The Therapeutics Development Network (TDN) Consulting Services Program is one of the best ways to harness the expertise of experienced CF clinical researchers. The following are some of the areas in which we can collaborate with study sponsors and principal investigators.
TDN consultants can review the preclinical components of regulatory submissions and advise on specific animal models or experimental data that will enhance investigational new drug applications.
Our goal is to help sponsors ensure that toxicology packages meet regulatory requirements and support the proposed clinical development program.
Specific areas of expertise include:
- Animal model development and characterization
- Development and/or review of pharmacokinetic data
- Regulatory issues related to small molecule and biologics development
Clinical Development Plans
TDN Consulting Group members have extensive experience reviewing overall clinical development plans, outlining the requisite studies and helping sponsors and investigators achieve their goals in an efficient manner. We can provide accurate information about current practices in CF clinical care and insight into how a compound might be utilized within the CF community.
Our goal is to ensure that the development program not only meets regulatory requirements, but also supports the appropriate labeling claims for the product and patient group.
Experienced personnel from the TDN Consulting Group can work with the sponsor and principal investigator to develop a clinical protocol. We will assemble a writing team that includes an experienced study manager and a biostatistician.
Principal Investigator Selection
If a principal investigator has not already been chosen, we can assist in identifying one to assist with protocol development and lead study conduct.
Statistical Design and Analysis
The senior biostatisticians at the TDN Coordinating Center (TDNCC) are experts in the statistical design and analysis of CF clinical studies.
Their focus is on developing the most efficient statistical methodology and clinical design for the orphan disease setting, where sample sizes must be limited. They also play an integral role in developing efficient analysis approaches and determining sample size requirements for novel CF outcome measures.
These activities are enhanced through use of archived data maintained by the TDNCC from previous and ongoing studies, as well as the CF Foundation national patient registry.
As part of the TDN Consulting Group, the statistical team uses this expertise to provide comprehensive input to sponsors and investigators on clinical development plans, endpoint selection and study design.
Novel CF Outcome Measures
Specialized procedures or assays are often required to measure outcomes in both proof-of-concept and confirmatory CF clinical studies. In addition, the use of standard clinical outcome measures (such as pulmonary function and exacerbation rates) in CF studies may require specialized techniques or standardization efforts.
TDN consultants can advise sponsors on the use and analysis of these outcome measures during discussions of clinical development plans and protocol design. We can also provide training to study teams on implementation of these outcome measures.
Here are some of our areas of expertise:
- Microbiology outcomes, including quantitative changes in bacterial load, identification of unusual CF pathogens and specialized susceptibility testing
- Inflammation outcomes, such as evaluation of sputum cytology and quantitative changes in inflammatory mediators, proteolytic enzymes and associated molecules in sputum, bronchoalveolar lavage fluid, urine or serum
- Nutritional and antioxidant outcome measures, such as vitamin levels and biochemical markers of nutritional status and oxidant-antioxidant balance
- Specialized pulmonary function testing for infants and toddlers including the raised volume technique, plethysmography, forced oscillation and multiple breath washout
- CFTR functional measures, including nasal potential difference, sweat chloride and intestinal current measurement
- Imaging-based measures, including radiographs, computed tomography and magnetic resonance imaging
- Mucociliary clearance
- Patient-reported outcomes
Study Implementation Support
When the time comes for study startup and implementation, the TDN Coordinating Center can be a key resource. The TDNCC offers a variety of support services, including overall study management, site monitoring, data management, biostatistics, research pharmacy support and medical monitoring.
Even those who already have a clinical operations team in place may want to take advantage of our CF expertise and insights into the CF community. For example, sponsors have found it valuable to arrange TDNCC support for key areas such as:
- Developing CF-specific aspects of study documents, including protocols, study manuals, consent forms, subject instructions, source documents and case report forms
- Reviewing draft documents to identify any CF-specific logistical issues
- Developing site study cost estimates
- Performing initial site assessments or advising on site selection
- Participating in investigator meetings
- Training site monitors who are new to CF
- Facilitating interactions between the sponsor or CRO and clinical sites, TDN National Resource Centers or other vendors
If you would like more information about our services or would like to begin the consulting process, please contact us at TDNCC@seattlechildrens.org.
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