Specialist Series: Filling the Unmet Need – Your Proposed Solution

Contributed by Ron Blackman

In our previous newsletter, we described how to identify the unmet need that will be served by your device or therapeutic, and here we will help you build the case that you have a solution to meet that need.  A number of points should be addressed and we will go through them individually.

What patient population are you targeting?

While you have already identified the market need, you may find for business, product development or medical reasons that it may be preferable to define a narrower niche for your entry to the market.  For example, by focusing on an indication for your drug that affects less than 200,000 people in the US, your therapeutic may qualify for orphan drug status which typically lowers the cost and development time due to smaller clinical trials.  Similarly, by choosing to address, for example, chronic cough rather than atopic dermatitis, even though the drug may alleviate both sets of conditions, clinical trials can be designed with simpler endpoints and less concern for a large number of extant competitors, again easing the path to approval.  This narrowing of focus means that your treatment group may be only a subset of the potential patients.  Delineating this group early in the device/therapeutic development process will help define which preclinical models should be run as well as what results are likely to generate early interest from potential investors.

What is the setting in which this technology would apply?

Similarly, you need to consider where your device/drug will be administered and by what means.  For example, a drug may delivered intravenously in the clinic over a few days or as a pill taken chronically at home.  This choice will greatly influence the preclinical and clinical development needs and strategies.  In addition, observing how patients are currently being treated will help identify product design requirements that your product needs in order to be successful.

What literature-based or experimental data do you have to support the expected benefit? 

To provide evidence that your device or drug will be efficacious and can successfully make its way to the market, it is best to build the case using a solid scientific foundation.  The background should be drawn from literature, reviewing the biology of the disease state and determining how your product will benefit the patient.  For therapeutics, when possible, you should also give a mechanistic explanation of the biological pathways involved and how your drug would act selectively to treat the disease state while minimally impacting healthy tissues and functions.

Most investors will want to see proof-of-concept (POC) data that extend beyond early discovery research.  Hence, preliminary work should already have been completed that shows the potential for success of the invention and sets the stage for the next steps of the development plan.  It is usually accepted that these studies be completed in models or indications that differ from the desired area of focus, but it will be necessary to explain how the results will translate to the indication of choice.  For greater interest from investors, additional research validating the commercialization potential of the drug/device is needed.  These need to be done in a model that is widely considered a “gold standard” model for the disease state, which is usually in a relevant animal model system but could also be bench testing or early feasibility clinical studies.  For therapeutics, the study should allow for the direct comparison to other drugs that have been tested in the same model.

At B-BIC, our Pilot awards are often used to provide resources for POC studies while our DRIVE awards provide a means to obtain results focused on the refined niche or indication driven by the chosen market need.

How does the expected benefit with this solution/product compare to that of the current standard of care?  

As part of this discussion, you should describe the current physician practice/standard treatment and discuss how the new drug/device will be an improvement worthy of a change in the clinical setting.  While patient health benefits are always paramount, realize that other non-health advantages such as cost savings, surgical time or convenience for implementation, site of administration, etc., can also be very important considerations for market adoption/penetration and should be addressed.

* This is Part 3 in our B-BIC Proposal / Business Plan series.  Click Here to see all articles in this series.