CWWeekly presents this feature as a spotlight on issues faced by executives in clinical research. This week, writer Karyn Korieth spoke with Ubavka DeNoble, M.D., corporate vice president at Parexel, who leads the company’s BioPharm unit, a dedi­cated team focused on the unique needs of small and mid-sized biopharmaceutical companies.

Q: Can you describe the current landscape for small and mid-sized biopharmaceutical companies? How has it changed?

A: One critical change is that the cumulative market cap for biotech has dropped over the last nine months. There are several explana­tions for what has happened, but one is that investors are now trying to find different ways to invest their money with the exception of two areas: new technol­ogy platforms and oncology. We now have a significant interest in drug development for rare diseases. For example, in 2015 alone, more than 40% of the 45 drugs approved by the FDA were approved for rare diseases.

Small companies also have intellectual properties, but not enough capital. They are struggling. The number of IPOs has weakened. During the last five years, even though the IPOs have raised money that is reasonable, it is not enough. IPOs are happening, but not at the level that they should be.

Another thing that will define the market is biosimilars, which is related to generics. Biosimilar approvals first started in Europe and have made their way to the U.S. The FDA has approved three biosimilars this year and there are many more on the waiting list. We have to think about how biotech companies feel about these changes because drug pricing is a big part of the game. Having biosimilars and generics approved is definitely creating anxiety within pharma.

Q: What are some of the specific challenges small and medium-sized companies face?

A: They are facing quite a few challenges right now. In order to raise capital, they struggle to make value-based arguments for their medicines. Then, those who are investing money—venture capital and pharma—are not diversifying their investments among therapeu­tic categories. At this point, money for experi­mental biotech, for example, is not at the level everyone would like it to be.

We anticipate that experimental biotech, which includes gene and cell therapies, will bring many changes to the industry. In biotech, there is a standard approach of optimizing the study protocol so that you have a good trial design, which eliminates things that are not necessary. You conduct proper feasibility so that you will have the right patient access and good patient recruitment. All of this is great. But going forward, what will play a critical role is predictive analytics and real-time data access. There are already many gadgets patients can use to provide real-data access. Many of the big players, however, are sometimes hesitant to join with small biotech companies doing this work. They prefer to be with more well-established companies.

When investigating rare disease treatments, genotyping and biomarkers are critical elements of drug development because these elements can help identify the correct targets and popula­tion for a drug. This is all part of the big picture relating back to tailored medicine. Biomarkers help to confirm that you are on the right path. But many small and medium-sized companies don’t have the capacity for this work in-house. Our company has positioned itself as a partner who can advise these small biotechs. That is a core philosophy at Parexel. Small biotech com­panies have the science and we have expertise in drug development.

Q: What role do you see for small and medium-sized companies in drug R&D going forward?

A: Our market research shows that 80% of new molecular entities (NMEs) come from small and medium-size companies. These companies are innovative and the intellectual incubators. Coupled with technol­ogy, they will have an impact on analysis and the way healthcare will be handled going forward. There is a new generation coming from academia who have a high level of curiosity, are very familiar with technol­ogy, have certain visions and ideas and are fear­less. For example, they have the ability to analyze electronic patient records. They have devices and connect patients to those devices to collect real-world information for companies that are developing drugs. They will use patient registries and genotyping to design clinical trials that will be very beneficial. In some ways, they are the leading force that will completely change how things are going to happen in the future.

Working with small and medium-sized com­panies is exciting. This is a sector that will likely exhibit the best growth. Shifts toward tailored medicine are definitely coming. There are amaz­ing stories of success, but you also have stories and a certain signal that the industry as a whole, especially investors, needs to make certain adjustments regarding a return on expecta­tions. What has happened with approved drugs recently is that there are huge expectations for them, but they didn’t reach the target control of pricing. It’s a big issue. It’s definitely going to create a very interesting environment.

This article was reprinted from Volume 20, Issue 41, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »