Federal Register / Vol. 60, No. 33 / Friday, February 17, 1995 / Notices 9339

in the agenda will be announced at the

beginning of the open portion of a

meeting.

Any interested person who wishes to

be assured of the right to make an oral

presentation at the open public hearing

portion of a meeting shall inform the

contact person listed above, either orally

or in writing, prior to the meeting. Any

person attending the hearing who does

not in advance of the meeting request an

opportunity to speak will be allowed to

make an oral presentation at the

hearing’s conclusion, if time permits, at

the chairperson’s discretion.

The agenda, the questions to be

addressed by the committee, and a

current list of committee members will

be available at the meeting location on

the day of the meeting.

Transcripts of the open portion of the

meeting may be requested in writing

from the Freedom of Information Office

(HFI–35), Food and Drug

Administration, rm. 12A–16, 5600

Fishers Lane, Rockville, MD 20857,

approximately 15 working days after the

meeting, at a cost of 10 cents per page.

The transcript may be viewed at the

Dockets Management Branch (HFA–

305), Food and Drug Administration,

rm. 1–23, 12420 Parklawn Dr.,

Rockville, MD 20857, approximately 15

working days after the meeting, between

the hours of 9 a.m. and 4 p.m., Monday

through Friday. Summary minutes of

the open portion of the meeting may be

requested in writing from the Freedom

of Information Office (address above)

beginning approximately 90 days after

the meeting.

The Commissioner has determined for

the reasons stated that those portions of

the advisory committee meetings so

designated in this notice shall be closed.

The Federal Advisory Committee Act

(FACA) (5 U.S.C. app. 2, 10(d)), permits

such closed advisory committee

meetings in certain circumstances.

Those portions of a meeting designated

as closed, however, shall be closed for

the shortest possible time, consistent

with the intent of the cited statutes.

The FACA, as amended, provides that

a portion of a meeting may be closed

where the matter for discussion involves

a trade secret; commercial or financial

information that is privileged or

confidential; information of a personal

nature, disclosure of which would be a

clearly unwarranted invasion of

personal privacy; investigatory files

compiled for law enforcement purposes;

information the premature disclosure of

which would be likely to significantly

frustrate implementation of a proposed

agency action; and information in

certain other instances not generally

relevant to FDA matters.

Examples of portions of FDA advisory

committee meetings that ordinarily may

be closed, where necessary and in

accordance with FACA criteria, include

the review, discussion, and evaluation

of drafts of regulations or guidelines or

similar preexisting internal agency

documents, but only if their premature

disclosure is likely to significantly

frustrate implementation of proposed

agency action; review of trade secrets

and confidential commercial or

financial information submitted to the

agency; consideration of matters

involving investigatory files compiled

for law enforcement purposes; and

review of matters, such as personnel

records or individual patient records,

where disclosure would constitute a

clearly unwarranted invasion of

personal privacy.

Examples of portions of FDA advisory

committee meetings that ordinarily shall

not be closed include the review,

discussion, and evaluation of general

preclinical and clinical test protocols

and procedures for a class of drugs or

devices; consideration of labeling

requirements for a class of marketed

drugs or devices; review of data and

information on specific investigational

or marketed drugs and devices that have

previously been made public;

presentation of any other data or

information that is not exempt from

public disclosure pursuant to the FACA,

as amended; and, deliberation to

formulate advice and recommendations

to the agency on matters that do not

independently justify closing.

This notice is issued under section

10(a)(1) and (2) of the Federal Advisory

Committee Act (5 U.S.C. app. 2), and

FDA’s regulations (21 CFR part 14) on

advisory committees.

Dated: February 14, 1995.

Linda A. Suydam,

Interim Deputy Commissioner for Operations.

[FR Doc. 95–4195 Filed 2–16–95; 8:45 am]

BILLING CODE 4160–01–F

National Institutes of Health

Technology Assessment Conference

on Gaucher Disease: Current Issues in

Diagnosis and Treatment

Notice is hereby given of the NIH

Technology Assessment Conference on

‘‘Gaucher Disease: Current Issues in

Diagnosis and Treatment,’’ which will

be held February 27–March 1, 1995, in

the Masur Auditorium of the National

Institutes of Health, 9000 Rockville

Pike, Bethesda, Maryland 20892. The

conference begins at 8:30 a.m. on

February 27 and 28 and at 9 a.m. on

March 1.

Gaucher disease, the inherited

deficiency of the enzyme

glucocerebrosidase, is the most common

lysosomal storage disease and the most

frequently inherited disorder in the

Ashkenazic Jewish population. In the

past decade there has been much

progress both in our understanding of

the molecular biology of the disease and

the ability to treat Gaucher patients.

However, many issues regarding

diagnosis, population screening, and

therapy for Gaucher patients do not

have clear consensus. Gaucher disease

is characterized by a remarkable degree

of clinical heterogeneity, ranging from

severely affected infants to totally

asymptomatic adults. Patients with

Gaucher disease have been classified

into three major types on the basis of

clinical signs and symptoms: Type 1—

non-neuropathic; type 2—acute

neuropathic; and type 3—subacute

neuropathic.

All types of Gaucher disease result

from the deficiency of the same enzyme,

glucocerebrosidase, and the diagnosis

can be made by measurement of enzyme

activity obtained from a tube of blood.

The most striking difference between

the types is the presence of neurologic

manifestations and the rate of

progression. Even within the different

types there is not a unique clinical

presentation. Some patients with type 1

Gaucher disease, which is by far the

most common type, may display

anemia, low platelets, massively

enlarged livers and spleens, and

extensive skeletal disease, while others

have no symptoms and have been

recognized only during screening or

evaluation for other diseases.

The gene for glucocerebrosidase on

chromosome 1q21 has been

characterized and sequenced. Multiple

mutations have been identified in the

glucocerebrosidase gene in patients’

DNA, several of which are encountered

frequently. While some patients with

similar clinical courses share the same

genotype, there are other examples

where patients with the same DNA

mutations have very different clinical

manifestations. It is still not clear to

what extent a person’s phenotype or

prognosis can be accurately predicted

on the basis of current DNA mutation

analysis. Furthermore, while the

availability of molecular techniques has

made possible early prenatal diagnosis,

heterozygote detection and population

screening for Gaucher disease, the

advisability and usefulness of these

techniques remains unsolved.

Gaucher disease has been

traditionally managed by supportive

therapy including total and partial

splenectomy, transfusions, and
9340 Federal Register / Vol. 60, No. 33 / Friday, February 17, 1995 / Notices

orthopedic procedures. Bone marrow

transplantation has also been

successfully performed. More recently

enzyme replacement therapy has

become available using a mannose

terminated form human

glucocerebrosidase. This therapy, often

costing $100,000 to $300,000 per adult

patient annually, has effectively

improved biochemical and hematologic

manifestations of this disorder in many

patients and has reversed

hepatosplenomegaly. The optimal

dosing for this preparation is still under

investigation. Also, other novel

strategies for enzyme therapy and gene

therapy for Gaucher disease are being

actively pursued.

The purpose of this Technology

Assessment Conference is to evaluate

current concepts concerning diagnosis,

genetic counseling, and management of

Gaucher disease. The conference will

bring together epidemiologists,

geneticists, pediatricians, neurologists,

obstetricians, orthopedists,

hematologists, genetic counselors,

clinical pathologists, others involved in

health care delivery, as well as

representatives of the public to review

available data and make

recommendations regarding population

screening, genetic counseling, and

current patient management as well as

for future research.

After 1-1⁄2 days of presentations and

audience discussion, an independent,

non-Federal panel will weigh the

scientific evidence and write a draft

statement that it will present to the

audience on the third day. The

statement will address the following key

questions:

What is the natural history of Gaucher

disease and what is the appropriate

technology to assess the severity and to

predict the progression of this disorder?

What are the roles of current

molecular and enzymatic assays for

ascertaining affected individuals and

carriers in various populations?

What are the indications for treatment

of patients with Gaucher disease and

what are the appropriate modes of

therapy?

What are the goals for and

consequences of treatment and how can

the therapeutic interventions be

assessed?

Under what circumstances could

genotype/phenotype correlations be

used for patient care and counseling?

What are the appropriate directions

for future research?

The primary sponsors for this

conference are the National Institute of

Mental Health and the NIH Office of

Medical Applications of Research. The

conference is cosponsored by the

National Institute of Child Health and

Human Development, the National

Institute of Diabetes and Digestive and

Kidney Diseases, the National Institute

of Neurological Disorders and Stroke,

the National Center for Research

Resources, and the National Center for

Human Genome Research.

Advance information on the

conference program and conference

registration materials may be obtained

from: Debra DeBose, Technical

Resources International, Inc., 3202

Tower Oaks Blvd., Suite 200, Rockville,

Maryland 20852, (301) 770–3153.

The technology assessment statement

will be submitted for publication in

professional journals and other

publications. In addition, the statement

will be available beginning March 1,

1995 from the NIH Consensus Program

Information Service, P.O. Box 2577,

Kensington, Maryland 20891, phone 1–

800–NIH–OMAR (1–800–644–6627).

Dated: February 10, 1995.

Ruth L. Kirschstein,

Deputy Director, NIH.

[FR Doc. 95–3991 Filed 2–16–95; 8:45 am]

BILLING CODE 4140–01–M

National Heart, Lung, and Blood

Institute; Notice of Meeting

Pursuant to Pub. L. 92–463, notice is

hereby given of the meetings of the

following Heart, Lung, and Blood

Special Emphasis Panels.

These meetings will be open to the

public to provide concept review of

proposed contract or grant solicitations.

Individuals who plan to attend and

need special assistance, such as a sign

language interpretation or other

reasonable accommodations, should

inform the Contact Person listed below

in advance of the meeting.

Name of Panel: NHLBI SEP on Pulmonary

Vascular Biology.

Dates of Meeting: March 9, 1995.

Time of Meeting: 8:00 a.m.

Place of Meeting: National Institutes of

Health, Building 31C, Conference Room 8,

Bethesda, Maryland.

Agenda: The panel will review the current

status of research in the designated areas,

identify gaps and make recommendations

regarding opportunities and priorities for

future contract or grant solicitations.

Contact Person: Dorothy B. Gail, Ph.D.,

5333 Westbard Avenue, Room 6407,

Bethesda, Maryland 20892, (301) 549–7428.

Name of Panel: NHLBI SEP on TB/AIDS.

Dates of Meeting: March 21, 1995.

Time of Meeting: 8:30 a.m.

Place of Meeting: National Institutes of

Health, Natcher, Building 45, Conference

Room C–1, Bethesda, Maryland 20892.

Agenda: The panel will review the current

status of research in the designated areas,

identify gaps and make recommendations

regarding opportunities and priorities for

future contract or grant solicitations.

Contact Person: Hannah H. Peavy, M.D.,

5333 Westbard Avenue, Room 6A09,

Bethesda, Maryland 20892 (301) 594–7428.

Name of Panel: NHLBI SEP on Critical

Care.

Dates of Meeting: March 28, 1995.

Time of Meeting: 8:30 a.m.

Place of Meeting: National Institutes of

Health, Natcher, Building 45, Conference

Room C–1, Bethesda, Maryland 20892.

Agenda: The panel will review the current

status of research in the designated areas,

identify gaps and make recommendations

regarding opportunities and priorities for

future contract or grant solicitations.

Contact Person: Carol H. Bosken, M.D.,

5333 Westbard Avenue, Room 6A07,

Bethesda, Maryland 20892, (301) 549–7428.

(Catalog of Federal Domestic Assistance

Programs Nos. 93.837, Heart and Vascular

Diseases Research; 93.838, Lung Diseases

Research; and 93.839, Blood Diseases and

Resources Research, National Institutes of

Health.)

Dated: February 9, 1995.

Susan K. Feldman,

Committee Management Officer, NIH.

[FR Doc. 95–3988 Filed 2–16–95; 8:45 am]

BILLING CODE 4140–01–M

National Heart, Lung, and Blood

Institute; Notice of a Closed Meeting

Pursuant to Section 10(d) of the

Federal Advisory Committee Act, as

amended (5 U.S.C. Appendix 2), notice

is hereby given of the following Heart,

Lung, and Blood Special Emphasis

Panel (SEP) meeting:

Name of SEP: Demonstration and

Education Research Applications.

Date: March 14–15, 1995.

Time: 9:00 a.m.

Place: Stouffer Concourse Hotel, Arlington,

VA.

Contact Person: Dr. Louise Corman, 5333

Westbard Avenue, Room 548, Bethesda, MD

20892, (301) 594–7452.

Purpose/Agenda: To review and evaluate

grant applications.

The meeting will be closed in accordance

with the provisions set forth in sec.

552b(c)(4) and 552b(c)(6), Title 5, U.S.C.

Applications and/or proposals and the

discussions could reveal confidential trade

secrets or commercial property such as

patentable material and personal information

concerning individuals associated with the

applications and/or proposals, the disclosure

of which would constitute a clearly

unwarranted invasion of personal privacy.

(Catalog of Federal Domestic Assistance

Programs Nos. 93.837, Heart and Vascular

Diseases Research; 93.838, Lung Diseases

Research; and 93.839, Blood Diseases and

Resources Research, National Institutes of

Health.)