www.terrapinn.com/orphandrug
Top 50 orphan drug thought-leaders
www.terrapinn.com/orphandrug
The top 50 thought-leaders in orphan drugs and terrapinn.com/loyaltyasia rare disease
The following report recognizes the top 50 thought-leaders in orphan drugs and rare disease. Thought- leaders include representatives from industry, patient advocacy groups, research institutions, government and media.
In addition to their biographies, I’ve included a brief paragraph highlighting the main reasons why they have been included in this report.
Please note, thought leaders will appear in alphabetical order and have been selected based on the following criteria:
• Research & methodology • Leadership • Awards & recognition • Community engagement & impact
Think we missed an important thought-leader? Let us know, we’d love to hear from you!
Andre Singer Conference Manager World Orphan Drug Congress USA
+1 646 619 1797 [email protected] www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
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is certified as a nursing assistant, and has had extensive training in emergency emergency in training extensive hadhas and assistant, is as certified a nursing
day operations for the Short Bowel Syndrome Syndrome Bowel Short the for operations day -
to - Founder and CEO, andFounder CEO, Jablonski , advances by comparing diagnostic studies in similar cases. similar in studies diagnostic comparing by advances Jablonski Southeast from graduated He recently technology. surgical andmedicine Born in 1986,inMissouri, Columbia,in Born small inchesofliving four only hehad that discovered was it birth, At life. entire his complications. medical chronic for risk extreme at him putting intestine, make to who continue gastroenterologists by case usedhisinternationally and is still odds five, five, - the the day Jablonski at age twenty age at supervising in disease. the for research accelerated Why we chose Andrew: chose we Why successful extremely been He’s disease community. rare the entire for inspiration amazing is an Andrew follow up care with long trusted professionals who are committed to his success to committed who are professionals trusted with long care up follow ofDirectors Board on SBS Foundation's, the participation through expressed a partnership into developed College with an Associates of Applied Science Degree in Business Administration, with a focus a withfocus Businessin Associates Administration, anwith Science ofApplied Degree College Today Andrew Andrew www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia on VI and and VI
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™ and eventually their partner their eventually and ™ for their son Ryan, called the Ryan Foundation. Foundation. Ryan thecalled sonRyan, their for
system, both through the the through both system, Dant multiple multiple BioMarin is best known for his work over the last 18 years to develop novel treatments for for treatments novel to develop 18last years the hisover work known for is best
: his position as Chief Medical Officer of Officer Medical position as Chief his Kakkis left left
and support to develop an enzyme replacement therapy ( therapy replacement enzyme an develop to support and model to patients a canine successful from translated therapy the get to TheI. struggle MPS by formed organization ofa new support patient financial critical to thedue succeeded Jeanneand Mark Dr. Dr. disorders. rare neglected Harbor at bungalow hisa in research work began He Kakkis and approval of approval and , a rare disease biotech with an impressive orphan drug pipeline. pipeline. drug orphan impressive with an disease biotech , rare a Kakkis President and Founder, and Founder, President . , has working to develop and advance rare disease treatments for over 18 years. He has guided guided He has years. 18 over for treatments disease rare and advance develop to working has
graduated from Pomona College, magna cum laude, and received a combined MD and PhD degree from the from degree PhDMDcombinedand a received magnacumand College, laude, Pomona from graduated in 1998 and held various positions including Chief Medical Officer from 2006 to 2009.from Officer Medical Chief positionsincluding 1998various in held and
Ultragenyx
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cystinosis
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AG, where he launched Germany's first biotech IPO in 1999. Before IPO 1999.biotechin Before first Germany's helaunched AG, where Research Foundation, the and Foundation, Research
cystine
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doctors and patients with the patients and doctors
line patient care, and at globalat and care, patient line - Myers Squibb and Eli Lilly. Eli Lilly. and Squibb Myers
- With a strong emphasis on leveraging the next next the leveraging on emphasis With strong a
Pfizer is a leader in the safe, effective and appropriate appropriate and effective safe, in the leader is a Pfizer Pfizer sure sure
group makes makes group
Hall is the Chief Medical Officer of Pfizer Inc. Trained as a psychiatrist, she hasshe as a psychiatrist, Trained Inc. ofPfizer Officer Medical Chief Hall is the -
the first clinical trials in humans onward. humans trials in clinical the first Hall: - Chief Medical Officer, Medical Officer, Chief
Prior to her work in industry, she led research projects for the National Institutes ofHealth Institutes National the for projects she ledresearch industry, inherwork to Prior ofCollege University Howard at of Psychiatry oftheDepartment vice chairperson was and Medicine. Freda Lewis Freda front research, medical academia, in roles leadership held Bristol Vertex, includingcompanies biopharmaceutical Hall was appointed by the Obama Administration to the inaugural Board of Governors for the for ofGovernors Board theinaugural to Obama theAdministration by appointedHall was - Hall leads the Pfizer Medical Division responsible for providing providing for responsible Division Medical Pfizer the Hall leads - Hall has been named as one of Savoy’s Top Influential Women in Corporate America 2012 America in was and Corporate in Women Influential Top as named beenHall hasoneSavoy’s of - Centered Outcomes Research Institute (PCORI), and in 2012 she was appointed chair of the Cures Cures ofthechair 2012in and she appointed(PCORI), was Institute Outcomes Research Centered - use of medications, from from medications, of use medical information they need to make informed choices. choices. informed make to need they information medical her drugs, orphan of generation Why we chose Dr. Lewis Dr. chose we Why Lewis Dr. selected as the Healthcare Businesswomen’s Association 2011 “Woman of the Year.” Year.” of the Association 2011 “Woman Businesswomen’s as Healthcare the selected Lewis Dr. She also serves on the Executive Committee of the Clinical Trials Transformation Initiative and on numerous other onnumerous and Initiative Transformation Trials Clinical ofthe Committee also serves SheonExecutive the Discovery, onDrug Forum ofMedicine’s School,The Institute Medical thoseof Harvard including boards, the Children. Save and Translation, and Development, Acceleration Network Review Board and a member of the National Center for Advancing Translational Sciences Translational Advancing for Center a Nationalmember andofthe Board Review Network Acceleration of Health. Institutes oftheCouncilNationalAdvisory (NCATS) In 2010, Dr. Lewis 2010, Dr. In Patient Freda Lewis Hall, Lewis Freda www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia by by
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funding funding sourced sourced democratize democratize - to to
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design personalized research projects for diseases so rare that no organization exists to to help. exists organization no that diseases so rare for projects research personalized design LouisandSt.in University theWashington member is at Linalso a schoolmedical faculty Dr. ever first oftheanalysis computational the led Jimmy Cheng Jimmy cutting leverage to community any enableto platform first theworld's Genomics Institute, 18 thewith of disease. Partnering rare any of understanding advance to biotechnology edge custom helpsRGI JohnsUCSF, Hopkins, and Yale, such as Harvard, institutions,medical top Founder and President, andFounder President,
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). Prior to Priorto ). AB
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solutions for patients with unmet unmet with patients for solutions commercialization of orphan drugs, drugs, orphan of commercialization Genzyme renowned not only for his broad broad his only for not renowned and and
in identifying identifying in Swedish Orphan Orphan Swedish the development development the founder of Catalyst Medical SolutionsInc. Medical of Catalyst founder -
and Internist at Massachusetts General Hospital in Boston and was thewas and Boston in Hospital General Massachusetts at Internist and
President & CEO, & CEO, President
Dr McDonough held various seniorwithinpositions held various McDonoughDr
Sobi Before Africa. and East Middleof Europe, as President latest Paediatrician Co and President Born 1970. Medical doctor, M.D. Harvard Medical School, B.Sc. in Biology and B.A.School,and in Medical B.Sc. Biology in Harvard M.D. 1970.doctor, Medical Born Carolina. ofNorth University from Philosophy Orphan of Officer Swedish Executive is Chief McDonoughDr
needs. medical medical having led Catalyst Medical Solutions, Genzyme and SOBI. He is He SOBI. and Genzyme Solutions, Medical led Catalyst having passion his but alsofor disorders in genetic experience Why we chose Dr. McDonough: Dr. chose we Why of experience has impressive McDonough Dr. Geoffrey McDonough, Geoffrey www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
.
. disease disease He
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work 2011 global
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fellowship in
.
commercial to and 2000
Genzyme and
Healthcare in Medical
treatments role
Care
of
October School
Clinic
in
Director
School
Dr. Meeker oversaw oversaw Meeker Dr. Genzyme’s
responsibility, including
President, Institute
Medical important
for organization,
Cleveland Medical
Genzyme an
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Business
.
as of the
changing therapies for patients I, MPS with patients for therapies changing as
- products,
increasing at Vermont Pulmonary/Critical California
CEO played
a 1994 of of
responsible about the risk/benefit ratios ratios about risk/benefit the Harvard
chapters the
management ®, life ®,
in
he at
and was
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4
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appointed Subsequently,
country positions
of
Rare Disease Business, Rare . University Officer,
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held Prize Genzyme
Boston
Myozyme role,
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Israel
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of Vice Vice
. ViroPharma
(R) from Eli Lilly and Company. Company. andEli Lilly from (R) , including leading efforts in raising raising in efforts leading including , Vancocin from 1997 2006.2006,to the In assumed he from ViroPharma Treasurer Treasurer , and , President and Chief Executive Officer, and Chairman of theof Chairmanand Officer, Executive Chief and President
ViroPharma’s Chief Financial Officer Financial Chief , Vice President, Chief Financial Officer, and Chief Operating Officer Operating Chief and Officer, Financial Chief Vice President,
, he was with KPMG LLP, independent certified public accountants, where he served as heserved where accountants, public certified independent KPMGwithhewas LLP, , . Milano has been instrumental in buildingin beenhasinstrumental Milano . Mr acquisitionofthe and $900 capital millionin nearly activities relations investor and developmentall inbusiness role critical a has He played Vincent Milanois Vincent 1996inas served and2008. as He the joinedcompany ofMarch Directors of Board President of role President, CEO and Chairman CEO of the Board, President,
ViroPharma to joining to . Milano received his bachelor of science degree in accounting from Rider College. Rider from accounting inhis ofscience bachelor degree received Milano . result of the company’s internal committment to advancing and translating science, and accelerating rare rare accelerating and science, translating and advancing to committment internal the company’s of result research. disease Under Vincent’s leadership, Viropharma has become one of the most prominent orphan drug biotechs in biotechs drug orphan prominent the most of one become has Viropharma leadership, Vincent’s Under is a This capability. fundraising its and amazing acquisitions, for its appetite for recognized market, the Why we chose Vincent: chose we Why Mr Prior manager. senior the company, and has contributed significantly to establishing the strategic direction of the company. ofthecompany. direction strategic the to establishing significantly has andcontributed company, the Vincent Milano, Milano, Vincent www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
will will hasa had
Research. He Research. Moscicki served as a staff as serveda staff
is of a member
U.S. Food and Food U.S. only drugs available in available drugs only - . His is board certified incertified His is board . Moscicki for worldwide regulatory and regulatory worldwide for Moscicki
newly created positionof created newly for Drug Evaluation and Evaluation Drug for immunopathology counter and prescription and counter training program in Allergy and Clinical Clinical and Allergy in program training - the Center Center the the - at at
. Prior to that, he was also hewas responsible that, Priorto . . In his previous role as Chief Medical Officer and Senior Vice Vice President, Seniorand Officer Medical as Chief role hisIn previous . Science Operations Science Operations , M.D. had been recently appointed for the for appointed recently beenhad , M.D. Sanofi for for Genzyme in its role to regulate over regulate to its role in has previously served as Head of Genzyme Clinical Development after the recent therecent after Development as servedClinical Head ofGenzyme previously has
Moscicki
CDER CDER :
Moscicki . Deputy Director for Science Operations, CDER, CDER, Science Operations, for Deputy Director
, Dr with integration all aspects of for responsibility worldwide hehad Affairs, Medical and Development Clinical Richard A. Richard Director Deputy lead help US. the Moscicki at Genzyme. He joined the company in 1992 as Medical Director. In his role at Genzyme, he was Genzyme, hewas at role hisIn 1992in Director. as Medical Genzyme. He thejoinedcompany at
79) at the Medical Center Hospital of Vermont. From 1979 to 1983, he held clinical and research research 1979and1983, clinical From to heheld ofVermont. Hospital Center Medical the79) at - received his medical degree from Northwestern University in 1976. He served his residency in internal 1976.internal inin He his residency served University Northwestern from his degree medical received
Moscicki Moscicki of patient registry optimization and collaboration to expedite trials. clincial expedite to and collaboration optimization registry patient of Why we chose Dr. Dr. chose we Why advocate an out spoken He is drugs. orphan developing experience years than 20 has more Moscicki Dr. Internal Medicine; Allergy and Immunology; and Diagnostic Laboratory Immunology. Dr. Dr. Immunology. Laboratory Diagnostic and Immunology; and Allergy Medicine; Internal ofthose societies membersocieties as is ofcommittees servedactive has andandanan professional numerous journals. professional several for supporteditorial has provided and 60 publications, thanHe has more USP. to advisor medicine (1976 medicine immunologyandclinical Schoolin Medical MGH Harvard at and fellowships faculty appointment at Harvard Medical School Medical since 1979. Harvard at appointment faculty Dr. involved in the development and approval of nine products. Prior to joining Genzyme, Dr. Dr. Genzyme, joining Priorto products. ofnine approval and development thein involved of the was andDirector Hospital Massachusetts General at physician Dr. NIH awards. several by supportedbeenhas MGH, research his At years. several for Immunology clinical research and medical affairs for for affairs medical and research clinical pharmacovigilance Richard Richard Administration Drug www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
in BI
led of the
Ulm, of and and
cystic cystic
global
IMID, of
of
a
also
launch Boehringer
on worldwide
He
centers
development
. fellowships
for and Research Development, Fibrosis,
Assurance
University candidates 1997
therapy Vertex’s
in Cystic different
Global
Clinical for approval C, drug numerous - in
Quality
gene completed
Einstein
of all
Non
Development, He and
of
. .
beginning Albert President
oversight Hepatitis
and and
successful
. the are
discovery disease
Vice management the
Japan,
at (CF) Development,
to University Chemistry
the Clinical R&D strategic
and
Research
label to include another group of another include group to label
and
led development
as
Executive the
Chemistry Vertex’ Organic 2011 KALYDECO
oversaw
provides Rochester well
As
Canada in of cardiovascular in
chemistry
for
.
for . at President,
he
D as
. Kalydeco’s Pharmaceutical
Ph areas
2003 Vice
which Mueller
a
. America, Theoretical Officer, Key
Vertex Pharmaceuticals Vertex
medicinal Dr
July .
and
Biophysics
,
in submission
responsible Senior
in
in
inflammation,
programs, Operations
South Diseases,
as
Affairs
was and degree
Scientific
research, and
Vertex
he Ingelheim
NDA/MAA served
research Chief a
Medical potentially expand expand potentially basic
), Professorship & North immunology,
joined
in
University to to Neurological where a
and in
of .
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and . Boehringer undergraduate Inc
discovery Dr holds Oxford
areas an
Mueller
positions
at
.
with portfolio
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also Regulatory Cancer, INCIVEK Dr Development drug Control,
both
Vertex, in Chief Scientific Officer, Officer, Chief Scientific
he time several
to
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held received worldwide
Pharmaceuticals, where
. Pharmacology
programs
coming
During to
.
Mueller
. also been leading leading the studies been also fibrosis. Fibrosis, Kalydeco. His leadership in strengthening the scientific collaborations with the Cystic Fibrosis Fibrosis Cystic with the collaborations the scientific strengthening in leadership His Kalydeco. Fibrosis, has He drug in 2012. the oflaunch successful the essential for was other stakeholders and Foundation Why we chose Dr. Mueller: Dr. chose we Why Cystic for drug orphan approved recently the of process development the entire oversaw Mueller Dr. Quantum Dr Germany, candidates, worldwide company’s research basis Prior Ingelheim Peter Mueller, Mueller, Peter www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
of to
and the
global Nader
. on
Board
Dr
the Executive healthcare healthcare .
the promoted to file its file to
representing
led the served of
companies
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his member formerly
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Poulenc
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trade
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of ,
division executive
Dr was
. University regulatory
he
Chamber Team BioNJ chief
medical
2008
including
and
for Vaccines
Joseph
chief Jersey , in the US in 2013 in the US , in .
the company into a leading global biotech focused on focused a biotech leading global into the company since
Pharmaceuticals St
as
Previously,
president, . Leadership medical
Pasteur New positions
Trustees
from of 2006
Noven
the
the
2007
been
in
at of
in
and
America has America
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NPS executive
Medicine Pharmaceuticals
of the MD, officer
Jersey
North in
North member of
. joined NPS operations
Hypoparathyroidism the New
of He and
Nader,
. of number on for for
Board
operating a
Doctorate chairman
and Tennessee
as
served held markets commercial Francois Directors industry chief of
Institute President and Chief Executive Officer, Officer, and Chief Executive President
Natpara French
serves
industry, his
drove the development and approval for NPS’S lead product, product, lead NPS’S for approval and the development drove University
Healthcare
the earned
currently
the
of
from
biotechnology Nader treatments for patients with rare disorders. Under Dr. Nader’s leadership NPS now plans plans now NPS leadership Nader’s Dr. Under disorders. with rare patients for treatments product, second Why we chose Dr. Nader: Dr. chose we Why Nader Dr. transformed ultimately which and US; Europe in Nader
. . Dr MBA Board Dr the François Nader, Nader, François www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
He rare disease disease rare Ways and Meansand Ways develop develop He He is also ofboththedean
. chairman ofNew Englandchairmanthe - Massachusetts Preserving Access to Orphan Drugs Acts, bipartisan legislation that that Acts, bipartisan Drugs legislation Orphan to Access Preserving
E. Neal was first elected to the United States House of Representatives in 1988.in House ofRepresentatives States United the to elected E. first was Neal
sponsored the the sponsored - Large Whip for the House Democrats. He He is a theco House Democrats. for Whip Large - the Barney School of Business and Public Administration at the University of Hartford in ofHartford University the at School Public ofBusinessAdministration andBarney the 1976. Richard Richard E. Neal was born in Worcester, Massachusetts on February 14, 1949 and was 14, 1949raised was andonFebruary Massachusetts Worcester, in bornE. was Neal Richard International of American He is a 1972 ofSpringfield.graduate thein City educated and a ofmemberwas andScience Political in hisDegree Bachelor's received he where College, from Administration Publicin Degree Masters his He received HonorSociety. National the US House of Representatives, ofUS House Representatives,
Neal is NealAt an
makes an important policy clarification and will remove a barrier to research and and research to barrier a remove will and clarification policy important an makes therapies. Why we chose Congressman Neal: Congressman chose we Why co Neal Congressman Congressional Caucus, where he continues to advocate for the unique regional interests of the six New England States. six New Englandtheof interests regional uniquethe for advocate to continues he where Caucus, Congressional Congressman avoid paying U.S. taxes. He has sponsored legislation that would increase the national savings rate by encouraging the encouraging by rate savings thenational increase would that He legislation sponsoredhas taxes. U.S. paying avoid for deductibletax expenses tuitionand care health to make worked has andaccounts, retirement ofindividualuse Subcommittee. MeansTrade and class middleHe is people. also a Ways memberofthe the Massachusetts delegation and the New England Delegation in the House thein Delegation New Englandtheand delegation Massachusetts the to offshore moving from companies American prevent a leadsponsorto beenhasNealoflegislation Congressman represents the First Congressional District of Massachusetts. He He ofMassachusetts. is a seniorpowerful memberofthe District Congressional First the represents Measures. onSelect Revenue Subcommittee theofMember Ranking theand Committee Richard Neal, Richard www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
in in at
Head, career
School
units Santaris
school
sciences
Global
pharmaceutical Medical
academic
and .
marketing business full companies
decision
and
2013 the
Officer global
INSEAD
clinical . Copenhagen April,
medical
of as the from in
oversaw
ASA
biotechnology early he
well Marketing Shire from
Euros
for His
as of
Holding .
University
Chief MBA
CEO
billion as Medicine the
an licensing products. products. licensing
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Biotech
pediatrics, of of
Shire PCI
where, gained
Specialty and Health, for
became
He Medicine Board
excess
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of
stage and in and stage in MPH Public the - at
of Directors of
medicine France
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was sales Doctor of General MBA,
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MD, with
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and emergency
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Denmark on qualified Ornskov
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in is China
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Chief Executive Officer, Officer, Executive Chief and medicine,
,
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Harvard
product Europe, Flemming Flemming Prior Strategic
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and an
roles schools
A/S
clinical research, and BD focused on late on focused BD and research, clinical - gained
currently
role, he is already starting to affect change at Shire, by combining R&D and business development with development business and R&D combining by Shire, at change affect to starting is already he role, pre Why we chose Dr. Dr. chose we Why the to new relatively While development. drug in orphan global leader a Shire, of is the CEO Ornskov Dr. He Pharma medical Fontainebleau, and included Flemming www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia rare rare
science; science;
2010, where she is where 2010, to founding the Rare Rare the founding to Prior U.S. FDA U.S.
is also actively involved in is involved also actively
Pariser in the Office of New Drugs in Drugs New of the Office in other stakeholders to further the development of treatments treatments of thedevelopment further to stakeholders other policy and guidance generation for rare disease product disease product rare for guidance generation and policy has worked at FDA since 2000. FDA at worked has
Pariser is the Associate Director for Rare Diseases in the Office of New Drugs at Diseasestheat ofNew Office Drugs Rare thein for is Associate theDirector
education; and, education;
: Pariser the Rare Diseases Program Diseases Program Rare the
for rare diseases. Dr. diseases. Dr. rare for she worked ONDwhere in Leader Team and sheOfficer a was Medical Diseases Program, disorders. genetic rare for ofproducts regulation and review onthe exclusively almost Anne Dr. Research. and Evaluation Drug for Center FDA’s governmental other developers, drug with and FDA within collaborations numerous and groups advocacy agencies, Pariser Associate Director for Rare CBER, Diseases, Rare for Director Associate , established established
specific training and and training specific - Pariser Pariser disease regulation. and review currently working to support, facilitate and accelerate the development of therapeutics for rare diseases. rare for therapeutics of development the accelerate and facilitate support, to working currently regulatory and biomedical of development the on concentrates Diseases Program Rare The Why we chose Dr. Dr. chose we Why Dr. Anne www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
U.S. FDA U.S.
oversees including the Orphan Grants Program, Orphan Drug DesignationDrug Orphan Program, Grants theOrphan including oversees
, MD, JD is the Director, Office of Orphan Products Development (OOPD) FDA,(OOPD) at Development Products of OrphanOffice JDDirector, is the, MD, Rao Rao
:
The provisions of the Orphan Drug Act of 1983 and other amendments to the Federal Food, Food, theFederal Act 1983to ofamendmentsDrug other and of Orphan the provisions The OOPD the administers, that programs major several Cosmetic Actand established Drug, Dr. which ConsortiaDevice Program. Pediatric and Use Device Program, Humanitarian Program, Gayatri demonstrate that products medical of evaluation and thedevelopment advance to working diseases. rare of or diagnosis,treatment prevention, for promise Rao Director, Office of Orphan Product Development, CDER, Development, of Orphan Office Product Director,
, Rao
graduated from the University of Medicine and Dentistry of New Jersey, New Jersey Medical School Medical and New Jersey ofNew Jersey, Dentistry andofMedicine University the from graduated comes to OOPD from FDA’s Office of the Chief Counsel where she provided advice on a wide range of issues ona advice wide range she provided Counselwhere of Chief theOffice comes FDA’s OOPD to from
Rao Rao Dr. Rao is the Director of the Office of Orphan Product Development at the FDA. She has led the efforts efforts the led has She the FDA. at Development Product Orphan of the Office of is the Director Rao Dr. development the nurture to program grants the office’s oversees and FDASIA, implement and launch to drugs. orphan and breakthrough new of Why we chose Dr. Dr. chose we Why related to orphan products. to orphan related earned both her law degree and bioethics masters degree from the University of Pennsylvania, where she where of Pennsylvania, theUniversity from degree masters bioethics and degree law herboth earned firminlaw a private for issues. school,law she Following worked related FDA and onhealthcare concentrated matters including matters, related healthcare otherand drug and onfood primarily focusing D.C., Washington, Dr. Dr. Dr. protection. subjecthuman and trials, clinical products, devices, combination medical to related Gayatri www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
access and and access NHS Trust.
improve patient patient improve
2008).
-
Royal Society of Medicineof Society Royal
1998) of the Committee on Safety ofMedicines; and on 1998)Safety oftheCommittee pharmacologist to the Newcastle Hospitals Newcastle the to pharmacologist - clinical clinical the continued development of new therapeutics for orphan conditions conditions orphan for therapeutics new of development the continued physician and physician
President and CEO, President 1992) and chairman (1993 1992)chairman and - was the Ruth and Lionel Jacobson Professor of Clinical Pharmacology at the University of theUniversity at Pharmacology ofClinical LionelJacobson Professor and was Ruththe He 1973the samepositionof timeto 2006the from heheld .At Tyne upon Newcastle consultant Sir Michael Rawlins was chairman of the National Institute of Health & Clinical Excellence Excellence Clinical ofHealth& Institute theofNational chairman was MichaelRawlins Sir School ofLondon theat 1999.in Professor He is also since Honorary itsan formation (NICE) the at Professor Emeritus and London,of University Medicine, Tropical and Hygiene Tyne. upon ofNewcastle University a leading global voice in the application of health economics to to health economics of the application in leading global a voice chairman (1987 chairman - reconcile patients’ need for need patients’ reconcile
with society’s need to manage the costs of healthcare sustainably, whilst preserving incentives for incentives preserving whilst sustainably, healthcare of costs the manage to need society’s with development. and research in invest to companies reimbursement for medicinal products. His contributions have helped advance the understanding how of understanding the helped advance have contributions His products. medicinal for reimbursement to Why we chose Sir Michael: chose we Why is Michael Sir chairman of the Advisory Council on the MisuseontheCouncil oftheofDrugsAdvisory(1998 chairman was He vice Sir Michael Rawlins, Rawlins, Michael Sir www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
NORD profit healthcare environments. environments. healthcare profit -
for -
is the President and CEO of the National Organization for Rare Disorders Disorders Rare for Organization theofNational CEO and is President the profit and notand profit
- Saltonstall is also committed to globalization of the rare disease patient community, as diseases community, disease patient oftherare to globalization is also committed
President and Chief Executive Officer, Officer, and Executive Chief President Peter Peter patients when expedited be can research and boundaries geographical notrecognize do patient with programs collaborative He established has involved. are counties many from Japan.and Europe in communities Peter L. Peter as a senior 30 years than more for served He having 2008 NORD joined(NORD). in after for bothin official , Saltonstall achieved best through joint efforts. joint through best achieved government agencies, drug and medical device companies, academia and investment communities. His communities. and investment academia companies, device and medical drug agencies, government be can disease patient the rare for advances that his view from stems build collaborations to efforts Why we chose Peter: chose we Why physicians, groups, the patient between relationships new has forged NORD leadership, his Under assistance to patients in need of medications that they cannot afford; and has recommitted to facilitate research research facilitate to recommitted has and afford; cannot they that ofmedications need in to patients assistance access assure patients. and by new therapies into Under Peter’s leadership, NORD also has updated and expanded its Patient Assistance Programs, which include which Programs, Assistance its Patient expandedand NORD also updatedhas leadership, Peter’s Under Peter Peter www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
and
compounds at a at compounds
Pharma Genzyme’s Duchenne , the Dutch Association of Dutch the, Nefarma for five years in various executive roles, roles, executive various in years five for
dystrophy and Huntington’s disease, using itsdisease, using Huntington’s and dystrophy
Genzyme myotonic , an innovative Dutch biopharmaceutical company focusing focusing company biopharmaceutical Dutch , innovative an Prosensa . He is also past Chairman of He is Chairmanalso . past
, Hans worked at , Hans worked Prosensa Biovitrum
muscular dystrophy, dystrophy, muscular Prosensa
is CEO of is CEO
key agreement with GlaxoSmithKline for part of its ofpart for GlaxoSmithKline with agreement key , Hans is currently Executive Board Member of the Dutch Top Institute Top ofMemberDutch the Board Executive , Hans is currently a million. changing therapy for Duchenne Muscular Dystrophy. Additionaly Additionaly Hans’ the led Dystrophy. Muscular Duchenne for therapy changing Schikan - Duchenne
joining Before of Development Strategic and GlobalMarketing for President Vice including at 17 years diseases.hespent Genzyme, Priorto genetic rare for ofproducts portfolio Hans diseases rare for treatments ofnovel commercialization and development discovery, the on like platform. modulationRNA Prosensa Chief Executive Officer, Officer, Executive Chief , from Utrecht University. Hans has given numerous presentations on orphan drugs, rare diseases and rare drugs, onorphan presentations numerous Hans has given University. Utrecht from
PharmD , both at corporate level and in country operations which included assignments in Asia and Europe. Europe. Asia inandassignments which included operations country in and level corporate at both , Schikan executive Director of Swedish Orphan of Swedish Director executive - firms efforts to establish establish to efforts firms 700 USD nearly of value Under Hans’ leadership, Prosensa has become one of the industry’s most promising biotechs, largely due due largely biotechs, promising most the industry’s of one has become Prosensa Hans’ leadership, Under its life of success the to Why we chose Hans: chose we Why has He a innovation. Aside from his role at at his role Asidefrom Non Industry. PharmaceuticalBased Research Organon Hans www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
to
get and was
they
many
Human
and
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before and
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Americans 2009 employers, collaboration
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Health of
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Magazine
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April
building of
the .
the elderly, and the rare disease disease population. the and rare elderly, the HHS on more
to historic
slow Department
the Governors
Sebelius
(HHS) to
of insurance coverage
Governor parents the
market,
Five
leadership, as to Secretary, Secretary,
the part
give
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of health patients Kathleen Services As and
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Sebelius’s medicines
America’s
Sebelius of
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- one to
Secretary
life
children, those with disabilities, disabilities, those with children, Why we chose Secretary Sebelius: Secretary chose we Why health enhance and America’s improve to efforts ambitious led has Sebelius Secretary office, taking Since vulnerable most nation’s the of services some to human ofdelivery the Secretary named occur, education Under bring Kathleen Kathleen www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
, of
and was
MGI
at Director panel
a SuperGen
by Shrotriya
for entrepreneurs .
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Dr
performance . Executive
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Medical winner being 15 President and Chief Chief and President
recognizes
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Year® most
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innovation,
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the
was
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and Shrotriya
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. Spectrum Pharmaceuticals as Pharmaceuticals Spectrum such & Vice Dr
. tumors. The drug drug The tumors. Entrepreneur Spectrum Pharmaceuticals Spectrum in
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businesses and ,
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turn Chief Executive Officer, Officer, Executive Chief
Research Previously Inc Pharma In September 2000, Dr. 2000, Dr. September In thisIn Officer. Executive Chief 2002, hewas August in appointed and Officer Operating structural coordinated and strategy businessin major changes hashe spearheaded capacity Inc. Pharmaceuticals,ofSpectrum theinformation culminating reorganization
to Shrotriya , Ernst
various
his
. excellence
for held 2011
cell lymphoma (PTCL) and other other solidand (PTCL) lymphoma cell Clinical
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he judges is
cited CNS
2013, with an FDA decision anticipated 2014. in anticipated decision with an FDA 2013, commitment -
Shrotriya years
demonstrate
18 Shrotriya
. by mid by Dr. Shrotriya led Spectrum to launch three drugs with emphasis on Belinostat, which is targeted to treat treat to is targeted which Belinostat, on with drugs emphasis three launch to Spectrum led Shrotriya Dr. T peripheral filing (NDA) Application Drug a New make to expects Spectrum and the FDA, by designations Track Fast Why we chose Dr. Dr. chose we Why independent specifically who personal Dr For Worldwide Rajesh www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
Party (PT). He (PT). has Party
Labour conquered the biggest poll for for poll biggest the conquered
1999,with He 4,229,706 has votes. - Suplicy
: Brazil 1983. - was elected Senator of the Republic from 1991 from oftheRepublic Senator elected was was the first elected senator in the history of the Brazilian ofBrazilian the history thein senator elected thewas first
Suplicy Senator, Senator, He is one of the founders of the Workers Party of Brazil (PT) and member of the Executive ofExecutive memberthe (PT) and ofBrazil Party of the Workers is He onetheoffounders ofParty. the Directory National the and Suplicy Since terms. three for Senate PTFederal ofleaderposition ofthethein the occupied Suplicy Deputy serve as State to elected He was first 20 years. than more for servant a public been 1979 from , has served as a Senator for the state of São Paulo. In 1991, he became the first member of the PTmember ofthe to 1991,Infirst thehebecame ofSão Paulo. state the for has as serveda Senator
Suplicy Suplicy Fund for Rare Diseases which will support research for rare and neglected diseases in Brazil. and neglected rare for research support will which Diseases Rare for Fund more than 13 million rare disease patients in the country. In 2011, he introduced the National Policy for for Policy National the introduced 2011, he In the country. in patients disease rare million 13 than more National the establish to an initiative on working and is also Rights, Disease Patients Rare of Protection Why we chose Senator Senator chose we Why serving to dedicated framework a policy creating in Senate leading Brazil’s has been Suplicy Senator take office as Senator. In the 1998 elections for the Federal Senate, Eduardo Eduardo Senate, theFederal 1998 theelectionsIn for as Senator. office take 6,718,463with votes. ofSão Paulo, ofhistory the second greatest theand Country thepositionin this 1991, Eduardo Eduardo www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
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to Governor entering
annual
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. the
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. industry considerably. As head of Medicare/Medicaid, Ms. Tavenner has a key role in addressing these in addressing role key has a Tavenner Ms. Medicare/Medicaid, of head As considerably. industry improve patients helping and challenges The The public Why we chose Marilyn: chose we Why Ms from service national (HCA) through assumed and Before former billion Marilyn Marilyn www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
which which
of
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its
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America .
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were. He founded founded He were.
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of
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biotechs Genzyme and Directors
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of in of
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at Erasmus University in The Netherlands and TheinNetherlands University Erasmus at
the
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1985
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. Mr Technology and Board Prior the Executive Founder and Former President, President, andFounder Former , studied economics economics the at studied
Termeer Termeer disease and biotech space thanks influence. his to thanks space biotech and disease who figured out how to build a great business by making drugs for rare diseases. An inspiration and and inspiration An diseases. rare for drugs making by business a great build to out how figured who rare in the companies successful other lead to on moved since have his protégés of many pioneer, is often said to have kick started today’s orphan drug biotech M&A frenzy. Henri is definitely a Henri is definitely frenzy. M&A biotech drug orphan today’s started kick have said to often is guy as the known be will always He space. drug orphan in the and world biotech in the mover/shaker Why we chose Henri: chose we Why what knew anyone before long pioneer biotech a was Henri
Mr. Mr. of Virginia. SchoolofUniversity the Dardenthe from of Business Administration his Master received Henri www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
Republican term pledge. In additionIn pledge. term -
Preserving Access to Orphan Orphan to Access Preserving the coalition ofthecoalition the the
-
. Philadelphia Inquirer wrote that hehas that wrote Inquirer Philadelphia
and fulfilled his three his fulfilledand introduced introduced . The .
Representatives, Representatives, president of the Club for Growth, owned and operated a small operated ownedand Growth, for Clubofthe president Pennsylvania
sized businesses making it easier for them to raise capital and create jobs. create and capital raise them to businesses making easier it for sized to the House to theof - along with two other Senators Senators other two along with was also was the helped write and enact the bipartisan JOBS Act, which cuts regulatory burdens on small burdens JOBS Act, which cutsbipartisan enactregulatory theand write helped He He medium and Sen. committees. Economic Joint and serves Budget; Finance;Banking; onthe senator The Committee Steering ofSenate chairmanthe is the Toomey Senator Pat Toomey is a leader on economic, financial services, and budget issues. is He oneconomic, is services,a budget leaderfinancial and Toomey Pat Senator responsibilityas a fiscal ofknown champion matters." money on voice as leading "a emerged US Senator, US Senator, , a bipartisan legislation to ensure that the development of treatments for rare rare for treatments of the development that ensure to , legislation bipartisan a was previously elected was previously Senator Patrick Toomey, Toomey, Patrick Senator and Kris Toomey live in Zionsville with their three children Bridget, Patrick, and Duncan. and Patrick, Bridget, children three theirwith Zionsville in live Toomey Kris and . Toomey Toomey . in front of innovative new therapies for rare diseases and conditions diseases and rare for therapies new innovative of front in Drugs Act of 2011 of Act Drugs placed not are hurdles unnecessary that regulatory ensures This legislation jeopardized. is not diseases Why we chose Senator Toomey: Senator chose we Why US Pat Sen service,his senator publicthe to servicesfinancial thein industry. worked and Valley, Lehightheinchain restaurant senators that advocates for innovative, conservative policies. conservative innovative, for advocates that senators Patrick Toomey, Toomey, Patrick www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
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registries. structuring many of GSK’s key partnerships in the rare disease space including Angiochem and Prosensa. Prosensa. and Angiochem including disease space rare in the partnerships key GSK’s of many structuring patient trials through clinical and optimize expedite to approaches innovative GSK’s alsooversees Patrick Why we chose Patrick: chose we Why in instrumental was 2010, and Unit Diseasein Rare GSK’s ofthe launching in involved was Patrick stage mature setting medicines Patrick Patrick www.terrapinn.com/orphandrugterrapinn.com/loyaltyasia
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Genzyme
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secured at
and
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Genzyme’s Brazil
leadership,
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diseases with an award. The company was honored honored was company The award. diseases with an Diabetes
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rare rare and he
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as well as initiatives to increase patient access to Genzyme treatments. Genzyme to access patient increase to as initiatives as well
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private
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to Vivaldi, Vivaldi,
received
had
Universidade
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pioneering the development and delivery of therapies for rare diseases, the main thrust thrust diseases, the main rare for therapies ofdelivery and development pioneering the doctor
Vivaldi Vivaldi
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Pennsylvania she oversaw oversaw she In
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to initiatives
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regulation
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.
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held
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medical
Woodcock Woodcock
San . .
Director, CDER, Director, her Path” consumers Dr Dr management “Pharmaceutical modernize University at
director
based treatments for multiple sclerosis and cystic fibrosis in her position as director of the the of as director position her in fibrosis and cystic sclerosis multiple for treatments based as launched the “Safety First” and “Safe Use” initiatives designed to improve drug safety safety drug improve to designed Use” initiatives “Safe First”and “Safety launched the -
received
Bucknell California
CDER
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from
also
Woodcock
. Office of Therapeutics Research and Review in FDA’s Center for Biologics Evaluation and Research (CBER Research and Evaluation Biologics for Center FDA’s in Review and Research Therapeutics of Office management within and and within management biotechnology Why we chose Dr. Woodcock: Dr. chose we Why Woodcock Dr. Dr degree University She Janet Woodcock, Woodcock, Janet
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Thirty out of the 50 thought leaders in this report have actively participated at the World Orphan Drug Congress USA. The conference has become the annual global gathering for influencers in orphan drug development.
Join us as we come together next year at the World Orphan Drug Congress USA 2014 from April 23-
25 in Washington, DC.
Hear from pharma, biotechs, government, payers and patient groups as they discuss:
• How to access emerging markets around the world • How to harmonize regulatory, reimbursement and patient access globally • How to diagnose rare diseases early • How to articulate the value of orphan drugs • How to access, maintain and relate to rare disease patients more effectively • How to manufacture low volume orphan drugs and sustain supply • How to launch orphan drug products successfully
For more information, go to www.terrapinn.com/orphandrug
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