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Planning for the Future: An NINDS Forum

Planning for the Future:  An NINDS Forum

May 16, 2008

Bethesda, Maryland

At the NINDS National Nonprofit Forum – the third of its kind to bring together NINDS and voluntary organizations that share the goal of stamping out neurological disease – the focus was on working together to accelerate research.  During the first half of the day, a series of presentations addressed how voluntary organizations can forge new collaborative efforts with NINDS and with each other.  During the second half, the NINDS Director invited representatives from the voluntaries to help set the stage for a new strategic plan at NINDS.  Below are highlights from the day's presentations and an open invitation to participate in the NINDS planning process.


NIH Update  - Story Landis, Ph.D., Director, NINDS

NIH Peer Review Self-Study
Since 2003, NIH's budget has flattened while the number of grant applications it receives each year has risen, placing increased pressure on investigators to secure funding.  To ensure that it continues to support the best science by the best investigators, NIH has spent the past year examining its peer review process, Dr. Landis said.  There is concern that that the best scientists do not participate in peer review. A second concern is that young investigators, who typically have less preliminary data and grant writing experience, are getting squeezed by budget constraints and will leave science.  A third concern is that most successful grant applications are resubmissions – applications that the investigators had to rewrite a second or third time, potentially unable to move forward with their proposed studies in the interim.  It is hoped that changes to the peer review system will reduce the administrative burden on reviewers and applicants while continuing to emphasize scientific excellence, fairness, timeliness, and integrity, Dr. Landis said.

The NIH Peer Review Self-Study began in June 2007, when NIH Director Elias Zerhouni, M.D., established two working groups – a working group to the Advisory Committee to the NIH Director and a Steering Committee Ad Hoc working group, of which Dr. Landis is a member.  Both working groups collected input for enhancing the peer review system from all stakeholder communities (including researchers, advocacy groups, professional societies and NIH staff) through an online Request for Information (RFI), as well as teleconferences and face-to-face meetings.  On June 12, after a consideration of the working groups' final reports and responses from the community, Dr. Zerhouni announced several planned changes to the peer review system, including a commitment to invest $1 billion over the next five years in investigator initiated, high risk, high impact research.  Other changes to be phased in during the next 18 months include: shorter grant applications with a new format that highlights potential impact over experimental detail; efforts to attract and maintain the best reviewers by offering flexible schedules, formal acknowledgment of service, and enhanced training; support for a minimum number of new investigators; and a permanent system for evaluating peer review.    For more information, see and

The Research, Condition, and Disease Categorization (RCDC) system
Dr. Landis also described a plan to standardize NIH's system for reporting how appropriated funds are invested to support biomedical research.  For many years, NIH has provided this data by summing information provided by individual Institutes and Centers who categorized their spending according to disease, condition and research area (  With advances in data- and text-mining, NIH leadership saw an opportunity to make this reporting process more reliable, consistent and transparent.

The RCDC ( uses most of the same categories that have been historically reported to Congress and the public, but changes the way NIH's research grants and contracts are sorted into those categories.  Like NIH's current reporting system, the categories are defined by experts at NIH.  (For example, the category "heart disease" might encompass "coronary artery disease," "myocardial infarction," "atherosclerosis" and other terms.)  The difference is that RCDC uses a new computer-based system to sort research grants and contracts into those categories.  Also, for the first time, RCDC will standardize the categories of research conducted in the labs and clinics at the 27 Institutes and Centers across NIH (the intramural programs).  The first RCDC report will be available in Spring 2009.  Although funding in certain areas may appear to increase or decrease, it is important to remember that RCDC changes only the way NIH reports its funding, not how it makes funding decisions.


Public-Private Partnerships – Walter Koroshetz, M.D., Deputy Director, NINDS

Dr. Koroshetz described several ways that NIH and voluntary organizations can work together to support talented investigators who are struggling for funding.

NINDS Funding for Translational and Clinical Research
The NINDS Translational Research Program funds research aimed at translating laboratory discoveries into new treatments for neurological disease (  The program uses cooperative agreements ("U" awards) to support high-throughput screening of potential therapeutics, animal efficacy studies, pharmacokinetics, pre-clinical toxicology work and other studies required for an investigational new drug (IND) application to the FDA.  These agreements are milestone-driven, and can be terminated if milestones are not met.  Dr. Koroshetz said that voluntary organizations can work with investigators to prepare and submit applications to the translational research program.  The SMA project, initiated by NINDS, is an example of a translational project where NINDS has successfully partnered with academia, industry and nonprofit groups.  The project has led to several promising lead compounds with the potential to treat spinal muscular atrophy (  NINDS also has programs to recruit and train clinicians to conduct research on neurological disease (see  These include the Mentored Clinical Scientist Development Award (K08) and the Mentored Patient-Oriented Research Career Development Award (K23).  These awards appear to be underutilized; since 2006, NINDS has seen a 30-40 percent drop in applications for them, Dr. Koroshetz said.  NINDS is working with medical societies to resolve this issue.  Meanwhile, voluntary organizations may be able to boost application rates by helping spread the word about K awards among their contacts within the medical community.

Database of Grants that NIH is Unable to Fund
Dr. Koroshetz also described a resource that that is being developed to allow voluntary organizations identify and support talented investigators whose grants were not funded due to NIH budget constraints.  The National Health Council (NHC;, a coalition of health related nonprofit groups, professional societies and businesses, is building an online database of unfunded NIH grant applications.  While NIH does not release information about unfunded grants, the database would allow investigators to voluntarily submit information about their unfunded grants, including the title, the abstract, requested budget, and score.  NHC member organizations can search the database at no charge, and non-member organizations can search it for a fee.  The database, Dr. Koroshetz said, would offer investigators enhanced access to non-government funding organizations, and would offer funding organizations the benefits of NIH peer review.  The database is expected to launch this summer.

The NINDS Gift Fund and the Foundation for NIH
Voluntary organizations can also pool their resources with those of NIH by participating in the NINDS Gift Fund or the Foundation for NIH (FNIH), Dr. Koroshetz said.  The NINDS Gift Fund ( accepts tax-deductible donations that are made in support of the Institute's mission to reduce the burden of neurological disease.  Donations may be directed to support: research that has been vetted by NIH peer review; the purchase of equipment and supplies for laboratories; scientific workshops and conferences; printing of materials about neurological diseases and stroke for the public, patients, and health professionals; and scientific staff recruitment efforts.

The Foundation for NIH ( is a nonprofit organization that supports the NIH mission by forging partnerships among NIH, other government agencies and the private sector.  For example, the FNIH Biomarkers Consortium ( is a partnership among the NIH, FDA, Centers for Medicare and Medicaid Services, and many for-profit and nonprofit organizations, formed to accelerate the development of biomarkers.  Biomarkers are molecular, biological, or physical characteristics that can be used to identify a risk for disease.  Identification of biomarkers could facilitate diagnosis and speed clinical trials. The consortium's Neuroscience Steering Committee ( focuses on developing biomarkers relevant to neurological and psychiatric disorders.


Collaborative Research: A Model – Ron Bartek, President, Friedreich's Ataxia Research Alliance (FARA)

Mr. Bartek described how FARA ( takes a collaborative approach to therapeutic development for Friedreich's Ataxia (FA) by seeking partnerships with NIH, industry and other nonprofit organizations.  One key to these collaborations, he emphasized, has been to identify and explore underlying mechanisms of damage and therapeutics that are shared by FA and other diseases.  For example, FA involves a breakdown of the cellular energy factories known as mitochondria – a process that occurs in many other neurodegenerative diseases.

Recognition of these common disease processes and potential therapeutic approaches has enabled FARA to enter fruitful collaborations with the Muscular Dystrophy Association, the National Ataxia Foundation, the American Heart Association and other nonprofit groups, Mr. Bartek said.  FARA's collaborative activities with these groups include co-review and co-funding of grants, co-sponsored workshops, the development of research portfolios, and the construction of patient registries.

Identifying common interests also enabled FARA to work with academic scientists and pharmaceutical companies to submit a successful application to the NIH Rapid Access to Interventional Development (RAID) pilot program, Mr. Bartek said.  This program supports the development of small molecule therapeutic agents – in particular, the high-risk, early stages of development that tend to lack industry support.  Examples of supported projects include good medical practice manufacturing, pharmacokinetic testing, and animal toxicology studies.  NIH-RAID projects are co-sponsored by one or more Institutes and the NIH Roadmap (the Common Fund), which supports research that cuts across disease-specific areas.  NIH-RAID applicants have a better chance of success if they can demonstrate that their proposed work is likely to have a broad impact on multiple diseases.  For more information, see


Collaborative Resources: A Model – Jonathan Jacoby, Chief Operating Officer, CollabRx

Mr. Jacoby described how Collabrx ( has developed online technology that can be used to accelerate therapeutic development.  The company provides management services, and has a web-based platform that makes it possible for a geographically dispersed team of scientists to collaborate within a "virtual biotech."  An online decision analysis tool helps the scientists collaboratively plan experiments and share data.  Meanwhile, funding organizations can use the online platform to track progress and set new priorities.

Mr. Jacoby is a co-founder and Chairman of the Hide & Seek Foundation for Lysosomal Disease Research.  His son has Niemann-Pick Type C, a lysosomal disease.  CollabRx was founded by Jay "Marty" Tenenbaum, Ph.D., a computer scientist and entrepreneur, after he was diagnosed with metastatic melanoma in 1998.  Thanks to surgery and immunotherapy, Dr. Tenenbaum is now cancer-free.


NINDS Strategic Planning – Paul Scott, Ph.D., Director, NINDS, OSPP

Dr. Scott described NINDS' new planning effort, which is intended to help the Institute more effectively pursue its mission to reduce the burden of neurological diseases.  A similar planning effort in 1999, Neuroscience in the New Millennium, led NINDS to initiate a series of infrastructure projects including core grants, GENSAT, and the microarray consortium, as well as scientific programs.  It also had a significant impact on how NINDS is organized. The Division of Extramural Research was created and organized into eight clusters around themes in neuroscience.  The new organization was also flatter, giving program directors more autonomy in responding to the needs of scientists and patient advocates.  To assist in the current planning process, NINDS plans to publish a Request for Information (RFI) to seek input from nonprofit organizations, the broader patient community, and other members of the public with an interest in neuroscience research.

One purpose of the 2008 Forum, Dr. Scott explained, was to bring NINDS together with nonprofit organizations to develop content for the RFI.  Toward that end, NINDS staff and the nonprofit representatives in attendance – representing more than 60 organizations – formed small breakout groups to generate topics and questions for the RFI.  In addition to generating ideas for questions incorporated into the RFI, discussions at the Forum highlighted a number of other interesting issues, many of which related to how NINDS and nonprofit organizations can better communicate with and inform each other on an ongoing basis about current activities, needs and opportunities.  In parallel to the current RFI and the strategic planning effort, NINDS staff will consider how best to work with the nonprofit community to follow up on this additional input from the Forum.

Last Modified December 16, 2015