Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.

For adult patients with a variety of advanced cancers (e.g. non-small-cell lung cancer, bladder cancer, breast cancer, and ovarian cancer) what are the comparative risks and benefits of using maintenance?

Does your question include a comparison of different health care approaches? (If no, your topic will still be considered.)

yes

If yes, explain the specific technologies, devices, drugs, or interventions you would like to see compared:

Drugs with promising pharmacological profiles that appear effective against advanced cancers may be administered as maintenance therapy or may be administered at the time of disease progression. The trade-off between these approaches is a guaranteed and timely exposure to the drug prior to disease progression versus the opportunity for a drug free interval prior to progression.

What patients or group(s) of patients does your question apply to? (Please include specific details such as age range, gender, coexisting diagnoses, and indications for therapy.)

This question applies to a variety of advanced cancers observed in patients of all ages and in both men and women. While in some cases maintenance therapy may be initiated as part of an effort for cure or long lasting remissions, current interest is in applying this treatment regimen to patients with a poor prognosis.

Are there subgroups of patients that your question might apply to? (For example, an ethnic group, stage or severity of a disease.)

The concept of maintenance therapy in solid tumors applies largely to patients who have advanced disease.

Describe the health-related benefits you are interested in. (For example, improvements in patient symptoms or problems from treatment or diagnosis.)

The most important health-related benefit is prolonged survival without detriment to the quality of life.

Describe any health-related risks, side effects, or harms that you are concerned about.

Significant toxicities are associated with the use of both standard chemotherapy and targeted molecular therapy. These are broad ranging and include systemic signs and symptoms, idiosyncratic local manifestations, and secondary effects due to damaging physical or immunological consequences of some of the therapies involved. Use of maintenance therapy only makes sense if these risks, side effects and harms are outweighed by improvements in survival accompanied by reasonable quality of prolonged life.

Appropriateness for EHC Program

Does your question include a health care drug, intervention, device, or technology available (or likely to be available) in the U.S.?

yes

Which priority area(s) and population(s) does this topic apply to? (check all that apply)

EHC Priority Conditions (updated in 2008)

• Cancer

AHRQ Priority Populations

Federal Health Care Program

• Medicaid
• Medicare

Importance

Describe why this topic is important.

Randomized clinical trials involving maintenance therapy for advanced cancers have been reported in more than 20 different types of cancer and the use of this treatment approach has become quite widespread. Part of the recent increase has been driven by the use of molecular targeted drugs or chemotherapeutic drugs which exhibit more favorable toxicity profiles. Use of these therapies has the potential to benefit or to harm large numbers of vulnerable patients. Inappropriate use is likely to create false assurance of benefit, may be a detriment to the quality of life, and may increase health care costs with no corresponding benefit.

What specifically motivated you to ask this question? (For example, you are developing a clinical guideline, working with a policy with large uncertainty about the appropriate approach, costly intervention, new research you have read, items in the media you may have seen, a clinical practice dilemma you know of, etc.)

The Technology Evaluation Center has been working on a report on the use of maintenance therapy for non-small-cell lung cancer (NSCLC). However, this is only one of the many potential indications for maintenance therapy. This topic addressed by AHRQ could be broad enough in scope to address many indications in maintenance therapy for the solid tumors that are emerging. Some emerging indications for maintenance therapy are in breast, bladder, and ovary.

Does your question represent uncertainty for clinicians and/or policy-makers? (For example, variations in clinical care, controversy in what constitutes appropriate clinical care, or a policy decision.)

yes

If yes, please explain:

The literature suggests a growing interest in maintenance therapy but the benefits and risks remain to be clearly delineated.

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?

Resolution of the research question will provide information of value to multiple stakeholders including researchers and research funders, third party payers, health care providers and patients.

Describe the timeframe in which an answer to your question is needed.

This is an area of intense current interest. More than half a dozen abstracts were presented at ASCO 2010 including studies of cancers of the lung, breast, and ovary. The answer to this question would be timely if provided in the coming year.

Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

While advanced cancers can affect individuals from all walks of life, there is a disproportionate burden of many of these in the elderly. Patients without access to centers of oncology excellence and underinsured patients will be particularly affected by lack of clear information about this health care issue.

Nominator Information

Other Information About You: (optional)

Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)

Resolution of the research question will provide information of value to multiple stakeholders including researchers and research funders, third party payers, health care providers and patients.

Are you making a suggestion as an individual or on behalf of an organization?

Organization

Please tell us how you heard about the Effective Health Care Program

BCBSA TEC is an evidence based practice center; In addition, Blue Cross Blue Shield plans make use of EHC reports