march 2016 sample pages pages | sample€¦ · this is kras, which was first approved in metastatic...

Trends and Innovations in Companion Diagnostics

March 2016

A FirstWord ExpertViews Report

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES

http://www.fwreports.com


Published March 2016© Copyright 2016 Doctor’s Guide Publishing Limited

All rights reserved. No part of this publication may be reproduced or used in any form or by any means graphic, electronic or mechanical, including photocopying, recording, taping or storage in information retrieval systems without the express permission of the publisher.

This report contains information from numerous sources that Doctor’s Guide Publishing Limited believes to be reliable but for which accuracy cannot be guaranteed. Doctor’s Guide Publishing Limited does not accept responsibility for any loss incurred by any person who acts or who fails to act as a result of information published in this document. Any views and opinions expressed by third parties and reproduced in this document are not necessarily the views and opinions of Doctor’s Guide Publishing Limited. Any views and opinions expressed by individuals and reproduced in this document are not necessarily the views and opinions of their employers.

Cover image: © Dan Race | AdobeStock

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES


All Contents Copyright © 2016 Doctor’s Guide Publishing Limited. All Rights Reserved

March 2016i


www.fwreports.com

ContentsExecutive Summary ................................................................................................................................. 1

Research Objectives ...............................................................................................................................3

Research Methodology ............................................................................................................................4

Definitions ....................................................................................................................................................5

Setting the scene .......................................................................................................................................8

Clinical and economic benefits of developing a CDx ......................................................... 21

Recent innovations in precision medicine and CDx ...........................................................29

Recent technological innovations in CDx ................................................................................. 37

Drivers and inhibitors for the uptake of CDx ..........................................................................45

Factors influencing successful/unsuccessful partnerships ..........................................50

Optimal and sub-optimal practice for developing CDx .....................................................58

Regulatory impact on the development and uptake of CDx ...........................................65

Managing stakeholder expectations on CDx programmes .............................................72

What needs to be done to encourage future innovation in CDx development? .78

Future opportunities in CDx and precision medicines ......................................................84

Appendix ......................................................................................................................................................92

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES



March 20163

www.fwreports.com


Research ObjectivesThis FirstWord Expert Dossier report provides a qualitative analysis of the key challenges and opportunities that pharmaceutical, biotech and diagnostic manufacturers face when developing a CDx to guide treatment decisions, improve patient outcomes, or obtain favourable reimbursement from payers for the use of precision medicines (PMs).

Key questions asked during the course of the research include:

1. What are the clinical and economic benefits of developing a CDx to support a PM?

2. Which fields have witnessed the greatest innovation in PM and CDx?

3. What needs to be done to encourage future innovation in CDx development?

4. What innovations have fuelled the development of more accurate and quicker CDx?

5. What are the main factors that drive or inhibit the uptake of a new technology that supports the prescription of PMs?

6. Which partnerships have proved successful/unsuccessful in the past, and why?

7. What are the optimal and suboptimal practices for CDx development?

8. How do you manage all stakeholder expectation during CDx development?

9. In what ways will the changes in regulation impact the future development of CDx?

10. Where do future opportunities lie for the industry to develop CDx to guide treatment with PM?

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES




March 20164

www.fwreports.com


Research MethodologyThe information for this report was gathered from multiple sources. In-depth research was conducted across multiple secondary resources, including in-house data, scientific journals, analyst reports, company presentations and conferences. Expert insight was obtained from telephone interviews (30-60 minutes) conducted during December 2015 – February 2016 with experts from pharma, biotech and diagnostic companies (n=9) in the US, Europe and Japan.

Experts interviewed:

nFrank Apostel, VP Companion Diagnostics, R-Biopharm AG, Germany

nJoachim Reischl, VP Head of Policy, Portfolio and Externalisation, Personalised Healthcare, AstraZeneca, EU

nRichard Watts, Vice President, Companion Diagnostic Partnerships, Global Business Development, QIAGEN, USA

nRob Seitz, Chief Technology Officer, Insight Genetics Inc., USA

nWilliam Pignato, Regulatory Affairs Consultant, Companion Diagnostic, USA

nDirector, CDx Development, Japanese Pharma Company

nDirector, Corporate Development, European Diagnostic Company

nCompanion Diagnostic Expert, Global Diagnostic Company

nCompanion Diagnostic Expert, Specialist Diagnostic Company

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES




March 201654

www.fwreports.com


Oncology has been the main therapy area to benefit and show success from the application of CDx. It has shown the highest number of regulatory approvals, however there are still failures based upon factors that cannot be controlled. According to Richard Watts of QIAGEN “in our partnerships we like to think of them all as successful in that we work well to meet the expectations of our partners. A success can be measured in many ways but ideally we will achieve an approval of an assay that then expands to other indications or can also be applied to other therapies to increase its clinical utility and expand its value and wider adoption. A good example of this is KRAS, which was first approved in metastatic Colorectal Cancer for Erbitux and then for Vectibix, it is now being explored in Non-Small Cell Lung Cancer as both a prognostic and predictive assay.”

Despite this considerable activity in CDx partnerships, not all will bear commercial fruit and partnerships can break down for a multitude of reasons. The Director of Biomarker Development at a Japanese pharma company highlights a number of reasons from personal experience, noting “you don’t get the A team. The partner is very bureaucratic, the research group is not flexible or does not have the ability to move quickly or you have a change in your clinical programme.” We asked our experts what factors are important to establish a successful partnership for efficient drug CDx development.

Start early, talk often and focus on the end game“When to start a partnership is a big area of controversy,” says the Director of CDx Development at a Japanese pharma company. Nearly all the experts we spoke with stressed the importance of aligning development between partnering products as early as possible. According to the Director, timing is key. “I think it’s a big mistake coming in late. There’s a misconception about cost, it’s not an up-front cost, you pay for work that’s done as milestone payments when you sign the contract.” He goes on to add “I think that spending half a million to a couple of million dollars early to save six months or maybe a year, getting a drug to patients is worth it – I always look at the endgame.”

Quoting a former senior FDA staffer, “Don’t let the diagnostic hold the drug hostage,” emphasises William Pignato. “I think the central issue that can’t be lost sometimes in good relationships with the partnership and difficult relationships; it’s all about the end game to get the drug approved in a timely fashion. It’s not about the diagnostic, although it is very important, it’s the drug’s approval that’s driving the relationship,” highlights William Pignato.

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES




March 201677

www.fwreports.com


The Regulators“Regulatory authorities are always a key stakeholder, then the pharma company who will take the lead on physician and patient education. They all need to be consulted in order to build a successful launch and commercialisation plan,” confirms the Companion Diagnostic Expert at a specialist diagnostic company.

It is also important to know the differences between approval pathways for a precision medicine and CDx and the region-specific requirements. It is also essential to know the type of regulatory approval required for the CDx, such as pre-market approval (PMA) or pre-market notification 510(k), given the significant differences in risk, costs, timelines, and level of evidence required to develop and commercialise as CDx.94 (See section on regulation, page 65 for more details).

On the whole, the experts believe the regulators are very supportive of CDx development and have put appropriate guidance in place to streamline the approval process. The Director of CDx Development at a Japanese pharma company notes that “from the FDA standpoint, they always say come early and come often. They honestly do mean that. The talks are very scientifically driven, non-confrontational and educational.” He also comments that “the worst thing instead of going early and often is going never because then you find out something late in the process that delays your project.”

According to the Corporate VP of a leading European diagnostic company, “in Europe, the whole process is very much country-dependent. The regulators encourage the development of drug diagnostics but too often this is disassociated from the validation of the drug itself.” The situation in Europe is currently more fluid that in the US and therefore developers need to understand that “in the EU there is really no governing body, it's all self-certification,” says the Director of Biomarker Development at a Japanese pharma company. Overall, it is best to assume a model of ‘early and often’ communication with the regulators, the experts conclude.

94 Quintiles (2014) Future best practices in oncology development. Published January 20, 2014 http://www.quintiles.com/library/white-papers/future-best-practices-in-oncology-development-critical-aspects-of-codeveloping-and-launching-an

SAMPLE P

AGES | SAMPLE

PAGES | S

AMPLE P

AGES



http://www.quintiles.com/library/white-papers/future-best-practices-in-oncology-development-critical-aspects-of-codeveloping-and-launching-an

Unique insight into current and future pharma market dynamics through quantitative surveys with physicians, providing essential data in major disease areas and on key industry issues .

Critical and unbiased intelligence derived from in-depth interviews with the world’s foremost thought leaders on the current and future treatment landscapes in major disease areas. Reports include three quarterly updates to ensure insights remain current.

Unbiased and concise analysis based on interviews with leading industry experts on important trends and challenging issues affecting the pharma industry today.

A personalised and comprehensive intelligence service delivering up-to-the-minute pharma news, insight, analysis, and expert views of importance to your company's success.

A personalised and comprehensive intelligence service reporting on the latest news and developments for the medical technology and diagnostic industries.

FirstWord delivers timely, need-to-know intelligence about your products, your competitors and your markets.

http://www.fwreports.com/firstview/

http://www.firstwordpharma.com/

http://www.fwreports.com/therapy-trends/

http://www.fwreports.com/firstword-dossier/

http://www.firstwordmedtech.com/

http://www.firstwordgroup.com/

march 2016 sample pages pages | sample€¦ · this is kras, which was first approved in metastatic...

Documents