Leveraging the Transition

Increasing regulatory scrutiny, greater patient and physician expectations around greater therapeutic effectiveness, and competition in the marketplace are putting pressure on pharmaceutical companies to develop new strategic plans for the transition from clinical research to commercialization, and for post-marketing research. It is the role of both the sponsor and their CRO partner to make the most of these critical steps.

What new challenges and opportunities are biopharmaceutical companies facing as they move their products from clinical development into commercial marketing?

It's a much more consumer driven healthcare market than lets say 20 years ago. Government policy makers, public and private reimbursement agencies, healthcare providers, hospitals, even patients are much more focused on healthcare costs. Therefore, the pattern of consumption of biopharmaceutical products has changed significantly. These changes among industries' customers have had a huge impact on how they implement their research strategies and then their subsequent investment in the projects that they conduct. For example, 20 years ago it would be common that a small to mid-size pharmaceutical company may invest millions of dollars to bring another beta blocker to the market to treat hypertension even if that beta blocker was the 10th or 12th beta blocker on the market. Now, regulatory agencies and reimbursement groups are asking, why do we need that 12th beta blocker on the market? Unless there is a significant benefit in either the effectiveness or a decrease in adverse events; the product really is not worth reimbursing.

What is the difference between late phase studies and clinical development trials?

Two significant ones have influenced most clinical development strategies as well as operational plans. The first difference is the target audience. In clinical development trials, the key audience is the regulatory authorities. Sufficient data on the product's safety and efficacy need to be provided in order to pass that regulatory hurdle, therefore the design follows suit. For late phase studies, long-term effectiveness and safety is still a concern of the regulatory audiences, however, the policy makers, reimbursement authorities, healthcare providers, and patients are new audiences for late phase studies. These audiences are much more interested in the value of the product they are receiving. The second significant difference is the research objectives in operational plans of the two studies. Clinical development trials must operate by a very strictly defined research protocol of good clinical practices and international conference on harmonization guidelines. Late phase studies, with a less strict research protocol, are allowed to operate within a broader set of guidelines. A responsible CRO partner will ensure that late phase studies are still conducted using good pharmacoepidemiology practices and help sponsors through study design choices and tradeoffs.

As stakeholders, what data do policy makers, reimbursement agencies, hospitals, and patients need to make their respective decisions?

The types of information will vary somewhat because these folks are making different decisions. Regulatory authorities will continue to focus on long-term effectiveness and safety. Reimbursement agencies, healthcare providers, and patients are interested in product characteristics that demonstrate the product's value. In other words, are patients getting the clinical outcomes that justify the cost of these new pharmaceutical products? Demonstrating value is often a key objective that is worked into late phase studies, in parallel with objectives of better defining long-term effectiveness and safety outcomes.

As investigator participation is key to many late phase trials, how do you keep them engaged?

This is a huge challenge for sponsor companies. The FDA in the United States and other regulatory agencies are requiring more post-marketing or late phase studies on a growing number of pharmaceutical products. These requirements are placing a huge administrative burden on physicians' offices and their staff. Because late phase studies can operate within broader guidelines, there is a way to minimize the potential burden on sites, and should be part of the design considerations of late phase studies. Web platforms for example, make communications easy between the sponsor and the physicians who are participating. There are data collection technologies that can assist physicians with their administrative tasks relating to maintaining medical records at their facility. Most importantly—especially to the nursing staff who supports physicians—if the technology can provide data back to the physician with individualized reports on their patients, they can often incorporate that information into patient care plans.

How important is technology in late phase studies?

Technology is extremely important as far as supporting and enhancing the strategic value of late phase studies. Technology is constantly changing and how it's applied is constantly evolving. For example, with social media being used more and more to support late phase studies, whether that's developing a patient community around patients who have the common denominator of participating in a late phase study, or just providing information out to physicians.

With considerable attention on global studies, how can sponsors prepare for marketing around the world?

The late phase area is strongly affected by healthcare policy laws, consequently the healthcare marketplace is constantly evolving. It is extremely important to have a presence in high growth regions, so that a sponsor can constantly monitor shifts in the healthcare delivery environment that may eventually impact the regulatory policies and reimbursement decisions related to their drug products. This will enable sponsors to be more proactive in their approaches rather than reactive to government decisions. As sponsors struggle to maintain regulatory compliance in different regions, it is critical that they have a partner with a deep understanding of the changes constantly occurring to help them proactively address those changes.

Neal Mantick is Global Head of Observational Research at PAREXEL International, e-mail: neal.mantick@parexel.com.