Contract Pharma: In the past, small/virtual biopharma companies have been hesitant to reach out to large CROs. How have large CROs become more receptive to them?
Dr. Ubavka DeNoble: Traditionally, small and mid-size biopharmaceutical companies believed they should work with companies of their same size to receive products, service and expertise uniquely tailored to their specific needs. Today, however, large CROs can offer smaller pharmaceutical companies a degree of optimization and regulatory expertise that smaller CROs may not be able to provide.
Large CROs are increasingly focusing on meeting the needs of smaller companies, in part because of the growing importance these companies are playing from an R&D perspective. In fact, 81% of new molecular entities (NME) originate with small and mid-size companies, so it is important for CROs of all sizes to connect to them. Since its founding more than 30 years ago, PAREXEL has served the needs of these customers. And last year, the company developed a specific biopharm unit to further meet the specific needs of small to mid-size companies.
Smaller pharmaceutical companies in particular can benefit from partnering with a one-stop-shop CRO because the company can provide regulatory expertise and operational optimization guidance to help advance a drug through the clinical process, in the most efficient way possible. A one-stop-shop, in addition to first-class strategic design end execution, helps small and medium companies meet the needs of market access and reimbursement agencies at launch time.
CP: Are you seeing an increase in demand from the small and mid-sized/virtual sector?
UD: Yes. According to the FDA’s Center for Drug Evaluation & Research (CDER), small companies had a lower approval rate (50%) than large companies (79%) and mid-size companies (81%). Additionally, large companies received first-cycle approval 58% of the time, compared to 41% for small companies. Therefore, smaller companies can especially benefit from the regulatory expertise that large CROs possess.
In addition to product strategy and planning, CROs bring strategic regulatory expertise to the table. This includes disease-specific regulatory experience and the ability to communicate with regulatory authorities, helping companies take advantage of holding pre-IND meetings, end of Phase I meetings, end of Phase II meetings, and pre-NDA/BLA meetings. Understanding market access strategy and reimbursement agencies that commercialization consultancy teams at large CROs possess is a key element for success. Additional values associated with a one-shop-stop relate to preparation for regulatory audits and inspections, especially inspection of manufacturing facilities and equipment.
CP: In what areas do small and mid-size companies require more assistance? What challenges do they face as opposed to larger companies?
UD: Small and mid-size companies have smaller teams and a greater need for outsourcing. In addition, their pipelines are limited and the future of these companies often depends on a certain drug to win not only marketing authorization, but also to gain access to the right markets.
A number of small and mid-size companies won’t see the development of a drug through to market access. They often license the drug out or partner at a certain stage. For those clients it’s essential to gain the desired results (e.g. proof of concept) in a lean and smart way. These clients are interested in PAREXEL’s EPD (Early Product Development) services and its BioPharm Unit, to help them develop a clear strategy and effective product plans. This way, small/virtual clients can move products through the entire drug development cycle more efficiently.
CP: What specialized services are offered as part of the BioPharm unit?
UD: PAREXEL’s BioPharm Unit offers services through three primary focus areas: Innovative and effective study execution, which offers techniques for recruiting patients, offering consideration of new emerging direct-to-patient and GP study models, access to established sites, an alliance network for accelerated start-ups, and an integrated technology solution. The latter is important for small and mid-size companies as it allows them to control project progress and their extended arm at PAREXEL in real time via smart technology. We call it the “trust, but verify” scheme.
Also, through dedicated teams, each client has a member of PAREXEL executive management and a dedicated project leader/portfolio directors assigned to them.
Finally, aligned incentives, where PAREXEL is invested in the client’s success. The company provides protocol/study assessment and consultation, monthly executive management check-in calls and semi-annual face-to-face meetings and introductions to venture capital firms or funding partners. Aligned incentives are tied to key milestones.
CP: Are these services associated with strategic partnerships/alliances? (i.e. discounting clinical in exchange for share in commercialization success)
UD: We believe in taking an operational risk approach to services, given that we can provide input on a project versus taking a commercial risk. Essentially, we invest in the setup of studies, rather than the financial aspect. Our clients understand the operational risk approach and appreciate the value it adds. An optimized project leads to time gains, which in turn translates to commercial gains.
CP: What is your perspective on the future of pharma/biopharma R&D? Where do you see opportunities for advancement?
UD: The entire industry needs to improve the drug development process. To do so, we believe we will see more partnering between pharma/biotech and CROs to make the process more time — and resource — efficient. In addition, there is a growing trend toward more personalized medicine supported by companion diagnostics to make sure the patient really benefits from a particular drug. We also must consider how to incorporate genomics into clinical trials and help the development process. Identifying a subpopulation of patients who will be high responders and obtaining approval for targeted population will be important for the industry’s future direction. Lastly, we will likely see an increase of early patient studies to capture early efficacy signals from real patients. That often goes hand in hand with finding the right biomarkers to develop certain selection criteria to ultimately benefit patients.
Kristin Brooks has been the associate editor at Contract Pharma since 2004.