There are a slew of specialist and boutique companies set up to provide high quality services and to meet the changing needs of the pharma industry.
From the emergence of big data analytics to huge advances in precision therapies and trial technologies, the world of clinical research has transformed in recent years, leading to closer and more strategic partnerships between sponsors and contract research organizations (CROs).
Pharma and biopharma companies are increasingly relying on CROs for their specialized expertise, global reach, data capabilities, and cost-effective solutions to navigating the evolving landscape of clinical research and drug development.
As such, the age-old, binary question of whether it is better to engage specialist or full service CROs is being replaced by a need to align the aims and objectives of the project to the aims and objectives of the research partner.
Since the likes of Quintiles—or IQVIA as it is now known—Parexel and PPD were founded in the 1980s, the CRO has been a major life sciences player.1 By taking on tasks such as clinical trial management, logistics, statistics, and data management, they have helped expand the capabilities of their sponsor partners and accelerate drug development timelines.
As of February, this year, there were 2,823 CROs in the United States alone,2 and with this growth has come evolution. Along with the big players, there are now a slew of specialist/boutique companies, many of which were set up to provide high quality services and to meet the changing needs of the pharma industry.
Over the past decade, for example, the balance between large pharma companies and smaller biotech businesses has shifted. Between 2011 and 2021, the share of the industry-wide pipeline owned by the top 15 pharma companies shrunk from approximately 40% to 20%, whereas a significant proportion of advanced molecules in development, across areas such as cell and gene therapy, monoclonal antibodies, and DNA and RNA therapeutics, are in the hands of biotech.3
These smaller companies do not necessarily have the specialist research expertise or connections needed to drive their research programmes, so naturally look to outsource to fill the gap. However, data from McKinsey & Co suggest biotech businesses are dissatisfied with the offerings of the big CROs. They feel they do not provide enough strategic advice or adequately support the integration of technology point solution providers, and that there is a “misalignment of incentives,” the survey found.3
At the same time, there is a trend for “big pharma” to bring more research activity back in house, in a bid to retain control and regulatory oversight over an increasing number of complex trial designs, and their spiralling number of datapoints.3,4 Furthermore, drug developers of all types are constantly looking for new ways to make trials more efficient, both in terms of time and costs.
Although the changing world may decrease the value of the one-stop-shop CRO, specialist organizations with the willingness to engage in new ways still offer all the same benefits of streamlined, optimized drug development. They can provide the support of the thought leader-level experts and innovators, who are best placed to help their pharma/biopharma partners navigate these complex challenges.
We often talk of drug development as a monolith, but each research area will have its own complexities, challenges, and regulatory considerations.Specialist CROs often have therapeutic area expertise. They can provide specialized knowledge and experience in specific disease areas, ensuring clinical trials are designed and executed with a deep understanding of the therapeutic and regulatory and compliance context, contributing to faster, more cost-effective studies.
An important obstacle in the rare disease space, for example, is the limited number of eligible study candidates. Medical device developers face an entirely different set of challenges, from selecting meaningful endpoints to ensuring access to a consistent supply of high-quality components.
Furthermore, each of these areas operates within an entirely different, complex, and fast evolving regulatory framework. A CRO with specialist focus will have the relevant experience across all functions, meaning they will fully understand the complexities and intricacies of development in the given area from day one.
For example, in rare diseases, they may have the expertise to guide discussions on broadening eligibility criteria and increasing recruitment timelines, as well as adopting innovative trial designs, such as the inclusion of historical control arms or Bayesian methods, all of which involve careful analysis and skillful adjustment that can only be delivered by specialists. They can advise on the optimal outcomes to ensure data are information rich.
From the medical device perspective, a specialist CRO will understand device-specific considerations, such as the regulatory requirements for different types of tools or instruments, and how the type of data they collect impact trial design and data analysis. They will be able to advise on the formal calibration and validation processes, and whether the required analysis should be based on sensitivity/specificity, agreement, or repeatability/reproducibility, depending on the device itself and existing gold standards.
Such deep familiarity will enable the specialist CRO to help their sponsor partner ask the right questions, design the right protocol, and mine the right insights. This streamlines and optimizes the development pathway from its inception right the way through to market, resulting in accelerated timelines, cost efficiencies and a higher chance of success.
An important area in which working with CROs can expand capabilities is in data science, analysis, and biometrics, which is particularly pertinent in light of the skills shortages facing the industry.
According to market analysts Market and Market, just a handful of global organizations currently have sufficient in-house expertise to translate large volumes of data into valuable insights.5 With data science being a rapidly growing discipline, and life sciences being just one of the sectors vying for candidates, this talent squeeze is only expected to get worse. By merit of conducting complex, pioneering, and—ultimately, interesting work—CROs that specialize in biometrics often attract the best people, meaning they can help sponsors to plug that gap.
Combining therapeutic area expertise with the specialist skills needed to leverage advanced analytics techniques to push innovation—and the industry—forward is crucial.
This benefits big pharma as much as it does smaller biotech business that lack in-house data science expertise. Of those larger companies that are bringing research activity back in house to retain oversight, for example, many are “in-sourcing” CRO data experts to work on the data within their systems, giving them the best of both worlds. At the same time, biotech organizations are outsourcing larger percentage of their services, as clinical trials become larger and more complex. As such, they are looking for flexible, responsive partnerships with specialist CROs who have the breadth and depth of capability to help them to achieve their long-term goals.
The desire for more efficient studies, and advances in omics, genetics, personalized medicine and computational power are all leading to more complex clinical trials. These fundamental shifts are changing the way sponsors work with CROs. Simply, this new model requires a more specialized service that generalist organizations just cannot provide.
But by replacing the one-stop-shop model of years gone by with a razor-focused alignment of challenges and goals, the two stakeholders can make sure the right skills are in the right place at the right time, and keep clinical trials humming for many years to come.
About the Author
Prof Jennifer Visser-Rogers is vice president, Statistical Research and Consultancy at specialist CRO, PHASTAR.