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Effective Health Care Program

Topic Suggestion Description

Date submitted: June 17, 2009

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

For patients with PKU, there is now a pharmaceutical drug to treat this condition. In the past is was treated by medical foods that eliminated the harmful substance phenylalanine. The cost of the new drug is $70K year. The cost to treat someone with medical foods is $7K per year. This drug will be reimbursed at a cost that is 10 times that of medical food. Why?

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


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

The cost and effectiveness of treating PKU patients with a pharmaceutical drug vs the cost and effectiveness of treating PKU patients with medical foods.

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.)

Ages 0 and up. Coexisting diagnosis would be pregnant women with PKU using the drug vs pregnant women using medical foods.

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

Those with the disease PKU.

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

This is a life long disease patients do not improve, they are able to maintain a healthy life without mental retardation. But they can maintain that healthy life in a cost effective manner on medical foods.

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

All drugs have side effects. Medical foods do not.

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.?


Which priority area(s) and population(s) does this topic apply to? (check all that apply)
EHC Priority Conditions (updated in 2008)
  • Pregnancy, including preterm birth
AHRQ Priority Populations
  • Women
  • Children
  • Individuals with special health care needs, including individuals with disabilities or who need chronic care or end-of-life health care
Federal Health Care Program
  • Medicaid
  • State Children's Health Insurance Program (SCHIP)


Describe why this topic is important.

Because as a country we do not have infinite dollars for healthcare. We should be making wise cost effective decisions about healthcare. We should not automatically gravitate toward pharma if there are cost effective substitutes.

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 cost of the two alternatives. Pharma vs. medical food (these are not supplements but rather foods designed to treat a specific disease approved by the FDA as able to make a medical claim).

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.)


If yes, please explain:

Potential Impact

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

It will prove that one treatment option is as effective as the other at lower cost.

Describe the timeframe in which an answer to your question is needed.
Describe any health disparities, inequities, or impact on vulnerable populations your question applies to.

The cost of this drug would make it prohibited in the Medicaid population. Therefore we would have a two-tier system of healthcare with only those with medical insurance able to have the drug.

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)

It will prove that one treatment option is as effective as the other at lower cost.

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


Please tell us how you heard about the Effective Health Care Program
Adjuvant Treatment for Phenylketonuria (PKU)