Preventing the “Rescue Trial Syndrome”

Working with clinical development in a biotech company is associated with limitations – and opportunities. The limitations are usually related to the fact that resources are not readily available. They have to be specified and defined, they have to be tailored to the specific project, and they need to be raised through external funding which may influence timing, amount, and availability despite the need. The opportunities, however, are right in front of you. It is the state of mind, and level of experience reached in the team at the time, that enables you to see the opportunities and eventually exploit them.

Most biotech employees who work in clinical development start their career in large pharma companies where they learn the basic skills of the business in an environment governed by corporate structures, politics, and mindset. Making the move to a small  or even virtual, i.e. almost non-existing organization, is like moving to a small farm in the countryside from your penthouse apartment on the 25th floor. You suddenly have to deal with a lot of issues you never could imagine.

Symptoms…

Once you have made the transition and survived , it suddenly comes clear how the different environment opens up new opportunities. It is a requirement in clinical development to have systems and process, structures and plans in order to be successful in the end  (but nobody has ever claimed that there is only one set of systems or processes, structures and plans to adhere to). Being able to shake off the corporate history and mindset when you enter the biotech field is essential for your success.

Trial startup activities and strategies is one area where traditional corporate thinking appears to be difficult to change. The industry statistics on recruitment feasibility and speed are clear, and everybody in the field has been there, slow study start up and lacking recruitment seems to have become the industry norm.

Prevention and a new way of doing the same thing

In my current company and team we felt that the time was right to try to do things differently. The company is a virtual start up with a team of highly experienced drug developers. We started in an empty office and as a virtual start up company you would typically focus on choosing a vendor with relevant therapeutic experience to do the job for you. However, since we were about to embark on a project in an indication without any precedence of industry-sponsored drug development trials, we felt that following the traditional path of vendor identification and selection, contracting, protocol writing, site identification, and feasibility through questionnaires eventually leading to study start up activities would just not give us the speed and kick-off we needed. And certainly not the quality and expertise, since no CRO could claim to have a track record of previous experience within this particular indication.

So we decided to restructure the process and instead focus on site identification, feasibility analysis, pre-screening activities , and last but not least  investigator relations as an entirely separate activity requiring its own focus, resources, and strategy. In parallel, we developed and wrote the protocol, performed vendor identification, selection, and contracting. In essence, we as the sponsor, took full responsibility for investigator/site identification and selection and the vendor kicked in with formal site qualification and study startup activities once the identification and pre-selection work was done.

The process and the soft skills

During the process we used feasibility questionnaires, but only as a tool to efficiently screen for and identify sites. We never relied on the information collected from the questionnaires during the selection process. Each site was selected based on a combination of experience, resource availability, rapport and quality of interaction during the selection process, perceived genuine interest to participate, and willingness to adapt to the study specific requirements – and last but not least, documented pre-screening records provided before the final selection decision. This activity in itself requires early planning  and resourcing at a stage while you still work on protocol details. But keeping a close dialogue with the site during the process at the senior medical director level turned out to leverage flexibility and collaboration enabling us to be successful.

In closing

With our focused approach and parallel processes we managed to recruit the first patient 6 months after initiation of the project, and an additional 5 months later the study (a 300 patient phase II trial) was fully recruited with 100% of the sites recruiting an average of 20-25 patients.

These days, where 97% of all trials do not fulfill recruitment projections, it seems unique to be able to accomplish such a success, but when it comes down to it, nothing is really new.

It’s all about presence, focus, and willingness to invest what it takes. And being a small team in a virtual company, working with a completely new indication, does not mean you can not turn the way you do things upside down – and be successful.

About the Author

Christian Meyer, MD, PhD, is the Chief Medical Officer at Cardoz AB and is an experienced biotech executive. Dr. Meyer also writes on his own blog.

Featured Image: www.freedigitalphotos.net

Tagged as: feasibility, Investigator, site identification, virtual start up