Title: FDA PROPOSED DISCLOSURE RULE
1FDA PROPOSED DISCLOSURE RULE
2An Example of Complexity Gene Therapy Products
Ex Vivo Transduced CD34 Cells Expressing
GammaC-R for X-SCID
3Gene Therapy and Xenotransplantation Products
Come From Nature...
- Natura enim non imperatur, nisi parendo Nature
cannot be ordered about, except by obeying her - Novum Organum (1620), Sir Francis Bacon
- in nature there are no rewards or punishments
there are consequences. - Some Reasons Why (1881), Robert Ingersoll
- For every intended consequence, there are a
hundred mostly unknown unintended consequences
that we must address... - FDA reviewers lament
4Consequences From Biologics
- Jenner, 1796
- Prevention of smallpox, but spread of syphilis
- St. Louis, 1901
- Diphtheria antitoxin, children died with tetanus
- Salk, 1950s
- First successful killed vaccine, large scale
manufacturing failure
5Consequences From Biologics
- RSV Vaccine, 1960s
- Killed vaccine, vaccinated died
- U Penn, 1999
- 18 year old patient dies in experimental gene
therapy trial - Rotavirus Vaccine, 2000
- Prevention of infantile diarrhea, but causes
intussuception
6Proposed Rule on Public Disclosure
- Availability for Public Disclosure and Submission
to FDA for Public Disclosure of Certain Data and
Information Related to Human Gene Therapy or
Xenotransplantation - Published for comment 1/18/01 (66 FR 4688-4706
docket number 00N-0989)
7SCOPE and PURPORSE
- Gene Therapy and Xenotransplantation
- Unique areas of clinical research with potential
for unique public health risks and modification
of the human genome - Complexity of products
- Provide consistent amount of information for
public discussion and public access - Enhance patient awareness and consumer protection
- Ensure accurate up to date informed consent
- Allow FDA to fully participate in public
discussions
8WHAT IS NOT DISCLOSABLE
- Patient information
- Trade secret information (must be productive
process, not idea) - Limited amount of commercial confidential
information, based on experience with RAC, DHHS
Xeno Group and Xeno Subcommittee
9WHAT IS DISCLOSABLE, 601.52
- (1) Product and patient safety data and related
information. - (2) The name and address of the sponsor.
- (3) The clinical indications to be studied.
- (4) A protocol for each planned study...
- (5) Written informed consent form(s) as provided
in 50.27 of this chapter.
10WHAT IS DISCLOSABLE, 601.52
- (6) Identification of the biological product(s)
- (7) IND safety reports, as provided in 312.32...
- (8) Information submitted in the annual report...
- (9) The regulatory status of the IND, such as on
hold, in effect, inactive, or withdrawn... - (10) Other relevant data and information that the
Director, CBER, determines are necessary for the
appropriate consideration of the public health
and scientific issues, including relevant ethical
issues, raised by human gene therapy or
xenotransplantation.
11HOW WILL THIS INFORMATION BE DISCLOSED? 601.53
- Official and redacted version of each submission
to an IND - Redacted document to not contain patient
information trade secrets, and certain limited
confidential commercial information - FDA Public Docket for each IND
- Redacted documents to specific docket through
life of IND
12lessons from the past enable the
futureWhat can be imagined, will be done
with hope humility compassion