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

More research is needed related to the viable treatment of pulmonary fibrosis. My mother died in 1971, and my brother, in 1995; treatment options have not progressed at all, except for the option of a transplant. Thousands suffer, and die each year from this disease. It seems that if a more effective treatment option could be found, that healthcare costs could be reduced. Some medications are being used in the EU, and Canada, but are not FDA approved in the US. Currently, my sister is dying from PF, and I am in the early stages. Check out the PFF Inspire support site, or the Coalition for PF, to see just how many people, and families, worldwide, are effected by this terrible disease. Doctors are unsure what approach to take with these patients, as there is no definitive protocol. It is a hodgepodge of prednisone, imuran, N-acetyl-cysteine, various inhalers, oxygen, etc. Total confusion, and frustration, for patients, family, and health professionals.

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:

Prednisone/Imuran/NAC versus Pirfenidone, versus no treatment. Effects of coexisting autoimmune disease on progression, and outcomes.

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.)

All ages, genders, worldwide, who suffer with PF. effects of underlying autoimmune disease.

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

Generally, this is an adult onset disease; no pediatric cases.

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

Improvement of patient treatments, quality of life, and potentially, decreased health expenditures, related to hospitalizations, resultant infections,oxygen usage, rehab, surgeries, caregiver expenses, in home care, and equipment needs.

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

Suffocating slowly, inadequate treatment options, inevitable death, poor prognosis.

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)

• Arthritis and nontraumatic joint disorders
• Depression and other mental health disorders
• Functional limitations and disability
• Pulmonary disease/asthma

AHRQ Priority Populations

• 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
• Medicare

Importance

Describe why this topic is important.

Thousands die yearly form this disease, with little to no treatment options. The availability, and likelihood of a transplant, is low.

With an improved treatment option, healthcare costs could be decreased.

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.)

I am suffering from IPF, family members have had this disease, and died, there is a genetic component, and I am concerned for my family, advocating for all who have this disease.

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:

No real definitive treatment guidelines exist for PF. Many issues addressed in above statements. The variable approaches to care, create more confusion, and frustrations, for patients, care providers, and families.

Successful treatment with Pirfenidone in EU, has been delayed in the USA, because of FDA non-approval. There needs to be more research for viable treatment options.

Potential Impact

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

Influence congress to support research efforts. Improve care of this patient population, and potentially decrease health care expenditures, for this group.

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

Sooner, the better!!!! Whatever it takes.

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

Addressed in above statements. There seems to be little, to no interest, in researching treatments for this deadly, devastating disease, that robs many of their lives, often at an early age. Many social, financial, and healthcare impacts.

Patients, and health providers, struggle to make any "sense" of this. Patients feel angry, frustrated, and useless, forgotten, and devalued.

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)

Influence congress to support research efforts. Improve care of this patient population, and potentially decrease health care expenditures, for this group.

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

Individual

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