Collins Comments on Complementary Translational Research

No doubt to counter a less than enthusiastic response from Congress, Francis Collins has published a commentary in Science Translational Medicine addressing specific concerns raised in hearings (e.g., referring to “NCATS senior leadership—to be recruited in the next year—” and “make it abundantly clear that NIH is not attempting to become a drug development company”) and reinforcing the reality (i.e., resistence is futile) of his new National Center for Advancing Translational Sciences (NCATS):

With the establishment of NCATS in the fall of 2011, NIH aims to reengineer the translation process by bringing together expertise from the public and private sectors in an atmosphere of collaboration and precompetitive transparency.

He also restates the NCATS mission on its own:

to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of diseases and conditions.

and in partnership with the FDA:

Through this assembly of scientific and regulatory expertises and technologies as well as interdisciplinary cross-pollination, NCATS will catalyze the development of new insights that, when implemented, can have broad benefits across diverse translational projects.

Not surprisingly, he uses the Human Genome Project as the historical parallel of his new paradigm-shifting initiative and the untapped opportunities from GWAS to identify new drug targets. A better parallel might have been NCCAM, in that the approach (complementary/alternative) rather than the underlying disorder/biological system is the focus. He then notes that:

Early discussions with a variety of stakeholders have identified several components of translational science that are ripe for the new scientific approach offered by NCATS and will likely be the subject of early targeted funding opportunities.

It would be nice to know who these stakeholders were and the precompetitive transparent process by which they selected the 10 programmatic priorities covered by Collins: therapeutic target validation, chemistry, virtual drug design, preclinical toxicology, biomarkers, efficacy testing, phase zero clincal trials, rescuing and repurposing, clinical trial design, and postmarketing research. Now, these areas are interesting points of focus in terms of improving the efficiency of the development process … but they are already being pursued in the absence of NCATS and could have worked as Roadmap initiatives, which would have benefited from the expertise, resources, and strategic priorities of all the ICs.

He is a little less clear, still, explaining how NCATS will work with and facilitate (versus replace) industry efforts at drug development. Early on, he lays out the threat facing private research and development:

Faced with economic stresses and patent expirations, many pharmaceutical companies are reducing their investments in research, and biotechnology companies are finding it difficult to obtain venture capital for projects that need many years of support to achieve profitability.

Collins identifies the “middle zone” of translational research (“assessment of … potential to lead to a clinical advance; development of candidate diagnostics, devices, or therapeutics; optimization of the candidates in preclinical settings; regulatory assessment of the data to determine the potential for human use”) as the bottleneck industry cannot overcome alone:

The development of systematic approaches for target validation, the reengineering of rate-limiting and failure-prone steps in the therapeutic development process, and the urgent need to increase the critical mass of well-trained individuals to drive innovations are among the various translational challenges that are ill-suited for solutions derived solely from the private sector.

So he suggests that:

The new center will instead seek to invest in the kind of science that creates powerful new tools and technologies that can be adopted widely by researchers in public and private sectors to streamline and derisk the therapeutic development process.

Why creating a new center at the cost of a well-functioning center (NCRR) was required is not addressed … in fact, NCRR is never mentioned. Collins remains confident the “whole” of NCATS will be greater than the sum of its existing parts. The one new program, if the amended NIH budget (NCATS added, NCRR subtracted) is approved by Congress, the Cure Acceleration Network (CAN), remains poorly defined. His table refers to CAN as supporting “translational solutions to high-need medical problems” with “much-needed flexible funding authorities” … and a lot more money per award (up to $15M) made to “academic and private-sector consortia, with such projects managed “actively and aggressively by using mechanisms similar to those used by DARPA.” Hmm. Hope this doesn’t mean the NIH intends to follow the DoD down the path of funding black projects as well …

6 Comments »

  1. curie said

    still can’t figure out why no one is asking collins why not create focused virtual sub-unit within each existing institute to handle their respective translational aspects. and why not link all such (virtual) sub-units within each institute to allow increased interactions among institutes. who is better to handle translational research for a disease more than the respective institute itself. (virtual) sub-units will increase funding (or will stop unnecessary funding reduction or division to handle translation), more jobs within each institute.

  2. AH said

    IMO, Francis Collins’s commentary is, as his usual, as persuasive as he can be. However, this idea of “centralization” of translational science in the new NCATS -to catalyze therapeutic advances and success to the public benefit and the national economic enterprise- is much less convincing.

    Centralization might appear potentially more effective. Except that looking at diverse experiences (in different contexts), one is left with the impression that centralization goes, hand in hand, with less participation and less openness. Promises that “things would be different in this case” are easy to make but much more difficult to effect.

    No reason to not predict and/or expect that decentralization (as presently), would not achieve the main goal as well (if not better) because there is decisive impetus, proven realities and almost universal consensus in the need for effective therapeutics.

    The analogy of NCATS with the Human Genome Project is somehow useful but not totally comparable. Their contexts were/are entirely different in spite of some shared anxieties.

  3. guess said

    Very nice article in Translational Science by Francis Collins: “Reengineering Translational Science: The Time Is Right”

    We all concur “ Opportunities to advance the discipline of translational science have never been better. We must move forward now. Science and society cannot afford to do otherwise. “.

    Centralizing the opportunities doesn’t sound like challenging the status quo…. “For from history we had learned, how many, many times the hopes were turned down”..

    Coordinating efforts and achievements in a diverse reinvigorated translational spirit might sound like “the heights are too high to climb”.

    Let us prove that we can climb really high from our diverse realities.

    We all laughed when Dr Francis Collins said that “we could all fly high”,
    We all grumbled when he proposed the new NCATS,
    We all argued that flying in just ONE plain will not do the most for all,
    We went forth and backwards,
    With the same ONE cry (NO!)

    We roared at him
    Wanting us
    to buy in just the one NCATS
    ‘cause’ it’ll prevent us
    from our challenging the status qua

    We would like translate from our places
    Come on guys, let’s take a bow,
    So no more, more,
    No more status quo !!!!.

  4. writedit said

    Interesting review in Nature Reviews Drug Discovery of how new drugs approved from 1999-2008 were identified (phenotypic vs target-based screening).

  5. […] link from writedit pointed me to a review of drugs that were approved in the US with an eye to how they were […]

  6. BugDoc said

    “make it abundantly clear that NIH is not attempting to become a drug development company”

    O rly? I don’t know if this is true for every field, but I’m sure seeing a LOT more RFAs with an emphatic focus on drug development in my institute (NIAID).

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