A Decade of Change: Innovation R&D Innovation: The Key to ... · The biggest paradigm shift over...

38 March 2011 � PharmaVOICE

help enable the development of targeted ther-apies for additional applications,” says BradThompson, CEO of Oncolytics Biotech, abiotechnology company. “There’s no questionthat personalized medicine will be the biggestmarket shaper in the next 10 years. The in-dustry continues to advance its understandingof the genetic basis of many diseases, with theultimate goal of developing treatments thatcan treat individual patients more effectively.Oncolytics has several trials that are lookingspecifically at patients pre-screened forKRAS- and EGFR-activated tumors and areworking hard to realize the benefits personal-ized medicine may offer in the future.”

A Decade of Change: Innovation

hen the scientific discover-ies and pipeline-buildingprocess of many successfulbiotechs are layered to-gether, the results are

likely to be dramatic. Experts predict there willbe a number of game-changing product candi-dates in development in the next decade.

“We view ourselves as part of the ‘multipliereffect’ of innovation emerging from biotechcompanies,” says Doug Ringler, V.MD., CEOof Tolerx, a biopharmaceutical company devel-oping novel therapies to treat autoimmune dis-eases and cancer by normalizing the immuneresponse. “Within the short-time span of adecade, a biotech company, such as Tolerx, canbring its first drug to near-commercial realityas a potential breakthrough for patients, as wellas develop a pipeline of additional drug candi-dates based on a novel approach.”

The biggest paradigm shift over the lastdecade has been the move of biologics, particu-larly monoclonal antibodies, to the center ofdrug discovery and development at almostevery major pharmaceutical company and fornearly all indications, says Bassil Dahiyat, CEOof Xencor, which engineers biotherapeutics.

“Biologics create a totally new set of oppor-tunities to treat diseases that were not availablein the small-molecule-only mindset of the past,”he says.

Within the last 10 years, there has been in-credible growth in the development and ap-proval of biologics, particularly monoclonalantibodies (mAbs), for the treatment of cancerand autoimmune disease.

“The progress that has been made to effi-ciently engineer and manufacture mAbs will

W

Pharmaceutical companies arguably put innovation on the back burner over the past decade as they looked to trim costs and

meet shareholder expectations. Going forward, experts predict that by shifting the focus to biologics, genomic advances,

personalized medicine, and specialty markets, innovation will have a multiplier effect on the industry.

R&D Innovation: The Key toLong-Term SUCCESS

“ The future is going to bebased on personalized medicines based on each person’s unique DNA and disease. ”

JOHN CLINE / unithink

“ In the next 10 years, the industry will continue totransform through theidentification and implementation of new development models. ”

JOSEF VON RICKENBACH

Parexel International

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39PharmaVOICE � March 2011


Simon Moroney, Ph.D., CEO of Mor-phoSys, a biotechnology company devel-oping therapeutic antibodies, predictsthat monoclonal antibodies will remainone of the leading drug categories withinthe biotech and pharmaceutical industry.

“Monoclonal antibodies can be used astools for diagnosis and treatment of a vari-ety of different diseases,” he says. “Sophis-ticated technologies that enable a newgeneration of antibodies with very preciseproperties, allied with a deeper under-standing of target biology, will producedrugs that will proceed through develop-ment more quickly and with higher suc-cess probability. Patients will benefit fromtherapies that are tailored to their needs.”

Innovation at the Molecular Level

Dr. Ringler has no doubt that the biggestchange in the next 10 years will be how muchcloser companies will get to creating “cures”

Biotech Predictions for 2011

» Biotech and the Capital Markets: Thebiotech industry did benefit from the return

of investor confidence in the second half of

2010, with the Burrill Biotech Select Index

outperforming the Dow Jones Industrial

Average on an annual basis. Expect to see the

biotech industry continue to outperform the

general markets as the financing

environment continues to improve in 2011.

» Biotech IPOs: The biotech IPO window willremain open despite the fact that the 17 new

biotech issues that debuted on the U.S.

market in 2010 were plagued by lackluster

receptions (selling fewer shares below the

pricing range) and their average annual

market performance was down by 13%. By

the end of 2011, at least 25 biotech IPOs,

possibly more, in the U.S. will be completed.

» Capital: The industry has achieved a steadystate in terms of financing, raising about

$15 billion annually. This situation will continue

in 2011. The industry’s collective market cap

will also remain at its present $360 billion level

as market value growth will be offset by

acquisitions.

» Biosimilars: Healthcare reform carries provisions instructing the FDA to create a

pathway for biosimilars. Expect to see both

biotech and pharma companies take an

interest in the drafting of new regulations

governing the development of biosimilars.

» Converging technologies impact healthcare: Expect to see a greater emphasis

on prevention and wellness, as well as a

greater understanding of human genomics

and the advent of molecular diagnostics, and

the convergence of information, wireless, and

medical technology promise to make

personalized medicine an ever-present reality

in the way doctors and patients approach

healthcare.

Source: Burrill & Co. For more information, visit burrillandco.com.

“While the days of mass-market blockbusters may beover, this term will be redefined, and the newblockbusters will be specialized therapies. ”KEN RIBOTSKY / The Core Nation

“ RNA as a novel and widelyapplicable drug target hastransformative medical potential. ”CHRIS GARABEDIAN / AVI BioPharma

for many diseases, as opposed to treating theconsequences of the diseases.

“With the innovations coming frombiotech and therapies that identify, target,and repair the root cause of the disorders, thedrugs emerging in the next decade should bemuch closer to cures because they will lead todisease remission, relapse prevention, and cor-recting or halting disease processes,” he says.

There is little dispute that the public re-lease of a rough draft of the human genome in2000 was the single biggest game changerwithin the first decade of the 21st century forthe life-sciences industry, says Michael Parks,executive VP, public relations and marketingcommunications, at Vox Medica, a healthcarecommunications agency.

“This achievement spearheaded by TheHuman Genome Project and Celera Ge-nomics gave new meaning to the possibilitiesof preventive medicine, where initiating trueprevention could begin before a child is evenborn through genetic testing.”

One genomic area that holds promise, ac-cording to several thought leaders, is RNA.

“The rapid progress in understandingRNA as a therapeutic target and innovativechemistries with the potential to deliverhigh-potency, low-toxicity, and easily adapt-able drugs, point to RNA as a novel andwidely applicable drug target with transfor-mative medical potential,” says Chris Garabe-dian, president and CEO of AVI BioPharma,a biopharmaceutical company discoveringand developing RNA-based therapeutics forrare and infectious diseases. “Directly attack-ing RNA, the carrier of genetic information,rather than enzymes or receptors like manytraditional therapeutics, makes heretofore un-druggable targets accessible.”

By a process of alternative RNA splicing,close to 90% of genes produce several differentproteins from the same gene, he explains.

“Manipulation of alternative RNA splic-ing can not only reduce or eliminate expres-sion of undesirable proteins, like other RNA-based technologies, such as siRNA andantisense, but also restore production of de-sired proteins,” Mr. Garabedian says. “This(c

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“In addition, there have been other majordevelopments over the course of the past 10years,” she says. “Carving out ZFN and iPStechnology has been exciting. Both are juststarting to have an impact. ZFN- or gene-edit-ing as well as iPS technology are the game-changers. These, plus a highly sensitive andmultiplex protein detection system will re-shape the research market and allow us to seethings that haven’t been detectable before. Inaddition, I believe that companion diagnosticsand the personalized medicine sector will even-tually find a prominent place in the market.”

Zinc finger nucleases (ZFNs) are a class ofengineered DNA-binding proteins that facil-itate targeted editing of the genome by creat-ing double-strand breaks in DNA at user-specified locations. Double-strand breaks areimportant for site-specific mutagenesis inthat they stimulate the cell’s natural DNA-re-pair processes.

David Smoller, president of researchbiotech at Sigma Life Science, says while inthe last 10 years the sequencing of the humangenome made a big impact on the industrywith RNAi driving the understanding of thisgenome, adult stem cells with the power ofZFNs will change the future of the industryand the understanding of biology.

“At the same time I would not underesti-mate the power of the genetically modified ratusing ZFNs,” he adds. “The rat could help rev-olutionize how we evaluate drugs for safety anddrug-drug interactions and open doors to un-derstanding the brain and the diseases that ef-fect the brain, such as Alzheimer’s, Parkinsons,

etc. Finally, nextgen sequencing will have aprofound effect on personalizing medicine.”

Experts agree that biopharmaceuticals willbe the dominant force in terms of marketgrowth in healthcare.

“Over the next 10 years, the creation andadvancement of new technology platformswill allow for the rapid, methodical, and sys-tematic study of new biologic drug candi-dates,” says Bill Newell, CEO of Sutro Bio-pharma, a biopharmaceutical company.“These novel technologies will circumventmany of the current limitations of biologic


technology is particularly attractive for thetreatment of rare diseases, such as Duchennemuscular dystrophy where repair of defectiveRNA and restoration of missing dystrophin(protein) are essential for clinical benefit.”

The discovery of RNAi has had a huge im-pact, agrees Helge Bastian, VP, researchbiotech/global marketing, business develop-ment and strategy for Sigma Life Science, andhas provided a better understanding of biol-ogy and shaping the landscape of the industry.Sigma Life Science offers an array of biologi-cally rich products and reagents that re-searchers use in scientific investigation.

“ Biologics create a totally new set of opportunities to treatdisease that were not availablein the small-molecule-onlymindset of the past. ”

BASSIL DAHIYAT / Xencor

BACK TO THE FUTURE

THE FUTURE OF BIOTECH

TERRENCE TORMEY

CEO and President, Prevention

Pharmaceuticals

» THEN (2001): [Biotech]companies looking to do their own mar-

keting and sales recognize they don’t

have the internal resources necessary, so

they’re going to outsource those func-

tions — everything from the marketing,

to the selling, to the ancillary services.

» NOW: With few exceptions, biotech companies have not lived up to their

potential in product commercialization.

Biotech CEOs are more interested in

raising monies and quickly exiting. As a

result, the very day a major pharma

company expresses an interest in their

NDE, they say, “sold.” I had once believed

that these CEOs and their investors would

come to understand that by outsourcing

sales, marketing, manufacturing, etc., they

could hold onto the NDE and enjoy

perhaps hundreds of millions of dollars of

revenue for their company. Instead, these

otherwise extraordinarily bright

M.D.s/Ph.D.s. are foregoing their

advantage. Major pharmaceutical

companies, on the other hand, faced with

product pipelines that are as dry as a west

Texas oil well, are quickly buying up

biotech companies and/or their

products.

“ Today, patients are treatedmore and more with targetedtherapies and the one-size-fits-allapproach to drug developmenthas become a thing of the past. ”

DR. SIMON MORONEY / MorphoSys

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drug discovery and development and will en-able the realization of novel therapeutics pro-viding important new treatment modalitiesthat are not realizable today.”

The continued adoption of molecular med-icine — facilitated by informatics systems thatenable collection, aggregation, analysis, anddissemination of data — is poised to com-pletely reshape every part of the healthcare sys-tem, from research and clinical development,to how products are reimbursed, to the practiceof medicine itself, says Marcia Kean, CEO ofFeinstein Kean Healthcare, a communicationsand business consulting firm.

“For biomedical innovators, the challengewill be to use data/knowledge more efficiently— and pre-competitively — to develop newproducts that prevent and change the courseof disease, leverage companion diagnostics toidentify the appropriate patient populationfor their drugs, and engage with an increas-ingly data-empowered group of consumers toaccelerate and improve clinical development.”

One of the big game changers in the last 10years has been the discovery and developmentof Novartis’ breakthrough cancer therapyGleevec. The drug was discovered using an un-derstanding of molecular pathways, and itworks by specifically targeting proteins in can-cer cells, but leaves healthy cells unharmed.

“This has been a huge breakthrough forcancer therapy as Gleevec has an 86% overallsurvival rate at seven years, the longest ob-served overall survival in blood cancer,” saysJoe Jimenez, CEO of Novartis, a globalhealthcare company. “Gleevec’s discovery hasalso led us to further innovations; for examplewe developed Tasigna, which studies show

performs better than Gleevec in slowingdisease progression for newly diagnosedchronic myeloid leukemia patients.

“I see this as a real turning point forthe industry because it literally transformedchronic myeloid leukemia; patients had a lifeexpectancy of three to five years once diag-nosed in the chronic phase, before Gleevec,”Mr. Jimenez continues. “Now it may be pos-sible for CML patients to expect to have a nor-mal life expectancy.”

While not a biologic, the introduction ofthalidomide in the last decade for the treatmentof multiple myeloma was another gamechanger, according to Brian G.M. Durie, M.D.,co-founder and chairman of the InternationalMyeloma Foundation and co-chair MyelomaCommittee, SWOG. He is also the medical di-rector of Aptium Oncology Myeloma Consor-tium (AMyC) Aptium Oncology, which pro-vides solutions for oncology care.

Thalidomide was introduced as a sedativedrug in the late 1950s. In 1961, it was with-drawn because of teratogenicity and neuropa-thy. Today, there is a growing clinical interestin thalidomide, and it has been re-introducedas an immunomodulatory agent used, in com-bination with dexamethasone, primarily totreat multiple myeloma.

“Thalidomide opened the doors to a dra-matic new paradigm of treatment, demonstrat-ing that non-cytotoxic approaches could workin the treatment of multiple myeloma and re-lated blood cancers,” Dr. Durie says. “Thispaved the way for other novel therapies, thethalidomide-relatives Revlimid and pomalido-mide, and the proteasome inhibitors Velcadeand more recently carfilzomib. This sea-changein treatment provides extended remissions withimproved quality of life for patients. Ten yearsfrom now we expect to see pharmaceuticaltreatments replacing stem cell transplants, and


“ Almost all new agents under development in cancer have a specifictarget identified on a molecular basisand therefore these agents, even in theearliest trials, are much more likely toclinically benefit the patient. ”

DR. STEPHEN JONES / US Oncology

“ Science has moved rapidly,and R&D scientists, regulators, and providers arenow chasing treatments andcures for a variety of diseaseswith a rising bar. ”MICHAEL KLEINROCK / IMS Health

“ The continued adoption of molecular medicine is poised to completely reshape every part of thehealthcare system. ”

MARCIA KEAN / Feinstein Kean Healthcare

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43PharmaVOICE � March 2011

we expect to identify at least the first cohort ofpatients who can be cured with a combinationor sequence of pharmaceuticals.”

According to Patrick Flochel, Ernst &Young’s Life Sciences Leader for EMEIA, whatwill separate winners from losers in the next10 years will still be the scientific excellencethat companies will be able to emulate.

“However, for science to fuel the commer-cial success of life-sciences companies, compa-nies will need to be able to develop the agilityto run successfully different business modelssimultaneously to deliver the best health out-come to different stakeholders in differentmarkets,” he says.

Julian Parreño, senior account director atDatacore Marketing, which combines data-driven intelligence with one-to-one market-ing innovation, says creating more stream-lined R&D organizations, includingde-risking initiatives for clinical candidatesentering Phase III, and live licenses, will becritical to discovering and developing the suc-cessful compounds of the future.

“In an increasingly cost-focused environ-ment, demonstrating the innovativeness ofnew products will also be a key factor,” he ex-plains. “The definition of innovation variesamong multiple stakeholders — researchers,investors, payers, regulators, providers, manu-facturers, and patients — but in the end, newproducts priced in balance with the value de-livered equals innovation. The industry needsto focus on investing in developing medicinesthe market will buy.”

Rob Likoff, co-CEO of Group DCA, an in-teractive agency, adds that innovation in theface of increasing government interventionand oversight will be key to the industry’s suc-cess in the next decade.

“The market can’t support 10 versions ofthe same drug any more,” he says. “Compa-nies that innovate in the lab, in operational ef-ficiencies, in communication outreach will bethe survivors. The danger communicators faceis getting caught up in the technology andoverlooking the true driver of this transforma-tion: the healthcare profession’s need to re-ceive content of value. Content needs to be thefocus. Companies that understand this andthat tap the efficiencies of multichannel com-munications, effectively pairing personal withdigital outreach, will win.”

The Genome

Ten years ago, the excitement of unlockingof the human genome had many thinkingthat personalized medicine couldn’t be far be-hind. Then reality set in; it has proven to bemuch more difficult to take the discoveriesstemming from the unraveled genome in thelab and translate them into therapies. There isexcitement, however, again, as many believethe industry is just one generation of medicineaway from taking a huge leap forward.

“The mapping of the human genome, inmy opinion, is the most significant advance-ment in healthcare,” says Michael Parisi, man-aging partner at Ogilvy CommonHealthWorldwide, a healthcare communications net-work. “Originally funded by the U.S. Depart-ment of Energy, this initiative was undertakento gain critical knowledge about humans thatwould enable us to better understand the causesof disease and, more importantly, help developmedications to treat disease. It was truly the be-ginning of personalized medicine. And thepromise of personalized medicine and the de-velopment and use of diagnostic tests to better


“ Genetic tests and profilingare improving the ability ofphysicians to match patientswith the most beneficialtreatments. ”

DAVE FISHMAN / Snowfish “While the fruits have yet to be fullyharvested, the mapping of thegenome has paved the way for a newworld of genetic testing and personalized medicine. ”

MATT GIEGERICH / Ogilvy CommonHealth

“ The progress that has beenmade to efficiently engineerand manufacture mAbs willenable the development of targeted therapies. ”

BRAD THOMPSON

Oncolytics Biotech

“ The market can’t support 10 versions of the same drug anymore. Companies that innovate inthe lab, in operational efficiencies,in communication outreach willbe the survivors. ”

ROB LIKOFF / Group DCA

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characterize diseases and select treatment arethe game changers of the future. I hope we’llsee the routine use of companion diagnosticsand targeted therapies change the face of seriousillnesses around the world.”

Jed Beitler, chairman and CEO Worldwideof Sudler & Hennessey, a global healthcaremarketing and communications organization,agrees that the mapping of the humangenome has been one of the biggest game-changers in the life-sciences field.

“Not only has it opened the doors to betterunderstanding of diseases and their origins, butit has also led to the development of better di-agnostic tools and therapies to more preciselydetect and attack these disorders,” he says.

Matt Giegerich, chairman and CEO,Ogilvy CommonHealth Worldwide, a health-care communications network, says while thefruits of this mind-boggling project have yetto be fully harvested, it has paved the way fora new world of genetic testing and more effec-tive and efficient personalized medicine.

Rather than the traditional organ-basedapproach to disease and the traditional one-size-fits-all approach to developing therapeu-tics, academe and industry have shifted theirfocus to unraveling the underlying mecha-nisms of disease at the molecular level andcustomizing medicines to molecular sub-groups of patients, Ms. Kean says.

“Today, almost all life-sciences companiesare using information on genetic variation andits effects to delineate disease more preciselyand to enhance the safety and efficacy of newtreatments,” she says. “And, more and more ac-ademic medical centers and other pioneering

vasive breast cancers — is Herceptin, whichwas developed as a monoclonal antibody tothe oncogene HER2.

“The routine use of this treatment hasmarkedly and forever favorably affected thenatural history of this type of formerly deadlybreast cancer,” Dr. Jones says. “The oncologycommunity welcomes the targeted approachto treatment in years to come.”

Biomarkers and targeted therapies areclearly the biggest game changers and indus-try drivers of the past decade and for the nearfuture, says Kevin Coker, VP of U.S. oncologyresearch at US Oncology, a division ofMcKesson Corp.

“By targeting therapies to individual geneticmarkers, scientists can more precisely targetunique cancer types,” he says. “This new infor-mation leads to better trials and helps guide ex-pectations on product life-cycle management.As the course of disease is better understood, itbecomes more predictable, which leads to betteroutcomes for the patient and, ultimately, lowercosts to the healthcare infrastructure. Althoughpersonalized medicine is still in its early days,this all holds great promise.”

No question about it, personalized health-care will be the game changer for the industryover the next 10 years, says Scott Cotherman,CEO of CAHG, an integrated marketingcommunications network.

“The application of genomic informationto diagnostics and therapeutics is one of themost significant developments in health andwellness,” he says. “The promise of personal-ized healthcare — driven by increased under-standing of the human genome, advances in

“Many view cancer as theposter child for how our industryhas made game-changing innovations to treat disease. ”

DAN JUNIUS / ImmunoGen

healthcare providers are reshaping their infra-structure to accommodate these changes.”

One research community that is eager tocapitalize on the therapeutic potential of ge-nomic/targeted therapies is oncology.

“Many view cancer as the poster child forhow our industry has made game-changinginnovations to treat disease,” says Dan Junius,president and CEO of ImmunoGen, whichdevelops targeted anticancer therapeutics. “Asof 10 years ago, we became armed with morein-depth knowledge of cancer at a biochemi-cal and cellular level, and this has led to tar-geted therapies for specific types and stages ofcancers, rather than a one-size-fits-all ap-proach. After the progress of the last decade,there are now more than 20 targeted cancertherapies on the market, with a pipeline ofmore than 100 in clinical studies as potentialnew cancer drugs.”

“Almost all new agents under developmentin cancer have a specific target identified on amolecular basis and therefore these agents,even in the earliest trials, are much more likelyto clinically benefit the patient,” says StephenJones, M.D., medical director and co-chair ofthe breast cancer committee with U.S. oncol-ogy research at US Oncology, a division ofMcKesson Corp. “This is a far cry from 10years ago when new agents rarely helped any-one. Despite the promise of this approach —and we all recognize that this is the future —there are relatively few approved agents basedon molecular mechanisms or targets.”

One targeted therapeutic that has changedthe natural history of one type of breast can-cer — HER2 positive disease, 20% of all in-

“ One of the big game changers inthe past 10 years has been the discovery and development of thebreakthrough cancer therapyGleevec. ”

JOE JIMENEZ / Novartis

“ Over the next 10 years, thecreation and advancement ofnew technology platforms willallow for the rapid, methodical,and systematic study of new biologic drug candidates. ”

BILL NEWELL / Sutro Biopharma

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molecular diagnostics, and targeted therapies— is exciting for the industry, as it holds thepotential to enhance wellness, provide bettertreatment outcomes, and improve overallquality of care.

“These opportunities, however, will bringchallenges for the industry as it works throughissues such as identifying the right biomarkers;conducting clinical trials with smaller patientpopulations; managing co-approval pathwaysof companion diagnostic/therapeutic offerings;and optimizing value propositions in the face ofmore sophisticated payers and benefit man-agers,” Mr. Cotherman continues.

If the future is going to be based on per-sonalized medicines based on each person’sunique DNA and disease, then the safety andtesting around these drugs will change theface of research and development, contendsJohn Cline, CEO of unithink, an electronicclinical research organization.

“The next 10 years in the e-clinical spacewill be about figuring out how to apply tech-nology to allow for the rapid design and test-ing of highly unique individualized medi-cines,” he says. “The second challenge will behow to manufacture those unique medicinesin a cost-effective method.”

Personalized Medicine

The amazing breakthroughs witnessed over

way for completely new treatment optionsand today, patients are treated more and morewith targeted therapies and the one-size-fits-all approach to drug development has becomea thing of the past.

“Understanding the differences betweendiseased and healthy cells has furthered thedevelopment of groundbreaking targeted


“ Companies will spend moretime thinking about how bestto leverage informatics andcompanion diagnostics to improve the economic viabilityof niche drugs. ”

DR. STEPHEN DECHERNEY

MedAssurant

“ Innovation that improvesoverall efficacy and patient outcomes, while decreasinghealthcare expenditures, willclearly drive the shape of themarket over the next 10 years. ”

MICHAEL PARKS / Vox Medica

the past 10 years have been incredible; thedecoding of the human genome, whichopened the door for personalized medicine,and the proliferation of companies that

comprise the biotech industry have been truegame changers, says JeanMarie Markham,CEO of Clinlogix, a next-generation CRO.

“Yet we are only peering into the possibili-ties of tomorrow,” she says. “It is humblingwhen one takes in the genius of what has beenaccomplished. Despite the challenges, the in-dustry’s ability to take these head on, adapt, andfocus on the future and envision what can beparamount to success; it’s this resiliency, deter-mination, and freedom that fuel our collectiveimagination and feed innovation.”

Novartis’ Mr. Jimenez says the key for thefuture will be developing algorithms that willallow us to unlock information in the millionsof terabytes of genetic data available and tocreate new molecular tests that will play anintegral role in our targeted therapy approach.

“Advances in biology and bioinformaticsare already enabling unprecedented analysis ofhuman genes and proteins, and we are startingto genetically identify which patients will re-spond positively to particular therapies,” hesays. “At Novartis, we recognize the possibili-ties for this area and have established a dedi-cated Molecular Diagnostics Unit, focused onmatching the right patient with the rightdrug. We have a rapidly expanding portfolio ofdiagnostic programs and are aiming for multi-ple launches over the next few years.”

MorphoSys’ Dr. Moroney says a clearer un-derstanding of human diseases has paved the

“ Thalidomide opened the doors to adramatic new paradigm of treatment,demonstrating that non-cytotoxic approaches could work in the treatment of multiple myeloma andrelated blood cancers. ”

DR. BRIAN DURIE / International MyelomaFoundation

“While RNAi drove the understanding of the genome,adult stem cells with the powerof ZFNs will change the futureof the industry and the understanding of biology. ”DAVID SMOLLER / Sigma Life Science

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therapies, such as monoclonal antibodies,most notably in cancer,” he says. “Today, sev-eral mAbs are blockbuster drugs.”

With the emerging niche-buster paradigm,focused on therapies that address specific dis-

ease areas, commercial success depends onmoving a larger number of compoundsthrough the discovery and developmentgates into the market, says Josef von Rick-enbach, chairman and CEO of Parexel In-ternational, a global biopharmaceuticalservices provider.

“This will take a different kind of R&Dstrategy, and there will continue to be an in-creasing focus on identifying winning com-pounds and validating those compoundsthrough to proof-of-concept,” he continues.“Overall, in the next 10 years the industrywill continue to transform through theidentification and implementation of newdevelopment models and ways to achievegreater efficiencies in the R&D process.”

Dave Fishman, president of Snowfish,which provides commercial insights tohealthcare, life-sciences, and biotechnol-

ogy companies, believes that the days ofmulti-billion dollar products with near uni-versal applicability are drawing to a close.

“Physicians have long-recognized that anygiven drug may in fact be more effective in cer-tain patient types than in others,” he explains.“Looking ahead, genetic tests and profiling areimproving the ability of physicians to match

patients with the most beneficial treatments.While targeted therapies will significantly im-prove the approach to patient care, the industrywill be challenged to ensure that the optimalROI is obtained given the smaller markets forthese products. Companies will need to have agreater understanding of market segments andthe opportunity they represent in order to aligninvestment with expected returns.”

Stephen DeCherney, M.D., senior VP ofpharmaceutical and life sciences at MedAssur-ant, a medical informatics solutions provider,agrees that without the blockbuster, compa-nies are and will pursue niche products thatare specifically targeted to patients of differentgenotypes and phenotypes.

“Employing the model of a rifle — person-alized medicine — versus a shotgun — block-buster drugs — over the next few years, com-panies will spend more time thinking abouthow best to leverage informatics and compan-ion diagnostics to improve the economic via-bility of niche drugs,” he says. “The challengeremains that the technical, regulatory, and eco-nomic models for developing, approving, andprofiting from personalized medicine have yetto be fully developed. However, this providesample opportunity for first-mover advantage.”


“ The decoding of the humangenome, which opened the doorfor personalized medicine, and theproliferation of companies thatcomprise the biotech industry,have been true game changers. ”

JEANMARIE MARKHAM / Clinlogix

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cal and economic edge required to compete inan increasingly more complex and demandingpayer environment,” he says.

The growing interest in and dedication todeveloping personalized treatments — docu-mented by the recent Tufts Center for theStudy of Drug Development study on the topic— has the most potential to transform health-care, says Edward Abrahams, Ph.D., presidentof the Personalized Medicine Coalition, whichbrings together people with a commitment topersonalized medicine.

“The industry’s commitment to personal-ized medicine will improve treatments in thenext decade, especially if the FDA and CMSsend the right signals to encourage greater in-vestment in the development of safer, more ef-ficacious drugs,” Dr. Abrahams says.

Despite a decade of critical challenges —safety concerns, quality problems, compliancefailures, and pipeline difficulties — the indus-try is now truly research-based and driven bynew knowledge of the human genome, mo-lecular mechanisms of disease, treatment reg-imens, and the emerging power of personal-ized medicines, says James Macdonell, VP ofPatni Americas, a global IT services company.

“These advancements allow us to developand deliver products with unprecedentedknowledge and control of quality, efficacy, and


“ Going forward, specialtypharma is the road to innovationthat can change the human condition. ”

GIL BASHE / Makovsky + Company

“ I hope we’ll see the routine useof companion diagnostics and targeted therapies change the faceof serious illnesses around theworld. ”MICHAEL PARISI / Ogilvy CommonHealth

“We view ourselves as part ofthe ‘multiplier effect’ of innovation emerging frombiotech companies. ”

DR. DOUG RINGLER / Tolerx

marketing and promotion as com-panies will no longer have theneed to market more broadly.

The next 10 years will provide even deeperinsights into the biological underpinnings ofdisease leading to novel drugs that are moreand more targeted at these fundamentalsources of disease.

“Both biotech and pharma companies arewell-positioned to convert this deep and fo-cused translation of science and an expandingexperience base into ever-more-targeted drugsthat open the possibilities for significant im-provements for patients across a wide range ofdiseases,” ImmunoGen’s Mr. Junius says.

Jay Bigelow, CEO of MicroMass Communi-cations, a behavior science-based communica-tions agency, says as the FDA starts to approvetreatments for patients with acceptable geneticprofiles, the industry will reinvest in new drugdevelopment and this will reopen the market todrugs that were previously denied.

“The life-sciences industry will respondwith new business models that are built onpartnerships and hybrid relationships withtheir managed care, physicians, and patientcounterparts,” he says. “This business modelwill focus on patient outcomes, an emergingrole for medical liaisons and transparent in-formation sharing.”

Mr. Parks says innovation that improvesoverall efficacy and patient outcomes, whiledecreasing healthcare expenditures, willclearly drive the shape of the market over thenext 10 years.

“As companies look to fill the estimatedgap of $16 billion in annual revenue over thenext five years alone, there will be far morecollaborations between companies that canhelp drive innovation that adequately ad-dresses patient needs, while finding the clini-

Mr. Cotherman says the industry faces mar-keting and sales challenges as physicians —and their patients — are woefully uneducatedand unfamiliar with genomics-based medicine.

“Marketing communications agencies thatpossess deep and broad knowledge of thisemerging area and its stakeholders will bewell-positioned to help their clients thrive inthis new era of healthcare,” he says.

Mr. Beitler say because much of the health-care industry is now focusing on specialtydrugs that target smaller medical needs-basedpopulations, there will be dramatic changes in

“The business model for developing and commercializing therapieswill, and must, look very different in the future. ”

TIM SHANNON / Canaan Partners

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safety for targeted populations,” he says. “Evenas first treatments emerge, the critical issue ofeconomic feasibility of personalized medicine isproving positive. For example, monogenicneonatal diabetes is now being successfullytreated by personalized medicine, in a cost-ef-fective manner, with improved outcomes.”

Specialty Markets

In the last 10 years, the shift in innovationto more niche, orphan, and specialty pharma-ceuticals has changed the market landscape

from addressing unmet needs in large,chronic populations to focusing on unmetneeds in smaller patient groups, says MichaelKleinrock, director of thought leadership atIMS Health, a market intelligence company.

“This has dramatically impacted the struc-ture and delivery of healthcare and healthcarefunding globally, and ultimately underliesthe emergence and cementing of the patentcliff for large global pharmaceutical compa-nies,” Mr. Kleinrock says.

Chris Bogan, CEO of Best Practices, a re-search and consulting firm, agrees that overthe decade, once-dominant primary-carebusiness and treatment models have evolvedtoward specialty-care targeted therapeutics.

“This mega-shift has commenced slowly,but it is having profound consequences,” hesays. “The mass-market primary-care ap-proach tried to produce blockbuster productspromoted widely across general and specialtypractices. Large sales forces, costly educationand promotion programs, and large non-re-sponder groups for whose treatments insurersreluctantly paid were all part of the landscape.Increasingly, specialty pharma models haveblossomed. They are geared to deliver tar-geted therapeutics or treatments best-suitedfor specific conditions, specific sub-popula-tions, and specific genotypes.”

(Editors’ Note: See the April 2011 issuefor more on the specialty market.)

Ken Ribotsky, president and CEO of TheCore Nation, a family of healthcare marketingand medical communications companies, saysnow that the human genome has been unrav-eled and that there are technologies availableto influence what happens at the genetic level,big pharma companies are starting to investmore on therapies to treat orphan and rare dis-eases.

“There is a shift happening — from treat-ing mass diseases to specialized diseases affect-ing smaller segments of the population,” hesays. “And there’s tremendous potential forhuge financial gains with these emerging ther-apies. The global orphan drug market is ex-

pected to reach $112.1billion by 2014. Whilethe days of mass-market

blockbusters may be over, this term will be re-defined, and the new blockbusters will be thesespecialized therapies.”

During the most recent decade, the great-est paradigm shift in our industry, accordingto Stephen Wray, president and CEO of Cadi-ent Group, an interactive marketing com-pany, has been the shift in focus to specialtymedicine.

“From the perspective of the industry’spipeline composition, the change in this di-rection has been monumental in reshaping thecompetitive landscape,” he says. “As a result,traditional rules for engaging with healthcarepractitioners, forging relationships with pa-tients and caregivers, and gaining market ac-cess have been dramatically altered.”

Tim Shannon, venture partner at CanaanPartners, a global venture capital firm, be-lieves one of the biggest game changers of thepast decade has been the emergence of rarediseases into the academic, regulatory, andcommercial mainstream.

“This is evidenced by Sanofi-Aventis’ ef-forts to acquire Genzyme, but also by thestated intentions of Pfizer, GSK, Novartis,and other big pharma companies to becomeactive in these areas,” he says. “These initia-tives will set the stage for new commercial de-livery models that will be much better posi-tioned to support the accelerated emergence ofpersonalized medicine products. The newmodels will look more like those of rare dis-eases than those of statins and blockbusters ofthe past. The most respected and valued com-panies in the next 10 years will be those thatnegotiate this transition successfully. Thebusiness model for developing and commer-cializing therapies will, and must, look verydifferent in the future.”

While the enactment of the Orphan DrugAct was more than 25 years ago, the biggestimpact has been finding a workable financialmodel for treatments for rare diseases, says GilBashe, executive VP of Makovsky + Com-pany, an independent global public relations,investor relations and branding consultancy.

“Balancing the high cost of discovery,development, and manufacturing with aprice payers are willing to shoulder hasopened the door to treatments — fromGleevec to Soliris — never imagined possi-ble and lives are being reclaimed,” Mr.Bashe says. “Going forward, specialtypharma is the road to innovation that canchange the human condition.” PV


“ The rise of personalized medicine will reopen the floodgates of innovation andbring new drugs to the market. ”

JAY BIGELOW / MicroMass

“ Biomarkers and targeted therapies are clearly the biggestgame changers and industry drivers of the past decade and forthe near future. ”

KEVIN COKER / US Oncology

“ The scientific excellence thatcompanies will be able to emulatewill separate winners from losers inthe next 10 years. ”

PATRICK FLOCHEL / Ernst & Young

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A Decade of Change: Innovation R&D Innovation: The Key to ... · The biggest paradigm shift over...

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