value chain insights for nextgen therapies · may 2016. imlygic. apr 2010. nov 2015. aug 2016. aug...

May 8, 2019

Value Chain Insights for NextGen Therapies

Copyright © 2019 Deloitte Development LLC. All rights reserved. 2

Agenda

1

2

3

4

Introductions

NextGen Overview

NextGen Treatment Journey

Key Insights & Implications


Introductions

Hussain Mooraj

NextGen Practice Lead

Sanjay Srivastava, PhD

CAR-T Value Chain & NextGen Lead


To uncover stakeholder needs for managing the complex NextGen therapies, we visited major transplant centers globally and stood up a NextGen IWG1

F o u n d i n g M e m b e r s o f I W GG l o b a l P r i m a r y R e s e a r c h S c o p e

*Not Confirmed

1: Industry Working Group

Copyright © 2019 Deloitte Development LLC. All rights reserved. 5Patient Connect /Elevate the Patient ExperienceCopyright © 2019 Deloitte Development LLC. All rights reserved. 5

NextGen Therapies Overview


NextGen therapies are of two types – gene and cell therapies

Gene Therapy* Cell Therapy**

Allogenic Autologous

*Source: Research Gate publication (link)**Source: Apceth MSC platform (link)

NextGen Therapy Approaches

Exam

ples

of

man

ufac

ture

s

One healthy donor

One tissue source

Master cell bank

Several batches cell product

Treatment of several patients

One patient

One tissue source

One batch of cell product

Treatment of one patient

PCV

https://www.researchgate.net/publication/281779310_Emerging_and_future_therapies_for_hemophilia

http://www.apceth.com/allogeneic-msc-platform


Stakeholder interactions for NextGen are significantly greater than those with traditional pharmaceutical products

A cross-constituent collaboration where many more players within the ecosystem are needed to provide enhanced value to the patient and sustainable competitive advantage to the LS

company

CAR-T Cellular Therapy Model

• Source of T-cells, which are the raw material for the CAR-T product

• Drives the therapy process and is the bio-reactor• Is very fragile and immuno-compromised, who

requires resources to cope with the demands of the therapy

• Genetically re-engineer the harvested T-Cells to recognize the cancer cell receptor

• Sponsor of CAR-T Platform to enable the therapy process and enhance its customer experience when interacting with LS Company

CoE / Infusion Center

LS Company

Community Oncologist

Aphe. CenterPatient

Patient

LS Company

ApheCenter


• Conduct apheresis and cryopreservation• Ship harvested cells to LS Company • Coordinate with infusion labs and LS Company for

scheduling• Share patient data and associated analytics

• Conduct apheresis and cryopreservation• Ship harvested cells to LS Company • Infuse re-engineered T-Cells • Maintain and share patient treatment data with

ecosystem players

• Act as a referrer as well as long-term care giver• Coordinate with Aphe Center, infusion center and LS

Company for patient therapy – evaluation, scheduling, apheresis, infusion, post-careCommunity

Oncologist


And the role of healthcare providers and treatment center does not change when the therapy moves from the clinical development to commercial stage

Clinical trial

Commercial

Pharmacy

Autologous Therapy (e.g. CAR-T)Typical Pharma (e.g., pill/infusion)

Wholesaler/SP

LS Company

Hospital

LS Company

Hospital

CoE

Patient

Patient

Aphe./ Cell Lab



LS CompanyPatient

Aphe. / Cell Lab



Patient LS Company


Several Cell & Gene therapies have been approved for various indications and over 60,000 patients have been treated with these therapies

Nov

201

2

Glybera*

Provenge

Aug

201

7

Kymriah

Oct

201

7

Yescarta

Mar

201

8 Luxturna

Onpattro

May

201

6

Strimvelis*

Imlygic

Nov

201

5

Apr

201

0

Aug

201

6

Aug

201

8

Gendicine

2003 2012 2016 2018 2019+ 2010 2014

Zalmoxis*

Gintuit

Mar

201

0

Oct

200

3

Mar

201

8

Alofisel*

Gene therapies

Cell therapies (Autologous)

LEGEND

Cell therapies (Allogenic)

Oncology

Other TAs

* Products only have EU approvals


Pharma and biotech companies continue to invest heavily to build their NextGen product portfolio

Note: The percentages indicate the proportion of studies for a certain intervention/therapy and the number inside the doughnut hole indicates the grand total of on-going studies registered on Clinicaltrials.gov

LEGEND

Stem cells

CAR-T cells

TCR-T cells

NK T cells

TAA/TSA

TIL

Data as of December 10, 2018

USA and China are the leaders in the development of cell therapies

Stem cell & CAR-T therapies represent the largest share of the global cellular therapy market

USA has the largest number of Stem cell therapies under investigation (300+) followed by EU (80+)

With increase in activity, FDA expects 200 cell & gene therapy IND applications each year by 2020 and 15-20 approvals each year by 2025

Copyright © 2019 Deloitte Development LLC. All rights reserved. 11Patient Connect /Elevate the Patient ExperienceCopyright © 2019 Deloitte Development LLC. All rights reserved. 11

NextGenTreatment Journey

“ When something goes wrong, it’s a different conversation to have with the patient and the care team. We need to have enough information to tell them how we will move forward ”–Hematologist-oncologist / Transplanter


Commercial cell and gene therapy journey has several key Moments that Matter (MoM) that need to be managed

Patient JCAR017 Onboarding Apheresis & Cell Transport

Cell Mfg and Supportive

Care

Chemo and Infusion

Insurance coverage and eligibility

complete(stage gate)

Hem/Onc evaluates potential CAR-T therapy to use

Patient Aphe date confirmed with Aphe

Center and Mfg. manufacturing.

Pat

ien

tTr

eatm

ent

Team

at

Infu

sion

Sit

e (H

em/O

nc,

N

urs

e C

oord

inat

or)

Ap

her

esis

C

ente

r

Nurse coordinator

preps patient for apheresis

Patient receives education on Mfg. CAR-T therapy, and its benefits

and risks

Patient/family consent to the therapy and benefits verification

and enrolment in PSP

Patient undergoes Mononuclear cell

collection

Hem/Onc receives regular updates on cell status and final shipment date from

Mfg.

Hem/Onc receives Mfg. product from Pharmacy/Cell

Therapy Lab on day of infusion and educates patient

about infusion and monitoring

Post 30 days Patient follows

up regularly with Hem/Onc at out-

patient clinic

Aphe tech. conducts venous assessment

and shares the result with the infusion site

Coordinator submits Mfg. enrollment

(Rx, BV, PO consents, etc.)

Refractory/relapsed DLBCL patient consults with

Hem/Onc at CAR-T Certified site for further treatment

Long Term Monitoring

Patient is monitored for 30 days at infusion

site/hospital/out-patient

Hem/Onc assesses patient for on-going

bridging / supportive care

Nurse coordinator

preps patient for infusion

If confirmed, Hem/Onc & Nurse

coordinator educates patient and family, about the CAR-T

therapy

Cell Tracking and Monitoring

Post-Infusion Monitoring

Infusion Preparation

Site Certification

Therapy Order Placement

Site Onboarding

1st Line / 2nd

Line Treatment

Patient undergoes 1st

line treatment and it fails

Patient undergoes 2nd line treatment

2nd line treatment relapse

Site leadership and Mfg. confirms site Pre-boarded status

Assessment of CAR-T enabling capabilities and

site training

Definition of site processes and

testing

Site certified and ready to deliver CAR-T therapy

Patient/caregiver returns totreatmentcenter for follow-up visits as part of the registry requirements

Patient & Cell Management

Long-term Follow-up

Treatment Options Discussion

Patient Reimb. & Logistics Support

Patient undergoes lymphodepletion

Patient is infused with

CAR-T therapy

If necessary, IR appointment for

Catheter placementCoordinator determines Aph

date with Aph Center and Mfg.

Key moment that matters Patient Site Hem/Onc Nurse / Finance case ManagerAphe CenterKey Treatment Flow Treatment Flow Sub-

Process

T-cells collected and shipped from apheresis

center to Mfg.

If needed,Bridging Chemo

Site Pharmacy receives final product

Hem/Onc writes prescription for CAR-T

therapy

Site certified and ready to collect patient cells.

Aphe. Planning & Scheduling

Nurse coordinator requests CAR-T product and schedule internal resources/patient for

administration

1

3

4

56

7

8

9 102

Patient wait time between Aph and

Infusion

Post Infusion Monitoring


Management of the MoMs require treatment centers to wear multiple hats

Be a Supplier• Site can identify and

enroll the right patient• Site can produce a high

quality raw material

03

01Be a Custodian of Regulations• Site maintains compliance

with REMS program requirements

.04

02Be a Partner• Facilitate the

orchestration of patient and cell journey

Be a Customer• Site is prepared to

receive and administer the therapy appropriately (as specified in label)“…Collection,

processing, manufacturing, and infusion are always about coordinating and updating each

other…”– Hem-onc

Physician


Several treatment center’s stakeholder needs and pain points emerged from our global site visits

• Getting patients to certified CAR-T therapy delivery centers

• Require digital engagement portals for order placement at the sites

• Use a scheduling mechanism to lock key therapy dates and timelines

Therapy Access

• Chain of custody• Identification and traceability• Labeling and packaging control• Follow more complex receiving, on-site

storage and handling processes

Supply Chain & Manufacturing

• Acquire prior authorization with hubs to confirm treatment funding source

• Billing and reimbursement mechanisms are mostly unknown and are yet to be determined

• Business case/profitability for the treatment centers

Pricing & Reimbursement

• Method of reporting AEs• Compliance with outcomes reporting• Stringent REMS and 15 year follow up• Maintaining the chain of identify & chain

of custody throughout the patient journey

Regulatory

• Patient access to the treatment and logistics support

• Multi-modal customer support model• Training and on-going certification• Patients and their families need to feel

some sense of control during treatment

Patient & Staff Experience

• COEs report significant dissatisfaction with all elements of current CAR-T onboarding / site certification process:

• Contracting is considered one of the biggest pain points

• Need to manage multiple SOPs for the same process

Site Certification

For example, to deliver CAR-T therapy, treatment centers need to have > 150 distinct requirements in place


Treatment centers can take few steps to be prepared for delivering NextGentherapies for the patients

Define auditable and FACT compliant processes and capabilities with change control for the processes often mandated by the manufacturers

– Scheduling, inventory, and logistics– Apheresis– Cell management– Cryopreservation– Cell receipt and storage– Therapy infusion– REMS and post-approval registry

Implement standard processes that site needs in place, but are not dictated by manufacturers

– Clinical assessment and therapy selection– Financial, billing, and reimbursement– Patient support– Clinical monitoring and support therapy– Lymphodepletion– Management of adverse events

Maintain FACT accreditation to demonstrate robust chain of identity and custody, cell, and supply chain management capabilities


In addition, treatment center can develop processes that comply with FDA requirements and improve site certification experience

• Treatment center must assign an Authorized Representative (AR) to manage REMS at the treatment centers, and have a process for selecting and onboarding AR

• AR must ensure everyone on-site managing the CAR-T patient undergoes REMS training; centers must have REMS training and assessment processes and ensure that all required data is documented and ready for external audit

• Manufacturer must ensure treatment center documents treatment milestones, training, Toci, and CRS and Neurotoxcity data

Processes necessary to comply with FDA mandates


What are treatment center stakeholders saying – guiding principles for providers and manufacturers

B E I N N O V A T I V E

Engagement with patients will require creative solutions, including partnering with non-traditional players, existing registries and thinking beyond IT

1

K E E P I T S I M P L E

Allow flexibility in procedure where possible prevent workarounds and non-compliance; not all sites are the same

2

Staff and physicians will appreciate a final process that enables them to collect a better product, not one that tells or controls the apheresis itself. Leverage existing SOPs

D O N ’ T R E D E S I G N W H A T I S A L R E A D Y I N P L A C E4

3I M P R O V E T R A N S P A R E N C Y

Provide broad access to calendar availability, cell status, and alerts and notifications to improve scheduling and coordination across stakeholders

“It’s not just about the new technology. It’s about Execution.”

value chain insights for nextgen therapies · may 2016. imlygic. apr 2010. nov 2015. aug 2016. aug...

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