Followup
DATE: May 8, 1998
TO: Interested Parties
FROM: Stephen D. Nightingale, M.D., Executive Secretary
Advisory Committee on Blood Safety and Availability
SUBJECT: Recommendations of Advisory Committee on April 28, 1998 - Follow-up 8/3/00
I. RECOMMENDATIONS FOR THE SHORT TERM
1. The Food and Drug Administration, the International
Plasma Producers Industry Association, and individual manufacturers and distributors
of plasma derivatives and their recombinant analogs should, on a monthly basis,
collect and disseminate standardized information on production, distribution,
and demand for intravenous immunoglobulin, clotting factors (recombinant and
plasma-derived), and alpha-1 antitrypsin.
Monthly reports distributed by IPPIA (now
PPTA) since 10/21/98.
2. The Department of Health and Human Services
should explore, in collaboration with industry, health care providers, and
appropriate consumer groups, methods to optimize and standardize allocation
of available products in an equitable manner, including management of emergency
supplies and programs that distribute products directly from manufacturers
to registered consumers.
Emergency allocation (for IVIG) and direct
distributin (for alpha-1 antitrypsin) programs have been established through
direct collaboration between consumers and industry.
3. Industry should discuss triage of specific
plasma derivatives to specific patient groups with the Food and Drug Administration,
the Federal Trade Commission, health care providers, and appropriate consumer
groups in order to promote accountability to the public for these practices.
Most triage programs are at local level
(e.g., hospital formulary); FDA "Dear Doctor" letter January 1998, followup
in JAMA 1999 (Nov 3);282:1613; ongoing industry discussions (including Infectious
Disease Society of America meeting on Antibiotic Shortages 10/3/00).
4. Industry should explore with the Food and
Drug Administration the possibility of importing additional supplies of intravenous
and intramuscular immunoglobulin preparations.
Extensive discussions ongoing; FDA workshop
on Clinical Comparability Studies for Plasma Derivatives scheduled for October
11-12, 2000.
5. Industry should explore with the Food and
Drug Administration strategies for reallocating partially processed plasma
materials from one manufacturer to another in order to optimize production
of alpha-1 antitrypsin and other plasma derivatives.
This has occurred.
6. Industry should explore with the Food and
Drug Administration labeling and disclosure strategies which would increase
product availability without compromising public safety and trust.
Change in policy regarding classic CJD
(Guidance for Industry: Revised Precautionary Measures to Reduce the Possible
Risk of Transmission of Creutzfeldt-Jakob Disease (CJD) and new Variant Creutzfeldt-Jakob
Disease (nvCJD) by Blood and Blood Products - November 23, 1999) resolved the
issue this recommendation addressed.
7. Industry and government should explore the
impact of a temporary decrease in exportation of plasma derivatives while
they are in short supply in the United States.
Recommendation received by Department.
II. RECOMMENDATIONS FOR THE LONG TERM
1. Every effort should be made to make recombinant
clotting factors available to all who would benefit from them, and all barriers
to conversion from human to recombinant clotting factors should be removed.
This appears to have been accomplished.
2. The National Institutes of Health should
convene a Consensus Conference on the use of recombinant clotting factors
for patients with bleeding disorders.
Referred back to Advisory Committee, which
considered the issue on August 27 and 28, 1998.
3. Industry should explore strategies for the
development of reserve supplies of plasma derivatives and for their allocation
during shortages.
Accomplished as noted under I 2. and I
3. above.
4. The National Institutes of Health and industry
should immediately evaluate alternative dosage schedules and alternative delivery
systems for alpha-1 antitrypsin therapy, including prophylaxis strategies
and strategies for treatment during acute exacerbations of disease, and accelerate
the development of gene-based products and gene-directed therapies for alpha-1
antitrypsin deficiency.
Dose schedule trials have been limited
by availability of product; investigation of alternate delivery systems continues,
as does investigation of gene-based therapies.
5. The National Institutes of Health and industry
should support the continued evaluation of the use and appropriate dose of
intravenous immunoglobulins for indications where its benefit requires further
delineation, and the results of these evaluations should be rapidly disseminated
to the public.
Dose schedule and new indication trials
have been limited by availability of product; FDA workshop on Clinical Comparability
Studies for Plasma Derivatives scheduled for October 11-12, 2000.
6. Industry should work with the Food and Drug
Administration to expand capacity sufficiently to meet anticipated demand
for plasma derivatives.
Industry members have announced plans
to expand capacity.
7. Industry and government should jointly explore
the antitrust implications of efforts to share data in order to prevent shortages.
Done. Major concern is in projection of
demand.
SDN August 3, 2000
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