For a pre-clinical stage small biotechnology company, the transition from being a totally research-driven organization to a more developmentally driven entity focusing on planning their first clinical trial the task can seem a bit daunting. As with all small grant and/or equity funded companies, cash flow is always a consideration. Not having the expansive budgets enjoyed by larger companies, it is essential that the clinical and regulatory process be completed in as efficient and cost-effective a way as possible.
First and foremost, it is important to outline a strategy for preparing for this first-in-man clinical trial
What data does the company have to support an investigational new drug (IND) filing? Is the current data set sufficient to initiate a discussion with the FDA? And importantly, does the company have a pertinent list of questions that will facilitate moving forward with the full IND package?
Since it is extremely unlikely the company will have any in-house regulatory expertise this brings us to the second big question:
What is the Optimal Partnering Strategy to Achieve These Clinical Goals?
If the company elects to work with a clinical research organization (CRO), then the choice of a partner in this area becomes critical to all future actions. Many CROs can be classified as full service and provide all of the needed components, including regulatory support, protocol writing and assistance in dealing with the FDA.
It may, however, be advantageous to secure independent regulatory advice prior to engaging a CRO. This is to provide for a more balanced and impartial input as to the required tasks prior and during engaging with the FDA. Financial and logistical considerations will play a part in the decision process here as it obviously involves additional management bandwidth to deal with multiple vendors. This could be offset by potential savings from not spending on unwanted services from the CRO.
Which brings us to the next important consideration:
Who Shall we Partner With?
There are over 1100 CROs in the world (credit: Wikipedia), so picking the right one would seem to be intimidating to say the least.
Let us look at the playing field. The market is dominated by 10 or 11 major players who are global in reach and account for some 56 percent of total worldwide revenues. Very impressive, but they do not maintain this kind of leadership by dealing mainly with small, early stage companies and so, and no offense meant here, even if they took your business it is unlikely you will get the kind of attention afforded to, say, a GSK or Merck. The first thing to do is narrow down the field perhaps by investigating companies headquartered, or at least with a significant presence, in your home state that are of a size more in line with a small biotechnology company. A bit of diligence is required here to elicit just how well the shortlist of CROs fit with respect to services provided and compatibility. A great asset in this decision process is attendance at relevant conferences, preferably local or regional, where one can meet in person with several potential CRO partners and get a feel for their capabilities and culture.
Finally, let us assume you have decided on an independent regulatory consultant and also picked a suitable CRO to meet your strategic clinical development goals:
Two words guaranteed to bring fear to even the most hardened executive. No matter how full-service the chosen CRO claims to be, they need a reasonable level of oversight. You may have done a great job of picking a compatible CRO and a regulatory consultant who both share your vision and can implement your strategic clinical plan, but don’t rest on your laurels yet!
The kind of service and results you can expect are directly proportional to the amount of effort you are prepared to invest in managing this whole process. Interactions between the regulatory consultant, the company, the regulatory agency and the CRO are complex and need to be managed to ensure the company’s best interests are at the forefront. In most cases, this cannot be delegated to scientific staff and the executive(s) will most likely not have the time or probably the expertise to properly fulfill this obligation.
Consider at this point bringing in-house some serious project management skill, someone dedicated full time to managing this complex interaction between multiple parties. Timely and constant communication will, in many cases, identify and correct minor problems before they become serious issues. Remember, you are not the only client the CRO is working with (at least we hope this is the case!), and so you are essentially competing for their attention at every stage of the process. A formal reporting structure and reasonable milestones will be a great asset in ensuring the program is moving forward at a reasonable pace and moving in the right direction.
In summary, this is not meant to be an exhaustive playbook for a clinical program, but rather a series of questions you should be considering to make the process manageable.
A good measure of success in choosing and managing a CRO and the regulatory process is that the only thing you have left to worry about is the science.