Cynthia A. Challener, Ph.D.
The first issue of Pharma’s Almanac in 2017 explores the current market landscape and the impact of Big Data and covers a whole host of other topics:
The 2017 Nice Insight Life Sciences Private Equity/
Venture Capital Investment Survey highlights some
interesting trends in the way investors of all kinds
currently look at opportunities in the life science and
healthcare sectors.
Don Barbieri
Patient noncompliance is extremely costly in terms of reduced patient health and lost revenue opportunity. SPI Pharma offers excipient technologies and drug development services supporting the formulation of patient-friendly oral dosage forms designed to address this crucial issue.
Andrew Warmington, Ph.D.
CONTINUOUS MANUFACTURING
OFFERS OBVIOUS ADVANTAGES TO
THE PHARMACEUTICAL SECTOR BUT
HAS NOT YET EVOLVED INTO A
MAJOR ALTERNATIVE TO BATCH.
ARE THINGS ABOUT TO CHANGE?
Arne Grumann
Targeted particle engineering for solid dosage drug formulations
has become an imperative given the challenging compounds in
the pharmaceutical pipeline, the growing interest in inhaled delivery,
and the move to continuous manufacturing. Contract development
and manufacturing organizations (CDMOs) with extensive knowledge
and experience with optimization of crystallization and particle
engineering processes provide customers with improved product
quality, lower costs, and accelerated development and
commercialization timelines.
Anil C. Nair, Ph.D, Kenneth F. Wertman, Ph.D., Marcel Patek, Ph.D., Paul R. August, Ph.D
The pharma landscape is constantly changing, especially as many
players in the industry move toward slimmed-down in-house R&D. This
includes embracing the “growth pharma” model in which drug discovery
programs are obtained via acquisitions. Many big pharma executives
have left their posts to spearhead lean, high-risk, high-reward biotech
firms, start-ups and virtual companies, which lack wet labs and other
means of obtaining experimental results.
Patrick Hatem, Kim Noll
Requirements for the manufacture of controlled
substances impact all aspects of drug development,
manufacturing and distribution. CDMOs that have
an in-depth understanding of the regulations,
long-standing positive relationships with regulators
and a track record of successfully working with
controlled substances can accelerate product
development and commercialization so that
treatments are made available more quickly.
Adam Covitt
Acquiring pharmaceutical
pro-cessing equipment and
successfully integrating it
into operations is not as
straightforward as it may
seem. Whether used or new,
everything is up for consideration
and evaluation:
vendor, type, specifications, performance,
reliability, capability, service life,
price and, importantly, availability. Oral
solid dose (OSD) manufacturing, especially
at commercial scale, is particularly dependent
on the operational efficiency of
a familiar but increasingly complex train
of processing equipment to mass produce
tablets and capsules. More than ever, the
success of a particular OSD product rests
on operational excellence and the ability
to deliver high-performing variation-free
process.
Nigel Walker, Catherine Hanley
WHY IS CULTURAL INTEGRATION
FOLLOWING A MERGER SO IMPORTANT
TO THE SUCCESS OF THE NEWLY
FORMED ENTITY?
Márcio Temtem, Ph.D., João Vicente, Ph.D.
Solubility in physiological fluids is a prerequisite for high bioavailability of drug substances. For many APIs that exhibit poor water solubility, bioavailability can be enhanced by preparing the product in an amorphous rather than a crystalline form. Amorphous compounds, because they lack long-range order, typically have higher apparent solubilities and faster dissolution rates than their crystalline counterparts.
Mark R. Bamforth, MBA, Richard O. Snyder, Ph.D.
Gene therapies are designed to treat diseases by modifying genetic information, including correcting genes that function improperly or adding normal copies of defective genes. They have the potential to address and potentially cure a wide range of ailments, including various inherited and acquired diseases such as cancers, neurological diseases, infections such as HIV, metabolic diseases, ocular diseases and cardiovascular diseases.
Gwenaël Servant, Ph.D.
Responsiveness has always been a key expectation for contract manufacturers providing services to the pharmaceutical industry. Today, however, with increased competition, the growing importance of evidence-based medicine, the expanding focus on orphan drugs and rare diseases and the rising use of accelerated approval pathways, responsiveness alone is insufficient for meeting the needs of sponsor companies. Flexibility combined with responsiveness has become essential
At the heart of That’s Nice is the Nice Insight division, which provides data and analysis from proprietary annual surveys as well as custom primary and secondary research of many kinds. Nice Insight is tasked every year with conducting a series of surveys, which offer unique insight into what companies in the pharmaceuticals and biotechnology sectors see as the key trends and drivers in their business.
The global pharmaceutical market continues to grow
at a healthy rate. According to research firm Evaluate
Pharma, the global drug market will expand at a compound
annual growth rate (CAGR) of 6.3% from 2016 to
2022, with biologics accounting for 50% of the top 100
products by the end of the study period.
The 2017 Nice Insight Clinical Research Organization
(CRO) Outsourcing Survey1 includes input from 608
outsourcing-facing pharmaceutical and biotechnology
executives. The majority of the respondents are key decision
makers; nearly one-third (31%) hold corporate or
management positions, with an additional 12% responsible
for clinical trials operations and management.
Pharmaceutical excipients are used in combination
with APIs in multiple medicinal products, including
tablets, capsules, oral liquids, transdermal patches,
implants and inhalers. Traditionally, these excipients
were used to add bulk to formulations. However, they
are increasingly used for functional reasons, such as
improving the wetting and organoleptic properties of
a drug or providing stability.
Pharmaceutical buyers have much lower budgets when
it comes to buying intermediates than excipients or
outsourced services, but their needs remain highly diverse
in terms of type of product and chemistry. However
basic this segment of the industry may seem at
first glance, buyers’ needs are driven first and last by
quality.
Modern pharmaceutical manufacturing equipment and
associated analytical instrumentation is the fundamental
‘hardware’ required to produce safe, effective, highquality
medicinal products. To meet the challenges from
increasingly downward price pressure, stringent regulation,
shifting markets and outcome-based insurance
reimbursement, modern manufacturing processes
rely heavily on innovative, integral, flexible equipment
to achieve quality, efficiency and cost-effectiveness.
Clinical supply chain logistics form a crucial component
of clinical trial infrastructure. The effectiveness
and efficiency of a supply chain is directly related to
the outcome of clinical trials.
Nigel Walker
It can help in disease pattern analysis by bringing information together in new ways; it facilitates drug discovery by enabling advanced search capabilities for analyzing millions of publications, patents and clinical trial documents; it can also help in clinical trials management by profiling patients, evaluating drug readiness or even identifying adverse effects before they are reported.
Nice Insight conducted
seven annual studies
to survey the pharma
landscape and
measure what buyers
in the market want. These surveys
were deployed to respondents in
the diverse and dynamic sectors
of CDMO, CRO, Equipment,
Intermediate, Excipient, Private Equity
Venture Capital and Supply Chain.
That being said, each space has
winners and losers. Respondents to
the study ranked companies on key
drivers, including Quality, Reliability,
Affordability, Productivity, Innovation
and Regulatory. The best of the best
in a wide range of categories are
honored here, with commentary from
key players on what it means to be in
the Top 5 for 2017.
Benchmarking with Nice Insight Annual Studies:
Company profiles with Deep Dive Customer
Awareness and Perception Ratings
Syed T. Husain, Catherine Hanley
The pharmaceutical industry looks very little as it
did even a decade ago. Not only has the focus shifted
from the development of blockbusters to drugs
aimed at indications for smaller patient populations,
the majority of new clinical candidates now come
from medium-sized and smaller companies, including
those that consist of two employees and a patent.
Ed Godek, Stephen Sirabian
Because oral solid dose (OSD)
pharmaceuticals will remain
the dominant dose form, the
pharmaceutical industry will
continue to be responsible for
the high-quality manufacture of
hundreds of millions of individual
doses every day, every year for the foreseeable
future. But as the world’s uptake of
solid-dosage pharmaceuticals continues to
grow, so does the perception that drugs are
too expensive and increasingly unaffordable,
and even though there is evidence to
the contrary, there has never been more
pressure on pharma to produce its products
at the lowest cost possible.
Kevin Haehl
Headlines on exploitive pricing
practices are just the most recent
examples of pharma’s corporate
social “irresponsibility”
presented for public vilification.
Right or wrong, pharma remains
a perennial target and a popular
bogeyman among politicians, the media
and a broad range of interest groups.
Marga Viñes
The goal of technology transfer according to ICH Q10 is to “transfer product and process knowledge between development and manufacturing, and within or between manufacturing sites to achieve product realization.” This knowledge “forms the basis for the manufacturing process, control strategy, process validation approach and ongoing continual improvement.”
Ariette van Strien, Daniel Bell
Marken is a leading patient-centric supply chain logistics organization with a complete focus on the pharmaceutical and life-sciences industries. Its state-ofthe- art GMP-compliant network includes 10 GMP-compliant depots and 45 worldwide logistic hubs for clinical trial material and investigational medicinal product storage and distribution, in addition to direct-to-patient (DTP) services and 50,000 monthly shipments of time- and temperature-sensitive drug and biological shipments at all temperature ranges in more than 150 countries, Marken also offers biological kit production; ancillary material sourcing, storage and distribution; shipment lane verification and qualification; and GDP, regulatory and compliance consultancy services.
Carl Carlson
Although single-use, disposable technologies (SUTs) have been around for decades, continued development and implementation of this innovative process technology is needed to help accelerate the advancement of biopharmaceutical drug development.
Tim Tyson
The pharmaceutical marketplace is more global
than ever and, as companies fight for market share
in this increasingly competitive landscape, contract
development and manufacturing organizations
(CDMOs) are becoming more important than ever.
Don Barbieri
Oral solid dose (OSD) form medications remain the
industry juggernaut. Though biopharmaceuticals
continue to grow and evolve, offering new and unique
treatment options, small-molecule drugs maintain their
dominance with OSD medications leading the charge.
Considering OSD drugs date back to 1,500 B.C., the
industry has had time to perfect the form factor, improve
manufacturability and enhance the efficacy of one of the
most portable and easily ‘packageable’ dosage forms.1
Christa Myers, Todd Vaughn
The Facility of the Future has to be more agile,
flexible and efficient than it is today. How to get
there? By innovating — introducing new processing
concepts, analytics and control — and pulling it
together with attention to flow and ergonomics.
Nick Bykerk, Val Dittrich
Driven by the demand for biologics, most of which still
require parenteral administration, the sterile injectable
market is projected to continue its 6% annual growth
through 2020.1 Though biologic injectables account for
52% of this market, which is projected to hit $363 billion
this year, they are not the only drugs filling this pipeline.2
Small-molecule injectable drugs, primarily those falling
under the categories of oncology and anti-infectives,
also command 38% of the overall market.2 The divide
between innovator drugs and generic, however, is perhaps
more indicative of how the market is responding to
the current competitive landscape.
Emilie Branch
The human body harbors up
to 100 trillion microbial organisms
throughout the gut,
skin, oral cavity, respiratory
system and urogenital tract,
among other organs and systems.
Collectively these microorganisms
are known as the microbiota, and the sum
of all of their genomes is known as the human
microbiome.1
These organisms play
an important role in human health, including
metabolism and immunity, and are
implemented in the gut-brain axis where
the central nervous system communicates
with the gastrointestinal tract.
Flexibility, responsiveness and operational excellence
are increasingly being demanded by drug developers.
The challenge faced by companies like Vetter Pharma
International is to deliver these to customers. Oskar
Gold, Senior Vice President Key Account Management
and Marketing/Corporate Communications, says that Vetter
believes that partnership is always the key to success, with operational
excellence being decisive in both the development and
commercial phases.
Steve Kuehn
If you were at the American Association
of Pharmaceutical Scientists
(AAPS) Annual Meeting in Denver
in November, you surely would not
have missed the That’s Nice booth.
There was a large company team, pop art
imagery, the opportunity to stand in a
miniature wind chamber picking up fake
money in order to win a cash prize or research
services, a white picket fence,
large paper flowers and, above all, that
yellow Lamborghini.
What do CROs/CDMOs need
to do to ensure that they
provide effective global
support but with locally oriented services?
What impact do you think the move to
cloud computing will have on the ability of
contract service organizations to better QQ meet the needs of their customers?
What will be the
impact of the
requirements
that drug
manufacturers
gain a similar level
of supply chain
understanding for
excipients as they
must have for APIs?