EARLY-STAGE CMC &
CLINICAL DEVELOPMENT
Our consultants develop programs to assess drug efficacy, safety, tolerability, pharmacokinetics, pharmacodynamics, and dose-response from first-in-human through proof-of-concept clinical trials. We establish go/no-go decision points while ensuring the generation of comprehensive, robust data sets to achieve regulatory endorsement. Our strong emphasis on critical CMC activities ensures the timely supply of validated API and drug product for clinical and nonclinical studies. NGT provides the following Strategic and Operational services:
Strategic
- Portfolio planning
- Evaluate options for accelerated development and approval, including:
- Orphan drug designation and rare pediatric disease designation
- Fast track, breakthrough therapy, accelerated approval, priority review designation, and EU’s accelerated assessment program (PRIME)
- Consolidation of phases
- Establish that the desired drug product attributes target the disease and that the development plan targets the desired clinical outcome
- Accelerate timelines and minimize development costs through innovative clinical trial strategies that consider the following:
- Unmet medical need and place in therapy
- Clinical pharmacology, biomarkers, and pharmacodynamics
- Clinical endpoints, patient populations, and comparator groups
- Safety/tolerability
- Disease natural history and published literature
- Evaluate dosage formulation (e.g., oral, iv, fixed-dose combinations) for commercial readiness
- Determine whether API and drug product manufacturing processes will support clinical trial supply demands
- Regulatory engagement strategies tailored to file INDs, initiate human studies, rapidly advance development, and achieve approval
- Begin addressing market access and reimbursement challenges
- Plan for advancement of the program from early phase to late-phase development and beyond
Operational
- Analyze nonclinical study data and reports
- Create Target Product Profiles (TPPs), Product Development Plans (PDPs), and project Gannt charts
- Develop pharmacokinetic/pharmacodynamic plans and design/oversee clinical studies
- Author study synopses and protocols, and prepare sponsor case report forms
- Interpret clinical, pharmacokinetic, and pharmacodynamic data
- Develop commercial API manufacturing process and advise on appropriate technology platforms
- Develop commercial formulation and dosage manufacturing processes and advise on appropriate manufacturing technology platforms
- Identify and manage outsourcing resources, e.g., CROs, clinical study sites, safety/tox studies, CMC development, and API/DP manufacturing (or support/complement internal operations)
- Liaise directly with regulatory agencies on behalf of the sponsor
- Author briefing materials and prepare for/participate in regulatory meetings
- Prepare and facilitate submission of regulatory documents, including Investigator Brochures, Investigational New Drug (IND) and Clinical Trials (CTA) applications
CONTACT
Michael K. O’Brien, PhD,
President & CEO
860-207-8135
[email protected]
CASE STUDY- Leveraging Early-Stage Clinical Data, Enabling the Start of Phase 3
Situation
After completing Phase 2 clinical trials for a small molecule cardiovascular asset, a pharmaceutical company wished to advance a new salt form of the active moiety, possessing superior physiochemical properties, into Phase 3. The company hoped to leverage data from a Phase 1 PK/PD comparator study with the FDA to mitigate the need for larger Phase 2 clinical studies.
Solution
NGT subject matter experts reviewed the data in the clinical study report (CSR), researched public-domain literature, and consulted with Key Opinion Leader’s. A report was written outlining approaches that would hold up to regulatory scrutiny.
Impact
The client accepted NGT’s recommendations, commencing dialogue with the FDA, and laying the groundwork for entry into Phase 3.
