Small biotech companies around the world are at the forefront of developing innovative new health solutions. But due to their size, many don’t have the in-house expertise or manufacturing capacity to move their molecules seamlessly through the drug-development journey on their own. Most need to partner with outside contract development and manufacturing organizations (CDMOs) to fully develop their drugs and get those life-saving medications into the hands of the patients who need them.
To explore the needs of small biopharma companies as they develop such partnerships, in January 2019 BioPharma Dive’s Brand Studio surveyed industry leaders on their drug-development concerns. Three issues emerged as their principal priorities: getting drugs to the clinic quickly, finding expert help, and having adequate funding.
Part one of this article series focused on the first priority, exploring what small biotech companies might look at when assessing how fast a partner can get their drug to clinical trials.
In part two, we’re looking at the second priority: how finding the right partner to provide expert help can move a company further along the drug-development journey, enabling a seamless scale-up from Phase II trials to commercial supply, including the all-important submission of a Biological License Application (BLA).
Here are three steps to consider when assessing potential partners:
1. Assess the company’s track record in getting partners to BLA
Many CDMOs make big promises, but can they deliver? A crucial step in selecting a partner, who can get your molecule to BLA, without compromising speed or quality, is assessing their track record, says Dr. Stéphane Varray, head of commercial development launch, mammalian & microbial development & manufacturing at Lonza.
“Ask the company how many BLAs they have supported, and whether they were successful with those filings,” says Varray. Get the details on how those processes were handled, any problems they ran into along the way, and how they solved them.
Inquire what their plan would be for your molecule. Dive deep into the details. What steps are they going to take? Are they really going to be able to commit to the timeline they are presenting to you? Does their plan seem realistic, or do they seem to be cutting corners that could lead to problems?
Also look at their global expertise. Do they have experience in working with regulatory bodies in different geographic areas? Have they worked with the U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA), as well as regulatory agencies in other countries?
2. Consider the company’s experience and flexibility in delivering a supply of your drug
Another important area to assess is determining if your potential CDMO partner can quickly scale-up production of your drug, if needed. Have they done this in the past? Is their manufacturing business model flexible enough to meet your expected needs, as well as any rapidly changing needs?
To get a sense of this, ask what their manufacturing capacity is now, and if they have plans for expansion. Do they have capacity from 1k all the way through 20k to accommodate demand volatility? Ask if they have the ability to reserve capacity for customers ahead of time. Can they develop a customized modular solution to meet your needs?
In addition, key features to evaluate are the technology and process design they will use – for both drug substance and drug product. For example, are they using single-use process technology that can enable quick adaptation to new processes, formats, titers, lower contamination and raise efficiency? Do they use tools in the drug product manufacturing, such as machine-learning algorithms, to decrease drug substance waste?
3. Evaluate how the CDMO will minimize your risk
Small-biotech company leaders place a high value on partners that can minimize errors and risks in the drug development journey. In the BioPharma Dive Brand Studio survey, when asked what factors are most important in choosing a CDMO partner, the second most important response was finding a company that will help them to “avoid unexpected delays or poor quality work.”
A key factor in minimizing risks is simplifying processes. When evaluating potential partners, ask about their ability to manage the entire life cycle of your molecule. Will they handle everything in-house, end-to-end, or will they outsource some steps in the process? Adding additional partners adds risk; tech transfers and other complications raise the risk of errors that can slow down your timeline, generate rework and lead to a significant loss of revenue.
Eliminating tech transfers — managing all clinical and commercial production under one roof, using the same assets — minimizes risks. In addition, a company with deep expertise can further minimize risks by recognizing when a particular process might not be the best option for your product; that allows them to make changes early on, before any costly delays and re-configuring occur.
Another important factor is finding a company that has the depth of knowledge needed to do problem-solving. Biological systems can be unpredictable. You want to choose a CDMO that has the ability to solve problems quickly, should they occur.
Finding the right partner to take your company further on the drug-development journey is a critical part of ensuring a successful launch of your molecule and getting it to patients. Take the time to assess your own internal capacities and where you need help, and then find the best experts that will work with you for the long haul and can meet all of your eventual needs.
Welcome to Ibex™ Solutions
Ibex™ Solutions consist of three CDMO offerings: Ibex™ Design, Ibex™ Develop and Ibex™ Dedicate that span the complete biopharmaceutical lifecycle - from preclinical to commercial stages, from drug substance to drug product, all in one location. The variety of solutions offerings provide the flexibility of a complete program - from gene to drug product, or the option to drop in at a later stage depending on where you are in your journey.
- Ibex™ Design is a pioneering gene to vial package delivering drug product for your clinical trials within 12 months. Also included is a manufacturing slot reserved for your clinical resupply needs.
- Ibex™ Develop accelerates market readiness and can help you achieve BLA submission in 22 months. It offers tailored capacity to flexibly reach your clinical and commercial expansion requirements.
- Ibex™ Dedicate is a technology agnostic supply solution that helps you save up to 30 months-time to market and control investment risk with tailored business and ownership models.
Learn more at www.ibex.lonza.com.
BioPharma Dive’s Brand Studio surveyed industry leaders on their plans for developing new drugs. Close to 200 people responded to the survey. The majority of them (61%) were from small/start-up or mid-sized biotech companies, while 25% were from large pharma companies. Most (60%) are developing biologics/large molecules, and plan to develop their drugs by themselves or with a contract development and manufacturing organization (CDMO) partner, rather than selling or licensing their drugs to another company.
When asked about their “biggest concern about taking a molecule through early development,” speed was clearly the top issue for many. Thirty-seven percent of respondents ranked “We are worried about moving quickly enough, without adding risk to our program,” as their top concern. The second highest-ranked issue was “having enough funding to take our project to the next phase.”
Speed was also a primary concern in selecting a CDMO partner. Of survey respondents, 37% ranked “Speed: We need a CDMO that can provide accelerated timelines without adding risk to the project and delivers on stated timelines,” as a top issue, second only to expertise (41%).
Likewise, “Being able to move forward quickly,” was ranked the top concern by 46% of respondents, when asked their thoughts on what was important when considering applying for an accelerated approval pathway