for internal use only 1 breakout materials mdi lad rww

For Internal Use Only 1

Breakout Materials

MDI

LAD

RWW


LAD Breakout Objectives

Provide introduction to Label as Driver as a process for ensuring our Planned Label is competitive and drives the Integrated Medicine Plan

Value of LAD

Process & tool to create a Planned Label

How Planned Label will evolve from DP to DP

Begin to apply LAD to your asset

Determine critical success factors of the Label Sections for your asset

Begin to establish Planned & Fall Back Labels

Discuss next steps for your asset


Label as Driver: beginning with the goal in mind….

Figure out what you want your label to say

Figure out what you need to do to be able to say it

Discuss your ideas w/FDA as you go along (if appropriate)

Use “label as a driver” process (LAD) to guide and prioritize

asset team activities


The expected outcome of the Label as Driver process

The Label Claim* is driven from our Unmet Need (patient, payer, provider, approver or care-giver) and linked to the Value Proposition

There is clarity to the exact wording we strive for Label Claim and how that compares to competitor claims

The Label Claim is the most competitive for COMPANY – able to address multiple needs (pricing/access, detailed aids, formulary, etc).

There is clarity to how the Label Claim will be achieved thru our Integrated Medicine Plans (clinical, regulatory, commercial, access)

* See LAD Glossary for definition* See LAD Glossary for definition


LAD Glossary Terms we will use:

Process Elements: Label as Driver Process: the set of activities which assist in identifying and

achieving the Planned Label for COMPANY. LAD Grid: Tying the unmet need, to the actual Label Claims, to the plan/roadmap

to achieve Product Profile and Planned Label. An on-going contract that captures the activities associated with specific Development activities and the Regulatory likelihood of success.

Label Claim: the exact wording/statement proposed in the final label that assists COMPANY in getting regulatory approval, pricing/access, and promotions, etc. The label claim can come from various label sections not just indications.

Planned Label Claim: the most competitive Label Claim we strive for. It must be realistic and have a high probability of success.

Fall Back Label Claims: alternative Label Claims if the Planned Label is not achievable.

Use of Product Profile versus TPP Product Profile: used to capture the key attributes of a product to be successful.

Typically created by Commercial Development to communicate key commercial attributes required. One can not promote from a Product Profile, as they are not written as label claims – particularly in early development.

Target Product Profile (TPP): discussion document used with FDA. Contains label claim statements that are promotable.

Terms we will not use: Target Label Learn Grid


Label As Driver Process

1. Define the Opportunity(what)

3. Receive Endorsements

A Well Differentiated, Value-Added Label, and a Sustainable Product

Deliver on important unmet medical need

2. Develop Roadmap

(how)


Label As Driver Process: 3 Major Activities in the overall Process

1.Define the Opportunity

(what)

3. Receive Endorsement

2. Develop Roadmap

(how)

LAD is an ongoing process to be done at each DP and as market or asset data changesLAD is an ongoing process to be done at each DP and as market or asset data changes

Articulate unmet customer need

Identify which label sections are critical success factors

Conduct competitive labeling assessment

Create MDI

Establish “planned” & “fall-back” position options

Develop & market test label claims

Present summary to Governance (BU/PRC) for endorsement with the target label as the core of the presentation

(Note: Allow for flexibility depending on BU/Governance)

Determine how to achieve the Label Claims (statements)

Data sources and endpoints

Trials/Study Designs Regulatory assessment

Identify why we can/can’t achieve desired product positioning

Use the LAD Grid as a contract to achieve the Label Claims


“As a PATIENT, I need...”

“As a PAYER, I can only reimburse if…... "

“As a PROVIDER, I see current methods lacking in this area…..it is a great unmet need that I would prescribe if available….

Speak from the perspective of a patient, payer, and provider.. Try to limit inputs to 2-3 key points per customer.

This is not asset specific at this time, just what the unmet need is.

PROVIDERS

PAYERS

PATIENTS

Summary of Unmet Customer Need

“As a REGULATOR, there is a need for a safer or more effective solution in this area…REGULATORS

“As a CAREGIVER, there is a need to be able to administer more simply…..CAREGIVER

8


(what)


2. Develop Roadmap:

Integrated program(how)


Promotional Claims/ Differentiation

• Which claims differentiate from competitors

• Which claims are key to acceptance as SOC

Revenue drivers

• Development program to encompass all target patient populations in revenue forecasts

• Critical issues for revenues e.g. duration of treatment

Market Access • Label must align with value proposition and value drivers

Fit with Clinical Practice

• Dosage, administration convenience

• No onerous requirement e.g. special monitoring

• Use with likely concomitant therapies

Additional Factors

• Requirement for diagnostic or biomarker

Red Flags • Acceptable limits for AEs, contra-indications

Label Sections *1: Indications and Usage2: Dosage and Administration3: Dosage Forms and Strengths 4: Contraindications 5: Warnings and Precautions6: Adverse Reactions 7: Drug Interactions 8: Use in Specific Populations9: (Drug Abuse and Dependence)10: (Over-dosage)11: (Description)12: (Clinical Pharmacology)13: (Non-clinical Toxicology) 14: (Clinical Section)15: (References)16: (How Supplied/Storage and Handling)

Identify Critical Success Factors


(what)


2. Develop Roadmap:


* Note: this should be done on global basis, based on asset & market needs* Note: this should be done on global basis, based on asset & market needs


COMPANY Competitor 1 Competitor 2 Competitor 3Critical Label Section X

– Specific claims – Specific claims –Specific claims –Specifi claims

Critical Label Section Y

– Specific claims – Specific claims – Specific claims – Specific claims

Critical Label Section Z

– Specific claims – Specific claims – Specific claims - Specific claims

Competitive Assessment

From the LAD process, highlight the competitor claims on only the critical

label sectionsProvide the conclusion or outcome

of this competitive assessment.

Conclusion……………………


(what)


2. Develop Roadmap:



LAD Worksheet

Dose

(size, type – tablet, LFC,

etc., number)

Regimen

(qd, bid, feed effect, time of

day, etc)

Route of Administration

(oral, patch, inhaled,

injected, etc.)

Packaging / Device options

Storage

Conditions (for biologics /

sterile pharms only)

PRESENTATION

Medical/ClinicalInput

Regulatory Input

Clinical and Regulatory to comment on Marketing’s inputs:

• Can what is being asked for be achieved through a clinical program?

• What is the relevant regulatory commentary regarding key requirements, precedents?

• What are risks we need to consider?

• If significant risk exists, are there mitigation options to consider?

“Must Have” Attributes

“Nice to Have”Attributes

Commercial Development & Access to populate “critical needs” and “other needs” columns as pre-work to the

LAD team kick-off:

• “Must Have” are items that must be achieved or the product is in jeopardy. These attributes represent the absolute minimum hurdles for commercial success

• “Nice to Have” should be thought of as upside options, or marketing/OR's wishes. We will value these and decide whether to pursue them later in the process. Note: These may be of extremely high value so should not be thought of as


(what)


2. Develop Roadmap:



Medicine A is indicated for the treatment of patients with [x]

Planned Claim: (best label claim)

Fall-back (1): (2nd best label claim)

Fall-back (2): (3rd best label claim)

Fall-back (3): (worse case label claim)

Establish Planned & Fall-backs Critical Claims

Outline in exact label claim wording your desired label and various fall back positions.

This is an opportunity to discuss the benefits of the best label claim, along with the

challenges/risks to achieve it.This can be similar to or based on a BOP (Best, Optimistic, Pessimistic profile) - but

put into language as we want the label claim to be written.


(what)


2. Develop Roadmap:



Label as Driver for PRODUCT “X”

Executive summary

Key differentiated /value claims to be outlined here

Product Vision

Projected positioning of the product to be entered here.

Label Section that provides differentiation eg. Indications, etc

Product Profile Claim / Labeling Statement Plan to Achieve

(Data Source and Endpoints)

Regulatory Assessment / Competitive Benchmarking

Ex: Treatment of specific diseases

n Specific language for indicationn Endpoints to be studiedn Population to be studied

n Study design (e.g. placebo/active controlled, X number of subjects, duration)

n PRO plansn Measurementsn Desired outcome compared to

SOCn Benefit/Risk Assessment

n Regulatory acceptability of Indication

n Regulatory adequacy of clinical database to support claims

n Regulatory assessment of comparators, dose, population, endpoints, SAPs and Benefit/Risk

n Consistent with guidelines?n Precedents, standards

(SBAs/EPARs/labels)n Agency acceptance of PROs /

tool validationn Discussions required

Ex: Prevention different aspect of disease

n Specific language for indicationn Endpoints to be studiedn Population to be studied

n Study design (e.g. placebo/active controlled, X number of subjects, duration)

n Different toolsn Benefit/Risk Assessment

Label As Driver (LAD) Grid – Label As Driver (LAD) Grid –

Describe the unmet need & value

proposition

Describe the unmet need & value

proposition

Describe how to achieve the claims

Describe how to achieve the claims


(what)


2. Develop Roadmap:


Include the exact wording in the

planned claims

Include the exact wording in the

planned claims

Provide robust Regulatory

assessment & Probability of Success

Provide robust Regulatory

assessment & Probability of Success


LAD Grid Example

PRA-027 Uterine Fibroids’ LAD Grid Development Strategy

TPP Claim / Labeling Statement Data Sources and EndpointsRegulatory Assessment /

Competitive Benchmarking

Value Proposition: For women who want relief from symptomatic fibroids, PRA-027 offers reduction of symptoms, and is a uterine-sparing treatment that reduces or delays the need for fertility-limiting invasive procedures. Unlike other PRMs, GnRH analogs, or procedures, PRA-027 therapy does not cause treatment-limiting endometrial changes, intolerable hypoestrogenic side effects, or expose women to the risks and costs of invasive procedures.

Positioning Statement: For women who want relief from symptomatic fibroids, PRA is the first nonsteroidal, oral therapy that unlike PRMs, GnRHs, or surgery, offers long-term resolution of anemia and symptoms, and reduces or delays the need for fertility-limiting procedures.

Target Audience: Women with symptomatic uterine fibroids, seeking long-term, effective, well-tolerated, uterine-sparing therapy.

Unmet Need: There is no long-term drug therapy available for treatment of bleeding or symptoms associated with uterine fibroids.

Key differentiation for PRA-027 vs. Lupron (GnRH Agonists): PRA-027 will have a simpler oral dosing regimen vs. provider administered injection. PRA-027 will not cause intolerable hypoestrogenic side effects, including osteopenia. PRA-027 will be safe to use for long-term suppression of symptoms.Meaningful point(s) of differentiation vs. surgery and potential PRMs, not yet on market No or fewer treatment-limiting endometrial changes PRA-027 does not diminish future fertility or expose women to the risks and costs of procedures Improved bleeding control [basis for belief? Bob M] More rapid onset of activity [basis for belief? Bob M] Fewer hypoestrogenic side effectsDisclaimer: Positioning Statements are forward-looking marketing tools that do not necessarily reflect the approved

promotional message for the product.

14


(what)


2. Develop Roadmap:



Label Claims – Life Cycle

If successful, the key claims (promotions, TV commercials, print ads, etc.) we will deliver are:

█ - High POS █ - Medium POS █ - Low POS

AtLaunch

0-2YearsPost

Launch

LongTerm


(what)


2. Develop Roadmap:


for internal use only 1 breakout materials mdi lad rww

Documents

competitive label

target label

valueadded label

final label

planned label claim

appropriate use label

label claim statements

alternative label claims