ATMP Development Gets A Boost From Outsourcing Best Practices
By Louis Garguilo, Chief Editor, Outsourced Pharma
Developing novel molecular and biologic therapies may require inventive, highly personalized CDMO relationships, but these innovative partnerships should be built on established outsourcing principles.
A grounding in best practices has never been more important. Our industry continues to develop a widening array of these so-called Advanced Therapeutic Medicinal Products (ATMPs).
In fact, according to a market report and analysis from Nova One Advisor, the global advanced therapy medicinal products CDMO market is estimated to have reached $9.35 billion in 2025, and projected to $53.11 billion by 2035 (at a CAGR of 18.97%) into 2035.
The complex simplified for our purposes here, programs such as cell/gene, xRNA, CARx, and other pursuits broadly require:
- innovation (often complexity) of molecule design
- deep specific knowledge, and also a broad, systemic understanding of biologics and chemistries
- advanced analytics and methodologies
- meaningful experimental production runs, and successful scale ups
- replicable, high-quality processing/manufacturing
- uncompromising fill/finish production
These attributes place increased demands on your CDMOs, and ironically perhaps, bring us back to the least novel part of ATMP development: outsourcing fundamentals.
You have every reason to feel a certain distinction in working on breakthrough science. Just don't let that excitement crowd out the outsourcing disciplines that ultimately determine whether or not that science reaches patients.
The Song Remains The Same
Led Zeppelin got it mostly right. Outsourcing best practices can evolve, but they do so around an enduring set of principles that apply regardless of program complexity or novelty. Here is a look at three starting points.
1. CDMO Search And Selection Criteria
Basic background components in your evaluation of external partners include these constants:
- Historic Perspective
- How long has this CDMO been serving customers – particularly customers of your size and disposition.
- Has this CDMO excelled at handling novel and challenging programs similar or at least adjacent to yours? Are they a “go-to” for ATMPs?
- Track Record
- What is the CDMO’s official regulatory-compliance record and customer reputation. How have they handled interactions with (specific or global) authorities?
- How do they manage customer site visits, audits and regulatory/quality inspections?
- Scientific/Technical Prowess
- Do they have the requisite expertise and experience (i.e., appropriately skilled and tested workers)? Are they an innovative, creative, crew?
- Do they possess the proper instrumentation/equipment, and enough redundancy of which so you won’t be fighting other customers for timely access?
- Location
- Can the CDMO clarify within which facility/production suite/lab each component of the work they will do for you will occur?
- Are there redundant facilities? Are they in different geographic settings?
- Corporate Culture
- Can you grasp whether the CDMOs corporate culture, strategic alignment, short- and long-term goals are compatible with yours? If not, investigate further.
- More elementally but important to your mental health, do you get along and enjoy working with them?
Quality Review might deserve our separate attention.
Some experienced professionals say that at least for name-brand CDMOs, one need not overly fret over quality (at least not to the degree many do).
CDMOs who have been around, answer well the questions asked above, and hold a track record and history … must possess the requisite level of quality to perform their work for customers.
If they didn’t, they’d never had made it so long as an external provider.
Yet other professionals – specifically because we are focusing on ATMPs – believe a better thought process is to never take quality for granted. No CDMO gets a pass.
All deserve your dive into a prospective CDMO’s quality systems, QA and QC departments; discuss strategies for maintaining quality control throughout all activities, from tech transfer to large-scale production.
Think forward, think together, consider all quality and regulatory aspects:
How will you and your CDMO implement quality initiatives to successfully keep up with oscillating regulatory landscapes for obtaining novel-drug approvals?
Editor’s Hint: Whether or not your CDMO will allow such deep analysis as per above prior to your contracting with them can be a telltale of how it might be to work with them long term.
2. Manage the Start
The foundation of a successful relationship has as its base an assimilation of expectations.
In other words, start your due diligence by comparing what you anticipate will unfold against the realities of the CDMO. A fundamental place to begin is with questioning the flow of communication:
- What does the CDMO consider its best practices for effective collaboration? What are your best practices?
Understanding that relationships often hinge on challenges:
- How will development setbacks, deviations, unexpected production delays, failed experiments/batches, be handled?
- What is the governing/oversight structure/hierarchy for managing the organization-to-organization interdependence and problem-solving?
There are roles and responsibilities on both sides, and a third “r” as well – reasonableness. (Practiced on both sponsor and provider sides).
3. Operational Challenges Start At Transfer
Requiring specialized expertise and standard services involves adjudication of capabilities and capacities of your external partner. All judgements – compromises and must haves – add up to a determination of the best overall business, facilities and personnel you can contract.
Once a CDMO is selected, operational challenges begin with the “tech package” and project/program transfers. Much is written and discussed about this initiating work-phase, and with ATMPs and novel projects, advice can run long and at times overly complicated.
We’ll avoid that here, and address two points.
- Transfers (of all kinds) should be a collaborative, communicative, knowledge (and to the extent possible) data-driven process directed by agreed protocols/costs, and clear “transfer success validations.”
- Transfers require a budgeting of realistic timelines, along with contingency strategies should those timelines slip.
Innovation requires new thinking and approaches. It also heightens the need for disciplined execution. With ATMP development – as well as with any program – starting out on a path lined with outsourcing principles may prove your greatest competitive advantage.