Trial Design: Prep Work

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Surprises are usually a bad thing in clinical research, which is why start-ups creating their study strategy should prepare early. From literature searches to working with key opinion leaders and regulators, thorough planning will keep things on track and to budget as the trial edges closer. Life sciences start-ups should plan for clinical trials from the inception of their research. But, in reality, many of these businesses often leave it to the last minute – causing unnecessary delays and increasing the potential for failure. Some simple steps, taken early in the development process, will streamline the path to trials and avoid needless setbacks.

Literature Search

The first piece of advice is: do a thorough literature search. It is remarkable how many companies do not carry out a  good search of previous, relevant studies, to find out what the medical community has already produced, and then to consider what the next step in the clinical evolution will be.

As a product is developed, it is important to know basis data, such as what has been recorded, what needs to be covered in the preclinical plan, what is required to gain approval and, ultimately, what should be done to support the marketing  goals and objectives. It is easy to dive straight into the development work, and not take the time to perform a good search. However, there can be significant consequences: companies that fail to do a rigorous search at the start of the process will often find a delay or roadblock as they near clinical trials.

Early Planning

The next step is to prepare early for trials, and there are a number of reasons for this. The right preclinical experiments must be planned in order to support the ultimate clinical trial design. It is also necessary to know where the study should be performed and how much money the study will cost. A company needs to have a clear idea of how many patients will need to be enrolled, how many sites set up. When raising money, these are basic things that have  to be accounted for.

For medical device companies, it is vital to engage with potential end-users very early in the development process: after all, they will be the ones using the device. Furthermore, end-users are a source of valuable feedback on the design, indication, and even end-user interface which is incredibly important in this market.

The likely impact of delays should be considered. Most start-ups burn $100,000 to $500,000 per month – which is a lot of money, particularly if there is a fixed budget to some degree because the company is venture capital financed. If preparation is left to the last minute, the business may be looking at delays of up to six months or more waiting for a meeting with the regulatory agency or negotiating trial design with the regulator. This cost rises due to longer time- to-market and other lost opportunity liabilities.

Avoid Roadblocks

One mistake that companies make in creating their strategic development and clinical trial plans is not understanding that everyone else is working to their own plan. In the US, for example, the FDA works to its own plan, as does the Centers for Medicare and Medicaid Services. A strategy that fails to factor in the near impossibility of controlling external partners and agencies will find serious roadblocks. An additional hurdle comes from a lack of knowledge about the regulatory approval process – getting expert regulatory and reimbursement guidance early can remove this possible hindrance.

One of the common causes for failure in clinical    trial planning is a shortage of experience in the leadership team.Inexperienced leaders can fall into the trap of collecting too many opinions and trying to please everyone with something that should be relatively simple. Another is to design a plan that tries to do too much with one study. The research fails because it is overly diluted and does not meet the primary endpoint goals; it is trying to gather more information than is needed. One way to sidestep this problem is to use the right advisors and key opinion leaders (KOLs), who can guide the company as it creates its trial strategy.

The Right Team

Finding the right advisors and management team is vitally important to ensure everything works to plan and progressing as expected. The following recommendations should be taken early:

Find a KOL who is ‘plugged into’ the product or technology. This person should not necessarily work as an investigator, but rather as a champion of the product or technology. For instance, they might be a retired physician who sits on many boards, attends numerous meetings a year, and who can promote

a company’s products as being the next wave of technology.

The second set of advisors should be KOLs who are clinically practicing and are influential within the specialty. These opinion leaders can influence other investigators, champion the clinical trial, and rally the troops when help is needed in enrolment or safety issues, etc

Ensure the internal management team – whether it  is a very small team or not – is experienced and familiar with the issues and problems that will be faced on the path to trials. These individuals also need to know how to manage external vendors and collaborators

Companies need to look for external partners that have been through this process, and can advise on potential risks and mitigate issues that might arise. This will help the development process and business operations run smoothly and get it across the goal line as planned

Seek out investors who realise they will not recoup their monies in a year or two; they have to be seasoned life sciences venture capitalists who know the process and that it can   be a five year road to market

If there is a ‘magic bullet’ that can help a company move more smoothly to the clinical trial phase, it would be planning, focus and a management team that understands what is needed. If the business is not as strong as it should be in one of these key areas, the time to fix it is early in

the process.

By Nuala Ronan