Pharmaceutical Competitive Intelligence for the Regulatory Affairs Professional

93 Pages·2012·0.79 MB·English

by  Raymond A. Huml (auth.)

SpringerBriefs in Pharmaceutical
Science & Drug Development
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Raymond A. Huml
Pharmaceutical Competitive
Intelligence for the Regulatory
Affairs Professional
Raymond A. Huml
Quintiles Transnational Corporation
Durham, NC, USA
ISSN 1864-8118 ISSN 1864-8126 (electronic)
ISBN 978-1-4614-3681-2 ISBN 978-1-4614-3682-9 (eBook)
DOI 10.1007/978-1-4614-3682-9
Springer New York Heidelberg Dordrecht London
Library of Congress Control Number: 2012937040
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Foreword
It is a great privilege and honor to have been asked by Dr. Huml to write the foreword
to this publication.
Ray Huml and I originally met 10 years ago, when we were both tapped to assist
PharmaBio Development, Quintiles’ new venture capital arm established in 2001,1 as
members of its due diligence teams evaluating potential partnership investments.
Ray, a well-respected North Carolina veterinarian and certi fi ed regulatory authority,
provided the due diligence teams with his regulatory and toxicologic expertise, while
I was charged with contributing medical and scienti fi c knowledge and experience.
Ray and I both discovered a shared love for the excitement and challenge of the
due diligence process, jointly participating in the placement of over half a billion
dollars in private equity capital over the course of my half decade with Quintiles.
Also, during the past 10 years we have collaborated on numerous articles and a
book detailing the processes we helped develop for PharmaBio-methods speci fi c to
the successful evaluation and funding of pharmaceutical and medical device invest-
ment opportunities.
There is a famous scene in the 1991 movie, “City Slickers,” wherein Jack
Palance, playing Curly Washburn, an experienced, wizened old cowhand, explains
to Mitch Robbins, the young city slicker, played by Billy Crystal, that the secret of
life is “just one thing.” When Mitch excitedly inquires, “What’s the one thing?”
Curly responds with a wry smile, “That’s what you’ve got to fi gure out.”
Having made the transition to full-time private equity funding work for Quintiles,
Ray has found his true calling—his “one thing.” The fi t is absolutely perfect, and
Ray has shone in this role, becoming one of the most accomplished venture capital
experts I have ever met. Now one of Quintiles’ fi nest due diligence team leaders, he
demonstrates uncanny scienti fi c, clinical, regulatory, fi nancial, commercial, and
1 PharmaBio was subsequently rebranded NovaQuest, but ultimately evolved into its current,
Quintiles Capital Solutions, entity, and separated from NovaQuest, which spun off as a separate
venture capital fund, NovaQuest Capital Management, LLC.
v
vi Foreword
legal judgment, combined with inspiring leadership skills, and impeccable, unwav-
ering ethical sensibilities—an extraordinarily rare combination in the private equity
funding arena.
Even more remarkable, functioning with high energy in an extremely competi-
tive domain where many sacri fi ce their personal lives in pursuit of career success,
Ray manages successfully to assign equal priority to his role as loving husband and
devoted father, participating fully in the lives of his two remarkably accomplished,
yet incredibly centered and well-balanced, children.
Yet, with all that, he still fi nds time to provide advice, encouragement, and moral
support to his friends. On this topic I speak from experience.
Finally, I would like to take this opportunity to acknowledge Dr. Dennis Gillings,
founder, Chairman of Quintiles, for his remarkable prescience in creating PharmaBio/
Quintiles Capital Solutions. Through its unique private equity partnership invest-
ment model, Quintiles alone amongst its peers offers many promising, innovative
companies in need of fi nancial support an alternative to traditional private equity
funding models, which force recipients to relinquish equity and control in return for
development capital. Each partnership deal focuses on collaboration rather than
control, melding Quintiles’ fi nancial resources and enormous pharmaceutical, bio-
logic, device development and commercialization experience with its unparalleled
operational expertise to custom craft a win–win solution wherein its partner achieves
maximum value from its intellectual property while Quintiles achieves consistent
long-term return on investment (ROI). Indeed, the values I acquired while at
Quintiles have informed my own subsequent career path choices.
With Dr. Gillings’ visionary long-term approach, Quintiles’ private equity divi-
sion has developed a unique playbook of pharma and device investment strategies.
This playbook combines innovative and thorough due diligence techniques (many
of which are detailed here in Dr. Huml’s treatise) crafted speci fi cally for the phar-
maceutical and medical device industries with term sheets, customized for each
partnership, which seamlessly blend royalties, licensing revenue, and milestone
payments, among other repayment options, with capital investment, loan guaran-
tees, CRO/CSO (contract sales force), and regulatory and management consulting
services. Their “special sauce,” though, is the importance placed on post-deal part-
nership alliance management, an extremely important, but oft neglected, compo-
nent of successful pharma/device deals. The end result is a remarkable and enviably
consistent “hit rate,” with impressive long-term ROI.
With thoughtful study of the principles enumerated here by Dr. Huml, anyone
involved in pharmaceutical, biologic, or medical device development funding, either
as provider of resources or as recipient, can markedly improve his success rate.
Morrisville, NC, USA Ross M. Tonkens
Acknowledgments
I would like to express my gratitude to Dr. Dennis Gillings, CBE, and Chairman of
Quintiles Transnational Corporation; Tom Perkins, Executive Vice President of
Quintiles Corporate Development; and Dr. Michael Arlotto, Senior Vice President
of Quintiles Corporate Development for the opportunity to serve as a due diligence
project leader and, in various roles and capacities, helping evaluate every major
executed transaction from PharmaBio’s inception through its evolution into
Quintiles Capital Solutions, a process spanning over 10 years.
This brief bene fi ts from an ever-evolving, output-driven due diligence process
that has at its core competitive intelligence that resulted in commitment of more
than $2.7 billion in capital to various large and small product partnering opportuni-
ties in the USA and Europe.
In addition, I would like to thank my friends Dr. Rick Turner, Senior Director of
Integrated and Translational Cardiovascular Safety at Quintiles, for his introduction
to Springer and coauthoring several papers cited in this brief; Allen Baum, patent
attorney at Brinks Hofer Gilson and Lione, who helped me understand some of the
challenges associated with patent expiration and regulatory exclusivity and also
coauthored several papers cited in this brief; Peter Kim, Senior Director of
Commercial Due Diligence at Quintiles and a master at forecasting product-based
sales; and Dr. Ross Tonkens, Director of the Science and Technology Acceleration
Division at the American Heart Association and former Global Head of the
Cardiovascular Therapeutics Division at Quintiles for encouraging me to publish
and for providing me with true mentorship over the last decade. I would also like to
thank the R egulatory Affairs Professionals Society who supported previous publica-
tions on topics related to the process of due diligence. Finally, I would like to thank
Dr. Jill Dawson, a consultant to Quintiles Corporate Communications, for her edito-
rial assistance with this brief.
It is hoped that this brief will provide the S pringer reader with a broad enough
overview of the due diligence processes to understand how to fi nd and interpret
competitive intelligence to offer input to upper management pharmaceutical strate-
gic and investment decisions.
vii

Contents
1 Introduction to Competitive Intelligence ............................................... 1
1 Definition ............................................................................................. 1
2 Introduction: Why Perform CI? ........................................................... 2
Reference ............................................................................................. 3
3 Regulatory Guidance, Advisory Committee Meetings,
and Case Examples .............................................................................. 3
3.1 Regulatory Guidance .................................................................. 3
3.2 Advisory Committee Meetings ................................................... 4
4 Case Examples ..................................................................................... 5
4.1 Case Example I: Cardiovascular Risk ......................................... 5
4.1.1 Introduction ................................................................... 5
4.1.2 QT Prolongation as a Cardiac Safety Biomarker .......... 7
4.1.3 Regulatory History of Formalized Cardiac
Safety Assessment ......................................................... 7
4.1.4 Regulatory History of Formalized Cardiovascular
Safety Assessment for Antidiabetic Drugs for T2DM... 10
4.1.5 FDA’s December 2008 Guidance for Industry .............. 12
4.1.6 Continued Discussion in the Literature of the
Cardiovascular Safety of Avandia and Actos ................ 13
4.1.7 July 2010 FDA Advisory Committees’ Meeting
on Avandia ..................................................................... 14
4.1.8 EMA’s January 2010 Draft Guidance............................ 14
4.1.9 Potential Ramifications for Future Global
Development of Antidiabetic Drugs for T2DM ............ 14
4.1.10 Summary ....................................................................... 15
References ............................................................................................ 15
4.2 Case Example II: Patient-Centric Services ................................. 16
4.2.1 Introduction ................................................................... 16
4.2.2 Privacy Challenges of Launching an Integrated
PCS Platform ................................................................. 19
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x Contents
4.2.3 Remote Patient Monitoring ........................................... 20
4.2.4 Regulatory Implications and Challenges ...................... 20
4.2.5 HIPAA ........................................................................... 21
4.2.6 HITECH ........................................................................ 21
4.2.7 Social Media and HIPAA .............................................. 22
4.2.8 510(k) Classification ..................................................... 22
4.2.9 Health and Wellness Programs ...................................... 23
4.2.10 REMS Programs ............................................................ 23
4.2.11 Latest FDA Regulations for Mobile
Medical Applications ..................................................... 24
4.2.12 Regulatory Overlap ....................................................... 25
4.2.13 Summary ....................................................................... 26
References ............................................................................................ 26
4.3 Case Example III: Biosimilars .................................................... 26
4.3.1 EU Biosimilar Status ..................................................... 27
4.3.2 Approved Biosimilars in the EU ................................... 28
4.3.3 Status of FOBs in America ............................................ 29
4.3.4 Interchangeability .......................................................... 30
4.3.5 Exclusivity ..................................................................... 30
4.3.6 Approved Biosimilars in the USA ................................. 31
4.3.7 Summary ....................................................................... 32
4.3.8 Chemistry, Manufacturing & Controls (CMC)
Concerns for Biosimilars ............................................... 32
4.3.9 Ef fi cacy Comparisons, Preclinical Safety,
Pharmacokinetics, and Clinical Studies
for Biosimilars ............................................................... 35
4.3.10 Summary ....................................................................... 37
References ............................................................................................ 37
2 Overall Perspective of Due Diligence
Investigations and Processes ................................................................... 39
1 Experience of Risk-Based Transactions ............................................... 39
2 The Process of Due Diligence .............................................................. 40
3 Outcomes of Due Diligence ................................................................. 41
Reference ............................................................................................. 41
3 The Regulatory Functional Review: Primary Roles ............................. 43
1 Introduction .......................................................................................... 43
2 Transaction Types ................................................................................ 44
3 Time Commitment ............................................................................... 44
Reference ............................................................................................. 44
4 Output and Expectations ...................................................................... 44