PHTD 2012 Spring Meeting - units.sla.org

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16
Emerging
Medical
Ventures
Cumulative Percentage Change from 2011
“The Pink Sheet” DAILY
Daily, in-depth analysis of the
key developments shaping the
biopharma industry.
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Where Are They Now?
Checking In With Four Alzheimer’s
Disease Start-Ups
1 VALUATION WATCH
Biotech IPOs: Rewards Worth
The Risk
3 VENTURE ’ROUND
INSIDE:
The Next “Surge”:
BY MARC WORTMAN With its driving theory in question and a sharp
Quanticel/Celgene: The Importance Drug Shortages
aversion to risk infecting the space, Alzheimer’s disease drug developers
Of Linking A Financing And An Exit ■ page 30
face deep investor skepticism even as the disorder gains prominence
as one of the gravest public health threats to an aging population.
As VC Industry Contracts, Limited
Yet four previously profi led companies have found their way forward
Partners Look To Specialists
in challenging times.
Jumpstarting US Device Trials: FDA
Makes Effort In New Draft Guidelines
Mobile Health Promises
vol. 6, no. 11 december 2011 elsevier business intelligence www.ElsevierBI.com
24 To Uproot, Uplift Medical Devices 10 START-UP QUARTERLY
BY MARY STUART The health care industry is being swept up in a
STATISTICS, Q3 2011 C O L U M N S
technological twister, again. Ten years ago, Vytorin it was the Internet. Revived? Today,
it’s mobile technology. How are medical device Merck’s companies, cholesterol drug start-ups is poised to add a unique claim to improve outcomes Free Speech ............................. 2
and investors harnessing this power to create in as better many devices, as 19 million improved Americans, even 14 though CAPITAL there still MATTERS isn’t data to
clinical outcomes and richer fi nancial returns? answer critics about whether ezetimibe actually Morningside adds clinical Group: benefit Billionaire to Gerald FDA Beat ................................. 26
conventional statin therapy. How did Merck persuade Chan an Bets advisory Early committee On Chinese Biotech
to support the claim? The company didn’t; Oxford’s SHARP study team did.
CMS Beat ................................ 32
■ page 6
VC-Backed GI Start-Ups
61 SCIENCE MATTERS
Personalizing Standard Street Smarts .......................... 36
PROFILES See Opportunity The In Public IBS Policy Implications Chemotherapy And of A Potential New
38 The ghosts of failed drugs like Zelnorm and Lotronex still haunt the fi eld, but private
Combination Treatment Strategy Pointed Elsevier Business View Intelligence ........................... 43
the Abbott Split
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companies are confi dent they can satisfy safety-focused regulators – and ink lucrative
partnerships, too – by focusing on novel, more selective, Abbott’s targets. decision to spin-off proprietary pharmaceuticals as a stand-alone
company is all about Wall Street: investors cheered 62 ON the THE move MOVE for “unlocking Interventional Cardiology
41 Altheus expects chemical synergy to improve ulcerative value” colitis. by removing the giant shadow cast by Humira Who’s over Going the Where diversified In Biopharma REVA: In Bioresorbable Stents,
base business. But it is also an important touchstone for all the key public
43 AltheRx’s solabegron: a safe and highly selective therapy for IBS and OAB.
And Medtech Start-Ups “Patent As Things reform Change, is a huge Will The win.”
policy themes playing out in the biopharma space after health care reform.
45 Ardelyx modulates mineral absorption to treat IBS.
Value Remain John Lechleiter, The Same?
■ page 14
CEO, Eli Lilly & Co.
70 EXECUTIVE SUMMARIES By Stephen Levin
47 Second Genome mines the microbiome to discover GI targets.
Hospira and FDA
Start-Ups Across Health Care Hospira’s new CEO Mike Ball was charting a global expansion and touting
a move into biosimilars. Then the lingering 64 quality control discussions with
50 InfraScan’s portable head scanner detects intracranial FDA hematoma. caught up with the company and forced their way to the top of his d a s h b O arD
agenda. That may be an important step for the company
51 PlaqueTec pursues vulnerable plaque biomarkers and druggable targets. DEALS
in the long-term.
■ page 36
Recent Financings Of The Part D Problem
54 Prosonix aims for robust and accurate respiratory drug delivery.
Private Companies And Costs of the Medicare Rx program are projected to soar
57 Veracyte pioneers molecular cytology.
Tech Transfers Between over time.
A New Blueprint for Market Access
Academia And Industry
60 EMERGINGS IN BRIEF: 480 Biomedical, Alafair Biosciences, The access Blaze and Bioscience reimbursement environment is becoming more
Cumulative Growth of Net Medicare Parts A, B, & D (2011-21)
and Oncobiologics
challenging as public and private payers scrutinize the value and cost
80%
of pharmaceuticals more than ever. Increasingly, robust proof of clinical
and/or economic value is being required as a condition of coverage.
60%
Pharmaceutical companies must make organizational changes to adjust.
Part D
Part B
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Biopharmaceuticals
Endovascular Devices
J&J’s Zytiga –
Lombard Medical:
The Anatomy Of
At Long Last, Turning
A Cancer Launch
The Corner In EVAR
By MichaeL GoodMan
By david caSSak
BIOPHARMACEUTICALS
Biosimilars:
A New Kind Of
Innovation
For several recent entrants, the biosimilars
game isn’t just about creating copies; it’s about
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promoting and branding a new kind of hybrid
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M E D I C A L D E V I C E Q U A L I T Y C O N T R O L
Founded 1996 Vol. 15, No. 11
November 2011
news this month Three Barriers To Device Quality And
A new FDA report finds three
major barriers to medical device
quality: a misalignment between
FDA investigator priorities and the factors
that truly drive product quality; a lack of
information on how products compare
with one another in terms of quality; and
manufacturers’ failure to use appropriate
risk management tools at a time of
increasing device complexity. The report,
“Understanding Barriers to Medical Device
Elsevier Business Intelligence
What FDA, Industry Might $1,015 A Year Do About Them
Shawn M. SchMitt sm.schmitt@elsevier.com
DA is reassessing its facility inspection strategy after hearing that industry
believes the agency places manufacturer adherence to the Quality System
F Regulation ahead of product quality. ‘‘The Gold Sheet’’
Manufacturers’ perceptions that they must throw an exorbitant amount of resources
at low-risk problems found during agency inspections is just one of three
PHARMACEUTICAL & BIOTECHNOLOGY QUALITY CONTROL
roadblocks to quality outlined in “Understanding Barriers to Medical Device Quality,”
an FDA report released Oct. 31.
November 2011
“What FDA inspects for becomes important, and what’s important gets done [by
PUbLISHeD
moNTHLY
Vol. 45, No. 11
Quality,” lists possible solutions for FDA and manufacturers], so it’s critical that we are inspecting for the right things,” CDRH
industry. “Identifying and addressing bar- Director Jeffrey Shuren said.
news this issue Opportunities to Statistically Mine
riers to medical device quality can improve
“We want to make sure ... we’re not focusing on the minor stuff so we don’t divert
public health outcomes while also improv- companies’ attention to those things that matter What less,” large he said. samples “Instead, could [we tell need FDA PAT Data for Release Testing Explored
ing a company’s bottom line and allowing
Elsevier Business Intelligence
to] get them focused – and ourselves focused – on what matters most.”
CDRH to better utilize its resources,” CDRH
The streams PUbLISHeD
of process analytical data that
$1,935 A Year
Joanne S. eglovitch J.eglovitch@elSevier.com
Director Jeffrey Shuren said. A goal for FDA One way FDA might modify its inspectional approach drug makers is to instruct increasingly WeeKLY
its investiga- produce could
tors to place more emphasis on QSR requirements support that regulatory greatly affect compliance the quality by showing
anufacturers, compendial authorities and FDA officials recently explored
is to collaborate with industry to create a
set of robust, commonly understood quality of devices – such as design controls and supplier that processes controls – are and validated less weight and batches on
ways the pharmaceutical industry could use sophisticated statistical meth-
practices ...........................Cover
areas of the regulation that are not as quality-centered. releasable. A recent PQRI meeting explored M ods to unlock the regulatory compliance potential of vast amounts of pro-
how to make it happen. ............................Cover cess analytical data it is generating.
‘‘The Further, inspections could be structured to concentrate more on products with a
FDA quality report Gray singles out Sheet’’ ®
history of quality failures, such as infusion pumps. Inspections also should target Discussions at a recent Product Quality Research Institute workshop focused
seven areas where manufacturers can
known company-specific quality weaknesses, the A coming report out notes. party for China’s GMPs mainly on batch release testing for content uniformity. Manufacturers typically
boost quality ...........................5
m e d c a l d e v c e s a n d d a g n o s t c s China is sending inspectors abroad to see
rely on a compendial end-product testing method that can demonstrate compli-
The report is an attempt by CDRH to understand why device quality problems
Warning Letters
how the rest
ance of a 10- to 30-tablet sample, but that does not address the likelihood that
continue to occur. Offering a menu of action items, it is Vol. of
intended 37, the No. world 50 complies with
as a starting the sample actually represents the batch.
December Respironics cited 12, for 2011
its new GMPs so they can learn how to in-
MDR violations in point for the agency and industry as they work to remove quality hurdles.
spect that kind of quality into China’s do- They talked about alternatives for statistically analyzing these small-sample re-
relation to its Trilogy Continuous Venti-
Manufacturer use of any recommendations in mestic the quality industry. report What is will completely this mean for sults to gain greater batch-wide insights.
lators; SterilMed was selling its Femo- voluntary; FDA has no authority to prescribe to multinational firms how to corporations? make their products. ...................... 8
Stop Gold compression device without
But the main focus of the Sept. 12-13 sample size workshop in Bethesda, Md., was
top news CardioMEMS’ Heart Failure Monitor
Continued > Page 3
FDA approval ......................10
on tapping the treasure trove of process analytical data that constitutes much
Foiled At panel By FDA Inspection FDA deferring CMC changes ‘wish list’ larger and therefore more representative data sets.
CMS pre-payment Close-Out reviews Letters ..................12
In Their Words FDA explains why it’s setting aside industry’s
The existing compendial approach under USP General Chapter 905, Uniformity
Anxiety may be unfounded Findings Of Study Bias
‘wish list’ as it focuses on clarifying draft
News In Brief
of Dosage Units (UDU), for tablets and capsules is also not relevant in the new
Zach Miners guidance on downgrading CMC changes to
The jury is still out on whether Medicare
z.miners@elsevier.com
This quality initiative will allow small firms to dedicate their
quality-by-design manufacturing paradigm where in-process testing and control
Former FDA Office of Compliance Direc- “
annually reportable. PhRMA complains the
pre-payment reviews will significantly
tor Tim Ulatowski joins Becker Consult- n FDA panel limited meeting resources last week on to CardioMEMS areas that Inc they .’s draft PMA know would for its are actually Champion priorities increase supplement to fil- is emphasized, they say.
reduce procedure volumes and device
ing, while former compliance office implantable FDA heart – and failure that monitor is as was beneficial dominated to not them ing by requirements..................................................12
the as firm’s it is to clinical
consumption, but hospital execs say
firms like
USP officials say that they are mulling over the possibility of creating a new chap-
official Larry Spears jumps from Becker A data but by findings from FDA’s pre-market clinical-site inspections pointing
that outcome is unlikely .10
medtronic and Johnson & Johnson.
ter addressing content uniformity of large samples. Meanwhile the European
to Deloitte; AAMI continues to address to bias in that data FDA wants generics more equivalent Pharmacopeia issued a proposal this year outlining new requirements for large
”
“alarm fatigue” .....................14
The result: CardioMEMS appears to be facing an uphill battle at best for ap-
– Steven Silverman, director, CDRH Office of Compliance
samples.
Diagnostic patent or law of nature?
proval That is a disappointing surprise not only for Atlanta-based
No more holding
CardioMEMS,
your nose and trying to
but also for St Jude Medical, which holds a 19% ownership
choke
stake
down
in the
smelly,
firm and
bulky generics. FDA One approach pharmaceutical manufacturers are talking about is adopting the
Mayo v. Prometheus case argued
has touted the technology as a major opportunity for its business
says it wants
(See
them
“St Jude:
the same size, taste and American National Standards Institute E2709 standard, otherwise known as the
before Supreme Court
CardioMEMS’ Heart Failure Data Reflects Major Opportunity”
smell
— “The
as the
Gray
reference
Sheet,”
listed drugs. ............14 CUDAL method, which ASTM approved in 2009.
The court To struggled sign up last for week FREE to map ONLINE Feb ACCESS, 14, 2011 .) go to: www.ElsevierBI.com/publications/The-Silver-Sheet Officials say that CUDAL is a highly effective method in identifying nonconform-
out rules for the types of method
ANDAs will need more stability data ing material for both large and small sample sizes. This method can be used to
claims, particularly for diagnostic
FDA is drafting guidance that will call for ge- assess the likelihood that particular sample results are representative of entire
methods, that should be eligible for
nerics firms to submit up to 12 months of sta- batches, and could play a role in process validation studies and in lot release.
patent protection 12
bility data with their ANDAs, instead of just the
FDA officials also pointed to published simulation studies showing that the ASTM
three months of data currently required. And
E2709 method is superior to the USP 905 chapter for detecting batch defects.
New on the mHealth scene
industry has a lot of questions about it. ...........15
Qualcomm launches wireless
Other statistical approaches for handling large sample sizes discussed at the
health business
Telecommunications firm unveils Qualcomm
Life, a new health care sub-
What ICH Q1A means for generics
conference included parametric tolerance interval test (PTIT) and nonparametric
counting test methods. Both approaches can be used to ensure dose uniformity
Current stability testing requirements vs.
at lot release.
what ICH Q1A requires. ......................................18
sidiary The company’s first product,
pictured below, is a platform that will
CardioMEMS’ Champion heart failure sensor is implanted via catheter to continuously
Continued > Page 3
allow wireless medical devices to send
take pulmonary artery pressure measurements.
patient data from the home to a cloud-
To sign up for FREE ONLINE ACCESS, go to: www.ElsevierBI.com/publications/The-Gold-Sheet
based storage site .20
FDA’s Circulatory System Devices panel voted 6-4 that the benefits of Champion
do not outweigh its risk at the Dec 8 meeting in Gaithersburg, Md
And in spite of the fact that the trial met its primary endpoint of reducing
heart-failure hospitalizations, panel members voted 3-7 against device effectiveness
due largely to a level of interaction between the sponsor and clinical site
investigators that FDA said went beyond the protocol outlined in the firm’s PMA
submission
This “auxiliary management,” as panelists came to call it, included telephone
calls and certain e-mails offering medical therapy advice from CardioMEMS nurses
to study investigators Most panel members agreed they could not tease out the
clinical benefits of the device from the benefits of the sponsor-generated therapy
recommendations
“There are confounding factors that make it impossible to know if the device alone
can explain the benefit,” concluded David J Slotwiner, of Long Island Jewish Medical
Table of Contents > page 3
Story continues > Page 5
To sign up for FREE ONLINE ACCESS, go to: www.ElsevierBI.com/publications/The-Gray-Sheet
Elsevier Business Intelligence
Photo: CardioMEMS top stories
Table of Contents > 3
P R E S C R I P T I O N P H A R M A C E U T I C A L S A N D B I O T E C H N O L O G Y
January 2, 2012
Research Policy
NIH Repurposing Of Failed
Compounds Could Begin Soon
NIH’s National Center for Advancing
Translational Science could launch its
first project in early 2012 with an agreement
with one or more drug companies
to repurpose compounds that the firms
have abandoned. Negotiations with industry
are very close to bearing fruit,
says Kathy Hudson, an NIH deputy director
and NCATS’ acting deputy director.
The repurposing project would the first
uniquely NCATS program..........................11
Manufacturing
Ranbaxy’s $500 Million
GMP Settlement Would Be
Second Most Expensive For
Rx Manufacturing Violations
Ranbaxy Laboratories’ consent decree
with FDA and agreement to set aside
$500 million to settle a related Department
of Justice investigation appears
to put the Daiichi Sankyo subsidiary on
pace to pay the second highest settlement
for pharmaceutical good manufacturing
practice violations, according to
data compiled by “The Pink Sheet”.......25
European Health
Technology Assessment
Grunenthal CEO Views German
Reimbursement Hurdle As Fair, As Pain
Drug Palexia Rolls Out Across Europe
German reimbursement authority’s strong
emphasis on drugs’ added benefit is logical,
Grunenthal’s Harald Stock says, and all
health care systems will eventually move
the same way.....................................................8
Looking AheAd To 2012:
To sign up for FREE ONLINE ACCESS, go to: http://ThePinkSheet.ElsevierBI.com
$2,450 A YEAR (print and online)
Founded 1939 | Vol. 74, No. 1
ACOs Get In Gear, Accelerating The Need to Prove
Biopharma Products’ Overall Value In Health Care
F
Scott Steinke s.steinke@elsevier.com
or any biopharma manufacturers standing on the sidelines as Medicare
accountable care organizations take shape, the time has arrived to
get involved.
The first group of ACOs began operations Jan. 1, and from day one, their
focus is on improving patient outcomes while also reducing the growth of
health care costs. Manufacturers that connect with ACOs and convince them
that their products contribute to both of those goals stand to gain share in
what could be a very influential program.
Improving outcomes while reducing costs is a familiar phrase in the health care
world. But with the creation of ACOs under the Affordable Care Act, Medicare is aiming
to put that phrase into practice by allowing all of the providers who interact with
Medicare beneficiaries – from primary care and specialty physicians to hospitals and
long-term care facilities – to organize into groups that will coordinate care. Through
the associated Medicare Shared Savings Program, ACOs will share in the cost savings
achieved or be penalized for cost over-runs based on a spending benchmark, all
while having patient outcomes measured to ensure care is improving.
Despite early reservations that the accountable care program would have few
takers, 32 ACOs, which CMS is calling “Pioneer ACOs,” started up Jan. 1. Other
Key Points
Story continues > Page 4
• ACO providers are responsible for overall patient outcomes and the related
costs, so physicians must consider the overall value to patient care of the
drugs they prescribe.
• ACO P&T committees are likely to develop formularies and treatment guidelines
for their physicians, so it will be essential to present the committees with good
data on a product’s value to overall patient care.
• Manufacturers should look for opportunities to partner with ACOs, both
to ensure the overall success of the ACO model and to ensure expected
outcomes for particular products are achieved, such as through working
on patient adherence.
• High-priced oncologics could be particularly vulnerable in ACOs to the need
for high-quality data on value, since cost benchmarks cannot be adjusted to
account for new therapies and cancer care is not a quality measure.
• The Medicare ACO model could spill over into other health care settings, with
successful ACOs potentially contracting to provide services to managed care
organizations serving commercial or Medicaid plans.