Guide for executives in small and emerging pharmaceutical and biotech companies to select contract development and manufacturing organization (CDMO) and contract research organization (CRO) partners

1. CDMO AND CRO OUTSOURCING AND
PARTNERING
GUIDE FOR EMERGING & START-UP
PHARMACEUTICAL/BIOTECHNOLOGY
COMPANIES
Irach B. Taraporewala, Ph.D.
Sitara Pharmaceutical Consulting, LLC

3. Need for Outsourcing in Start-Up Companies
▪ Start-Ups have limited bandwidth for achieving targets
▪ Limited Personnel, Lab Space, Equipment, Buildout Capability
▪ Often virtual companies or off-shoots of academic labs
▪ ~ 70% of new drug products under development emanate
from small or start-up companies

4. Stages of Drug Development

5. Outsourcing & Partnering Requirements
▪ API Synthesis
▪ Formulation Development
▪ Preclinical Pharmacology & Toxicology Testing
▪ GMP Manufacturing (Phase I through Commercial)
▪ Analytical Testing and other laboratory services
▪ Packaging and Labeling; Sterilization or Terminal Sterilization
▪ Clinical CRO for Set-Up and Conduct of Clinical Trials

6. Why have a CDMO Partner rather than In-House
Buildout ?
▪ Cost Efficiency
▪ Infrastructure
▪ Experience and Expertise
▪ Capabilities - Faster Time to the Marketplace

7. Ideal CDMO Partner
• Correct Product Quality
• Correct Product Amounts
One that can consistently deliver…
• Meet Correct Timelines
• Ship Product Correctly

8. ▪ Identify Clinical Candidate (API)
▪ Develop Synthetic/Manufacturing Route – Scalable
▪ Analytical Method Development for API
▪ Stability Studies for API
▪ Formulation Development based on Route of Administration
▪ Formulation Stability Studies
▪ IND-Enabling GLP Preclinical/Toxicology Studies
▪ Stage-consistent GMP Manufacture Testing of Drug Substance
(API) and Drug Product for Phase I-III Clinical Trials
▪ Commercial Manufacture
Stages in Product Development

9. Criteria for Evaluating a CDMO Partner
• Range of Services
• Geographical Location
• Capabilities
• Experience & Expertise
• Regulatory Experience
• Confidentiality
• Do services match your needs?
• Convenient to your needs?
• Right Equipment, Scale?
• Personnel Qualifications/Relevant Experience
to your product
• Track Record with Regulatory Bodies
• Safeguards to Confidentiality
Criterion Parameters

10. Criteria for Evaluating a CDMO Partner
• Scale - Size Match
• Scheduling
• Flexibility
• Communications
• Cost
• Personality
Criterion Parameters
• Capability to manufacture at the right scale?
• Available to execute when needed?
• Adaptable to change?
• Transparency, Sensitive to needs
• Meet your budget?
• Can you envision working with
them?

11. Establish In-House Readiness for CDMO/CRO
Partner Selection
▪ Identify potential technical/regulatory/logistical challenges that may be specific
to your project. Identify how the tech-transfer will be done from the company
end.
▪ Get a checklist of criteria for partner evaluation prepared
▪ Do a preliminary search of potential “best-fit“ partner candidates. Narrow down
to two or three
▪ Issue a Request for Proposal (RFP) to the shortlisted vendors. Provide as
detailed a technical description and scope and desired timelines of the
project, so the CDMO or CRO can adequately address the issues in the
proposal
▪ Establish In-House Selection Committee - Multidisciplinary
• Project Management
• Technical/Medical/Regulatory/Finance

12. Evaluation of Proposals in the RFP Process
▪ Specific Related Experience
▪ Team Member Experience/Training
▪ Cost Justification
▪ Approaches to Perceived Challenges
▪ Responsiveness and Communication
Skills
▪ Quality of Service/Consistency of
Service
▪ Quality Systems
▪ Financial Stability/Pending M&A
▪ Regulatory Body Inspection History; GxP
Compliance
▪ Data Capture/Data Management
▪ Problem Resolution and/or Escalation Process
▪ Client Management and Communications
▪ Risk Management/Subcontractor
Management/Statistical Analysis
▪ Hidden Costs

13. QA Audit of Finalist Contenders/Site Visit
▪ Look at Quality Management Systems (QMS)
▪ Evaluate experience and track record with GXP Compliance
▪ Evaluate ability to meet timelines/flexibility in scheduling
▪ Assess general staff attitude; meet specific team and ease of communication
▪ For CDMOs evaluate lab cleanliness, general order and GMP Compliance in the
facility. First impressions count
▪ Look at SOPs
▪ Analytical Instrumentation assessment, specific project equipment
▪ CAPA; Root Cause Analysis Procedures
▪ Business Aspects – Past Profitability, Ability to Sustain Growth, Financial
Stability

14. ▪ Can they do it?
▪ Have they successfully done this before?
▪ Do they have the correct infrastructure, support to meet the project needs
for process, analytical testing, equipment
▪ Support with Regulatory Documentation needs
▪ Are the costs commensurate with the tasks and justifiable?
▪ Do they have the right personnel and team (Technical Expertise,
Experience level, attentiveness, communication skills)?
▪ Can they provide references from past or current “satisfied
customers”?
Final Assessment

15. ▪ Delivery of Goods/Services properly spelled out. Check that the
requirements in the RFP are all met
▪ Written Quality Agreement and Assigned Responsibilities of Sponsor and
Contractor
▪ Quality Commitments to GXP are addressed. Transparent QA/QC
functions. Sponsors and Contract Facilities Are Both Responsible for cGXP
compliance
▪ Legal Restraints and US/Foreign Regulatory adherence are spelled out
▪ Contractually stated that if and when the CRO/CDMO contracts any
services out to third parties that the level of control/quality is not
compromised
Contract Review

16. ▪ Sponsor company is not expected to hand-hold the CRO/CDMO or
vice versa. It has to be a real partnership of a fine balance
▪ The company can delegate GXP regulatory tasks to the CDMO/CRO,
but not the legal responsibility
▪ Short-term/long-term project commitment
▪ If the internal QA system at the CDMO/CRO is deficient in some
manner, the sponsor can bring in rectification
▪ Adaptability of the CDMO/CRO to accommodate any program
change/deviation in scale/timelines/technical or product
requirements
Other Contract Considerations

17. Tech-Transfer Process to CDMOs
▪ Fully open lines of communication are essential
▪ Involve the bench scientists who initially developed the compound or
product – they often have valuable product knowledge
▪ Provide the CDMO as much technical information as you have to aid the
product scale-up and development process; including chemical and
thermal stability, solubility, hygroscopicity, light sensitivity or analytical
methodology data you may have as sponsor in the initial development
phase
▪ Have frequent conference calls/meetings during the tech-transfer
phase

18. Full Service CDMOs versus Specialty CDMOs
▪ Full-service CDMO partners can provide support through all stages of drug development
and formulation for all types of drug substances and project timelines
▪ Full service CDMOs can ensure continuity of the project over the full drug development
cycle. Smoother or quicker transition from development to scale-up to commercialization
▪ Full service CDMOs may have better infrastructure for providing consistent regulatory
documentation and smoother transition through the different stages in drug development
▪ Greater ease of the tech transfer process with a single full service CDMO
▪ Specialty CDMO may be considered when your product is in a specialty niche process that
requires specific non-standard manufacturing or specialized purification available at a
specialty CDMO (highly potent material handling, high pressure reactions,
specialized ultrafiltration/chromatography purification), especially in earlier
stages of drug development
▪ Batch Size/Lag Times can be a deciding factor

19. ▪ Relevant technical expertise in your type of preclinical studies
▪ GLP Compliance record
▪ Regulatory Track Record with USDA and FDA
▪ AALAC accredited/Animal welfare
▪ Timelines to study start and finish – in-life and post-study histopathology
studies, final reporting
▪ Adequate Personnel/Facility Resources/Communication
▪ Data Management – QA/QC
▪ Cost of studies and hidden costs
Pre-Clinical CRO Selection Criteria

20. Clinical Studies CRO Selection Criteria
▪ Relevant technical expertise in your therapeutic area of interest in the
appropriate clinical phase
▪ Credentials/experience of actual study personnel working on your trial
▪ GCP Compliance Record; Regulatory Track record with USDA and FDA
▪ Anticipated Timelines to study start and finish – patient accrual rate estimates
▪ Adequate Personnel/ Facility Resources/Communication
▪ Data and Quality Management – QA/QC and Clinical Monitoring
▪ Geographical reach and access to patients in your therapeutic area of interest
▪ Range of Services – Data, Regulatory Documentation, Biostatistics –
In-House or subcontracted?
▪ Cost of studies and hidden costs

21. Establishing Partnerships with Big Pharma
▪ Start the process early on to get the potential big pharma partner interested
in your program
▪ Investigate potential companies who may have an interest in your
technology/drug product/ therapeutic area.
▪ Identify and connect with their Business Development personnel in your
therapeutic area
▪ Keep them abreast of your progress periodically as your program develops,
sharing non-confidential data
▪ Get your IP portfolio in order before initiating any actual in-licensing
▪ If the big pharma develops specific early interest in your product,
establishing a co-development plan is an option

22. itaraporewala@outlook.com
THANK YOU!