1.
NCATS: Who moved my cheese?
This
year, the National Institutes of Health will establish its new National
Center for Advancing Translational Sciences (NCATS), created to speed the
translation of basic discoveries into therapies that will improve public health.
In light of our current fiscal reality (and limitations), we breathed a sigh of
relief to see it up and prioritized in the appropriations process. Solutions can
be tough to develop, and tougher to implement. How will NIH translate its
translational focus to its cadre of basic research-focused scientists? Will
NCATS strengthen and streamline existing efforts at the 26 other institutes and
centers? Will it help to move discoveries out of the lab and to patients more
quickly? NCATS is one way to get moving (and, as we know, "movement in a new
direction helps you find new cheese").
The stakes are high with more than 100 million patients waiting for meaningful
treatments options. But with Dr. Francis Collins as the visionary behind this
effort, the same man who defied expectations by completing the Human Genome
Project ahead of schedule and under budget, we like the odds.
2.
FDA: The science of failure.
Companies
usually turn to the FDA to help them succeed (and with 35 approvals in FY2011,
it helped many succeed), but perhaps it can also help them fail, faster.
Commissioner Hamburg seems committed to having FDA play a role in improving
R&D efficiency for all. FDA is sitting on mountains of data, and she has
made it a priority -- through the agency's Strategic Plan for Advancing Regulatory Science and specific
efforts like the PACES Initiative -- to figure out how to share more of it,
particularly from compounds that were not approved, to help researchers and
companies learn from failures. Will the industry see its self-interest in this
and allow data to be shared -- and recognize that there is a business case for
opening up the file cabinet?
3.
Intellectual property: Keep it simple, stupid.
Everyone
in medical research seems to agree that intellectual property is a significant
issue slowing progress, from disagreements over the value of discoveries to the
time it takes for agreements to be negotiated, and the aversion to standardized
contracting. We say, why does this need to be so complicated? Can't we come up
with a simple set of guiding principles or values that we all agree to abide by
-- something along the lines of Google's "Don't be evil"? Ideas, anyone?
4.
Cures on the stump.
Last
time we checked, 2012 was a presidential election year. Will life sciences make
an appearance on the agenda? The Obama Administration is working on a National Bioeconomy Blueprint to "harness biological research
innovations to address national challenges in health, food, energy, and the
environment" -- oh, and of course to "grow the jobs of the future." What
commitments will the candidates make to support this critical economic driver --
whether NIH funding, tax policies for industry, workforce training, etc.? Can we
make innovation in the life sciences a topic du
jour in the election season?
5.
2012: Year of the Patient?
TIME
magazine notably called 2011 the "year of the protester." Could 2012 be the
"year of the patient"? We offered up a roadmap to effective activism in 2011, built on the example
of the HIV/AIDS advocacy movement. We identified its ingredients for success as
attention, knowledge and solutions, community, accountability and leadership.
Many of us point to the successes this movement created in HIV/AIDS drug
development. Thirty years later, will more groups seize the mantle and make 2012
as much about changing the research and regulatory paradigm as about raising
more money and awareness?
6.
Reimbursement is the new approval.
Even
if we are successful at getting new treatments through the R&D and approval
process, the question remains whether payers (government and private) will be
willing to pick up the tab. Insurance coverage decisions -- and insurers
themselves -- are playing a role farther upstream in the R&D and investment
process, as companies begin to craft their research priorities and programs to
maximize the possibility of reimbursement. Can payers position themselves as part of the solution to getting
truly innovative therapies to patients? CMS is currently seeking comments on a
revamp of its "coverage with evidence development" program. Could this become a
model for other insurers to get patients access to new treatments while
continuing to generate data about its effectiveness? Will smaller companies
start reading the tea leaves and work with payers to avoid "failure to
launch"?
7.
HIT: If we build it, will research come?
The
federal government has succeeded in driving a doubling of providers' adoption of
health IT over the last two years through its stimulus funding. But their
interest in ensuring all this data is available for research to drive new, more
effective treatments for patients is still not evident. "Meaningful use" of
electronic health records (EHR) to qualify for federal funding seems to have
been an effective carrot/stick combination for driving adoption of EHR systems
for patient care. Let's use it in the service of research by requiring that using EHR
systems for clinical trial screening and matching be included as a measure for
meaningful use in the next round of definitions. Unlike the "Field of Dreams,"
just because we built the infrastructure, research use will not magically
appear.
8.
CTSAs: Who you gonna
call?
The
first five years of NIH's Clinical and Translational
Sciences Awards (CTSA) program are over, and by many accounts the focus at
most of the 60 institutions has been on standing up the program on their own
campuses, not on maximizing the power of the network. That may start to change
in 2012 with the creation of a new Coordinating Center at Vanderbilt, which is already ramping
up development of tools and resources, such as a shared IRBs, contract language,
IP database and research data capture. Its director, Gordon Bernard, wants to
make it a "virtual institute of drug development," a one-stop shop for
companies, investors or nonprofit funders looking for academic partners. At
Partnering for Cures 2011, he invited participants to contact him with their
needs, saying that until the right partners are in place "we'll own that
problem." So give him a call.
9.
Putting old wine in new bottles.
Enthusiasm
continues to grow in academia and among patient groups for the idea of
repurposing existing compounds. Companies, if they're interested at all, are
looking for some sort of exclusivity mechanism to allow them to make money. NIH
sees this as a central opportunity for NCATS (see #1), and NIH's TRND program is piloting a repurposing effort with the Leukemia & Lymphoma Society
and the University of Kansas as partners, with the aim of recruiting an industry
partner. But pilots won't be enough; there needs to be funding sources
identified, IP policies sorted out and regulatory pathways clarified. Could 2012
be the year we create a business model to support repurposing? Tick tock goes
the clock. Patients are waiting.
10.
Convergence of venture capital and venture
philanthropy.
enture
capital investment in life sciences is on the wane, but venture philanthropy
investment in drug development is on the rise. We're hearing discussion in several quarters
about the idea of blending venture capital and venture philanthropy in some sort
of hybrid investment fund. The wider world of social entrepreneurship has been
experimenting with business models and forms of capital all along the spectrum
from not-for-profit to for-profit for some time; the field of medical research
is slow to catch up. We'll be watching for the launch of one or more of these
efforts in 2012. Look for more discussion of financial innovations for
translational research from FasterCures in
2012 as well.
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