The effort will be supported by a Broad Agency Announcement (BAA) officially released Dec. 15, 2014.
A rare (or orphan) disease is generally considered to have a prevalence of fewer than 200,000 affected individuals in the United States. Emerging viral diseases can be defined as infections that have newly appeared in a population, or have existed but are rapidly increasing in incidence in that population or geographic range.
The study of serious rare or emerging viral infections in these populations provides unique and special challenges and treatment options are frequently sparse. Consequently, the development of effective therapies or therapeutic strategies for rare viral diseases or viral diseases in special populations remains a significant unmet medical need.
Natural history studies that focus on understanding the range of manifestations, disease progression, disease presentation in various populations and the establishment of biomarkers of clinical progression and correlates of clinical outcome are being recognized as critical to facilitate effective product development programs and overcome issues that have led to stalled or failed product development strategies.
For the purposes of the forthcoming BAA, the targeted patient populations are those for which there is a significant unmet medical need for therapeutic options for rare/emerging viral diseases that are not being addressed by the pharmaceutical industry or other significant clinical initiatives, including: children (neonates through adolescents); the elderly, transplant recipients, and pregnant women.
The following are some examples of viral diseases of interest which pose an increased risk to special populations:
- Congenital Cytomegalovirus (CMV)
- Serious Enteroviral Diseases
- Epstein-Barr Virus (EBV)
- Neonatal Herpes Virus
- Human Herpesvirus-6 (HHV-6)
- Viral Encephalitis
- Lassa Fever Virus
Eligible clinical studies under the BAA include:
- Interventional trials of safety and effectiveness for treatments
- Natural history studies of the target viral diseases to assist in the design trial endpoints
- Validation of biomarkers or surrogate markers of clinical responses and safety
- Validation of diagnostic tests for predicting clinical response to therapy
- Proof of principle studies to further product development
- Exposure/exposure-response studies to optimize therapies
- Assessment of the emergence of resistance to antiviral therapies
- Analysis of existing databases or patient charts
Contracts awarded under this mechanism will not support surveillance or transmission studies. Further details are available under Solicitation Number: BAA-NIAID-DMID-NIH-AI-2014028.