perspectives on pain points, unmet needs, and disruption

DRAFT

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Perspectives on Pain Points, Unmet Needs,

and Disruption in Precision Oncology

DeciBio Consulting, LLC

725 Arizona Ave, Suite 202

Santa Monica, CA 90401

Phone: (310) 451-4510

Email: [email protected]

www.decibio.com

DeciBio Contacts:

Andrew Aijian: [email protected]

Colin Enderlein: [email protected]

Companion Diagnostics Forum 2019

Princeton, NJ

Disclaimer

Some of the companies listed in this document may be

DeciBio Consulting Clients or Customers; However, this

project is entirely self funded by DeciBio Consulting

DRAFT

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DeciBio’s mission is to provide insights that drive disruption and innovation in the precision medicine and life

science industries; to this end we conducted internal research to assess the following two questions

1. What are key pain-points and unmet

needs in the precision medicine ecosystem

today and how do these translate into

opportunities for precision medicine

stakeholders?

2. What are potential scenarios for the

evolution of the precision medicine

landscape and what are the potential

implications for various stakeholders in

these scenarios?

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0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Landscape Vertical Business Function

N = 22N = 22

Dx Specialist

Tx Specialist

Director

Clinician / Patient

Specialist

VP / CXO

Clinical /Patient

Journey

Specialist

We conducted >20 stakeholder interviews with senior executives, oncologists, and KOLs in a variety of settings

Example Interviewee Demographics (N = 22)*

Notes: * Multiple stakeholders were able to speak firsthand about several landscape verticals; not all organizations shown

Source: DeciBio Interviews

RWD /

RWE

Specialist

Dru

g D

ev.

Sp

ecia

list

RW

D / R

WE

Sp

ecia

list

Clin

ica

l / P

t

Jo

urn

ey

Sp

ec.

Dx

Sp

ecia

list

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science industries; to this effect we conducted internal research to assess the following two questions





stakeholders?





these scenarios?

DRAFT

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Unmet

Need /

Opportunity

Demand

(High)

Supply

(Lowi)

Favorable Economics

Supply

(High)

Demand

(Lowi)

Less Favorable Economics

Ideal Innovation Scenario (“First Principles Innovation”) Less Ideal Innovation Scenario (“Reverse Innovation”)

Many companies with innovative technologies or approaches have a hard time gaining market traction due to

poor visibility of market needs or inability to find a “key application”; the objective of this research is to identify

unmet needs in precision medicine to help drive “first principles innovation”

Need?

Need?

Need?

Need?

Solution

(technology,

product,

service)

Solution

(technology,

product,

service)

To pinpoint precision medicine painpoints, we first created a map of the precision medicine landscape,

highlighting the key steps and processes, and pressure-tested it with interviewees from various backgrounds

Care Team JourneyBasic R&D

Translational R&D

Clinical Trials

Regulatory

Reimb. / Coverage

Regulatory

Reimb. / Coverage

Clinical Trials

Translational R&D

Basic R&D

Dev. Timeline / Resource Coordination

Deal / Partnership Terms Management

Partner Identification

Planning/ Design

Recruit./ Enrollment

Biomarker/CDx Dev.

Data Management

& Analysis

Planning/ Design


Biomarker/CDx Dev.

Data Management

& Analysis

Clinical Trial

Diagnosis

Primary Care / Screening

CDx / Treatment Selection

Treatment Response Monitoring

Surveillance

Sample Collection

Sample Prep

Analysis

Reporting

Decision Making

Coverage / Reimb.

Sample Collection

Sample Prep

Analysis

Reporting

Decision Making

Coverage / Reimb.

Treatment Availability

Auth. / Coverage

Dosing

Treatment Administration

Toxicity management

Response Evaluation

Resistance detection / Testing

Ongoing response evaluation

MRD monitoring / detection

RWD/EGeneration

RWD/EAnalysis

RWD/EIntegration

RWD/E Privacy Protection / Management

Implementation of RWE into R&D

Physician Education /

TrainingPatient

Communication

Care Team Coordination /

Communication

Patient Data

Management

Palliative Care / Other

Dx. Mfr.

Drug Mfr.

Dru

g /

Dia

gno

stic

Co

-Dev

elo

pm

en

t

Drug Development

Diagnostics Development

Patient Journey

RW

D/E M

anage

me

nt

Data management

& analysis

Basic R&D

Translational R&D

Clinical Trials

Regulatory

Reimb. / Coverage

Regulatory

Reimb. / Coverage

Clinical Trials

Translational R&D

Basic R&D

Dev. timeline / resource coordination

Deal / Partnership Terms Management

Partner Identification

Planning/ Design


Biomarker/CDx dev.

Data management

& analysis

Planning/ Design


Biomarker/CDx dev.

Clinical Trial

Diagnosis

Primary Care / Screening

CDx / Treatment Selection

Treatment Response Monitoring Surveillance

Sample Collection

Sample Prep

Analysis

Reporting

Decision Making

Coverage / Reimbursement

Sample Collection

Sample Prep

Analysis

Reporting

Decision Making

Coverage / Reimbursement

Treatment Availability

Auth. / Coverage

Dosing

Treatment Administration

Tox. management

Response Evaluation

Resistance detection / Testing

Ongoing response evaluation

MRD monitoring / detection

RWD/EGeneration

RWD/EAnalysis

RWD/EIntegration

Health Data Privacy Protection / Management

Implementation of RWE into R&D

Physician Education /

Training

Patient Communication

Care Team Coordination /

Communication

Patient Data Management

Dx Mfr.

Drug Mfr.

Care Team Journey

Dru

g /

Dia

gno

stic

Co

-Dev

elo

pm

en

t

Drug Development

Diagnostics Development

Patient Journey

RW

D/E M

anage

me

nt

Stakeholder feedback reveals multiple pain points and unmet needs distributed throughout the precision

medicine landscape, the needs / pain are fragmented with no single “node” representing a large majority

6

1

Pain Point / Need Mentions

Significance Weight

100

0

1 The models we use to study cancer and discover new therapies and biomarkers are too simplistic

2 Precision medicine creates unique, and increasingly challenging, barriers to clinical trial participation

3The current commercial and technological health IT infrastructures are fundamentally incapable of

enabling of the potential of RWD/E

4 The tools and methods for educating clinicians on precision medicine are lacking or inadequate

5Numerous siloes exist within and between the different branches of the precision medicine landscape

impeding progress

Aggregating feedback from interviewees reveals key pain point and unmet need themes, and thus,

opportunities, for stakeholders in the precision medicine landscape

High-Level Paint Point / Unmet Need Takeaways

• Cancer research models are too simplistic

o The lack of in-vitro models that reflect systems biology hinders drug discovery

▪ “…2D tissue culture is worthless, 3D spheroids are better, and organoids are

even better, but these are still not sufficient…”

▪ Gene expression and multi-omics tools are a step in the right direction, but

price and TAT are too high and data analysis is too intensive to support high-

volume use necessary for dynamic, iterative testing of a systems model

❑ Complex data sets and intensive analytical pipelines can create

disconnects between the people asking the questions (biologists) and

those analyzing the data (bioinformaticians), creating inefficiencies

• Advancements in proteomics have not kept pace with genomics

o “…Proteins are the ground truth, but we still can’t do a 10,000 plex protein assay;

some aptamer techs get us partially there, but we need advancements in

proteomics to make strides in discovery…”

• Research sample acquisition and management is inefficient

o Even major pharmaceutical and biotech companies have difficulty securing and

managing (accurately annotated) research samples; sample challenges can add

weeks to months to research project timelines

▪ “…Everyone talks about cool analytical technologies, but samples are a huge

problem…liquid biopsy compounds this because now you have to acquire

and track samples from multiple time points…”

9 / 22interviewees identified drug

and / or diagnostic translational research as

pain points

~14%Of the total weight of all the pain points identified

1Drug and diagnostic translational R&D represents one of the biggest collective pain point areas, driven

primarily by the need for better disease models and diagnostic tools that capture systems biology complexity

• Precision medicine clinical trials are-, and will become, increasingly

challenging to execute

o Increasingly complex biomarkers and trial protocols will raise the burden of

clinical trial participation on patients, making enrollment more challenging

o More complex biomarker and treatment strategies (e.g., rare / composite /

multiplex markers and rational combination therapies) require more patients

to reach data significance, increasing the time and cost of trials (and drugs)

▪ “…We need to do rational combinations for cancer like we do for

antibiotics, but the current clinical trial and regulatory frameworks are

not built to support the types of trials we need for precision medicine…”

• However, fundamental barriers exist for clinical trial enrollment

o Despite the promise to facilitate trial enrollment, the quality and quantity of

RWD/E is lacking; access to data is hampered by red-tape, legalese

▪ “…Drug developers have a poor line of sight into clinical trial recruitment

due to lack of good data / access to data… ”

o Additionally, more importantly, many patients have a negative perception

and / or limited access to clinical trials

▪ “…Many patients think a clinical trial means they have a 50% of getting

nothing more than a placebo; they see it as being a guinea pig…”

o Patients prefer to be treated in their communities; for the most part, a patient

is willing to travel up to 50 – 80 miles to participate in a trial; doing so

creates a large burden on the patient and their family

8 / 22interviewees identified

clinical trial-related activates as pain points


Aggregate enrollment targets for newly initiated

cancer trials in the U.S. have averaged ~500,000

patients annually over the past 3 years,

corresponding to 25 – 30% of all newly diagnosed

cancer patients in the U.S.;

Current enrollment rates are 4 – 6%

The rising stakes and requirements for precision medicine trials erect barriers that exacerbate an already

challenging patient recruitment problem; the current trial system may not support the types of trials needed

2

v

2

0 - 509

510 – 1,650

1,651 – 5,000

5,001 – 17,800

17,801 – 115,000

While access to precision medicine testing is generally considered good, access to precision medicine care /

treatment is limited, to a large extent, geographically

Population by Zip Code

Trial site willingness

to travel radius

(max ~80 miles)

While many companies focus on clinical trial matching, there is a large share of the population that does not have easy access to

precision medicine clinical trials, creating a huge need for remote clinical trial solutions

Phase 1

Phase 1/2

Phase 2

Phase 3

Trial Phase

Map of Currently-Recruiting “IDH1” Trials*

* Note: Excluding AML, for which an FDA-approved therapy is available; Source: ^ doi: 10.1016/j.conctc.2018.08.001

This distribution of trials

is representative of many

other precision therapies

“…There is a last mile problem in

precision medicine…”Studies suggest that 25% of trials failed to enroll

a sufficient number of patients, and 18% of trials

closed with less than half of the target number of

participants after 3 or more years^

Match

Match

• The current health IT infrastructure is not designed for RWD/E

o “…EMRs were primarily designed to facilitate billing, not cataloging and

sharing medical information, and are fundamentally incapable of enabling

the full potential of RWD; we need systems built specifically to capture

and share clinical data…”

o Numerous barriers exist to the utilization of RWD

▪ EHR interfaces are not designed for efficient, standardized data

collection

▪ Clinicians do not have the time / incentivization to enter data into

charts / EHRs in an RWD-friendly way

❑ “…In one study, we found >70 different ways clinicians referred

to or mentioned ‘leukocytes’…”

▪ Almost every stop on the patient journey has a custom / different

EHR, which doesn’t easily speak to any other EHR

• Numerous competing commercial interests are erecting barriers to the

advancement of RWD/E

o “…HCPs are becoming increasingly possessive of their data for

commercial reasons, making it hard to access…”

o The commercial fragmentation leads to data fragmentation, and varying

degrees of data quality; this lack of standardization and quality may

“poison the well” for RWD if it creates more confusion than clinical value

The current commercial and technological health IT infrastructures are perceived as fundamentally incapable

of capturing and integrating health information in a way that enables the realization of the potential of RWD/E

3

12 / 22interviewees identified drug

and / or diagnostic translational research as

pain points


KOLs believe we are 3 -

5 years away from

achieving the scale and

quality of RWD

necessary to start

realizing meaningful

clinical value

Ref

Lab 2

• For the most part, the patient journey is highly decentralized, with data being generated across multiple different sites and EHRs

o “…At each handoff much of the valuable data generated is lost; there is a lot of redundancy in data generation because care

providers don’t seek out, can’t access, or don’t trust data generated in previous steps in the patient journey…”

• Many RWD/E aggregators rely on a network of partnerships with individual hospitals of providers, however, any given hospital / provider

in the network likely represents only a small slice of a patient’s data

o Much of the emphasis is placed on acquiring and integrating clinico-genomic data, but there are many other untapped data sets

Ref

Lab 1

Most RWD/E efforts rely on stitching together disparate clinical and genomic data sets with varying degrees

of overlapping patient data; this approach does not generate a comprehensive picture of a patient’s RWD

Summary of Common RWD Aggregation Approach Current Focus of RWD Aggregation

3

RadiologistPCP /

SpecialistSurg. Onc Pathologist

• Family history• Clinical chemistry labs• Baseline vitals• Symptomatic vitals• Physical health

• Scans / images • Operative reports

(gross tumor data,

surgical staging)

• Path reports

(diagnosis, staging,

typing, biomarker

expression)

• Slide images

Each of these providers may be at a different site with a

different EHR / PACS / LIMS system

• Treatment data

• Response /

outcomes data

• Toxicity data

Med /

Rad Onc

• Biomarker data • Biomarker dataRWD

Aggregator

9 / 22interviewees identified CDx /

Treatment Selection and Physician Education as pain

points


• The pace of change in oncology is too high for clinicians to keep up with

o “…many community oncologists are generalists and see multiple cancers, it

is almost impossible to stay up to date with every change to the SOC…”

▪ Precision medicine leads to many “grey areas” for clinicians; e.g.,:

❑ “…Start patient on chemo while waiting for CGP test results?...”

❑ “…What if there are 2 potentially clinically-actionable variants?...”

❑ “…What if you have a VUS in a known oncogene?...”

▪ Currently, clinician education efforts are more passive (e.g., websites

and webinars) than proactive (e.g., field medical people in the offices)

▪ Additionally, hospital labs, which are often perceived as cost centers,

are not receiving the resources necessary to execute on the

accelerating demands of the precision medicine imperative

• There is a lot of marketing “noise” from precision medicine test vendors

o “…There are too many test options, each with a salesperson pitching a

different story / value proposition; it is difficult to tell what is necessary and

unnecessary and what is validated, reimbursed, etc.…”

o The lack of clear guidance / standardization on how to implement precision

medicine creates a barrier to adoption (no one makes treatment decisions

they are not well-informed of)

The inability of providers to keep pace with the clinical and financial resources necessary to support

precision medicine limits broad adoption; better tools are needed to support clinician decision-making

4

v

In 2019 YTD there

have been 92 updates

to 26 different NCCN

guidelines, an average

of ~3.5 per guideline

per year (so far)

Significant siloes persist throughout the precision medicine ecosystem; these siloes create inefficiencies and

friction that hinders advancement and adoption of precision medicine

Dru

g /

Dx

Co

-Dev

elo

pm

en

t

Care Team Journey

• Misaligned incentives and timelines, and challenging partnership

coordination can create tension between drug and diagnostic co-developers

o Some stakeholders perceive precision medicine to be largely reactionary

(e.g., only when needed) rather than a proactive effort by drug-developers

o Delayed dialog / engagement between drug and diagnostic developers around

CDx development creates multiple issues:

▪ Dx vendor / technology evaluation is rushed, potentially resulting in the

selection of a suboptimal technology in terms of performance or

accessibility

▪ Inability to align on terms and timelines can force drug developers to take

on risk and proceed without a biomarker to get to market sooner

▪ “…The timeline is fast.…each party has different internal timelines and

multiple siloes of teams that delay decision making; almost all deals are

de-novo with little leverage provided from previous deals…”

• Similarly, misalignment between oncologists and pathologists can lead to

suboptimal, delayed, or unnecessary (i.e., redundant) care

o “…The care journey is very fragmented, a lot of people switch care throughout

their journey, and at every handoff, all of the valuable information is lost.…”

o There is a disconnect between the person who orders the test (oncologist)

and the person who implements the test (pathologist), which creates

confusion

Only ~33% of all pain

points identified were

outside of interviewees’

own branches of

participation

5

12 / 22interviewees identified these

siloed areas as pain points


Disease models that better capture

systems biology

Decentralized clinical trial solutions;

clinical trial patient education

Patient-centric RWD management

solutions

Multi-source / multi-modal RWD

integration (beyond genomics + clinical)

Sample access, information, and

management solutions

Next-generation proteomics tools

Interfaces that better support RWD

capture

Despite the paint points that exist, all stakeholders interviewed are optimistic overall about the future of

precision medicine; the pain points and unmet needs represent tangible opportunities for market participants

Opportunities for Precision Medicine Stakeholders

DRAFT

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science industries; to this effect we conducted internal research to assess the following two questions





stakeholders?





these scenarios?

DRAFT

18

Considering what changes will occur in the distant horizon (5-10 years) is a valuable lens through which to

understand where growth will most rapidly occur vs where we may see ongoing bottlenecks

Future Changes: In 5-10 years, where do you expect to see the most significant changes?

Specific Interview Questions

• Which verticals / nodes of the precision

oncology landscape do you expect to

change most in 5-10 years? Change least?

• What will be the key drivers of change

across each of the verticals?

• How will future changes address /

exacerbate current pain points?

• Who will benefit most from these future

changes? Who will benefit least /

experience new barriers?

• Will any nodes be added or removed from

this landscape map?

• Under your vision for the future landscape,

what are some potential scenarios

highlighting these changes?

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Across precision medicine, the Dx space is expected to see most evolution over 5-10 years; RWE / health AI

changes expected, but long-term vision still abstract

Stakeholder Feedback Highlighting Key Changes / Trends

Expected for Precision Medicine in a 5-10 Year Timeframe

# of Mentions Theme* Precision Med. Vertical

12 Dx Improvement Dx

11 Tx Improvement Tx

11 Screening Dx

10 Patient Empowerment Clin

9 Multi-Modal Data Linking RWE

9 Trial Improvement Tx, Dx

8 Bearish on Precision Med. All

7 RWE Drives Research Engine RWE

6 Pharma as key Precision Med Driver Tx

5 Cost / Payer Access Improves All

5 Organic Precision Med Expansion All

5 Value Based Care RWE

5 Other All

5 LBx Emerges as Standard of Care Dx

4 Trend of Healthcare Consolidation Clin

4 Physician Education Clin

4 Tumor Agnostic Movement Tx, Dx

4 Dx / Tx Parallel Development Tx; Dx

3 Dx Precision Med Tools Reflexive Clin

2 EMR Improvement RWE

2 Trial Headwinds Tx 0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Distribution of Expected Changes by PM Vertical**

Dx

Changes

Tx

Changes

Clin

Changes

RWE

Changes

Key Takeaway – Significant disruption is expected across all verticals of precision medicine; however, key areas with defined evolutionary steps map closely to

current pain points (i.e. Dx, Tx), while changes in other areas are less concrete

Note: * Themes in Orange will be explored further; ** Expected changes have been normalized based on total stakeholder distribution by precision medicine vertical

Source: DeciBio analysis, Primary Research

DRAFT

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The Dx space is not only expected to see the most changes, but changes were highlighted by specialists

from each vertical

~35% of Key Changes

Expected in Dx Space*;

~75% of Claims Come

from Non-Dx Specialists

~25% of Key Changes

Expected in Tx Space*;

~80% of Claims Come

from Non-Tx Specialists

~20% of Key Changes

Expected in Clin / Pt Journey

Space*; ~55% of Claims Come

from Non-Clin Specialists

~15% of Key Changes

Expected in Clin / Pt Journey

Space*; ~65% of Claims Come

from Non-RWE Specialists

Note: * Expected changes have been normalized based on total stakeholder distribution by precision medicine vertical


Bubble size corresponds to

# of key change mentions

DRAFT

21

Dx & Tx improvements were the most identified changes, with implications spanning all precision medicine;

while changes are expected, stakeholders are uncertain about funding mechanisms

Dx Development RWE / Health AITx Development Clinical / Patient

To

p L

an

ds

ca

pe

Ch

an

ges

Ke

y S

cen

ari

o

• Screening to become routine, driving

detection of earlier stage disease

• Improved Dx frontloads data for

multiple Tx lines

• Algorithm refinements may be basis

of Dx dev. given WES availability

More Tx’s will enter the market,

particularly tumor-agnostic ones

guided by mutation markers;

expanding PM* access across

settings as more Txs reimbursed

~51 Mentions of

Change in this Space

Diagnostics overall will improve,

driving an increase in CDx

offerings, which will be key to

expanded Tx access; mutation

panels may trend towards WES

~38 Mentions of


• The need for physician education

rises amidst more Tx / Dx choices

• Tumor agnostic marker approvals

will emerge

• Precision medicine will expand in

community settings

~28 Mentions of


Patients will become increasingly

empowered to guide care journeys;

better education and advocacy

groups are driving patients to

proactively request PM*

• An increasingly holistic medical

approach will be expected, with

improved continuity of care

• Patient brand awareness becomes

driver of product adoption

• Physicians expected to field more

questions

~21 Mentions of


Linking the multitude of data sources

from individuals & populations

increasingly prioritized; EMR

improvements key, and data

consolidation likely pharma driven

• EMRs may focus on pt. data /

outcomes vs billing

• Pt. data from wearables and lifestyle

factored in

• New outcome measures / biomarkers

emerge

Dx Stakeholder Tx Stakeholder RWE Stakeholder Clin. Stakeholder

0 20 40 60

Note: * PM = Precision Medicine; ** CH = Community Hospitals


Mentions by

Stakeholder

Type

17 13 10 11

0 20 40 60

13 6 10 9

0 20 40 60 0 20 40 60

6 5 4 13 6 4 7 4

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Following diagnostics improvements, key developments include screening expansion that could drive a shift

towards earlier cancer treatment, and large panel / whole exome tests see expanded use

Dx Development

Key Change – Diagnostics Improvements –

Identified by 12 Stakeholders

Subtopic 1 - Screening for Early /

Pre-Cancer Expands

Routine screening will be performed in

primary care settings (perhaps as a

liquid biopsy); cancer will be detected at

earlier stage and can be quickly referred

Subtopic 2 - Large Panel Testing

Becomes Standard

Comprehensive genomic profiling /

WES may become routine for all

mutation-based testing; a single test

could inform multiple rounds of Tx


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Increased screening is one of the most expected outcomes to emerge from upcoming Dx improvements;

cancer will be detected earlier, directly impacting future Tx & Dx development towards earlier stage disease

Dx Development

Sub Theme – Dx Developments Lead to Expanded Early Screening


Screening for

Early / Pre-

Cancer

Expands

Pharma

Catching patients with earlier disease could shrink

the metastatic population for systemic therapies;

refocus into adjuvant or pretreatment

Large potential screening

population may force payors to put

pressure on screening assay prices

Market for adv. Cancer CDx testing may face

pressure, driving Dx developers to pursue new

tests (i.e. recurrence monitoring, site-of-origin)

Screening could be key in shifting treatment from systemic to localized approaches; overdiagnosis poses key risks

Likely / Near-Term Somewhat Likely / Mid-Term Uncertain / Long-Term

Dx

Investment in new metastatic cancer

drugs may decline; precision treatments

for early cancer could expand

Payors

Patients

Increased screening could actually lead

to overdiagnosis; patients may undergo

costly & unnecessary follow-up / Tx

Overall cost per patient could

increase as cancer is treated like

a chronic vs acute illness

“…Rather than adjuvant, use tests to tailor Tx or change

course will be key; great opportunities for neo-adjuvant or

maintenance Tx as we detect earlier cancer…”

-SVP, Guardant Health

Current technical limitations (namely

test specificity) could lead to 10s-100s

of thousands of misdiagnosed patients

DRAFT

24

Large panel testing will frontload tremendous amounts of patient data, allowing multiple Tx lines to be

determined early; the technical requirements will like continue driving testing into centralized settings

Dx Development

Sub Theme – Dx Developments Lead to Expanded Use of Large Panels / WES


Large Panel

Testing

Becomes

Standard

Pathologists

All data needed for future Dx

development derived from

existing panels; new tests

based on algorithm refinement

Test / bioinformatics capabilities leads to

increased test centralization and

decreased in-house pathology

Future Dx development may focus on scalability and algorithm refinement vs platform / chemistry; cost may gate access


Health AI

“…WES & WTS will remove need for anything more to characterize cancer;

maybe the whole Dx process would disappear in place of signature refinement…”

-VP, Personalis

Patients

Single large test becomes

standard at first line, informing

multiple lines of Tx without retesting

Added data could help ID

patients eligible for trials

Pan tumor TKI’s see increased use as

more patients have known mutations

Pharma

Pharma fully underwrites CDx testing

as pricing per test becomes increasingly

marginal compared to Tx expense

Dx

Development priorities refocus on

batching and scalability for core labs

rather than decentralized workflows

“…Tx companies will start carrying strategy burden that Dx companies usually have;

lack of Dx uptake will limit their drugs, but they have resources to remove barriers…”

-KOL, Third Rock Ventures

DRAFT

25

Therapeutic Improvements are expected to drive access across care settings and improve survival, but could

also enhance education burden and create reimbursement complexities if payers are outpaced

Tx Development

Key Change – Therapeutics Improvements –


Subtopic 1 - Increase in the Number

of Tumor Agnostic Drugs / Indications

The number of new available drugs and

combination regimens will increase; new

approvals will be based on biomarkers

rather than tumor origin

Subtopic 2 - Improved Efficacy &

Survivorship

New treatments and catching earlier

disease will boost efficacy and extend

survivorship; cancer may be increasingly

viewed as a recurrent lifelong vs acutely

terminal disease


DRAFT

26

Even when patients have druggable mutations, they frequently cannot receive therapies if the tumor origin is

off-label; more agnostic approvals will expand therapy access and drive broader mutation testing

Tx Development

Sub Theme – Therapeutic Developments Lead to More Tumor Agnostic Approvals


Increase in

the Number

of Tumor

Agnostic

Drugs /

Indications

Physicians

Community settings will have a vastly

expanded Tx arsenal; trials will not

be the only option for late line Tx

The pressure and complexity for ongoing

physician education (especially oncologists in

community settings) will increase

Precision medicine could see uptick in community settings, driven by pan-tumor Tx; may increase education burden


Dx

Large panel tests will be ordered with

more regularity regardless of tumor origin

Pharma

More pan-tumor basket

studies will be initiated

Health AI

RWE will drive future R&D; speeds

time to clinical stages and pivotal

to guiding basic R&D strategy

Future pathologists could play the role of

key advisors on tumor boards as test

options become increasingly complex

“…The role of the pathologist will

become more important in driving

responsible testing; also educating

different stakeholders on findings …”

-CXO, PathGroupOverall cost of Dx expected to increase

as low-plex technologies replaced

DRAFT

27

Therapies will naturally become more efficacious, boosting survivorship and become curative vs palliative;

treating cancer as a disease you will fight at several points in your life is new for payor discussions

Sub Theme – Tx Developments Leads to Improved Long-term Survivorship


Improved

Efficacy &

Survivorship

Patients

More patients will be subjected to long-term

maintenance Tx and monitoring; could

increase cost per patient given prolonged Tx

Patient advocacy groups could swell in

numbers, helping drive education that

will propel patient empowerment

Payors and physicians will need to make fundamental changes to accommodate curative/long-term vs palliative treatment


Payors

Insurance providers struggle with expense

for minimal survival in late stage disease;

reimbursement discussions will refocus on

long-term vs acute care

Physicians

Primary care physicians will play

increasing role in long-term follow-up

care; education burden falls on them

Tx Development

Pharma

Drug development focuses on

further maintenance &

recurrence Tx development

Payors may not tolerate exponential price increases for

incremental survival benefits; possible that drugs only

covered if years rather than months of life added

DRAFT

28

Patient Empowerment through education and growing advocacy groups present growth opportunities as

patients proactively request precision medicine

Clinical / Patient

Key Change – Patient Empowerment –


Subtopic 1 - Patient Education / Advocacy

Will Drive Precision Medicine Demand

Increasing resources will be available to

patients at all stages of care; improved

survivorship will drive growth of both

established and rare cancer advocacy groups

Subtopic 2 - Patient Data Ownership &

Generation Sees Growth

New direct to consumer offerings for

testing and wellness (i.e. 23&Me, FitBit)

will serve as data sources; patients are

eager for clinical actionability of their data


DRAFT

29

An increasing number of resources are becoming available to patients, leading to proactive requests for

certain procedures or Txs; the onus will fall on clinicians to help facilitate care based on clinical evidence

Sub Theme – Patient Empowerment Through Education / Patient Advocacy


Patient

Education

Will Drive

Precision

Medicine

Demand

Patients

As patients conduct more research,

they are more likely to be influenced

by brand name tests & treatments

Patient education & advocacy could add some burdens, but will ultimately be a strong driver for precision medicine uptake


“…Telehealth will come online and play a big role in

expanding access to expert care teams regardless of

location; Consultations could be decentralized…”

-Patient Advocate

Physicians

Patients requesting specific treatments

/ tests could add further pressure to

ongoing physician education

Large panel testing could increase

/ become reflexive as patients

increasingly request advanced tests

to understand all Tx Options

Dx

Payors

Patient willingness to pay out of

pocket for additional information/

clinical actionability may increase

Clinical / Patient

Patients may expect increased

responsiveness from physicians; this will

add to clinician burden; offers opportunities

for software solutions

Software

Devs.

“…More patient becoming aware of precision med. options;

they will see commercials and ask for specific tests / Txs…”

-Oncologist KOL, USC

Patient advocacy groups could act as valuable

call-points for trial enrollment; endorsements

present new marketing opportunities

DRAFT

30

Driven by increased overall education and more technical resources, the rate at which patient data is

generated will continue to accelerate, creating new opportunities and challenges across the landscape

Sub Theme – Patient Empowerment Through Expanded Data Ownership


Patient Data

Ownership &

Generation

Sees Growth

Patients will play a larger role as RWD generators; handling these data will require EMR and education advancements


Clinical / Patient

Dx

Diagnostics / test developers will have

an increasing number of offerings to

generate data at all stages of care

Patient

Trends towards wellness and

medical education will drive

patients to acquire more data

from tests and self reporting

New digital biomarkers could emerge

as sufficient data from wearables and

lifestyle factors are consolidated

Health AI

EMRs will need to evolve in

order to accommodate these data

Physician

Pharma

Opportunities for Tx developers to

offer holistic care (i.e. pre-

cancer) will emerge as broader

patient data enters the ecosystem

Physicians will be ill-equipped to handle

self-reported patient data without better

tools (i.e. health data tracked on phone)

“…Current EHR is system fundamentally

flawed in ability to collect RWD & follow-

up; it Needs to be rebuilt from scratch…”


Some patients will want to

commoditize their data;

others will be wary of security

DRAFT

31

It is generally agreed that vast amounts of useful clinical information are already generated; multi-modal data

linking is the next step to unlocking new therapy R&D

RWE / Health AI

Key Change – Multi-Modal Data Linking–


Subtopic 1 - EMRs Evolve to Accommodate

New Inputs

Data from internal clinical channels (i.e.

pathology, radiology) sees better integration;

clinical notes and outcomes more closely

tracked in a curated format

Subtopic 2 - Value Based Care

Becomes Expected

As Tx costs continue to soar, public &

private payers will increasingly demand

‘value based pricing’; health outcomes

beyond clinic are more actively tracked


DRAFT

32

EMRs are a known chokepoint in the expansion / actionability of RWD; improvements to handle multi-modal

data inputs while reducing physician workload are a known need

Sub Theme – Multi-Modal Data Linking Supported by EMR Evolution


EMRs

Evolve to

Accommodate

New Inputs

EMR’s to be modified / rebuilt to be

based around data collection /

analysis, rather than billing

Few EMR improvements are expected to occur near-term; significant disruption may be needed to drive advancement


Payors

RWE / Health AI

Software

Devs.

Physician

New platforms ideally streamline

physician data entry by integrating

natural language processing

Patients

Data may become patient rather

than hospital centric, smoothing

referral, reducing redundancy

Payors to more proactively track

biomarkers and their links to efficacy;

potential to adjust premiums based on risk

Payors develop their own

EMRs; Track outcomes and risk

factors to proactively make

recommendations to patients

PharmaAs data landgrab

continues, legitimacy

around what constitutes

‘gold standard RWE’

becomes obscured

“…Could create a parallel EHR system; we can’t stay

in context in billing but need to make it actually

focused patients and what tests are conducted…”


DRAFT

33

Payers and patients will increasingly demand greater value in the face of increasing drug prices; RWE has

the potential to demonstrate added value, but will also increase pressure on pharma to deliver improvements

Sub Theme – Multi-Modal Data Linking Enables Value Based Care Assessments


Value-Based

Care

Becomes

Expected

Payors

Payers could be key advocates of

post Tx surveillance / monitoring

test development, improving

efficacy vigilance

Factors like adverse events and quality of

life may be tracked more closely as new

therapies enter with incremental efficacy

Incremental survival may no longer be acceptable to payors as RWE makes its way into health econ. evaluations


Dx

RWE / Health AI

RWE rather than clinical guidelines

used to set reimbursement prices

More confusion about

recommended clinical

steps / Tx expense

Pharma

New clinical outcomes / trial endpoints will need

to be adopted as incremental survival benefits are

no longer accepted by payers at list price

Physicians

Greater bifurcation

occurs as advanced

settings consolidate

RWE and entry

barriers grow“…As more data is generated and consolidated by Drs., leads to bifurcation; the educated

get smarter and more tuned into care, but others feel more distant from overall Tx…”

-Patient Journey Expert

Predictive biomarkers to be improved at the

expense of further limiting patient populations

Predictive biomarkers to be improved at the

expense of further limiting patient populations

DRAFT

34

Following changes that will permeate every level of precision medicine, there are several key considerations

for developers to keep in mind as they work towards future innovations

Physician Education Burden – Many precision medicine advancements risk adding to physician education / time

burden

Key Considerations for Precision Medicine Developers

1

Pricing for Long-Term vs Acute Cancer Care –Treatment may become episodic vs acute; adverse events less

acceptable, and value-based care will be key

2 Shift Towards Earlier Stage Disease – be prepared to treat healthier patients in community settings with localized

rather than systemic interventions

3

Data at All Points of Patient Journey Captured – Care needs to be taken to generate / record data in a

standardized manner; software needs to enable this4

Care Decentralizes / Testing Centralizes – Patients increasingly expect care with minimal traveling; technical /

bioinformatics requirements consolidate future testing5

DRAFT

35

Thank you for your time and attention – We are happy to answer any questions

Special Thanks To:

• Tom Fare and the PlanetConnect team

• Colleagues at DeciBio who contributed to this analysis

o Stephane Budel – Partner

o David Cavanaugh – Partner

o Fanny Anderson – Associate

o Seth Schachter – Associate

• All stakeholders who participated in primary research

For more information about DeciBio, visit us at www.DeciBio.com;

These slides will be made available via PlanetConnect; Please connect directly with us for additional insights

perspectives on pain points, unmet needs, and disruption

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