• Summary
• Why an NIH-RAID Pilot?
• What specific services are available for the pilot program?
• What the NIH-RAID Pilot is not
• Role of the NIH Institutes and Centers
• Eligibility
• Intellectual Property Rights
• Critical dates

Summary

The National Institutes of Health (NIH) Roadmap has established a pilot program called the NIH-RAID Pilot (Rapid Access to Interventional Development), similar to the National Cancer Institute’s (NCI) RAID program, to make available, on a competitive basis, certain critical resources needed for the development of new small molecule therapeutic agents. This program, part of the Translational Research component of Reengineering the Clinical Research Enterprise, uses resources of NCI's Developmental Therapeutics Program. The services provided will depend upon the stage of the project and the strength of the preliminary data. Services potentially available include: production, bulk supply, GMP manufacturing, formulation, development of an assay suitable for pharmacokinetic testing, and animal toxicology. Assistance also will be provided in the regulatory process, through access to independent product development planning expertise. In the program’s pilot phase, animal efficacy studies, and synthesis of recombinant proteins, monoclonal antibodies or reagents for gene therapy will not be supported. However, the NIH-RAID Pilot will now consider requests for services to support later-stage preclinical development of monoclonal antibodies, recombinant proteins, and gene therapy agents.Additionally, the NIH-RAID Pilot will now consider requests for manufacture of material for any clinical study. For more information see NOT-RM-08-005

The NIH-RAID Pilot is not a grant program. Successful projects will gain access to the government’s contract resources, as well as the assistance of the NIH in establishing and implementing a product development plan. Funds to support individual projects will come both from Roadmap funds and from individual Institutes, with Institutes assuming the bulk of support in the specific disease areas germane to their mission. This co-sponsorship is critical because of the resource and expertise needs and because the NIH-RAID Pilot cannot support the full developmental pipeline; an Institute partnership may therefore be important for subsequent translational efforts.

To obtain access to NIH-RAID Pilot resources, applications must be submitted electronically through Grants.gov using SF424. Applications are screened to determine whether the resources requested are appropriate for this program. Applications are then reviewed by the NIH Center for Scientific Research. The results of that evaluation along with supplemental information from the lead investigator will guide final Institute and Roadmap resource allocation.

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Why a NIH-RAID Pilot?

NIH-Roadmap consultants advised the NIH that a number of promising ideas for novel therapeutic interventions encounter roadblocks in movement from bench to bedside testing. Sometimes, translation is facilitated by alliances with corporate or other private sector partners, but high risk ideas or therapies for uncommon disorders frequently do not attract private sector investment. Where private-sector capacity is limited or not available, provision of developmental resources by the government can bridge the gap between discovery and clinical testing, so that more efficient translation of promising discoveries may take place.

The NIH-RAID Pilot is intended to reduce some of the common barriers between laboratory discoveries and clinical trials of new therapeutic entities. Projects in both the early and late stages of pre-clinical development are suitable for NIH-RAID proposals. The NIH-RAID Pilot will accept requests through 2011.

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What specific services are available for the pilot program?

The main tasks that are supported by the NIH-RAID Pilot are as follows:

• Synthesis in bulk of small molecules
• Synthesis of oligonucleotides
• Chemical synthesis of peptides
• Scale-up production
• Development of analytical methods
• Isolation and purification of naturally occurring substances
• Pharmacokinetic/ADME studies including bioanalytical method development
• Development of suitable formulations
• Manufacture of drug supplies
• Range-finding initial toxicology
• IND-directed toxicology
• Product development planning and advice in IND preparation
• Later-stage preclinical development of monoclonal antibodies, recombinant proteins, and gene therapy agents

The tasks necessary will vary from project to project. In some cases the NIH-RAID Pilot will support only one or two key steps for early stage preclinical development; in other cases it may be possible to provide assistance with most of the development tasks needed to file an IND.

The output of NIH-RAID Pilot activities, both products and information, will be made fully available to the originating investigator for support of additional studies or of an IND application and performance of clinical trials. Data and product will be transferred to the applicant under the terms of an NIH-RAID Pilot Material Transfer Agreement (see Intellectual Property below). For those projects approved for production of a clinical batch, the final vialed drug product will be delivered in a single shipment; the NIH-RAID Pilot cannot distribute drug product in multiple shipments or on a per patient basis.

Note: Again, while the NIH-RAID Pilot will now consider requests for services to support later-stage preclinical development of monoclonal antibodies, recombinant proteins, and gene therapy agents, during the NIH-RAID pilot phase the program is not able to offer support for preparation of monoclonal antibodies, recombinant proteins or gene therapy reagents. In addition, the NIH-RAID Pilot Program will not provide either in vitro or animal efficacy testing.

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What NIH-RAID Pilot is not:

• The NIH-RAID Pilot is not a complete drug development program or an unconditional commitment to develop a particular compound for the clinic. Development will proceed sequentially, in a logical order, and the start of one segment of the process (e.g., toxicology) will depend on satisfactory completion of preceding segments (e.g., formulation). Insurmountable difficulties in one segment may force abandonment of individual projects, as they do in any development program.
• The NIH-RAID Pilot does not support either in vitro or animal efficacy testing or human subject research. The investigator, with advice from the co-sponsoring Institute, needs to consider alternate sources of support for these components of the drug development process. For projects close to clinical application, the investigator needs to develop and document the strategy they expect to pursue for early phase clinical testing, including possible private-sector collaborations.
• The NIH-RAID Pilot is not meant to assist industry in its development projects in the absence of an academic partner. However, academic investigators may have collaborations with for-profit partners and qualify for NIH-RAID Pilot funding.
• The NIH-RAID Pilot is not intended to yield NIH-held INDs. It is anticipated that the clinical phases of testing will generally occur under investigator-held INDs within the originating (or collaborating) institution or by partnership with an industry partner.

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Role of NIH Institutes and Centers

All NIH Institutes and Centers are participating in the funding of this Roadmap program. The NIH-RAID Pilot projects will be co-sponsored by Roadmap resources and one or more Institutes. Applicants do not need to establish Institute co-sponsorship prior to filing a proposal. However, interested individuals may contact the NIH-RAID Pilot office or members of the NIH RAID Pilot Working Group for discussions about Institute co-sponsorship. When the preliminary work for the project has been supported by the NIH, requestors are also encouraged to contact their program officer to discuss suitability of the project for the NIH-RAID Pilot and potential willingness of the Institute to co-sponsor the project. Several Institutes already support services like those provided by the NIH-RAID Pilot through other mechanisms, and have elected not to co-sponsor requests to this program in those areas.

• NCI: Investigators pursuing the development of therapeutic agents for cancer will in general be eligible for the programs of the National Cancer Institute (NCI). See: http://dtp.nci.nih.gov/docs/raid/raid_index.html. For more information contact Richard Camalier (camalier@mail.nih.gov).
• NHLBI: The National Heart, Lung and Blood Institute (NHLBI) supports an integrated and coordinated program of research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders. The NHLBI plans and directs research in development and evaluation of interventions and devices related to prevention, treatment, and rehabilitation of patients suffering from such diseases and disorders. It also supports research on clinical use of blood and all aspects of the management of blood resources. The treatment goals of NHLBI include new therapies to improve the outcome of heart and lung transplantation and increase the supply of donor organs; to improve hematopoietic stem cell transplantation; to develop the scientific underpinning necessary to be able to repair or replace damaged or diseased tissues and organs with biological substitutes; to develop strategies to treat microbial infections that result from cardiothoracic surgery; and to identify and answer key questions related to cardiovascular therapy. For more information contact Traci Heath Mondoro, Ph.D. (mondorot@nhlbi.nih.gov).
• NIA: The National Institute on Aging leads a broad scientific effort to understand the nature of aging and to extend the healthy, active years of life. An important component of the NIA mission is focused on understanding, preventing and treating Alzheimer's disease and related dementias of aging. The NIA is interested in small molecule agents that increase the health span of the elderly by preventing or delaying one or more of the many adverse consequences that accompany aging. For example, these agents might prevent or reduce: muscle and/or bone weakening, oxidative stress associated with aging, the age-dependent loss of lean body mass, or metabolic changes in the elderly that increase disease risk. In addition, the NIA supports the development of agents that act as mimetics of caloric restriction. The Institute has a special interest in new small molecule therapeutic agents that would prevent the onset, or delay the progression, of neurodegenerative diseases of aging- particularly Alzheimer’s disease and related dementias, older age-related changes in neuroendocrine systems and their sequelae, and sensory, motor and sleep disorders of older people. For more information contact Chhanda Dutta, Ph.D. (DuttaC@nia.nih.gov).
  
• NIAAA: The mission of the National Institute on Alcohol Abuse and Alcoholism (NIAAA) is to generate new knowledge about the causes of alcohol-use disorders and their medical consequences, and to use this knowledge to develop prevention and treatment strategies. NIAAA is interested in requests for support to develop new medications and probes for alcohol use disorders with a particular focus on early intervention and the treatment of co-morbid disorders such as depression and anxiety. For more information contact Nanwei Cao, Ph.D. (caon@helix.nih.gov).
• NIAID: The National Institute of Allergy and Infectious Diseases (http://www.niaid.nih.gov/) comprises three Divisions, each with a specific biomedical focus.
  1. The Division of Acquired Immunodeficiency Syndrome (DAIDS) addresses the national research needs created by the advent and spread of the HIV/AIDS epidemic. Specifically, the Division supports research to increase basic knowledge of the pathogenesis, natural history, and transmission of HIV disease and research promoting progress in its detection, treatment, and prevention. Coinfections and complications of AIDS therapies are additional areas of major emphasis. Investigators pursuing the development of therapeutic agents for HIV/AIDS should consult the Resource Guide (http://www.niaid.nih.gov/daids/PDATguide/overview.htm) for instructions on how to access NIAID contract resources.
  2. The Division of Allergy, Immunology, and Transplantation (DAIT) focuses on studies of immune system function in the maintenance of health and in the production of disease. DAIT supports basic and clinical research to enhance understanding of the causes and mechanisms that lead to development normal immunity and immune-mediated diseases and transplant rejection. DAIT supports clinical studies and clinical trials to expand this knowledge base in order to improve diagnosis, treatment, and prevention of immune mediated diseases.
  3. The Division of Microbiology and Infectious Diseases (DMID) supports extramural research to control and prevent human diseases caused by any infectious agent except HIV. DMID supports research in bacteriology, mycology, virology, parasitology, biodefense, emerging infectious diseases, vaccine development, and microbial genomics. DMID supports a wide variety of projects spanning the spectrum from basic biomedical research, through applied research, including developing diagnostic tests, experimental drugs, and vaccines. NIAID is especially interested in development of novel therapeutic approaches for infectious or immune-based disorders that do not attract sufficient private sector research support.
     
     For more information contact Robert Goldman, Ph.D. (rgoldman@mail.nih.gov).

• NIAMS: The mission areas for National Institute of Arthritis and Musculoskeletal and Skin Disease (NIAMS) include arthritis, musculoskeletal disorders, and skin diseases such as rheumatoid arthritis, lupus, vitiligo, psoriasis, muscular dystrophies, osteoarthritis, osteoporosis, and many other diseases. For more information contact Gayle Lester, Ph.D. (lester1@mail.nih.gov).
  
• NICHD: The mission of the National Institute of Child Health and Human Development (NICHD) is to ensure that all children are born healthy and wanted, and to minimize disability through rehabilitation. NICHD has an interest in requests that will lead to treatment for reproductive diseases and infertility in men and women, the development of interventions that would treat obstetric-fetal conditions and/or pediatric problems and that would fully consider the unique pharmacological dynamics of pregnant women and children, and development of drugs or devices to assist the rehabilitative process. For more information contact June Lee, Ph.D. (leejun@mail.nih.gov).
  
• NIDA: The mission of the National Institute on Drug Abuse is to lead the nation in bringing the power of science to bear on drug abuse and addiction. Through a series of contracts administered by the Division of Pharmacotherapies and Medical Consequences of Drug Abuse (http://www.nida.nih.gov/about/organization/DPMCDA/index.html), NIDA supports the discovery and development of medications for the treatment of drug addiction disorders. For such medications, it may be advantageous for academic investigators and for NIDA to utilize NIH-RAID Program resources to support a major portion of preclinical development. The discovery and development of novel analgesics with little or no abuse liability is another area of NIDA interest; however, while NIDA provides substantial grant support for basic research that may lead to the discovery of such novel analgesics, their development generally is not supported by NIDA due to the large market for analgesics and the activity of the private sector in this area. For more information on partnering with NIDA, please contact David McCann, Ph.D. (dmccann@nih.gov).
  
• NIDCD: The National Institute on Deafness and Other Communication Disorders(NIDCD)(http://www.nidcd.nih.gov/index.asp) covers a broad range of communication disorders of hearing, balance, smell, taste, voice, speech, and language. NIH-RAID pilot projects of interest to the NIDCD would involve the development of new small molecule therapies directed to these communication systems. Especially important would be therapeutics for the treatment of hearing and vestibular dysfunctions due to diverse causes, including noise and ototoxins, imbalanced fluid and mineral composition, and loss of hair cells. For more information contact Ling Chin, Ph.D. ( lchin@mail.nih.gov).
  
• NIDCR: The mission of the National Institutes of Dental and Craniofacial Research (NIDCR) is to improve oral, dental and craniofacial health through research, research training, and the dissemination of health information. NIH-RAID pilot projects of interest to the NIDCR would involve the development of new small molecule therapies for oral cancer, Sjögren’s syndrome, orofacial pain, dysregulation of dental biofilms, and genetic disorders of the orofacial system. For more information contact Eleni Kousvelari, DDS, D.Sc. (kousvelari@de45.nidr.nih.gov).
  
• NIDDK: The mission areas for the National Institute of Diabetes, Digestive and Kidney Diseases (NIDDK) include endocrine and metabolic diseases, digestive diseases such as hepatitis and inflammatory bowel disease, kidney and urologic diseases such as kidney failure and prostate enlargement, and blood diseases such as the anemias. Projects related to Type I diabetes are supported through a separate initiative: http://www.niddk.nih.gov/fund/diabetesspecialfunds/T1D-RAID/. For more information contact Myrlene Staten, M.D. (statenm@mail.nih.gov).
  
• NIEHS: Human health and human disease result from three interactive elements: environmental exposure, individual susceptibility (genetics) and age. The mission of the National Institute of Environmental Health Sciences (NIEHS) is to reduce the burden of human illness and dysfunction from environmental causes by understanding each of these elements and how they interrelate. The NIEHS achieves its mission through multidisciplinary biomedical research programs, prevention and intervention efforts, and communication strategies that encompass training, education, technology transfer, and community outreach. In selecting projects for the NIH-RAID pilot, NIEHS is particularly interested in development of new therapeutic approaches for treating or preventing injury and disease caused by the intentional release of chemical agents by terrorists, and for environmentally associated disorders which do not attract extensive private sector research support. For more information contact Dennis Lang Ph.D.  (DL73v@nih.gov).
  
• NIMH: The mission of the National Institute of Mental Health (NIMH) is to reduce the burden of mental illness and behavioral disorders through research on mind, brain, and behavior with the goal of transforming prevention and recovery from mental disorders. NIH-RAID projects of interest to the NIMH would involve the development of novel small molecule therapeutics for mental disorders such as schizophrenia, depression, bipolar disorder, anxiety disorders, ADD/ADHD, autism spectrum disorders, obsessive-compulsive disorder, and PTSD. Projects related to the toxicology and safety testing of novel imaging ligands are supported through a separate initiative, the Toxicological Evaluation of Novel Ligands Program (http://www.sri.com:/pddd/nimh/nimh-tox.html). For more information contact Jamie Driscoll( jdrisco1@mail.nih.gov )
  
• NINDS: The mission of the National Institute of Neurological Disorders and stroke (NINDS) is to reduce the burden of neurological disease - a burden borne by every age group, by every segment of society, by people all over the world. NINDS is interested in receiving requests for NIH-RAID Pilot support that seek to develop new small molecule interventions for neurological disorders. The institute is responsible for research on many diseases of the nervous system, both rare and common, and a list of relevant disorders is available on the NINDS Web site: (http://www.ninds.nih.gov/disorders/disorder_index.htm) For more information contact Thomas Miller, Ph.D. (MillerT@ninds.nih.gov).

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Eligibility

The NIH-RAID Pilot is intended for use by academic discovery laboratories and not-for-profit organizations. It is expected that each application will have a Project Leader with an academic appointment in an institution with an NIH-assured Institutional Review Board or involve formal collaborations with a staff member of such an institution.

Can companies use the NIH-RAID Pilot?

The first goal of the NIH-RAID Pilot is to facilitate access of academic centers and investigators to opportunities for therapeutic development arising from their research. In the pilot phase, ideas arising solely from a corporate source without academic collaborators are not eligible. Nevertheless, it is recognized that partnerships with for-profit entities are critical in the development process, and products may be licensed to for-profit partners and still be eligible for the NIH-RAID Pilot. Project Leaders are also free to negotiate with companies for additional licensing opportunities while the NIH-RAID Pilot projects are under way. For awarded projects, representatives of the for-profit partner may attend meetings with the Developmental Therapeutics Program staff, but the primary point of communication will be the Project Leader.

Is NIH-RAID open to non-U.S. applicants?

Yes, provided the applicant meets all the other eligibility criteria. Additionally, please note that the U.S. DHHS Office of Human Research Protections (OHRP) requires that the recipient of any tangible product intended for human use have an OHRP assurance number, regardless of the recipient's location. If the applicant's institution does not already have an OHRP assurance number, their institution will need to obtain one before the NIH can manufacture vialed drug product.

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Intellectual Property Rights

It is expected that originating parties will have acquired or be in the process of acquiring intellectual property protection prior to involvement of the NIH-RAID Pilot. All intellectual property relevant to the project needs to be fully described in the application and the Technology Transfer Form .

As noted previously, most NIH-RAID Pilot tasks will be accomplished by the use of contracts in the Developmental Therapeutics Program of the NCI. Normally, NCI will not acquire intellectual property rights to inventions made by its employees with Research Material under NIH-RAID, unless the originating investigator and NCI agree that to do so would be in the best interest of the project. If the NCI does file a patent application, the originating investigator will be given the opportunity to negotiate for an exclusive license under procedures set forth in 37 CFR Part 404.

NCI contractors, under the Bayh-Dole Act, may elect to retain rights for a contribution they make that rises to the level of invention. However, NCI contractors have, as a term of their funding agreement, agreed to offer a first option to the originating investigator for license negotiation. Certain other contractors or subcontractors may be subject to a Determination of Exceptional Circumstances through which their rights in subject inventions made using Research Material may be assigned to the originating investigator. Should an invention occur, the originating academic party will thereby have acquired a valuable potential ally in commercializing the subject of the research and may have acquired additional intellectual property if the latter was derived from collaborative research.

NIH Material Transfer Agreements (MTAs)  will be put into place at an appropriate time after the approval of a project and will form the basis for sharing confidential information with NCI and the co-sponsoring Institute.

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Critical Dates

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