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How to get the most out of clinical trials



 

Introduction

Clinical trials are expensive.  It’s imperative that early stage companies maximize the benefits of any clinical trials that they are sponsoring. In addition to maximizing the benefit, they must also limit their liability and insure they comply with all regulatory requirements.

Purpose and Endpoints

All clinical trials will have a purpose, otherwise there would be no need for the trial.  But to maximize the value one must consider the possibility of multiple purposes.  Typically the most needed clinical trial would be one for regulatory clearance or approval to market.  And in the US, if this is going to be required a pre-sub(mission) with the FDA would be recommended to insure that the trial design meets the needs of the FDA to grant approval.  The FDA is evaluating safety and efficacy.  But depending on the size and scope of the study it’s possible the study could be used for more, such as making marketing claims about the device, or cost effectiveness in using the device.  Another possibility depending on the reimbursement landscape is to at least get some pilot data from the study for a more comprehensive study that could be used to gain reimbursement. 

De-Risking the Design

Even though a purpose for a study can be well known, how to achieve the endpoints is more obscure.  How many cases will be needed to demonstrate statistical significance?  It depends on how big the difference is from the standard existing technology and the new technology.  Or what if we’re try to prove there is no difference or at least it’s not inferior to existing technology? This can often be  the case for a 510(k) application.  In these cases doing a smaller pilot study to see what the trends are may be prudent.  As well as showing trends, in some cases it may show not to include a particular population or demographic as the device may not be as effective for the group.  I was working with one company where limiting the age of the population for the indicated use made a huge difference in the size of the study.  This was able to get them on the market earlier and less costly, and later they went back and did a broader study to expand the indications for use to a larger age group.

Regulatory Considerations

One must have regulatory clearance to sell a device in the US.  For all PMA applications and many de Nova and 510(k) applications clinical trials will be required.  A pre-sub meeting with the FDA is best way to determine what size, scope and endpoints would be necessary to gain regulatory approval.  Sometimes instead of a full clinical trial there may be the requirement for usability studies.  These insure that the device can be used safely and effectively by the caregiver.  This is typical for a 510(k) application.  Again, these types of studies can also be used for marketing purposes.  Keep in mind that the FDA requirements could be sub-set of broader range studies (or trials) and evaluate the additional cost of perhaps gaining some added value.  Depending on the competitive landscape this could be very advantageous for a 2nd or 3rd entry into a particular market.

Another aspect to consider, which is not directly related to the clinical trials, but is a regulatory requirement that should be considered and possibly discussed with the FDA, is post market surveillance.  How will you insure that over time the devices continue to work and be used as they are indicated and intended to be used?   While this is certainly less costly than a clinical trial, this data may also lend itself to marketing claims in terms of utilization and possibly effectiveness of the product depending on the data you choose to collect.

Market Dynamics

As mentioned the competitive landscape can be an influencing factor on possibly expanding a clinical trial or study to gain additional credibility in the market.  Sometimes, however, there is an interesting possibility of focusing on something very specific within a study.  I’ll call these sub-studies, meaning at study within a broader study or trial.  Regularly, these sub-studies do not have enough data to achieve statistical significance.  However, they may show enough of a trend that a presentation at a national conference is warranted and may ultimately lead to a larger study with that focus for either publication or additional claims.

Reimbursement

In a previous article we discussed the reimbursement landscape:  https://www.rivesconsult.com/post/reimbursement-strategies-for-early-stage-medical-devices.

Clinical trials for reimbursement are generally much larger and more costly than those needed to grant regulatory approval.  Most will require a multi-center study and frequently multiple studies that are published in peer reviewed journals.  The process is lengthy and time consuming.  There are two parts to achieving reimbursement.  The first is gaining a CPT or reimbursement code, but that doesn’t mean payors will pay for the device (or procedure).  The second is the RUC process, which stands for Relative Value Scale.  This is a committee based on societies of the AMA.  The typical process is for a company to work with an appropriate society that would have an interest in their device to present to the committee.  The studies would support the value claims to be presented.  So, this is far beyond safety and efficacy; this looks at the economics of the device (procedure).  Does it save money? If so, how, and how can it be proven?  Does it lead to better outcomes, shorter hospital stay, less chance of recurrence or readmittance?  Is it faster or more accurate?  These are the types of questions a study will have to prove in order to successfully gain reimbursement.

Case Studies

Within a study there may be the opportunity to highlight a case study.  This sounds simple enough, but it turns out it can be quite a bit tricker than one would think.  Due to HIPAA regulations and many times restrictions imposed by an IRB, gaining access to a specific case to highlight can be difficult if not impossible.  If this is something that can prove of value (and it very often is), then it must be considered, discussed, and have a pathway to achieve this before the study begins.  After the study begins it can be almost impossible to achieve.

Conclusion

In conclusion, a well thought out clinical trial design is critical for any company, but possibly even more critical for early stage medical device companies where budget constraints tend to be the highest.  Always consider all options from case studies, pilot projects, regulatory, reimbursement and marketing collectively to get the most out of what is likely an expensive and time-consuming endeavor.

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