Fundraising Success and Increasing Sales

QUESTION: We need to fundraise now, but how do we raise money without data – we have months to years before study completion, data analysis, and publication?

ANSWER: Engage an experienced, cross-functional expert to integrate all the data and market analyses to develop the most positive fundraising value proposition. This works most effectively if the person engaged is expert in clinical science and business, with experience as a SAB member, preclinical and clinical researcher, data and statistics analyst, regulatory strategist, certified medical writer, and CEO. This interim strategist would organize and analyze the company’s existing in vitro, ex vivo, preclinical, clinical, and disease incidence/market data, perform a literature review meta-analysis of competitive devices and incorporate these into a compelling White Paper* which has proven effective in fundraising.

QUESTION: We have not been able to achieve our sales ramp forecast; we received feedback that potential customers would buy our device but require data. How can we provide data before publication of our studies?

ANSWER: Not only are White Papers effective in fundraising, they have been used very effectively to increase sales. Therefore, have an experienced, multifunctional expert integrate all the existing data in vitro, ex vivo, preclinical, and clinical data, perform a literature review meta-analysis of competitive devices and incorporate these into a White Paper* that can be used by the sales force.

Cost Effective Strategy and Senior Staff Mentoring

QUESTION: These are very difficult economic times and it is a necessity for us to be extremely careful with our burn rate. This includes justification of both full-time employees and consultants to assure we are using our capital efficiently. How does engaging interim management such as a Chief Strategy Officer (CSO) fit in with this necessity?

ANSWER: It is more cost effective to utilize interim management because as an independent contractor there are no executive recruiting fees and overhead costs from benefits such as healthcare, etc. It is vastly more costly to miss milestones and to try repeatedly to overcome roadblocks.

The skill set of a multifunctional CSO is unique in that it enables a centralized approach for open, clear, and efficient lines of communication and strategic vision among all corporate disciplines thus providing savings through meeting milestones on time and planning around or overcoming roadblocks.

QUESTION: We are concerned that the senior staff in the targeted functional areas will fear being replaced or not having their ideas heard during the Fast Track Program when they report to the interim leader/CSO. How is this managed?

ANSWER: The cross-functional leader is an interim role by design, not intended to replace any senior staff/functional heads. The interim leader has, at one time, been in the shoes of the senior staff and in charge of their functional area. During the Assessment Phase of the Program, the interim leader gathers information from the senior staff and requests and needs their perspective.

During the Solution Plan Phase, the interim leader and senior staff brainstorm and agree upon a strategic plan and goals together. Finally, during the Plan Execution Phase, the interim leader works alongside the senior staff mentoring them and assisting them with execution to achieve the plan goals. As a result, by hiring a CSO, you help your senior team deliver faster, better decisions while building world-class execution capabilities throughout your company after the Program ends and the interim leader is gone.

Overcoming Technical & Clinical Science Hurdles

QUESTION: How do we solve a technical hurdle that is burning up so much time and resources, e.g., the device works on some patients but not others, or works initially and then the benefits fade, or the device won’t scale to larger sizes? Our SAB, physician experts, R&D engineers, consulting physicists, and statisticians have all worked on this, and the problem remains.

ANSWER: While these experts have deep vertical knowledge in their respective disciplines, they do not have the broad cross-functional ‘Big Picture’ view or approach provided by a multidisciplinary clinical scientist and inventor with a 60-company experience. This multidisciplinary approach has repeatedly proven effective in solving such problems.

QUESTION: We have been having difficulty gaining IRB study approval. Centers want evidence of safety relative to the standard of care.

ANSWER: Have an experienced, multidisciplinary clinical scientist integrate all the existing data in vitro, ex vivo, preclinical, and clinical data, perform a literature review meta-analysis of competitive devices and incorporate these into a compelling Risk vs. Benefit White Paper which has repeatedly proven effective in obtaining IRB approval.

QUESTION: The industry thought leader is the designer and investigator of our clinical trial. This is great if the trial is successful, but if it is not, the company may have to fold. How do we best reduce risk of trial failure?

ANSWER: While thought leaders have clinical specialty expertise, they do not have the scientific trial design, regulatory (e.g., IDE, GCP, IFU, guidance documents), statistical, and device market expertise provided by an experienced cross-functional clinical scientist, which is needed to reduce risk and has proven effective in designing successful clinical trials.

Solving Regulatory Roadblocks

QUESTION: FDA has been a roadblock for our IDEs, 510(k)s, and PMAs asking more questions each time we respond to their original questions. How do we negotiate a faster path through these regulatory hurdles?

ANSWER: I have had 100% success in negotiating with FDA. I believe it is due to being a clinical scientist able to design the “Valid Scientific Evidence” (21 CFR 860.7) of safety and effectiveness they require for trial initiation or product clearance. The reviewers and decision makers at FDA are scientists and physicians, so who better to negotiate with them than a clinical scientist who understands what they require.

QUESTION: FDA has refused to allow use of the trial efficacy endpoints, which we have found best measure the effectiveness of our device. The problem is that these endpoints have never been accepted for use by FDA before and are not the primary endpoints in the medical literature, however they scientifically define how our device performs.

ANSWER: Have an experienced, multidisciplinary clinical scientist analyze and integrate all the existing data in vitro, ex vivo, preclinical, and clinical data, perform a literature review meta-analysis of competitive devices and endpoints then make a Minimal Clinically Important Difference (MCID) determination to validate use of the new endpoints and incorporate these into a compelling Endpoint White Paper. This has been effective in getting FDA to accept previously unused trial endpoints as primary and secondary.

QUESTION: Our company’s existence depends on a successful clinical trial. We heard that the odds of trial success are improved by using Bayesian statistical trial design and analysis because it is more flexible than the usual traditional (Frequentist) statistical methods alone. Will use of a Bayesian design and analysis be a problem with FDA?

ANSWER: It is not a problem, actually the FDA is encouraging industry to use Bayesian statistical trial design**. Indeed, odds are much improved by using Bayesian rather than Frequentist design alone – I have worked with the FDA successfully gaining IDE approval for a Phase II/III Bayesian trial.

Some of the important benefits of using Bayesian statistics are: allowing midcourse changes in trial design e.g., learning from and using collected data during the trial; adjusting the procedure/dose; re-estimating the sample size per treatment group; stopping ineffective therapies sooner; altering the timing of interim analyses; obtaining better decisions faster; seamlessly integrating Phase II/III into one trial; imputing missing data; improving data quality; making patient recruitment easier; reducing risk in developing new products; and enabling more efficient product development.

* Rasor, JS. Producing a white paper: A parallel path making clinical data available before publication. Amer Med Writers Assoc. 1991 Dec;6(4):12

** Draft Guidance for Industry and FDA Staff Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. U.S. Department of Health and Human Services. Food and Drug Administration. Center for Devices and Radiological Health. Division of Biostatistics. Office of Surveillance and Biometrics. February 2010.