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CLINICAL TRIALS

Mr. Early: Will you be able to continue the clinical trials now underway at the planned levels with the FY 92 budget request? Are there any that will have to be curtailed or stretched out?

Dr. Williams: Ongoing trials and trials we plan to begin in FY 1991 will continue at the same level of effort under the FY 1992 budget levels. No ongoing trials will be curtailed.

Mr. Early: Are there any clinical trials that could be undertaken in FY 92 if additional resources were available:

Dr. Williams: Yes. If additional resources were available, clinical trials could begin on geriatric rehabilitation, malnutrition in geriatrics, growth hormone factors, pharmacological agents for Alzheimer's disease, muscle loss and cardiac function.

Mr. Early: Please provide for the record a list of approved but unfunded clinical trials, along with a brief description of the trial, its duration, the first year cost and the estimated total cost of the trial.

Dr. Williams: The following major new clinical trials would be ready for initiation in 1991 if funds were available:

STOP-IT (Sites Testing Osteoporosis Prevention/Intervention
Treatments)--Expansion

First Year Cost: $2,100,000
Estimated Total Cost: $12,290,000
Duration:

5 years Description: A set of clinical trials to test promising ways to maintain or increase bone strength in persons over 65. Companion studies on the causes of progressive bone loss in late life will be included.

Treatment of Cognitive Impairments/Behavior Disorders Associated with Alzheimer's Disease-- Expansion

First Year Cost: $1,500,000
Estimated Total Cost: $8,120,000
Duration:
Description: Testing of additional compounds aimed at
slowing, halting, or if possible reversing, the progressive
decline in cognitive function in Alzheimer's victims.

5 years

Prevention of Hypertension in Elderly

First Year Cost: $1,600,000
Estimated Total Cost: $8,660,000
Duration: 5 years
Description: This study will test the efficacy of weight loss
and sodium restriction following withdrawal of
antihypertensive medications. The significance of the
proposed study is that findings may identify a safe and
well-tolerated method by which to discontinue
antihypertensive drug therapy in elderly persons who are
currently being treated with blood pressure medications.

The following major new clinical trials would be ready for initiation in 1992 if funds were available:

Geriatric Rehabilitation

First Year Cost: $3,000,000
Estimated Total Cost: $9,360,000
Duration: 3 years
Description: There is a need for rigorous studies of hip
fracture rehabilitation strategies to decrease current
high rates of long term disability. Rehabilitation
techniques for both cognitive and motor deficits following
stroke need development and testing in older patients to
improve their outcomes. There is also a need for trials
of improved assessment techniques to detect moderate
levels of disabilities which could be alleviated by
rehabilitation.

Interventions Against Malnutrition in Geriatrics

First Year Cost: $2,000,000
Estimated Total Cost: $6,240,000
Duration: 3 years
Description: There is evidence that the number of
severely malnourished nursing home or homebound patients
far exceeds the malnourished hospital populations, but
data are lacking on the effectiveness of nutrient
supplementation or other strategies for these patients.
Many of these issues could be resolved by relatively
small-scale short term studies using available
interventions.

Systemic Effects of Chronic Treatment of Elderly with Growth
Hormone Stimulating Factors

First Year Cost: $275,000
Estimated Total Cost: $1,050,000
Duration: 4 years
Description: Preliminary studies suggest that hormone
supplementation or exercise may partially reverse changes
seen with aging in muscle mass and strength, skin
thickness, and bone density. Some older persons tend to
be both hormone deficient and sedentary. This trial will
evaluate the effects of graded exercise and replacement of
growth hormone and sex appropriate steroids in moderately
physically impaired older men and women.

Assessment of various pharmacological agents for Alzheimer's disease

First Year Cost: $500,000
Estimated Total Cost: $2,200,000
Duration: 5 years
Description: Intramural clinical trials to (a) evaluate
treatment efficacy of thyrotropin-releasing hormone; (b)
evaluate motor and cognitive abilities after
administration of haloperidol; and (c) measure performance
with a PET scan on a graded intensity test before and
after cholinergic drug administration.

Effects of Strength Training on Age- Associated Muscle Loss

First Year Cost: $400,000
Estimated Total Cost: $600,000
Duration: 2 years
Description: Significant differences exist between women
and men in muscle strength and body mass which become
greater with increasing age and may, in part, account for
the greater degree of frailty in elderly women. A program
of strength training in elderly women and men would be
conducted to identify the specific neural and endocrine
(hormone) controls of muscle function and their effects on
changes in muscle mass in adaptation to exercise.

Effect of Pacemaker Rate on Cardiac Function

First Year Cost: $175,000
Estimated Total Cost: $600,000
Duration: 3 years
Description: There is suggestive preliminary data that there
may be clinically significant changes in the circulation
of patients fitted with pacemakers because of
inappropriate setting of the pacemaker rate which may
eliminate nocturnal decline of heart rate. This trial
would evaluate the effect of pacing rate on cardiac
function in patients with demand atrial pacemakers.

Mr. Early: Doctor, what impact will the financial management plan have on your institute's ability to mount and fund clinical trials?

Dr. Williams : Implementation of our financial management plan does help ensure optimal use of all of our resources, including those for clinical trials.

RESEARCH CENTERS

Mr. Early: The budget request proposes an increase +2.5% for the centers program. What is the average increase in a center grant under the budget request?

Dr. Williams: 2.5%.

The average center award will increase by

Mr. Early:

How was the size of this increase determined?

Dr. Williams: In order to maximize funding for research project grants, a 2.5% increase was deemed appropriate for NIA's centers mechanism.

Mr. Early: What was the level of downward negotiations in the centers program in FY 89 and FY 90? And what is the estimate for FY 91?

Dr. Williams : In FY 1989 there was no downward negotiation in the centers program. In FY 1990, the level of downward negotiation was 15.2%. There will be no arbitrary downward negotiations for centers in FY 1991. The NIA will carefully examine each center grant and implement cost management measures on an individual basis.

Mr. Early: Doctor, will the NIA be able to fund noncompeting centers at commitment levels under the budget request? Competing centers at the peer reviewed levels?

Dr. Williams : Under the President's budget for FY 1992, the NIA will fund noncompeting centers slightly below commitment levels. The NIA will fund only one competing center under the current budget request; this center will be funded at less than peer reviewed level.

FINANCIAL MANAGEMENT PLAN

Mr. Early: Doctor, how does your Institute plan to administer the financial management plan with respect to research project grants? How do you plan to stay within the parameters that have been set? Research centers grants?

Dr. Williams : In 1991 beginning with the first council round, the NIA instituted cost-containment policies that eliminate the arbitrary downward reductions which were instituted in past years. Each potential competing grant is now carefully examined to determine what level of budgetary adjustments can be effected without damaging the science of the proposed project. NIA has instructed its study sections to pay special attention to proposed budgets in light of the cost-containment issue. It must be emphasized that each grant proposal is then considered individually by NIA staff, and that budgetary adjustments are made on a case-by-case-basis. Regarding non-competing awards, NIA staff ensure that future year costs budgeted for each award reflect an accepted inflationary increase of 4% from the first year adjusted cost, taking into consideration any study section recommendations concerning scientific needs for the future years of the project. Regarding the average length of award, NIA will continue to examine its grant portfolio and work to maintain our present average length of 3.9 years.

NIA study sections and staff apply the same level of scrutiny to the budgets in the centers applications as is given to the research project grant applications.

Mr. Early: Does the NIA plan to make greater use of "out of order" funding to achieve a lower cost per award?

Dr. Williams: Because of our desire to fund quality research as efficiently as possible, we have been and will continue to be watchful of quality projects that await funding. We use percentile criteria as a guide, rather than as an arbitrary cutoff for decisions on grant funding. Whether or not we make greater use of out of order funding will depend upon the quality of particular grant applications and Institute judgement of scientific need, balanced against available resources.

Mr. Early: What has been the reaction of your study sections to the financial management plan, and limiting the overall average increase in the cost of a grant?

Dr. Williams : Our study sections understand and appreciate the purpose of the plan, and take their charge seriously when

examining the proposed budgets. Cost changes for the future noncompeting years of a grant proposal are carefully examined to ensure that these changes reflect scientific need; requirements may differ from year to year depending upon the particular nature of the research.

Mr. Early: What impact will limiting the average increase in the cost per award have for your institute, particularly with respect to clinical research and clinical trials?

Dr. Williams: If individual grant budgets were to be unduly restricted, the cost of the research would have to be spread over a longer time, which would then slow the rate at which research results are obtained and also increase the duration of the studies. Limiting the increase would thus result in extended durations of studies and the potential of incurring extra costs because of limitations of economies of scale.

BIOMEDICAL RESEARCH AND DEVELOPMENT PRICE INDEX

Mr. Early: In general, Doctor, what do you see as the "down side" of using the BRDPI? What is your professional judgment on this?

Dr. Williams: The Biomedical Research and Development Price Index (BRDPI) is calculated to estimate the specific effect economic pressures have had or will have upon the theoretical, typical "market basket" that a biomedical researcher must fill in order to conduct research. Whereas the consumer price index is calculated to reflect aggregate price changes for gasoline, food, clothing, automobiles etc., the BRDPI reflects changes for items such as research chemicals, glassware, investigator salary levels, laboratory animals, etc. The BRDPI has risen faster than other measures of inflation, including the consumer price index.

While I believe that this index should be used as a guide for gauging cost increases associated with biomedical research, use of it should be individually tempered with considerations such as the specific research requirements for the outyears of a particular grant. For example, it is often the case that for applied clinical studies, significant cost increases will occur in the second year of the award because of transition into the patient accrual stage. Other grants may require the purchase of equipment in one year, and not in the other. Thus, while the use of the index is beneficial as a guide, in my professional judgement its use should not overshadow research needs and scientific judgement.

OUT OF ORDER FUNDING

Mr. Early: What is your professional judgment on out of order funding?

Dr. Williams: In my professional opinion, one has to take a balanced view of research need, opportunity, and available resources when making those difficult decisions as to which grants will receive funding, and which ones will not. Certainly, if several grants are judged to be of nearly equal scientific value in light of the NIA mission, and some of these are more costly

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