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 comparative effectiveness of interventions for neovascular, age-related macular degeneration(AMD)?

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:

Monotherapies of laser photocoagulation, photodynamic therapy, brachytherapy, external beam radiotherapy, intravitreal VEGF inhibitor therapy, systemic VEGF inhibitor therapy and intravitreal corticosteroid therapy.

Combinations of the above interventions also need to be studied to assess the optimal treatment stragegies.

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.)

The elderly population, with an avereage age of 74 years. Most patients are aged 50 and above. Bother genders are involved, as are all ethnic groups.

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

The elderly population is especially vulnerable and unable to adapt to the ravages of neovascular AMD as well as those who experience visual loss at a younger age.

Untreated neovascular AMD patients are typically unable to drive, read, sign checks, shop, perfrom the activities of daily living or live alone. Many require long-term care in nursing facilities.

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

What is the comparative effectiveness of neovascular AMD therapies, including all visual benefits and all adverse events? The comparative effectiveness methodology should include quality-of-life evaluation and allow comparability across all ophthalmic and non-ophthalmic interventions.

The considerable financial consequences of the disease must also be addressed and carefully analyzed.

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

Intravitreal VEGF and corticosteroid injections can cause retinal detachment, cataract, glaucoma and endophthalmitis. Photodynamic therapy can cause severe skin burns and visual loss. Systemic VEGF therapy can cause hemorrhaging and thrombosis.

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)

• Depression and other mental health disorders
• Functional limitations and disability

AHRQ Priority Populations

• Elderly
• 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
• Other

Importance

Describe why this topic is important.

Two hundred thousand US citizens per year develop neovascular AMD. More will develop the disease as the population ages. The incidence of dry (atrophic) AMD is 10% for people in their 70's, 20% for those in their 80's, and 30% for those in their 90's. Ten percent of each group will progress to wet AMD.

The quality-of-life associated with untreated neovascular AMD is similar to that of a person with a Rankin Grade 4-5 stroke who is totally dependent for care and is incontinent.

Depression is present with a high 30% incidence in the neovascular AMD group.

The cost to society for an untreated neovascular AMD patient exceeds $50,000 per year. If no treatment was given, the cost to society over 12 years could exceed 700 BILLION! Knowing the most effective intervention(s) is critical to help eradicate the human burden of disease, as well as the extraordinary financial burden.

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.)

Twenty years of research in macular degeneration, comparative effectiveness, cost-effectiveness and healthcare economics.

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:

Clinicians and patients will greatly benefit from comparative effectiveness data that elucidate the most efffective treatment strategies for neovascular AMD.

An untreated neovascular AMD patient costs the US society over $50,000/year. Over a 12 year period, the total cost to the US if neovascular AMD patients were unable to be treated would be a staggering $700 BILLION. This type of cost can severely impair US economic growth.

Potential Impact

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

Information will be disseminated in a white paper by the American Academy of Ophthalmology Vitreoretinal Ophthalmic Technology Assessment Panel, of which Dr. Gary Brown is the Methodologist.

These white papers are very well respected by the clinical community and would facilitate immediate dissemination and acceptance of the preferred treatment modalities. This translational strategy from the research arena to clinical implementation is anticipated to be very effective.

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

2-3 years, especially as new data become available

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

The elderly population is exceptionally vulnerable to neovascular AMD and often unable to adapt effectively to the ravages of the untreated or ineffectively treated disease.

Untreated and ineffectively treated patients experience increased depression, increased trauma, increased overall medical costs, extraordinary caregiver costs, and increased admissions to subacute and long-term nursing facilities. Lives are utterly destroyed by the disease, while patient and family financial resources can be decimated.

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)

Information will be disseminated in a white paper by the American Academy of Ophthalmology Vitreoretinal Ophthalmic Technology Assessment Panel, of which Dr. Gary Brown is the Methodologist.

These white papers are very well respected by the clinical community and would facilitate immediate dissemination and acceptance of the preferred treatment modalities. This translational strategy from the research arena to clinical implementation is anticipated to be very effective.

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