Synopsis of Conversation with Pharma/Bio Reps 29 Jan 2010
Reading Room Production, pp.450-453 · reading_room:email:00444
Page text: p.450, p.451, p.452, p.453 · original PDF
- Date
- 2010-01-30 13:57
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- email · email
- cc
- George Korch
- sender
- George Korch
Heidi, et al.:
I have summarized the conversation with the VP's and representatives from the pharmaceutical / biotechnology industries
regarding incentives promoting greater participation by industry in our public health emergency medical countermeasure
enterprise. Where possible, I identify follow-on actions that derive directly from the conversation. There are still important
conversations to continue with our commercial partners. Please feel free to modify or add information or perspectives that I
did not adequately capture. Also, I did not quote by name the discussions offered by our industrial partners.
Best regards
George
George W. Korch Jr.
Senior Science Advisor
Assistant Secretary for Preparedness and Response
Department of Health & Human Services
ble
Summary of meeting with VP's of pharmaceutical and biotechnology companies
29 January 2010
Eisenhower Executive Office Building
Dr. Zeke Emanuel opened the meeting with a problem definition statement that we as a
nation need medical countermeasures for public health emergency events ranging from
biodefense to emerging infectious diseases, but after years of investment, we still have
not been successful in meeting these needs. He indicated that to a certain degree, there is
a sense that the big pharmaceutical companies have opted out of assisting in this
endeavor, and while there are smaller, biotechnology companies attempting to fill the
gap, they lack the experience to complete the task. Ms. Heidi Avery added that this was
not to single out big pharma, but that the USG recognizes that it may not have created the
best conditions for being a good partner as well. She pointed out that the industry itself
has recently struggled with producing approved medical products for more highly valued
commercial products. The bottom line is that the President is seriously committed to this
problem and this was highlighted in his State of the Union message.
This meeting was being held therefore to start a dialogue with industrial partners (big
pharma and the biotechnology sector) to gain their perspectives on what government
actions can we take to lower the barrier to entry and to increase the attractiveness of
partnership, because without big pharma specifically, the whole thing will not work. We
need to hear their ideas.
Merck started by reminding folks that they had in fact bid on the contract award for the
cell culture derived smallpox vaccine, but had not been successful.
Merck then indicated that there are three areas of concern that must be addressed to
attract companies like theirs to the program:
1. Need to assure an adequate market
2. Need to make the regulatory path much more viable
3. Need to reduce legal liabilities (e.g. tort claims)
Markets
On the issue of adequate markets, this allows both big pharma and the biotechnology
companies to gauge issues on such things as return on investment, infrastructure and
operations costs, etc. Even if the market is destined just to be standard replacement of a
strategic stockpile, that constitutes a defined market need. NKT Therapeutics,
representing the biotechnology sector indicated that ability to define an adequate market
is a key variable for their ability as a small company to secure investment funding. (Note:
I interpret the relative order of these three items to actually be in a sort of priority order
for big pharma).
An issue that was cited as an example for how markets might be improved for these
products was the use of the Priority Review Voucher System applied to biodefense
products as is now afforded to industry for orphan products under the Orphan Drug Act.
If a system could be developed wherein the voucher was transferable or saleable to
another industry partner, it would represent a value proposition that could be
understood by the investor community as a potential opportunity for return on
investment. There was discussion regarding the theoretical vs. practical aspect of this
advantage for priority review, but agreement that another feature regarding 3 months of
granted exclusivity on review would be of value.
Regulatory Approval Pathway
On the issue of better defining the pathway to regulatory approval of the product, a
general description of the preferred process is that the FDA should be very specific in the
requirements that a company is expected to demonstrate for a candidate study, rather than
the current system where a company provides data for a study, and is then told whether it
meets the reviewers' approval. Dr. Zeke Emanuel pressed for more specificity on the
steps where this pathway could be made more transparent. This is an action item for the
industry to be further defined with more dialogue.
One concrete example of how better understanding of a path to approval would affected a
company's effectiveness in producing countermeasures was given by Novartis regarding
use of an adjuvant to increase immunogenicity of a vaccine. Adjuvants stimulate an
immune response at a lower antigen concentration, and potentially produce a broadened
response profile to similar antigens, thus increasing the manufacturing yield per unit of
production. "In general, you can have more product produced if the material is more
potent" was the phrase used to define this issue. In Europe, this pathway to use of
adjuvant through their regulatory agency permitted the use of adjuvants in influenza
vaccine, so what is the reason that the U.S. cannot seem to provide a reasonable pathway
to accomplish a similar allowance ? There is a need for harmonization of the
regulatory pathway with the EMEA (the European Medicines Agency) on issues such
as this. It would also permit global companies more opportunity for accessing larger
markets. Zeke Emanuel pointed out that the issue of adjuvants had to also account for the
perceptions of safety by the U.S. population and must be factored into the market issues
concerning this approach.
Merck added an example regarding lack of a clear understanding of the regulatory path to
have an anthrax lethal factor inhibitor approved.
•
Action item: identify what steps or processes are needed for international
harmonization in regulatory pathways, with adjuvants as an exemplar
•
Action item: industry needs to be more specific in what steps should be taken to
improve the FDA's study review process (prospective vs. retrospective feedback).
Liability
It was noted by Merck that adequate liability is afforded to the industry on childhood
vaccines (National Childhood Vaccine Injury Act), but that similar liability protections
are not provided for adult vaccines. Dr. George Korch asked for clarification regarding
whether protections under the PREP Act were not sufficient for the industry concerns
regarding liability of products for biodefense and pandemic disease threats. There was
no further discussion of liability issues at this time, and a due out to industry would be
more definition of the nature of liability concerns that remain. (Note regarding the Prep
Act: "Passed primarily to address the pandemic influenza threat, the PREP Act provides
liability protections after a Secretarial declaration of covered countermeasures for any
disease or health condition that the Secretary views as constituting a public health
emergency, either presently or in the future. Liability protections cover the manufacture,
testing, development, distribution, or use of the designated covered countermeasure
absent willful misconduct", source:
http://www.hrsa.gov/countermeasurescomp/prep_act.htm as accessed on 30 January
2010)
Links shared
- gov http://www.hrsa.gov/countermeasurescomp/prep_act.htm