COLLABORATIVE PROGRAM TO ACCELERATE SMA THERAPEUTICS DEVELOPMENT

RELEASE DATE:  December 17, 2002  

NOTICE:  NOT-NS-03-005

National Institute for Neurological Disorders and Stroke (NINDS)
 (http://www.ninds.nih.gov)

The National Institute for Neurological Disorders and Stroke (NINDS) is 
considering issuing a contract that would provide resources and 
expertise to help us accelerate therapeutics development for Spinal 
Muscular Atrophy (SMA). NINDS is attempting to identify competent 
sources to establish and coordinate pre-clinical research activities 
leading to a new treatment for SMA, and is seeking input from 
experienced sources into the design of a proposed statement of work for 
the project. 

Background

The NINDS wishes to develop a treatment for SMA, a devastating 
neurological disease of childhood. SMA is an ideal candidate for 
targeted therapeutics development for two reasons. First, it is caused 
by the loss of function of a known gene product. Thus, there are 
tractable strategies for developing treatments based on replacement of 
the lost function. Second, SMA is the most severe of the common 
genetically recessive neurological diseases. Currently, no effective 
treatment for SMA exists. 

The overall objective of the proposed contract is to accelerate the 
development of a treatment for SMA through the establishment of an 
innovative SMA translational research program. This program will pursue 
parallel research avenues for therapeutics development in a 
coordinated, rapid, milestone-driven process to identify a testable 
therapy suitable for clinical trials. Clinical trials will not be 
undertaken as part of this program. The contract will establish the SMA 
research program and coordinate its progress, implementing a fast and 
focused process in response to the NINDS strategic plan.

Organizations capable of fulfilling this effort must be able to: 1) 
Establish an SMA Research Program consisting of institutions capable of 
providing the scientific and administrative expertise necessary to 
perform therapeutics development studies for SMA. 2) Manage a strategic 
plan that must include processes for a) establishing research 
priorities; b) reviewing, modifying, approving and disapproving 
research studies; c) allocating resources; d) monitoring and evaluating 
progress and maintaining effective working relationships. 3) Provide 
input on research progress to a Program Oversight Committee. 

Information Requested

Information and advice in the following areas will aid in the design of 
a statement of work and solicitation for establishment and oversight of 
the SMA research program. We ask that organizations that have performed 
such services identify critical criteria to include in the solicitation 
and describe their capabilities to meet these criteria. This 
information can address performance measures, standards and incentives, 
as well as possible criteria for judging success. The information 
supplied in response to this request need not be limited to these 
areas. 

1. Establishing the SMA Research Program

The SMA research program will be established through subcontracts, 
awarded by the contractor, to individual research laboratories. The 
requirements for these subcontracts will be defined by the NINDS 
strategic plan for SMA therapeutics development. The contractor will be 
responsible for soliciting and reviewing research proposals with a 
targeted review and award cycle of approximately 4 months. We 
anticipate 3 standard application receipt dates per year. At the 
discretion of NINDS, up to 6-10 solicitations may be released per year, 
with 5-30 research proposals submitted per solicitation. The contractor 
will establish a scientific review committee(s) that could meet as 
often as 3 times per year. Based on the results of the scientific 
reviews and the priorities of the program, final award selection 
recommendations would be forwarded to NINDS. The NINDS will ultimately 
be responsible for final award selection (or: for approving all award 
selection recommendations). With respect to the establishment of the 
research program: 

i. Describe your capabilities for the efficient and fair 
solicitation and review of research proposals for a pre-clinical 
therapeutics development program.

ii. Describe the criteria and strategies you would apply in 
establishing a peer review committee and in managing data related 
to the reviews.

iii. Describe the expertise and level of effort of personnel 
necessary to solicit and review proposals according to the above 
schedule.

2. Subcontract Award and Management

Research conducted by the SMA network will be funded through awards 
issued by the contractor as subcontracts.  It is anticipated that up to 
6-12 subcontracts may be issued each year.

i. Describe the personnel and database requirements associated 
with awarding and managing multiple subcontracts, including 
quality control procedures.

ii. Describe your capabilities for evaluating the budgets of 
subcontract proposals.

iii. Describe your strategies for insuring that subcontracted 
research projects are completed on schedule and within budget.

3. Program Management and Evaluation

To maximize the rate of progress, researchers in the SMA network will 
be required to interact and collaborate with each other and to report 
progress regularly to an oversight committee selected by NINDS. The 
contractor will be required to develop a meeting schedule to facilitate 
these interactions. In addition, the contractor will be required to 
evaluate the progress of the subcontracted research against the goals 
of the NINDS strategic plan and communicate these evaluations to the 
oversight committee.

i. Describe your experience with strategic planning efforts.

ii. Describe your experience with the evaluation of the success 
or failure of scientific programs.

iii. Describe your experience with planning and conducting 
meetings.

4. Personnel

Please provide an overall assessment of the number and mix of 
personnel required to accomplish the tasks outlined above and the 
approximate level of effort required for each. Include a 
description of the scientific and technical expertise required.

This Request for Information is for planning purposes only and shall 
not be construed as a solicitation or as an obligation on the part of 
the Government. The Government does not intend to award a contract on 
the basis of responses nor otherwise pay for the preparation of any 
information submitted or the Government's use of such information. 
Respondents will not be notified of the Government's evaluation of the 
information received. Should a proposed solicitation materialize, no 
basis for claims against the Government shall arise as a result of a 
response to this request for information or the Government's use of 
such information as either part of our evaluation process or in 
developing specifications for any subsequent requirement. Responses 
will be held in a confidential manner. Any proprietary information 
should be so marked.

All respondents are asked to indicate the type and size of your 
business or organization, e.g., Large Business, Small Business, Hubzone 
Small Business, Small Disadvantaged Business, Women-Owned Business, 
8(a), Historically Black College or University/Minority Institution 
(HBCU/MI), educational institution, or other profit or non-profit 
organization.

Responses should be limited to 20 pages, should be identified with RFI 
No. NS-03-005, and are due by February 1, 2003. Please submit three (3) 
copies of your response to:

Patricia S. Denney
Contracting Officer
Contracts Management Branch
National Institute of Neurological Disorders and Stroke
NIH
6001 Executive Boulevard, Room 3287
Bethesda, MD 20892-9531
(For Fedex or courier, use: Rockville, MD 20852)  (The telephone number 
for the Contracts Management Branch is (301) 496-1813.) 

Email responses will also be accepted at: pd22n@nih.gov. NO FAXES 
PLEASE.

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