee) of the factors that the committee should keep in mind in recommending patient management topics, and a brief review of the homework exercise by Kathleen N.Lohr (of the Institute of Medicine [IOM] staff). Session II consisted of general discussion by the committee of the preliminary set of patient management topics, followed by a second round of “voting” on main patient management topics. Session III encompassed a further refining of the set of patient management issues and a discussion of main research strategies related to those issues. The executive session focused on the final recommendations the committee wished to endorse.

BACKGROUND READINGS

In preparation for the workshop, the IOM staff compiled a large set of background materials that was forwarded to the committee before the meeting. These readings were intended to establish the context of the HCFA



The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement



Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
Intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text on the opening pages of each chapter. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Do not use for reproduction, copying, pasting, or reading; exclusively for search engines.

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research Appendix A Background and Conduct of the Workshop STRUCTURE OF THE WORKSHOP This appendix describes the breast cancer workshop project and documents the materials developed or used as background for the committee discussions. The main elements of the project were background reading, information on Health Care Financing Administration (HCFA) data sets and analyses done for the workshop, and a committee homework exercise that yielded a “first round” set of patient management topics. The workshop was conducted in three public sessions and an executive session, in addition to the previous evening’s reception and background session. The first public session featured presentations by Regina McPhillips and Henry J.Krakauer of HCFA, a brief review by Kenneth I.Shine (chair of the study committee) of the factors that the committee should keep in mind in recommending patient management topics, and a brief review of the homework exercise by Kathleen N.Lohr (of the Institute of Medicine [IOM] staff). Session II consisted of general discussion by the committee of the preliminary set of patient management topics, followed by a second round of “voting” on main patient management topics. Session III encompassed a further refining of the set of patient management issues and a discussion of main research strategies related to those issues. The executive session focused on the final recommendations the committee wished to endorse. BACKGROUND READINGS In preparation for the workshop, the IOM staff compiled a large set of background materials that was forwarded to the committee before the meeting. These readings were intended to establish the context of the HCFA

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research Effectiveness Initiative and familiarize the committee with the issues to be discussed. They centered on Medicare data files, randomized controlled trials conducted by the National Cancer Institute, and recent reports of the findings of studies relating to use of services and efficacy of various treatment regimens (see the Bibliography, this volume). HCFA DATA Medicare/Medicaid Decision Support System The description of the Medicare decision support systems included a simplified version of the flow of data into the Medicare systems. That flow starts with entitlement and demographic data for about 33 million Medicare beneficiaries that are obtained initially by the Social Security Administration (SSA). Health care providers and contractors are the primary source of Medicare utilization data. Providers (e.g., institutional providers, home care agencies, suppliers, and physicians) submit bills to fiscal intermediaries (FIs, for Medicare Part A) and carriers (for Medicare Part B); they in turn adjudicate and then pay the bills and pass them into the system. These utilization data are merged with the SSA demographic information. From these main sources, several basic record groups are developed. Basic Record Groups The first record group is the Health Insurance Master (HIM) Enrollment record, developed from the SSA file; these data, which are updated daily, include dates of birth and death, sex, race, residence, dates of entitlement, and dates of enrollment into health maintenance organizations. This is a rich source of data for and identifying beneficiaries and drawing samples for follow-up research studies. The second file, the Provider of Service (POS) Record, contains considerable information on hospitals, skilled nursing facilities, home health agencies, independent laboratories, ambulatory surgical centers, and similar providers for Medicare. The third and fourth files are the Utilization Records for Medicare Parts A and B billing information, including hospital days of care, diagnoses, surgical procedures, physician visits, charges, and payments. The fifth main record group is the Provider Cost Report Record, which has cost, accounting, and other data from participating institutional providers. For effectiveness research, other “derivative” files may be important sources of information: MEDPAR (Medicare Provider Analysis and Review file), MADRS (Medicare Automated Data Retrieval System), and BMAD (Medicare Annual Data System for Part B). The SSA-based HIM file provides the beneficiary identification number and demographic information;

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research that information can be used to enter these files for more detailed utilization information. MEDPAR is a 100 percent file of Part A inpatient care (about 10 million admissions a year). Because it has person-level data with unique identifiers, it can be used to identify women who have received inpatient services related to the diagnosis of breast cancer. Among the information elements on this file are principal and secondary diagnoses and surgical procedures (ICD-9-CM [International Classification of Disease, ninth revision, clinical modification] codes), days of care, charges, and provider. This file is updated quarterly. MADRS is a newer, 100 percent file that links Part A and Part B data for all persons receiving inpatient hospital care; it presently exists for 1986 and subsequent years and is updated monthly. It allows the creation of episodes of care; Medicare-covered inpatient and outpatient care given to a beneficiary before and after a hospitalization can be identified. For this file, which contains about 250 million records per year, Part B (outpatient) data are in summary form only. The BMAD file is built on a 5 percent sample of beneficiaries and contains about 21 million records, which are updated annually. It contains somewhat more information than the MADRS file on all outpatient services for this sample, such as expenditures, place and type of service, visits, and procedures; the latter are coded using the HCFA Common Procedure Coding System (HCPCS), which is based on CPT-4 (Current Procedural Terminology, fourth version) codes. An example was offered of how the existing basic record groups might be used to conduct analyses related to breast cancer (especially to monitor trends and examine variations in use of services). First, researchers would select the ICD-9-CM code for simple mastectomy and then enter an inpatient file to extract all records for women who had services with that code. Then, because of the presence of unique beneficiary identifiers, the researchers could enter a file that contains information, for each beneficiary, on all institutional services and some summary data on outpatient care (i.e., MADRS). Third, to obtain more detailed information on physician and supplier services, researchers could then examine a file that contains considerably more detailed data on a 5 percent sample of beneficiaries (i.e., BMAD). Acquiring Additional Clinical and Outcomes Data HCFA can obtain additional clinical information (such as data on treatments administered and physiologic aspects of the disease itself) from selected inpatient medical records. One mechanism may be through the Medicare Peer Review Organizations (PROs) as part of the proposed Uniform Clinical Data Set. In 1987, HCFA’s Health Standards and Quality Bureau (HSQB) began a

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research complex project to develop a data set for use by the Medicare PROs and the wider research community; it was intended to contain far more detailed clinical data than was heretofore available in the HCFA data files. Known as the Uniform Clinical Data Set (UCDS), this project is part of a set of steps meant to expand and improve the ability of the agency to assure the quality of care delivered to Medicare beneficiaries, using the PROs as the principal mechanism. A second purpose of the UCDS is to permit the development of more and better information about what works in the practice of medicine—precisely the aim of effectiveness research. The availability of extensive clinical information collected in UCDS formats would support much more thorough and detailed analysis of patterns of interventions and of outcomes than is possible simply with billing data. Thus, for patients with particular medical conditions, such as breast cancer, a large body of information could be made available to the medical community and for intramural and extramural research. The basic operating premise of the UCDS is that relevant clinical data will be abstracted from medical records of all inpatient admissions that are reviewed by the PROs. (This currently amounts to about 20 to 25 percent of all Medicare admission in a year, or about 2.0 million to 2.5 million admissions; of these, about 3 percent are a truly random sample of admissions, and the remainder are cases mandated for review for various reasons.) PRO personnel will abstract medical records either on-site or at a central office using desktop or laptop computers. The total number of data elements available on the UCDS is about 1600, although not every data element is needed or relevant for every case. The contents of the UCDS fall into 10 major categories: Patient Identifying Information Patient History and Physical Examination and History and Physical Exam Findings Laboratory Findings Imaging Findings and Other Diagnostic Test Findings Endoscopic Procedures Operative Episodes Treatment Interventions Medication Therapy in Hospital Recovery Phase Patient Discharge Status and Discharge Planning Detailed guidelines that describe precisely the data to be acquired have been developed; for an example relating to breast cancer, see Table A.1. As of April 1989, the project was in a pilot-test phase. Field testing of the whole approach was expected to be conducted over the summer and fall

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.1 Selected Data Elements Related to Breast Cancer Recorded for the Proposed Uniform Clinical Data Set LABORATORY FINDINGS Cytology/History—Specimens taken outside of the Operating Room and specify date obtained. Cytology/Histology reports should either reveal the culture as 1= normal, 2=abnormal, benign, or 3=abnormal, malignant. Needle aspirate. Record admission value only for specimens taken in 1st 24 to 48 hours, as specified. For multiple tests, use worst value. OPERATIVE EPISODES This section refers to operating room procedures. Operative episodes are those which occur in the operating suites under anesthesia or which would generally be performed in the operating suites but were not because of emergency or patient condition. One operative episode may involve more than one operative procedure. For example: Laparotomy and Cholecystectomy and Splenectomy=1 operative episode=3 procedures=3 procedures codes.   If Operative Procedures were done, change F to T. For each operative episode, record the month and day. If the surgery occurred around midnight, record the date that anesthesia began. Date: ______________ ______________   (month) (day)   You are required to enter the procedure codes (ICD-9-CM) which apply to each operative episode. To access the computer screen to enter these codes, change F to T and enter all procedure codes which apply per operative episode. 8. Tissue Findings For each operative procedure record the pathology results of tissue removed as follows: No tissue removed F Inflammation F Infection F Normal tissue F Benign tissue change F Malignant tissue change F No pathology report: with tissue removed F The maximum number of tissue sample findings that can be recorded per operative procedure is 3.   SOURCE: “Resource Manual for Uniform Clinical Data Set (UCDS)” prepared by Case Mix Research, Queens University Department of Community Health and Epidemiology, Kingston, Ontario, Canada in association with Wisconsin Peer Review Organization (WIPRO), Madison, Wisconsin, 1988.

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research of 1989. An assessment and recommendation as to whether to go forward with this approach was expected early in 1990. HCFA is also working to develop mechanisms to collect quality-of-life and other patient outcomes data more directly. The possible use of a provider-reported functional status measure relating to cancer (the Karnofsky Index) was mentioned in connection with the Part B files. Breast Cancer Analyses Illustrating the Use of Medicare Data HCFA staff compiled an array of data tables from their analyses of Medicare files on breast cancer as well as a set of tables on acute myocardial infarction, coronary artery bypass graft, and angiography illustrative of types of analyses that might in the future be done with breast cancer data. These materials were distributed the evening before the meeting and were further elaborated in presentations at the workshop. It was acknowledged that the data were pertinent chiefly to the use of services, not to outcomes (with the exception of death and rudimentary measures of morbidity such as some complications of surgery). The first set of analyses concerned the incidence of mammography; in 1985, for instance, approximately 5 percent of nearly 18 million Medicare women received at least one mammogram. In this context, the fact that only diagnostic, and not screening, mammography was (and is currently) reimbursed is critical. As a crude indicator, HCFA presently considers paid mammography claims as diagnostic and nonpaid (rejected) claims as screening. However, before repeal of the Medicare Catastophic Coverage Act reimbursement for screening mammography was expected to be allowed as of 1990, so that this screening and diagnostic modality would then be a special target of investigation. A second set of analyses concerned temporal sequences of mammography and biopsy and mastectomy. For instance, data were presented suggesting that about 6 percent of mammographies lead to biopsy within 24 months (of the mammography), that about 4 percent lead to a mastectomy within 24 months, and that about two-thirds of women undergoing biopsy have mastectomies within 24 months. Most of these procedures were said to have been performed within 90 to 100 days of the mammography. (In the discussion, members of the IOM committee expressed a need for caution in interpreting the data in this way, because the standard clinical approach to breast cancer workup is to obtain a mammogram in conjunction with a biopsy for all patients with a palpable breast mass or symptoms.) A third set of studies on surgical treatment was based on the Part A MEDPAR files. Here the emphasis was on changes in the mid-to-late 1980s in the use of surgical interventions and types of procedures. For instance, in 1984 about 13 percent of patients admitted with a breast cancer diagnosis

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research had no procedure and nearly 60 percent had either simple or extended simple unilateral mastectomies; in contrast, in 1987 the no-procedure percentage had dropped to just over 2 percent and the combined unilateral simple-extended mastectomy figure had risen to 74 percent. The discussion brought out that the use of radiotherapy and chemotherapy does not routinely appear in these data files, so information on those forms of adjuvant therapy has to be sought elsewhere. In addition, the longitudinal data strongly indicate the shift in care to the outpatient setting; for data on these women, investigators would have to go to the 5 percent BMAD file. Outcomes of admissions for breast cancer constituted a fourth group of analyses. These focused chiefly on mortality rates for all breast cancer patients and for those with surgery (all mastectomy; unilateral simple/extended; radical; and subtotal) and on changes over time in those rates. The data suggested that postadmission deaths (at various points from the date of admission) have decreased profoundly. Data on readmissions within various time periods (30, 31 to 180, and 181 to 360 days of first discharge) were also presented; here they were viewed as an indicator of morbidity. A final set of analyses centered on risk factors, specifically on the time to death following admission for breast cancer or for resection of breast cancer as those measures related to demographic characteristics, other diagnoses suggesting coexisting chronic conditions, and type of treatment. More detailed severity-adjusted data on patients admitted for acute myocardial infarction were used as examples of how the risk-factor issue might be pursued for breast cancer. HOMEWORK EXERCISE The purpose of the homework exercise was to gain some sense of the committee’s views about high-priority breast cancer research questions to present at the outset of the meeting. It was conducted as a modified Delphi process. Before the workshop, committee members were asked to complete two questionnaire sheets. On the first, they were to list three management topics that should be accorded highest priority for effectiveness research; on the second, for each of the topics nominated, they were to specify the types of studies that should be undertaken or sponsored, keeping in mind the basic framework and scope of this approach to effectiveness research. A second round of voting conducted done during the workshop itself was reported in the main text. Table A.2 summarizes the topics nominated by committee respondents in the first round. The three areas mentioned most often were, in order of frequency, therapeutic options (sometimes as an all-inclusive term and sometimes with considerable specificity), screening (sometimes paired with prevention), and diagnosis (sometimes paired with staging). The emotional

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.2 Summary of Key Patient Management Topics: First Round of Homework Exercise Screening and prevention (4 mentions) Screening (3 mentions) Screening protocols Who should be screened Screening for breast cancer in 35- to 50-year-olds Algorithms for cost-effective selection of subjects and frequency of mammographic screening Alternative approaches to diagnosis (3 mentions) Diagnosis and staging (2 mentions) Staging of disease Staging: how long do patients survive Therapeutic approaches (6 mentions) Alternative surgical approaches/options (2 mentions) Primary treatment and follow-up Local/regional therapies for breast cancer Systemic therapy for breast cancer Role of biologic markers in selecting therapy and monitoring for reoccurrence Use of biologic variables in selecting systemic therapy Use of adjuvant chemotherapy Use of adjuvant chemotherapy even when axillary nodes are negative Management of metastatic disease Breast cancer as a chronic illness—treatment and care Rehabilitation Rehabilitative issues, use and type of prosthesis—reconstruction Emotional aspects Improved educational materials for physicians and patients Breast carcinoma education: would a standard curriculum in school be cost-effective

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research aspects of breast cancer were identified as a major feature cutting across all diagnosis and treatment issues. Finally, other topics mentioned at least once were related to rehabilitation and physician and patient education, including initiatives that might lead to earlier diagnosis and thereby reduce expenses to the Medicare program. Tables A.3 to A.8 give detailed information on issues raised by the first round of committee homework for six patient management topics: prevention and screening; diagnosis and staging; alternative treatment regimens; rehabilitation; psychological aspects of breast cancer, treatment, and recovery; and patient and physician education. The information in these tables represented the views of skilled clinicians, some of whom specialize in the care of patients with breast cancer, and experts in research and other disciplines needed for successful effectiveness research. The workshop format did not permit full discussion of the issues raised by these committee members, but the breadth of topics they recorded provides guidance for a rich research agenda for future years.

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.3 Summary of Research Activities Recommended for Prevention and Screening in Breast Cancer GENERAL PREVENTION ISSUES I. Study the Extent of Access to Prevention and Screening Modalities II. Examine the Role of Prophylactic Mastectomy in Prevention   A. Current use   B. Appropriate indications for use III. Planning for Longer-Term, More Complex Studies   A. Begin planning and undertake small-scale studies in preparation for larger-scale trials (20–30 years) to examine the potential for biochemical cancer prevention (e.g., trials employing antiestrogenic treatment such as tamoxifen to inhibit the action of estrogen on breast gland epithelium).   B. Among the issues that might be addressed are: the number of patients; the particular subsets to be studied with clinical risk factors; duration of therapy; the kind of antiestrogen; the potential for any toxicity for the antiestrogen; and how the data might be analyzed. SCREENING I. Monitor Trends in Use of Screening Techniques   A. Types of screening approaches specified     1. Physical examination by patient and/or by health care provider     2. Mammography   B. Monitor by     1. Patient sociodemographic characteristics (e.g., age, residence)     2. Patient health status characteristics     3. Patient compliance and motivation     4. Previous history (count) of mammograms     5. Provider type       a. area of expertise/specialty       b. referral patterns       c. overall screening practices for other diseases: Determine incidence of mammogram screening as a function of incidence of colorectal cancer screening by physician supply and characteristics, to answer the question: Are physicians who are educated to appropriate screening guidelines for mammography also screening appropriately for colorectal cancer (or vice versa)?   C. Frequency:     1. Determine what percentage of Medicare-eligible women obtain:       a. mammography in a given year       b. mammography in consecutive years       c. breast examination   D. Trends over time     1. Determine if the use of screening mammography correlates with the periodic recommendations of national organizations such as the Americal College of Surgeons, National Cancer Institute, and others II. Study Geographic and Regional Variations   A. Study the regional variation of age at first diagnosis as a function of

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research     mammogram screening programs in those regions to answer the question: Does mammogram lead to earlier first diagnosis?   B. Study mammogram screening incidence as a function of physician supply and specialty distribution to answer the question: Do areas with greater physician supply and specialty representation have higher or lower mammogram use?   C. Study variations in referrals for screening mammogram III. Assess the Outcomes of Screening   A. Determine outcomes in terms of pathological confirmation     1. Number of positive biopsies per total biopsies, by age group     2. Number of invasive and noninvasive carcinomas detected     3. Yield in women who undergo long-term annual screening (as opposed to short-term 5-year results of BCDDP); of particular interest: the yield of subsequent screening(s) if women are screened annually from age 50 to age 60   B. Study the yield/benefits/outcomes of routine screening across ages     1. Study HMO population(s) to determine the benefits of routine screening in terms of survival     2. Determine the frequency of surgical (biopsy) procedures following mammography and how many cancers are diagnosed, as an approach to investigating the problem of a cancer screening procedure potentially being done in a “medical vacuum”   C. Study the yield/benefits/outcomes of routine screening by age group     1. Examine role of mammography in women older than 60 and in “younger” Medicare women       a. stage of breast cancer       b. breast cancer deaths with and without mammography (stratified by frequency of mammography)     2. Examine role of mammography in 35- to 50-year olds (recognizing this is not doable with Medicare claims data but might be doable through extramural research) looking at points as noted in 1. above.     3. Conduct a controlled trial of annual screening by physical exam of breast alone versus mammography in the 65+ population to determine if a reduction in breast cancer mortality results from mammographic screening in this age group IV. Mount Studies Designed to Improve Screening Programs and Procedures   A. Examine/clarify criteria and guidelines for a screening mammography program     1. Study the relative contributions of risk factors (e.g., family history) and how they would guide a screening strategy, through either cohort studies or case-control studies     2. Determine appropriate screening intervals for various age groups and an appropriate maximal age, ideally through RCTs but possibly with computer modeling and only secondarily with case-control studies     3. Develop screening protocols that include establishing ages at which mammograms are no longer life-prolonging

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research   B. Assess who (health care provider or patient) requests mammography and how the results are processed     1. Does the patient initiate the request?     2. Which health care personnel eventually order the screening mammography?     3. Who is performing screening mammograms (i.e., hospital-based, free-standing, or dedicated mammographic unit)?     4. Who gets the results?     5. What is the frequency of subsequent screening mammography following the index procedure?   C. Develop methods to increase screening in the Medicare population that will reach patients     1. Mount a demonstration/pilot project to determine whether enclosing specific health information in Social Security checks changes behavior and increases the use of preventive breast services (mammography, breast exam)     2. Mount studies of psychological factors pertinent to screening and mammography     3. Give special attention to the more elderly and to minorities   D. Develop methods to increase screening in the Medicare population that will reach physicians     1. Study ways to increase compliance with screening mammography guidelines (this relates to several other points—reducing uncertainties about guidelines for screening, improving physician education, etc.)     2. Develop ways to provide information on the proven effectiveness of screening (disseminate information as a result of mass screening) V. Costs and Cost-Effectiveness of Screening and/or Mammography   A. Determine costs     1. Current program costs     2. Costs if all women were screened according to current ACS guidelines (and if appropriate surgical biopsies were then performed)   B. Develop algorithms/protocols to maximize the cost-effectiveness of screening based on age, family history, medical history, and outcomes     1. Extend existing work (non-HCFA) that has led to screening recommendations     2. Consider consensus conference format     3. Consider possibility of further clinical trials of breast cancer detection methods, to set standards for age (first screening, frequency), imaging techniques, number of views, and recommendations for biopsy   C. Analagous to 2: Study ways to decrease the costs of a screening mammography program     1. Study true predictive values of various mammographic features and alternative forms of biopsy

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.4 Summary of Research Activities Recommended for Diagnosis and Staging in Breast Cancer COMMENT: MANY OF THE TOPICS AND STUDIES NOTED HERE ARE LINKED VERY CLOSELY TO THOSE SUGGESTED FOR SCREENING AND PREVENTION, AND SOME POINTS LISTED ON TABLE A.3 ARE RELEVANT HERE BUT WERE NOT SPECIFICALLY MENTIONED IN THIS CONTEXT. I. General Issues Relating to Diagnosis and Staging   A. Identify elements of the diagnosis and staging workup that would be monitored and studied, using     1. Pathology data: histologic subtypes of tumor, histologic or nuclear grade of tumor, pathologic size of tumor, adequacy of surgical margins, hormone receptors, and flow cytometric data     2. Radiologic data: mammographic size of tumor, status of noninvolved breast, and “metastatic workup” including chest X-ray, CAT scan of chest and abdomen, bone scans, and other nuclear scans     3. Surgical data: type of biopsy, type of excision of primary tumor and/or mastectomy and details of axillary node sampling     4. Other clinical and health status measures not directly related to breast cancer physiologic measures   B. Examine issues (establish guidelines?) for adequacy of workup     1. What are the appropriate tests, radiologic screening approaches, etc?   C. Improve mammographic criteria for diagnosis     1. Study predictive values for specific combinations of features of lesions seen on mammography (ideally through multicenter study) and use information to develop better indicators of when patients should have biopsy or follow-up examinations   D. Clarify role of and techniques for biopsy in diagnosis     1. Adequacy of fine-needle aspiration biopsy and of excisional biopsy for palpable and nonpalpable breast lesions (RCTs of FNAB, especially for nonpalpable lesions, needed)     2. Determine variation in quality of cytology interpretation     3. Determine consistency of mammographic needle localization   E. Investigate alternative imaging methods     1. Develop better approaches for differentiating benign from malignant masses on screening radiologic exams, e.g., magnetic resonance (MR)     2. Develop and test new technologies (ultrasound tissue characterization, Doppler, monoclonal antibodies)   F. Develop approaches to improve physician interpretation of diagnostic tools (mammogram, MR, etc.)     1. Sponsor education for pathologists in breast cytopathology   G. Study the pathologic features of breast cancer as associated with     1. Prognosis     2. Infuence on primary therapy     3. Influence on adjuvant therapy recommendations

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research II. Monitor Access and Use of Diagnostic and Staging Techniques   A. Describe better the epidemiology of biopsy in diagnosing breast cancer     1. How many women have mammography before biopsy and how many have biopsy as initial procedure?     2. How many women have multiple biopsies?     3. What is distribution of biopsies as a function of physician supply?   B. Correlate the type of staging workup with recommendations by national organizations, e.g., NIH Consensus Development Conference   C. Analyze geographic, social-economic, and age-related variations III. Study Yield and Outcomes of Diagnosis and Staging   A. Correlate changes in length of hospitalization (as mandated by DRGs) and the timing and thoroughness of workup   B. Correlate results of workup and choice of:     1. Primary therapy     2. Adjuvant systemic therapy     3. Survival   C. Determine if staging data can be used to identify patients for whom heroic medical care is likely to be ineffective     1. Analyse data from registries, medical records, other supplementary sources, Part A and date of death information IV. Other Suggested Studies relating to Auxiliary Node Involvement   A. Evaluate axillary node dissection     1. Indicated in all patients?     2. Minimally adequate extent of dissection?     3. Extent of “axillary sampling” (useless term but is it done?)?     4. Relationship with tumor size? Influence on primary treatment options?   B. Evaluate (and perhaps develop) noninvasive tests to look for axillary nodal involvement or its prognostic equivalent; correlate with pathology data.

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.5 Summary of Recommended Research Activities Related to Alternative Therapeutic Approaches in Breast Cancer I. Monitor Access and Use   A. Types of services, interventions, and providers     1. By implication, all types of therapeutic interventions, but those mentioned specifically included       a. radiation therapy       b. systemic adjuvant therapy         1. chemotherapy         2. hormonal therapy       c. operative procedures generally         1. mastectomy and/or reconstruction         2. lumpectomy       d. combinations of therapies, including         1. mastectomy and postoperative radiation         2. lumpectomy and postoperative radiation         3. adjuvant chemotherapy         4. adjuvant chemotherapy and hormonal therapy     2. By implication, all types of providers, but those mentioned specifically included       a. medical oncology consultants       b. primary caregivers   B. Across time     1. With particular attention to the NCI Alert   C. By geographic areas (including small-area analysis)   D. By patient characteristics   E. By type of provider (e.g., community hospital versus teaching hospitals; ambulatory care settings)   F. By disease/tumor stage (e.g., Stage I and Stage II) and other biologic aspects of the condition   G. By supply of medical oncologists II. Study (and Compare) Outcomes Following Various Medical, Surgical, and Radiotherapy Interventions   A. Interventions warranting direct study and/or comparison (not RCTs, necessarily, but clinical demonstrations, case-control studies, etc.)     1. Surgical options alone       a. wide lumpectomy/wide excision, subtotal mastectomy, simple mastectomy, modified radical mastectomy     2. Radiotherapy in lieu of surgery     3. Conservative surgery and radiotherapy vs mastectomy     4. Adjuvant chemotherapy therapy     5. Adjuvant chemotherapy therapy with hormonal therapy     6. Systemic therapy used as substitution or as adjunctive therapy

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research     7. Use of biologic markers to select therapy, monitor for recurrence       a. types of markers: carcinoembryonic antigen (CEA), estrogen receptors (ER), progesterone receptors (PR)       b. examples of questions to be answered         1. Is CEA a reliable indicator of cancer cells in women who have received local therapy?         2. If yes, when the only sign of recurrence is elevated CEA does chemotherapy have an advantage over waiting until recurrence is symptomatic?   B. Outcomes include     1. Survival, local recurrence, time to first relapse, relapse-free survival     2. Various quality of life measures: physical health and stress; functional health, ability to care for self, etc.; psychological/emotional status and stress     3. Length of hospital stay for definitive local-regional therapy     4. Incidence of complications requiring hospitalization (treatment related), and length of hospital stay for complications     5. Longer-term complications   C. Control for various covariates, such as:     1. Age (e.g., >65 versus <65; younger versus older elderly), geographical location, SES and other relevant sociodemographic characteristics     2. General medical status and comorbidity     3. Specific aspects of breast cancer       a. stage of diagnosis (e.g., in situ carcinoma, invasive early-stage breast cancer, locally advanced breast cancer)       b. presence or absence of axillary node metastases     4. Develop better adjustors for prognosis (beyond age, sex, prior hospitization) for inpatient care and outcomes       a. determine if appropriate information on staging can be obtained from tumor registries and merged into Medicare Part A files   D. Include studies of the use of complementary/alternative therapies to minimize symptoms and to enhance quality of life, such as heat, relaxation, massage, diet, exercise, pet therapy, music III. Randomized Controlled Trials of Alternative Therapies   A. Of alternatives that appear to lead to similar outcomes   B. Of modified radical mastectomy versus local excision plus tamoxifen for Stage I and Stage II breast carcinoma in women over 70 with comparison of     1. Disease-free survival     2. Overall survival     3. Local recurrence     4. Return to preop functional status

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research   C. Of options for surgical treatment for intraductal carcinoma   D. Of adjuvant chemotherapy in women with no axillary node metastases   E. Of response rates to adjuvant and relapsed therapy according to numerous pathologic/physiologic/biologic variables, to identify appropriate therapies for different biologic subgroups of breast cancers IV. Other Specific Therapeutic Questions (Type of Study Not Specified)   A. Radiotherapy     1. Symptoms patients experience with primary radiation     2. Rehabilitation in patients who receive or do not radiotherapy     3. For primary treatment patients, should axilla be irradiated if they are not resected?     4. Indications for adjuvant radiotherapy (after mastectomy)   B. Adjuvant systemic therapy (adjuvant chemo- and/or hormonal therapy)     1. Use (percentage of Medicare recipients receiving adjuvant chemotherapy and/or hormonal therapy), by       a. node-positive or node-negative       b. estrogen receptor status       c other pathological characteristics, such as tumor size, flow cytometry, and similar markers       d. menopausal (pre/post) state       e. setting—protocol or nonprotocol       f. since 1985 consensus conference recommendation     2. Indications for selection and use of       a. hormone therapy       b. combination chemohormonal therapy       c. sequential chemohormonal therapy     3. Criteria for length of use     4. Toxicity     5. Rate of patient compliance—patients completing therapy as prescribed     6. Existing RCTs should be continued; additional ones funded V. Costs and Cost-Effectiveness Studies   A. Monitor and study distribution of total cost from diagnosis to death, by     1. Geographic region     2. Level of physician supply   B. Cost to Medicare of administration of tamoxifen to node-negative women for a minimum of 5 years per patient resulting in a 5 percent improvement in disease-free survival versus cost of treatment at time of recurrence VI. From Above Studies, Develop Clinical Indications for Selecting Therapeutic Options   A. Specification of selection criteria for main treatment decision, using     1. Factors relating to access, patient demographics (e.g., age), patient preferences, outcomes     2. Factors relating to clinical status, as listed in IV, above

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research   B. Specification of selection criteria for adjuvant therapy, using factors relating to selection of adjuvant therapy (patient demographics, pathologic and other factors relating to the cancer) VII. Other Issues Relating to Therapy   A. Management of breast cancer metastases to bone (especially weight-bearing bones)     1. Use and timing of palliative or reparative procedures   B. Linkages between diagnosis and treatment choices     1. Links to diagnosis aspiration cytology, incisional biopsy, excisional biopsy, needle localization biopsy     2. Links to pretreatment evaluation before definitive local-regional therapy-chest X-ray, bilateral mammogram, bone scan, lab, ER and PR, flow cytometry   C. Questions raised about surgical interventions     1. Indications for axillary node dissection (therapeutic or staging?)     2. What constitutes adequate excision of the tumor (for radiation patients)   D. Use of experimental techniques     1. In far-advanced disease, high-dose chemotherapy with autologous bone marrow transplantation       a. question of whether improves survival       b. costs     2. Whether new and untried investigational drugs should be made widely available widely to practitioners     3. Who should pay for experimental therapy?     4. When does a particular form of treatment cease to be experimental and thus become reimbursable by third-party payers? VIII. Other Issues Relating Mainly to Patient/Physician Factors   A. Special considerations of the patient and family with recurrent breast cancer, including     1. Symptom management and palliative care     2. Emotional and other burden on family (e.g., spouse dependency)     3. Financial burdens     4. Availability of special services (home care, respite care)   B. Physician and patient shared decision making     1. Survey representative populations (e.g., rural, urban) to determine whether surgical and/or other treatment options were discussed with the patient before treatment and whether factors of acceptability (e.g., inability to get to the hospital for daily treatment) influenced treatment choices     2. Survey representative surgeons for patterns of care to determine whether older patients are treated differently than the under-65 age group by virtue of age or functional status   C. Multidisciplinary approaches to patient management     1. Does it occur before primary therapy is carried out?     2. Who is involved (diagnostic radiologist, therapeutic radiologist, pathologist, surgeon, medical oncologist, other)?

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research     3. Who synthesizes the information and coordinates the effort?     4. Necessity of second or third opinion IX. Special Considerations of the Very Elderly Patient   A. In this context, what is the age definition of “very elderly”?   B. Prognosis and management options in older women     1. Is the natural history of breast cancer different in the older age groups?     2. Is recovery altered after primary therapy in older age groups?     3. Do the psychosocial issues differ?     4. Is breast reconstruction offered to older women?     5. Do they have adequate access to follow-up care?   C. Information and learning needs; compliance; reinforcement of self-care activities; physical abilities/disabilities; transportation   D. Possible special aspects of chemotherapy or hormone therapy     1. Organ system effect of chemotherapy     2. Tolerance (physical and psychological)     3. Dose attenuation   E. Should patients over the age of 70 years be excluded from clinical trials? TABLE A.6 Summary of Recommended Research Activities Related to Rehabilitation in Breast Cancer I. Monitor Access and Use of Prothesis and Reconstruction   A. Assess frequency of use and trends over time in use of     1. Surgical reconstruction (reconstructive plastic surgery) without prostheses     2. Prostheses II. Study Elements of Rehabilitative Care   A. Evaluate level and adequacy of information women receive before surgery including information about indications for immediate versus delayed procedures   B. Determine whether women who choose conservative surgery/radiation option receive the same information and support as those who choose mastectomy   C. Generally, evaluate the effect of different types, levels, timing of information on overall rehabilitation   D. Assess outcomes, costs, and cost-benefit (cost-effectiveness) of various options III. Study Aspects of Continuity of Care   A. Assess and monitor the coordination of care     1. How and by whom (patient, family member, nurse, physician)?   B. Evaluate adequacy of discharge planning and predischarge information     1. In ambulatory surgery centers     2. In very short hospital stays   C. Document use (or lack of it) of volunteer support services     1. According to type of health care providers

OCR for page 45
Breast Cancer: Setting Priorities for Effectiveness Research TABLE A.7 Summary of Recommended Research Activities Related to Psychological Aspects of Breast Cancer Diagnosis, Treatment, and Recovery I. Investigate the Emotional and Psychological Aspects of Breast Cancer   A. Using available data from current service-based codes, determine the prevalence of these complications of breast cancer (depression, anxiety, etc.)   B. Using chart reviews over a 12-month period, determine the prevalence of these conditions   C. Using data from A and B, determine whether these conditions are treated   D. Move to clinically based studies when patients with breast cancer are specifically assessed for these conditions   E. Mount treatment trials of interventions II. Develop Psychosocial Adjustment Indicators of Patients at High Risk for Adjustment Problems III. Determine the Ambulatory Resources for Those Patients Requiring Support TABLE A.8 Summary of Recommended Research Activities Related to Patient and Physician Education in Breast Cancer I. Physician and Patient Information Publications   A. Develop readable document with state-of-the-art information for physicians and patients on the comparative benefits of     1. Alternative primary treatments     2. Chemotherapy       a. types       b. timing     3. As a function of age, menopausal status, receptor status, lesion location, clinical stage, pathologic stage, etc. II. Breast Cancer Education in Primary and Secondary School Curriculum   A. Conduct randomly selected cohort study of educational interventions in cancer awareness among women 25 to 35, subjects to be followed for 50 years, with possibility of extending interventions to subsequent target population of schoolgirls (note: this recommended research effort was elaborated in great detail)