Post on 27-Mar-2020
transcript
Roadmap for Commercialization: Idea to Product to Market
2012 Hydrocephalus Research Conference
Tom ClementCEO, Aqueduct Neurosciences
32 years of Medical Device development experiencespanning Neurosurgery, Interventional Cardiology, Radiology, and Gastroenterology
I have equity in Aqueduct Neurosciences, Inc. I will be using Aqueduct’s company development and strategic planning as examples throughout talk
Background and Disclosure
Prepare a Business Plan to address major risks associated with commercializing inventions: Market Intellectual Property Prototype Development Regulatory and Clinical Strategies Reimbursement Team Funding
The goal is to convince investors to invest
You Think You Have Identified a Need for New or Improved Medical Device...
What’s Next?
Prod A
Prod C
Prod B
What is the problem you are trying to solve? Disease has suboptimal treatment – high complication rates, low
success rates, too expensive, not durable, etc. Condition has no current treatment Only some patients can be treated
How big is it? Prevalence, burden, current treatment
options statistics Revenues and “units” associated with current treatments
Who are the competitors? What do their products do well? Where is there room for improvement?
Start by Describing and Quantifying Target Market
Physician Treating or referring or both “User” or “influencer” Why would he / she change their practice?
Hospital Patient Who will pay for
the new device?
Determine: Who is the Customer and What are Their Needs?
Customer ‐ User
Customer ‐ Purchaser
Customer ‐ Influencer
Hydrocephalus is Common
Pediatric HC (38% of patients)• effects 1 in 500 live births in US (8,634
newborns annually)• average 2‐3 surgical revisions before
adulthood• most common brain surgery for children
“Other” adults with HC (36%)• former pediatric & newly diagnosed • acquired (tumors, trauma, meningitis)
Normal Pressure Hydrocephalus (NPH)
• underdiagnosed and under‐treated
• 26% of current patients or 375,000 people in US
• significant growth due to aging population
• up to 10% of dementia is misdiagnosed• only 25% of diagnosed are shunted
Treatment is surgical placement of a shunt to drain fluid
Neurosurgeons Are Frustrated• Alarming failure rates – 30% with in first year; 98% by 10 years
• 2‐3 revision surgeries for children before they reach adulthood
• ~ 50% of all shunt surgeries are revisions/ repairs /removals
• No diagnostic capability resulting in CT scans to diagnose problems
• 20 or more CT scans before age 20• Uncertainty significantly impacts
quality of life for child and family• Requires manual adjustment to ensure proper valve setting
• Pressure based designs are prone to over‐drainage (siphoning)
US Market ‐ Large & Growing
NPH
Pediatric
Growth in NPH driving market growth• Doubling population >65 years old• Increasing recognition of NPH
US shunt market grows to $298 million (89,000 procedures) by 2018
58,000 annual shunt procedures
Start by protecting your idea: Write it down or draw it Describe it Have a witness who knows the “space” sign your disclosure File a (provisional) patent
World is harmonized on “first to file” Inexpensive But, now you are “on a clock”
Are there patentable aspects to the idea? Are there existing patents that block the idea?
Then Take Care to Protect Your Intellectual Property
Develop and describe the concept(s) Build working prototypes to demonstrate the concept(s) How can you test / challenge the idea?
Develop tests and experiments that show AND challenge the design
Head to head comparison with leading competitor is important Develop bench tests that start building the foundation for method to prove the initial design and allow for comparison to previous designs
How Do You Know Idea Will Work?
Regulatory strategy is an integral part of overall business plan; approval for commercial sales and allowable marketing claims 510(k) or PMA path in US?
Are there are “predicate” or existing devices that could be used for 510(k) submission?
Are there existing devices that have CE Mark and marketed in Europe or other countries?
Will there be a requirement for clinical trials? [note: even a 510(k) may need clinical data] Registry vs. randomized End points acceptable to agency?
Develop a Regulatory Strategy
Did not visit FDA for a “pre‐IDE” meeting
Conducted 200+ patient, literature controlled study with a CRO, Core Lab, DSMB and oversight board
Established predicate devices (2‐3)
Submitted clinical data along with all the appropriate testing to the FDA in 510(k) Supporting the claims of safety and effectiveness that was “the same as
predicate devices”
Subsequent product iterations or new indications were submitted as 510(k) or Special 510(k)
Pathway Medical – Regulatory Path
It is important to understand what the value proposition for your technology is, especially from the insurers’ standpoint How are similar products reimbursed? (inpatient, outpatient, ambulatory center)
Does current reimbursement provide enough “room” for a new technology?
Engaging with a reimbursement specialist earlier rather than later, can: help with product development to ensure coverage, and if necessary, get the process started to obtain coverage and establish
new codes
Understand the Reimbursement Environment
Value Proposition of a Shunt with Reduced Failure Rates
Payer
• Reduced healthcare expenditures • Each shunt surgery costs $67,000• Average length of stay in hospital is 8 days • ~ 50% surgeries are for revising existing shunts
Patients• Reduction in complications• Fewer diagnostic CTs• Improved quality of life for patients and their families
Healthcare Professionals
• Diagnostic feedback of implanted shunt to: • determine if surgery is required • plan surgery for better patient outcomes
Are clinical studies required for regulatory submissions?
Studies for market development and demonstration of product performance
Once the study purpose is defined, develop the study design and statistical plan Patient population Number of patients Primary and secondary endpoints Time period for measurement, follow up points
Develop Clinical Study Plans to Support Overall Strategy
Early in the process, design controls and documentation is limited, but:
Once the product design is frozen, specific controls and documentation are required ISO certification audit, ultimately FDA audit
Quality assurance as part of clinical trials also important –Good Clinical Practices
Plan for Quality Systems
Quality Manual
Minimal structure ‐Early feasibility
+ Design History
Documentation & systems for prod development phases
Quality System
Preparation for regulatory submissionsPreparation for Commercialization
Probably the most critical aspect of developing a new product It is guaranteed that there will be difficult points during the
process; at those times you want to be side by side with people you trust
However, the team also needs to be able to disagree and have varying opinions
Build the team around people with complementary skill sets – technical, business and clinical
Put Together the Team
• Samuel R. Browd, MD, PhD, Chief Medical Officer• Pediatric Surgeon, Seattle Children’s
• Barry Lutz, PhD, Chief Technology Officer• Assoc. Research Professor, Univ. of Wash, Dept. of Bioengineering
• Tom Clement, MS, MBA, Chief Executive Officer• Medical device entrepreneur
• UWC4C support• ADD
• Engineering • Regulatory, Marketing, IP/Legal consultants• Operations, Quality Assurance
The Team
Also called “Go to Market” strategy What is the optimal sales model?
Direct sales Distributor Corporate partner
Limited launch vs. full launch Targeting the right customers
Develop Marketing Strategy
Pursue CE Mark in parallel with US 510(k)
Launch by conducting careful clinical studies in specific OUS markets
Initial focus on post‐market studies Demonstrate clinical, economic and product performance
Engage with key opinion leaders
Gather data for publication and possible submission with next generation SmartShunt
Aqueduct “Go To Market” Strategy
Evaluate Potential OUS Markets
Opportunistic Distributor
Revenue MarketsItaly/Spain
IndiaSouth Africa
BrazilAustraliaCanada
StrategicRegulatory Markets
Japan
ChinaKorea
Established Distribution MarketsGermany
Great Britain
Core questions for all start up companies: What is the current “valuation”? How much to raise and to what milestones? How much total $$ will it take to get to market? Expect “dilution,” but how can you manage it?
Is there grant money available? The answer to these questions will drive who you approach Angels Institutional Investors (VCs) Corporations
Financing the Company
How far do you plan to take the company Manufacturing? Clinical trials? Sales?
Distribution? Direct?
Who are “partners” or acquirers? IPO? (ha ha)
Define an Exit Strategy
Thank you
Questions?
Tom Clement, CEOAqueduct Neurosciences, Inc.tom.clement13@gmail.com