Does your company have the big 3 - Life Science Fast Track

TCN Life Science Venture Fast Track

Does Your Company Have The Big Three: Intellectual Property, Regulatory,

and Reimbursement? January 21, 2014

Panelists Therapeu(cs

Leslie Williams President and CEO ImmusanT

Emily Walsh Principal Tremont TherapeuPcs ConsulPng

Device

Nancy Briefs CEO InfoBionic

Nandini Murthy Regulatory Consultant ENEM ConsulPng

Moderator: KonstanPn Linnik Partner, Intellectual Property NuSer McClennen & Fish

Download slides: bit.ly/tcnLifeSci2014

The Big Three

IP Market Exclusivity (Patent & Regulatory ExclusiviPes)

Regulatory Safety & Efficacy

Payors Value ProposiPon (Pricing, Reimbursement & Market Access)


Patent Exclusivity

$

Loss of Exclusivity (LOE)

Generic entry Development�� 7-15 years

FDA Exclusivity


IP -‐ Market Exclusivity

Regulatory -‐ Safety & Efficacy

Payors -‐ Value ProposiPon

Ave. Exclusivity ~12.5 yrs, ave. Exclusivity necessary for ROI is at least 12 yrs

50% of Phase III trials fail for lack of efficacy over placebo

3 out of 4 approved drugs are not profitable

$


The Big Three: The EssenPals 1.  To get reimbursed, the use must be on the label; can’t rely

on off-‐label use or off-‐label promoPon

2.  There must be no generic subsPtute available; automaPc subsPtuPon is o_en required by law or by payors

3.  To get adequate exclusivity, patent claims must cover the approved use

4.  Clinical trials must be designed to support the label and the value proposiPon to payors


Examples 1.  Biogen’s BG-‐12 (MS or Psoriasis?)

2. Sepracor’s Lunesta (US) v Imovane (Europe)


Panelists Therapeu(cs

Leslie Williams President and CEO ImmusanT

Emily Walsh Principal Tremont TherapeuPcs ConsulPng

Device

Nancy Briefs CEO InfoBionic

Nandini Murthy Regulatory Consultant ENEM ConsulPng

Moderator: KonstanPn Linnik Partner, Intellectual Property NuSer McClennen & Fish


Nutter McClennen & Fish LLP • www.nutter.com 9

IP ProtecPon 1.  File early…don’t wait 2.  It’s all about the disclosure

3.  Know the prior art

4.  ArPculate your patent strategy

5.  Reserve funds to perform a freedom to operate ASAP

6.  Prepare for compePtors to use ambiguiPes in the patent system to their advantage

7.  Don’t skimp on choosing a law firm

IP….without a strategy, you won’t get funded


Reimbursement 1.  Link with PaPent Groups – they can put pressure on Washington

2.  ArPculate a sound comparaPve effecPveness argument 3.  Know if you can use exisPng codes or need a new code 4.  Hire a consultant if you need to 5.  It will take 3+ peer reviewed publicaPons (with significant numbers) to convince payors to

grant coverage

6.  You must show a reducPon in cost to the system 7.  Determine if private pay, Medicare or Medicaid cover your paPent populaPon

8.  Start dialogue with Payers including self-‐insured (large employers) early 9.  Find a champion early

Reimbursement….without a strategy, you won’t get funded


Regulatory 1.  Understand your desired path to approval 2.  Hire a consultant if you need one

3.  Communicate with the FDA early and o_en if possible -‐ look for guidance documents

4.  Understand and research the group at the FDA that will be responsible for the product

5.  Know your device will work clinically before submiong to the US FDA

6.  Don’t short cut criPcal aspects of development– go slow to go fast

7.  Budget appropriately (Pme & money)

8.  MITIGATE RISK wherever possible

Regulatory….without a strategy, you won’t get funded

Regulatory Path Preclinical Clinical Approval Market

Toxicology InvesPgaPonal New Drug ApplicaPon

Phase I Phase II Phase III New Drug ApplicaPon

Phase IV / Postmarket surveillance

safety safety dosing efficacy

safety efficacy side effects

Expenses $15.2 million $23.4 million $86.5 million

Time 21.6 months 25.7 months 30.5 months

1 to 6 years 6 to 11 years 0.6 to 2 years 11 to 14 years

Overall probability of success

30% 14% 9% 8%

CondiPonal probability of success

40% 75% 48% 64% 90%

Sources: Dimasi, Hansen, and Grabowski (2003).

Notes: The line marked “Overall probability of success” is the uncondiPonal probability of reaching a given stage. For example, 30 percent of drugs make it to phase I tesPng. The line marked “CondiPonal probability of success” shows the probability of advancing to the next stage of the process condiPonal on reaching a given stage. For example, the probability of advancing to Phase III tesPng condiPonal on starPng Phase II tesPng is 48 percent.

Overview

MedTech Regulatory Overview

Nandini Murthy

Regulatory Consultant

Email: [email protected]

Phone # (781) 710-‐5378

•  Did you have a Pre-Sub Meeting with FDA? •  Did the FDA identify any concerns or risks? •  If the path is 510(k), what is the predicate? Traditional or

De Novo? •  Indications for use? Aligns with business plan or

reimbursement strategy? •  Does it need a clinical? If so, what is the study design?

RCT? Observational? OUS studies suffice? •  Primary endpoints in the study? Will it support

reimbursement? Early adoption? •  Timeline for US approval? After CE Mark? •  Names of Legal Counsel, Regulatory Experts

Copyright ENEM Consulting LLC

Typical VC Questions

15

•  Majority of devices come to market via 510(k) •  Primary premise is one of Substantial Equivalence to

predicate devices •  Regulatory Strategy potentially counter to IP and

Reimbursement goals, where novelty of device may be emphasized.

•  Claims of benefit or superiority are limited when pursuing a 510(k) clearance path

•  The broader and more bold the claims, typically the longer and more complex the regulatory process


Regulatory versus reimbursement

16

•  Confusing the marketing need for regulatory requirement for a clinical study o  Devices are designed with intent, sometimes bench or

animal studies are adequate for 510(k) •  Starting the Clinical study prematurely, before regulatory

strategy has been identified o  Is it an equivalence study? RCT? Single Arm? o  Against a Gold standard? o  What is the patient population (intended use)?

•  Aligning Clinical study endpoints to marketing needs instead of regulatory validation

•  Assuming that Clinical Studies conducted OUS to satisfy EU launch will suffice for FDA. They might, but need to plan accordingly.


Common missteps

17

•  Regulated environment •  Moving target •  Details matter •  Planning and execution is critical •  Many FDA Submission requirements typically not

subject to negotiation (e.g. cannot negotiate an exemption from biocompatibility tests for patient-contact devices because of timeline commitments to investors)

•  There will be surprises during testing. Rely on experienced Regulatory and Project personnel

•  Manage expectations throughout process


Regulatory Summary

18

Develop a proprietary plaNorm for remote pa(ent monitoring

which leverages the cloud to reduce opera(ng costs

Data not Device Company

OUR VISION

Disrupt the $3B arrhythmia detec(on market with a lower cost, high quality cloud-‐based SaaS

solu(on for cardiologists

OUR MISSION

COMPANY FACTS •  Founded March 2011 – M2D2 UMass Lowell, MA

•  Team – Serial Successful Entrepreneurs

•  Exis(ng $3B Global Arrhythmia Detec(on Market

•  Innova(ve Proprietary Technology -‐ MoMe™

•  Reimbursement – ExisPng Codes in place

•  Regulatory – –  CE Mark

–  510(k) filed

MoMe™ Platform

Continuous Streaming ECG, Respiration, Motion

Cloud-‐Based solu(on

Mul(-‐pass Algorithm Analysis

INTELLECTUAL PROPERTY US Patent 8,478,418 (July 2013)

US Patent 8,620,418 B1 (Dec 2013)

•  Systems and methods for processing and displaying pa(ent electrocardiograph data

ABSTRACT •  A method is disclosed for displaying paPent ECG data.

The method includes receiving ECG data including an ECG waveform; receiving analyzed ECG data including arrhythmic events; generaPng an indicia of the detected arrhythmic event; and displaying the indicia of the detected arrhythmic event in relaPon to the ECG waveform at a posiPon associated with a Pme of the detected arrhythmic event. A system for displaying paPent ECG data is also disclosed.

US REIMBURSEMENT Larchmont Strategic Advisors

•  CPT Codes available and applicable

•  Holter, Event & MCT

•  Medicare reimburses all monitoring

•  Private Payors reimburse for Holter, Event and ~85% reimburse for MCT

Reimbursement for Arrhythmia Detection & Monitoring

REGULATORY

OUS -‐ CE Mark – Successful NoPfied Body Audit – CerPficate December 2013

US -‐ 510(k) Submission • Pre-‐IDE MeePng August 2012 • No Human Clinical Required • Clinical TesPng against recognized Data Bases •  Filed ‘Q4 2013

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Does your company have the big 3 - Life Science Fast Track