Clinical trial collaborations – an idea whose time has come

Clinical trial collaborations – an idea whose time has comeThe cost and time commitment to develop new drugs has increased significantly over the last few decades. A variety of factors come into play, but on average, in terms of total expenditures, it has taken from $3 to $5 billion and up to 15 years to bring a new drug to market. Even after all that time and money, with challenging regulatory requirements and other factors, the odds of a new compound making it from the lab to the pharmacy are miniscule. The chance for a new drug to make it to market is a sobering 1 in 5000.

Such expenses and risks shut out many manufacturing innovators. This leads to the potential for stagnation among drug developers, while potentially life-saving and health-changing ideas are left undeveloped.

It’s not a new issue or concern for the industry. Research and development (R&D) manufacturers have been trying to find better ways to not only build but also innovate, fund, and quickly deliver new drugs for decades. Over the past few years, however, new approaches have been emerging to better share the cost and risks.  The newest approach is that of clinical trial collaborations. Although clinical collaborations among pharmaceutical companies are not new, the clinical trial approach—in which two or more entities develop and conduct the trials and share costs—is something that has just recently emerged as a risk-sharing arrangement.

Such collaborations may grow with the announcement of a venture between Bristol-Myers Squibb and Celldex Therapeutics to evaluate the safety, tolerability, and preliminary efficacy of nivolumab, Bristol-Myers Squibb’s investigational PD-1 immune checkpoint inhibitor, and varlilumab, Celldex’s CD27 targeting investigational antibody in a phase 1/2 study.

Most of the trials will likely focus on higher-cost medications such as those for hepatitis C and oncology, but there is the potential for funding for smaller niche products and disease categories, something of great interest to patients and clinicians. Such collaborations can also include so-called companion diagnostic or device technologies.

Factors leading to growth

The clinical trial collaboration concept is driven by economic necessity built off of a new pharmaceutical business model emphasizing R&D as well as the need for greater overall operational structure when it comes to research, development, trials, and delivery. Despite their size, even larger firms are finding it challenging to bring new drugs to market. With development budgets slashed in the past few years, many larger firms may not have the new product innovation pipeline they did in the past.   Although there are benefits to the approach, there are also challenges to clinical trial collaborations. Such challenges include:

– Rigorously defined disease categories, such as oncology, enhance the development of targeted therapies but could slow trials, because of study placement, design, or FDA communication decisions.

– The incorporation of biomarkers could slow the process because of multiple registration requirements from FDA.

– The current drug development system in the United States has evolved to discourage research of two investigational agents belonging to different pharmaceutical companies due to legal, liability, and ethical issues as well as regulatory and intellectual property issues.

Yet another major obstacle to drug research is the task of enlisting patients to participate in the trials. This has always been a costly and time-consuming process, but has become increasingly difficult.  A recent Washington Post article over just the prior 5-year period and retention of participants is equally challenging.

Despite these issues, there is reason enough to continue the effort. Although smaller firms need the infrastructure, funding, experience, and marketing expertise, larger manufacturers also need the innovation and speed that smaller firms can bring. Pharmaceutical companies are not often nimble enough to move quickly from idea to trials. We are seeing considerable forward-thinking innovations coming from smaller biotech firms; big pharma can tap into this creativity.

Value to providers

Yes, there are still many questions and issues to address within clinical trial collaborations. For example, if the drug is successful, who will market and share in the revenue? When does the collaboration start and end? Who will carry the risk for development; will it be equally shared?  As the number of collaborations grow, issues will be addressed and resolved.  The benefits for providers within the industry of this new approach are clear and therefore make it a concept worth supporting. For clinicians, pharmacy and therapeutics committees, and pharmacy directors, it speeds up new product development or pipelines for marketing of new specialty drugs to meet unmet needs while simultaneously impacting hospital/health system budgets to purchase these new products.

There is still much we need to explore about these new types of collaborations, but in terms of development cost, speed to market, and the expansion of medical innovation to new drug therapies, clinical trial collaborations hold much promise.

Bernstein. L. (2014, May 21). Manufacturers seek badly-needed volunteers to test drugs and other therapies.The Washington Post. Article accessed 6/12/14.