Addressing manufacturing and technologies strategies to accelerate market entry is one of BPI’s highlighted themes for 2019. In partnership with our conference colleagues in Informa’s KNect365 division, this already has been a shared theme, reflecting the general goals of the industry and related advice from its regulators.
BPI’s summer 2018 preconference ebook included interviews by Dan Stanton (editor, BioProcess Insider) with speakers previewing their talks for the BPI Conference in Boston on 7 September 2018. Two of those conversations focused on ways their companies are reducing time to market. Then at the September event, a third interview on the theme was conducted by BPI’s senior technical editor, Cheryl Scott, at the BWB TV desk. Summaries are offered here, with links to the full recordings online.
Regina Au (BioMarketing Insights)
Stanton spoke with Regina Au (principal of new-product planning and strategic commercial consultant at BioMarketing Insights), who presented as part of the Speed from Gene to Market track.
Can you elaborate a bit on the topic of your presentation? In product development, your target product profile (TPP) is your commercial profile in determining which product specifications you need to meet as critical elements and how to meet the specific needs of your customers to be successful in the market by being better than the competition. Basically, I’m trying to bring commercialization into product development to derisk that process from the business end.
Who are your clients? It would be anybody who is developing a product or service from “big pharma” all the way down to a start-up company.
How would you serve those clients? Large multinational pharmaceutical companies have internal people to do this work — in fact, that’s how I got my experience. I’m just bringing that experience from “big pharma” out into the consulting world. Today with all the changes and companies acquiring other companies, people are streamlining everything. However, the industry does have a lot in its development pipeline. Companies may have more projects than human resources in terms of the people to work on them. Or a business unit conducting “due diligence” may not be high on the priority list.
If a company doesn’t have the internal people to do it, or if it needs to happen right away, or it doesn’t have in-house specialty expertise, that’s when it needs to hire a consultant. I have a lot of experience in the pharmaceutical, biotechnology, medical device, and diagnostic industries, so I can look at things from a broad perspective. And diversity is what everybody is talking about today: diverse viewpoints that help companies succeed.
You mentioned streamlining. Is this a risk to drug development? Yes, companies are streamlining their staffing. Where the risk comes in — particularly for companies who don’t do their due diligence — is that you may miss something. For example, when you bring commercialization into product development, you know your end game and you can work back from there. When you know what your end game is and work your way back, you can make sure that you cover all the pieces.
But companies that rush to get to the investigational new drug (IND) stage may not perform all the necessary due diligence. So they might come out with a project that doesn’t meet the terms of physicians, for example, or patients. And that will influence your TPP: People might not want to use the product you put out.
I’m guessing that, like in manufacturing, a mistake made in the early phases of development can’t be rectified later. It’s going to be very costly and very time consuming to do so. I’ll give you a simple example: a target or lead candidate that shows potential in terms of having activity. You still have to develop it to a TPP. You put together a commercial profile by analyzing the market and technology trends. Everyone usually thinks about competition, but people don’t always think about the technology trends or where the market is going.
Consider the technology trends. If your technology is going to the left, but everyone else’s technology is going to the right, then people might get the perception that yours is a better technology because it’s different, or you might be using an older technology when everyone wants what’s new. If you don’t take that into consideration, then you might end up getting your product approved only to have people say, “That’s nice, but we already have products that are in the same class. We want new technology because we know it’s supposed to work better . . .” or “That’s nice, but we’re fine with what we have.” Things like that can hinder your drug development.
If you go too far down the wrong path — if you go to marketing and discover that very few people want to use your product — then you just wasted 10–15 years (the average drug development time span to market) and $2.6 billion (the average amount of money spent). That’s an awful lot of money you’ll be spending, and you don’t want to find out that you don’t have a market for your product and people won’t be using it.
Have you ever come across an example of a company that has gone to that extreme and wasted so many years of development and money? I recently worked with a company that came out with a product and thought it would be great on the market. But the point is that people want newer and better products. So the product had potential in terms of working, but there were a lot of other products on the market for that indication already.
The question became, “Where do you want to position your product?” That’s an important part of your TPP and your indication. This company’s answer was: “Well, when everything else fails, they’ll use our product.” If you position your product that way, then that means patients will use everything else before they get to yours, so there is only a slim chance that they’ll end up using your product. In that particular class, other products work well. Not every product works for every patient, but it’s not going to be a large-enough market to bring the necessary return on investment so the company can spend more money on further R&D.
What should be the key take-aways from your presentation? I have three main points. The first is that your TPP influences the indication you pursue, which affects your regulatory path. The second thing is that you need to satisfy all your stakeholders, which will influence your TPP. In terms of indication, it will also influence your clinical trial designs because of reimbursement. Payers want your company to show that short- and long-term data and how the product will save the healthcare system money. And the final thing is early due diligence before you file. This allows you to make changes. Once you file an IND, everything becomes public. If you do it early enough and then change your mind, you can focus on something else.
When you think about how much R&D costs, it’s inexpensive to make changes very early on. Most (80%) of the dollars you’re going to spend will be on clinical trials. That’s why I’m emphasizing that you really need to make changes early. Work everything back to the beginning so you don’t miss anything. If you do that early enough, then you can make changes and they won’t be expensive. You don’t want to go through clinical trials and find out you don’t have a large-enough market for what the company needs. Is it a “nice-to-have” or a “must-have” product? Unmet medical need is critical.
Gene Lee (EMD Serono)
Stanton also spoke with Gene Lee (senior director and head of the protein and cell sciences group at EMD Serono). Lee was a session chair in the Speed from Gene to Market track, speaking about his company’s efforts to ensure seamless integration of projects between R&D and manufacturing.
Can you tell us a little about that work? Like many other companies pursuing novel biologics in the fields of oncology and immunology, EMD Serono has recognized that we need to bring our molecules into the clinic quickly. It’s a highly competitive space. The targets available to us for therapeutic intervention are limited. So we need to bring our molecules into the clinic quickly and answer the fundamental questions that our research teams are interested in.
Several years ago, the company embarked on a series of initiatives geared toward helping those research teams find more efficient ways to work together, using our resources more wisely, and homing in on the critical experiments to answer the question of whether our drugs will have the therapeutic benefit of interest. But the question remained, “Are chemistry, manufacturing, and controls (CMC) a bottleneck in our desire to build quickly into the clinic?” We focused initially on translational aspects of our molecules, the processes necessary to move projects from the preclinical stage into clinical trials. At a certain point, we also took a deeper look at CMC development processes and associated timelines. We found out through a number of industry surveys that compared with those of our industry peers, we were quite a bit slower.
In fact, we specifically had the Latham BioPharm Group consultancy survey a number of our industry peers asking some very specific questions to focus in on those areas that needed the most intervention and help us come up with a solution. We recognized that we needed to bring our molecules into the clinic more quickly than we were. Some interesting things came out of that survey that led us to initiate a strategic initiative, including participants from our R&D, manufacturing, and quality organizations. Over two years with a number of face-to-face meetings, we identified inefficiencies and likely areas for intervention. It was painful at times to identify our own processes that were not working well. But we worked through it and came up with a couple of recommendations. (More information about EMD Serono’s work is provided in the previous article.)
One nice outcome from this initiative is that we piloted our recommendations on three projects and in all three cases were able to shave six months off the CMC development timeline. And that put us right at the industry benchmark. So we felt confident that with additional attention and focus on those efficiencies, in a short time we should be able to accelerate even further and be faster than benchmark. But it’s a good start.
Eun Young Yang (Samsung BioLogics)
As part of the BWB TV interviews, conducted on site during the Boston event, Cheryl Scott spoke with Eun Young Yang, executive director, head of the CDO business team at Samsung BioLogics. Yang shared insights into why Samsung BioLogics is expanding to become a contract development and manufacturing organization (CDMO).
Why emphasize the “D” for development? Does this reflect market changes? We have been seeing increasing development needs from biopharmaceutical companies and for many biologics in the development pipeline. Our clients are looking for reliable development and manufacturing partners. They otherwise tend to develop a focus on R&D rather than on process development in manufacturing. By working with us, they can save on capital expenditures and use our development and manufacturing services. With our help, they can launch their products much faster into the market.
What kind of services are you adding, and what is your expertise in terms of molecules and product modalities? We are a one-stop service provider, integrating development and manufacturing. We provide cell-line development, process development, drug substance production, and formulation as well as analytical method development through clinical and commercial production. With end-to-end service integrating drug substance and drug product at one site, clients do not have to deal with multiple vendors — saving them both cost and time.
Biologics take an average of five to 10 years to go from discovery to product launch. How do your timelines compare with the average? Our development services provide 16 months from cell-line development to investigational new drug (IND) filing. We can fast-track that down to 13 months at a client’s request. For the commercial section, we’ve expedited the process of regulatory approval to 19 months, down from 21. Among other efficiencies we offer clients, we keep our cost of goods at about 40% less than that of other pharmaceutical companies.
What do you see for the future? We expect to see an increased need — perhaps 40–50% — for process development and manufacturing services by 2022. Our goal is to become a long-term strategy partner for process development and manufacturing for pharmaceutical and biotechnology companies.
S. Anne Montgomery is cofounder and editor in chief of BioProcess International. Dan Stanton is editor of BioProcess Insider. Cheryl Scott is cofounder and senior technical editor of BPI; firstname.lastname@example.org.