According to a new report by a Washington, DC advocacy group, "the US Government each year contributes around 45% of all investment in global health R&D and 70% of all government investment worldwide."
Also from the same report: “The US government’s investment in early basic (global health) research is so great that it now provides nearly two-thirds (62%) of global funding in this area,”
The report put out by the Global Health Technologies Corporation (GHTC) seems to define "global health" as communicable disease (and not, for example cancer or diabetes), so I'm a bit suspicious that the math behind the conclusions may be cherry-picked, but just accepting the figures my first reaction is that the high level of government support is BAD news for global health, and that the best thing that could happen would be for the US government to step back. Here's why I think that:
-Global health R&D is intended to benefit poor 3rd world patient populations, yet the bulk of the global health research is targeted to very a commercial market. According to the report, HIV R&D constitutes 57% of the global health R&D budget, as monitored by GHTC, so parsing the numbers would lead to the conclusion that there is already significant private sector research in HIV, just as there are very significant therapies having been discovered and commercialized by the private sector. Indeed, while the largest patient population for HIV therapies may be among very poor in Africa, this seems to me to be as much a distribution/manufacturing problem, and not an R&D challenge so much. If private sector HIV research were leading the way, the global health resources could be devoted to other research areas (cancer, etc.) and non-R&D assets like USAID could assume a bigger role in HIV treatment, especially in delivering HIV therapies.
-I don't like the idea that global health research strategy is largely driven by one large customer (the US Government.) Not to knock the people and funding process of the NIH/DoD/etc - and I know their research strategy isn't monolithic - but I would much, much rather see global health research strategy driven by a plurality of voices. Science doesn't progress as fast when one R&D catechism is dominant. Historically, much of our progress in life science is due to researchers with quirky or contrarian strategies swimming against the majority of researchers. No government body is very flexible in this regard.
(The best example I can think of here is Dr. Judah Folkman's pursuit of anti-angiogenesis. Once cast into the wilderness of cancer research by the cancer research establishment, Folkman's strategy is now an integral part of cancer therapy, as embodied by drugs such as Sutent and Avastin.)
-Likewise, unfortunately US global health policy is in part driven by politics, at least as significantly as the best scientific rationale or the aim of the most significant scientific impact. I'd rather more wise people steer global health R&D than the jokers in Congress.
-I favor global health R&D being driven by the private sector, with the government as a ultimate backstop, with the goal that global health R&D could be more market-oriented, but in an untraditional way. Market-oriented would traditionally be defined as financial demand tied to the patients' (or other payers) ability to pay. In the case of global health R&D, I think market-oriented would reflect (non-financial) patient demand, to include the amount of charity resources being devoted to a particular disease. I think by weighting R&D by this metric, we're more likely (for example) to place greater weight of clean water than TB, as the number of people with poor health due to a lack of access to clean water is many times greater than those at risk for TB. (The fact that clean water may be less of a biotech R&D issue and more of a civil engineering challenge underscores the point that the US government tends to mis-allocate R&D assets.)
-Finally, I'm annoyed by the idea that much of the first world is free-riding on America. The US accounts for ~27% of World GDP, and with US government being approximately 1/3rd of US GDP, this means that roughly 9% of world GDP funds 45% of global health R&D. I'm pointing the finger here at other first world powers to pull their weight. Unfortunately, to react to this point by cutting US federal global health R&D to equalize efforts puts others in jeopardy, but I highly doubt that other governments will spontaneously decide to pull their own weight in this area.
Showing posts with label NIH. Show all posts
Showing posts with label NIH. Show all posts
Monday, May 7, 2012
Thursday, April 12, 2012
NIH to repurpose failed leads?
(Let's call them "shelved" leads instead of the pejorative "failed." Some of the leads just lacked the organization will, expertise, or budget necessary to justify the risk of further investment.)
I find Collins' idea VERY interesting for a few reasons:
- this seems like a good role for government DD investment - the NIH can provide some very worthwhile assets & expertise, especially as leads may be repurposed from one disease class to another. (say, cancer to allergy application.) No pharma company has the breadth of expertise that the NIH does across their Institutes.
- this looks like a high leverage role for the NIH - theoretically, a small incremental investment in a shelved lead that is already proven safe in humans could have a HUGE return. However, the nature of the quest says that there will be ALOT of failure along the way, so much failure that pursuit of this mission by the private sector isn't economical.
-Politics: Any clinical success can make the NIH's mission more tangible to those who fund the NIH. The NIH does some great research, but since it is heavily biased towards early, basic research, not a lot of it can be used as a 'trophy' to gain more funding. Also, from the NIH perspective, a "win" from repurposing can come a lot sooner than a "win" from de novo drug discovery by NCATS, and by the nature of the repurposing work, the NIH can't step on any toes. If a pharma has shelved a program, how can they object to the NIH building off their earlier work? (I think we all know how pharma execs run from failed programs like cockroaches when the lights are turned on.)
-Also, while I'm not in love with the idea of the NIH taking a formal role in translational research, this seems like a smart way to dip a toe into drug discovery. Perhaps the NIH's DD experience to come from repurposing will help improve the DD regulatory process.
The devil is in the details, and in this case, it's the IP. What happens if a chemical patented by Company A for Disease X is found to be effective in Disease Y? Who owns the resulting IPR? There's no shortage of failed/shelved leads for the NIH to consider, so might they only pursue leads off patent or nearly off-patent?
The only question that I'd ask the NIH is "why does the NIH believe they have a higher probability of success with the "shelved" leads than the originating pharma team?" I can think of a few reasons, but I'd like to hear their rationale from them.
Friday, March 16, 2012
NCATS' vs. Calibr (public vs. private translational medicine)
The NIH's National Center for Advancing Translational Sciences - has been controversial since first promoted a year ago by Francis Collins, head of the NIH. The controversy is due, in essence, because the NIH isn't the right vehicle for drug discovery. (See Derek Lowe @ In The Pipeline for a very sharp, accurate, extended opinion.)
As NCATS is still being formed, there hasn't been a lot of news or action taken on their stated strategy, but they announced their initial partnership this week: NCATS and Eli Lilly will work together to profile almost 4,000 compounds either FDA approved or under investigation. The main goal seems to be to establish other possible applications for these compounds (What does this compound for kidney cancer do in CV models?)
As a first deal, the NCATS-Lilly partnership is a solid one, as disseminating the resulting info in the public will add to the characterization of existing drugs, and hopefully the development of new therapeutic applications, very possibly at a high speed, as many of the drugs in the NIH database will have already received she level of clinical scrutiny.
As an indication of NCATS' direction, however, this deal is underwhelming. It's an easy extension of NIH assets and activities that doesn't justify the dedicated infrastructure of NCATS. The partnership could have been undertaken by the existing extensive NIH compound profiling efforts (the NCI-60, for example), or externally as grant funded research.
(Given that some partnerships take more than a year to negotiate, one could wonder if the idea for the NCATS-Lilly partnership actually pre-dates NCATS.)
With the announcement of NCATS last year, the NIH announced the termination of the NCRR (National Center for Research Resources - I'd never heard of it either), and in principle, I like the idea of taking a look at current NIH missions and assets and restructuring when ideal. (Science changes regularly - shouldn't the NIH change with it?) However, I still don't see what the NIH is bringing to drug discovery/translational medicine that doesn't already exist. I hope NCATS' next deal proves that they are not a solution looking for a problem to solve.
(btw: congrats and thanks to Janet Woodcock and others at the FDA who are similarly progressively looking for opportunities to restructure the drug approval process. The current FDA/NIH leadership is to be commended for their commitment to progress and regulatory process improvement.)
In marked contrast is the just-announced private, not-for-profit translational medicine institute sponsored by Merck, Calibr (short for California Biomedical Research). There is a huge need for somebody to shepherd along promising molecules between their discovery in a basic research environment (universities) and when they are "ripe" for commercial development by a pharma/biotech company, and while NCATS is a step by the government to address this need, I think it is much more the responsibility of for-profit life science stakeholders.
Merck is seeding Calibr with $90M (likely feeling to Merck like a ~$60M expense, after accounting for the tax deduction), and will receive a right-of-first refusal for resulting technologies. The investment by Merck will also be leveraged by government grant funding of Calibr R&D. I'm sure Merck could put more exact numbers to it, but they'll probably get the benefits of say $120M in R&D over the next few years, for a $60M investment. This makes TONS of sense, and to any economic development folks out there, this also results in lots of new US jobs.
In contrast, NCATS' budget in 2012 will be $722M, with $553M to come from retitling existing programs. (In effect, NCATS = ~ $169M in incremental translational programs, less start-up costs).
Anyone want to bet which is ultimately more productive, per dollar, NCATS or Calibr?
(BTW: to make my criticism more constructive, what I'd instead nudge the NIH more towards is post-approval research. I think there's a need for impartially-sponsored Phase IV testing, and also a need for simply more Phase IV testing. As an example, consider a new compound approved for a particular solid tumor cancer. Post-approval, the pharma concentrates on marketing the new cure, and clinicians begin experimenting on off-label uses on an ad hoc basis. The NIH could better organize this research, expand upon it by scaling, and mitigate the inherent conflict of pharma-run Phase IV trials.
Alternatively, I'd be in favor of taking the NCATS $$$$ and using it to re-restablish R&D at an abandoned Pharma site. (Such as the old Parke-Davis/Pfizer campus in Ann Arbor, MI.) There's plenty of R&D talent, experience, and assets already available, with a big bang-for-the-buck.)
As NCATS is still being formed, there hasn't been a lot of news or action taken on their stated strategy, but they announced their initial partnership this week: NCATS and Eli Lilly will work together to profile almost 4,000 compounds either FDA approved or under investigation. The main goal seems to be to establish other possible applications for these compounds (What does this compound for kidney cancer do in CV models?)
As a first deal, the NCATS-Lilly partnership is a solid one, as disseminating the resulting info in the public will add to the characterization of existing drugs, and hopefully the development of new therapeutic applications, very possibly at a high speed, as many of the drugs in the NIH database will have already received she level of clinical scrutiny.
As an indication of NCATS' direction, however, this deal is underwhelming. It's an easy extension of NIH assets and activities that doesn't justify the dedicated infrastructure of NCATS. The partnership could have been undertaken by the existing extensive NIH compound profiling efforts (the NCI-60, for example), or externally as grant funded research.
(Given that some partnerships take more than a year to negotiate, one could wonder if the idea for the NCATS-Lilly partnership actually pre-dates NCATS.)
With the announcement of NCATS last year, the NIH announced the termination of the NCRR (National Center for Research Resources - I'd never heard of it either), and in principle, I like the idea of taking a look at current NIH missions and assets and restructuring when ideal. (Science changes regularly - shouldn't the NIH change with it?) However, I still don't see what the NIH is bringing to drug discovery/translational medicine that doesn't already exist. I hope NCATS' next deal proves that they are not a solution looking for a problem to solve.
(btw: congrats and thanks to Janet Woodcock and others at the FDA who are similarly progressively looking for opportunities to restructure the drug approval process. The current FDA/NIH leadership is to be commended for their commitment to progress and regulatory process improvement.)
In marked contrast is the just-announced private, not-for-profit translational medicine institute sponsored by Merck, Calibr (short for California Biomedical Research). There is a huge need for somebody to shepherd along promising molecules between their discovery in a basic research environment (universities) and when they are "ripe" for commercial development by a pharma/biotech company, and while NCATS is a step by the government to address this need, I think it is much more the responsibility of for-profit life science stakeholders.
Merck is seeding Calibr with $90M (likely feeling to Merck like a ~$60M expense, after accounting for the tax deduction), and will receive a right-of-first refusal for resulting technologies. The investment by Merck will also be leveraged by government grant funding of Calibr R&D. I'm sure Merck could put more exact numbers to it, but they'll probably get the benefits of say $120M in R&D over the next few years, for a $60M investment. This makes TONS of sense, and to any economic development folks out there, this also results in lots of new US jobs.
In contrast, NCATS' budget in 2012 will be $722M, with $553M to come from retitling existing programs. (In effect, NCATS = ~ $169M in incremental translational programs, less start-up costs).
Anyone want to bet which is ultimately more productive, per dollar, NCATS or Calibr?
(BTW: to make my criticism more constructive, what I'd instead nudge the NIH more towards is post-approval research. I think there's a need for impartially-sponsored Phase IV testing, and also a need for simply more Phase IV testing. As an example, consider a new compound approved for a particular solid tumor cancer. Post-approval, the pharma concentrates on marketing the new cure, and clinicians begin experimenting on off-label uses on an ad hoc basis. The NIH could better organize this research, expand upon it by scaling, and mitigate the inherent conflict of pharma-run Phase IV trials.
Alternatively, I'd be in favor of taking the NCATS $$$$ and using it to re-restablish R&D at an abandoned Pharma site. (Such as the old Parke-Davis/Pfizer campus in Ann Arbor, MI.) There's plenty of R&D talent, experience, and assets already available, with a big bang-for-the-buck.)
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