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

What is the evidence for use of "diagnostic image" determined progression free survival as compared to "symptom based" progression free survival or other outcome based measures, as a surrogate for use in determining effectiveness of interventions in the treatment of malignancies?

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:

Diagnostic imaging determination of progression free survival versus symptom based and overal survival as a determiner for effectiveness of a treatment intervention.

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.)

Broad ages and both genders but specifically individuals with a diagnosis of malignancy and a treatmentintervention being undertaken.

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

The question is not about a particular subgroup, but I would hope the review would explore if there is sound clinical information related to use of image based disease free progression as a substitute for functional outcomes/overall survivalfor any particular subgroup or for any particular malignancy.

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

The benefit would be to understand what evidence exists regarding the use of image based disease free survival in determining effectiveness of therapies for malignancies - with effectiveness defined by patient symptom improvement or overall survival (rather than by the diagnostic image alone).

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

The risk is that in using image based disease free progression as an intermediate outcome for overall effectiveness, clinicians may use biologic and pharmacologic interventions with their associated toxicities and cost in situations where neither symptom improvement nor overall survival is actually affected by the intervention.

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

• Minority groups
• Women
• Children
• Elderly

Federal Health Care Program

• Medicaid
• Medicare

Importance

Describe why this topic is important.

Recently significant recommendations are being made and FDA approvals granted for biologic and pharmacologic therapies based on diagnostic image based "disease free progression." This is despite the fact that these treatments are frequently for diseases with relatively brief overall survival rates and a lack of evidence that these interventions improve overall survival, or for that matter other functional outcomes. It is important to understand what is known about when image based disease free progression is a useful surrogate for sysmptoms, functional status, and overall survival.

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.)

My organization frequently is reviewing literature that cites image based disease free progression as the primary outcome and it remains unclear to us when is this of clinical significance.

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 issue of under what situations to use image based disease free progression to make clinical treatement and medical policy recommendations remains a major source of debate at clinical meetings, in CMS determinations, and in payor medical policies.

Potential Impact

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

By better understanding what is known about image based disease free progression as a surrogate for clinical outcomes for a wide array of malignancies, better care may be rendered to patients and appropriate coverage policies may be developed.

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

As image based pisease free progression is being cited frequently as a surrogate for clinical outcomes for an array of treatments and for an array of cancers, the sooner this is addressed, the better.

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

This question applies to all individuals with malignancies, but as medical costs escalate and with that disparities among sub groups have been shown to increase, identifying appropriate end points for determining the usefulness of high cost therapeutics is potentially an important issue in phelping reduce disparities.

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)

By better understanding what is known about image based disease free progression as a surrogate for clinical outcomes for a wide array of malignancies, better care may be rendered to patients and appropriate coverage policies may be developed.

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

I am a member of the stakeholder group