Orphan Drugs Summit 2015

17th – 18th September 2015, Carlsberg Museum & Business Centre, Copenhagen, Denmark

5th Edition

Find out more at www.orphandrugssummit.com@ PL

US Join us on:Orphan Drugs Executive Platform

Twitter: @PharmaDivision

a

event

Fostering the right relations and bringing the right discussions to collaboratively shape the future of orphan drugs

Partners

Editor’s note:Changing times ask for solid relationships.

Times are changing. A renewed interest from big pharma in the rare disease landscape has awakened due to large-scale patent expirations, competition from generics & biosimilars, anemic pipelines, escalating clinical trial costs and a global health-care reform. This means that the traditional blockbuster model has become less viable while the revenue-generating potential of orphan drugs has shown to be huge with a greater return on investment than non-orphan drugs. According to EvaluatePharma, the orphan drugs sales will grow at an annual rate of 11% and constitute 19% of the total share of prescription drugs by 2020, totalling 176 billion dollars.

The rare disease landscape is very complex due to the large amount of stakeholders involved. Despite their different interests they have one goal in common: getting an orphan drug approval that will help save or improve lives. But there are many challenges on the road to orphan drug designation:

• Complexandchangingnationalandregionalregulations

• Clinicaltrialdesignandfinding&keepingpatients

• Thelackofacentraldatabasedesignedspecificallytolistpatientregistries,which asks for close stakeholder engagement

• Partneringandestablishingfinancingforfuturedevelopment

• Establishingafoundationforpricethatisbalancedandsustainable

• Achievinganefficientandtimelyaccesstomarketwithequalaccessforpatients around the world

• Achievingtimelyandcorrectdiagnosistoenablehigherqualityoflifeandmoretime and information for developers

Changingenvironmentsaskforachangingconference.Withthehelpoftheaudiencewe have been able to map the complex orphan drugs ecosystem, resulting in a whole newconceptattheOrphanDrugsSummit2015.Thisconferenceisspecificallyde-signed to enable future stakeholder interactions, which is much needed. Each session bringstogetherdifferentgroupsofstakeholdersonspecificallyselectedtopicstohelpthembuildrelationshipsandreachtheirgoals.Withtherightrelationsandtherightdiscussions we can help shape the future of healthcare by collaboration.

Flip the pages to see your ecosystem map and what sessions are specially designed for you. Start building your solid relationships right here, right now!

Welcometothe5th annual Orphan Drugs Summit.

Sincerely,

Maaike Gerritse EditorialContentDirector,OrphanDrugsSummit

The Advisory Board is instrumental in setting the tone and direction of the event, thanks to the accumulated years of experience of all the members within the Orphan Drugs

community.

Advisory Board - 2015

Robert Derham Founder & President

CheckOrphan

Anders Waas CEO

Tikomed

Marlene E. Haffner MD, MPH, CEO, Haffner

Associates, LLC (former Director of OOPD at US

FDA)

Dr. Hoss A Dowlat Vice President

Regulatory Affairs, Global Strategy

PharmaBio Consulting

Donald Macarthur Principal

Justpharmareports

Elizabeth Vroom Founder and President

Duchenne Parent Project

Conferenceataglance

12:00 Registration opens

12:45Chairmanopenstheconference

13:00 Plenum session


14:30 Debate Debate Panel

15:00 Networking break & 1-to-1 meetings

15:30 Debate CaseStudy

16:00 Round table Masterclass Round table



18:00 Networking cocktail

19:00 Networking dinner

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11:00 Focus session Round table Research Discussion

11:30 Masterclass Masterclass

12:00 Debate Roundtable Roundtable

12:30 Networking lunch

13:30 Roundtable Casestudy Paneldiscussion

14:00 Masterclass Research Discussion Debate

14:30 Masterclass Debate




17:00Chairmanclosestheconference

17:15 One for the road

Day 2

Day 1

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08:45Chairmanopensday2

Summit conceptTheOrphanDrugsSummit2015istailoredaccordingtostakeholderneeds.WiththehelpofCopperbergsOrphanDrugsSurveywehavebeenabletospecificallydesignthisconferencetobringtherightstakeholderstogethertodiscusscriticaltopics.Thesurvey report can be downloaded via www.orphandrugssummit.com. All stakeholders are visualised with the use of icons. All sessionsarecodedaccordingtotheseiconstoshowyouwhatsessionsarespecificallydesignedforyou.

Take a look at the agenda and see what we have in store for you!

Regulatory bodyPayer

Drug developer

Hospital representative Researcher

Industry association

Patient organisation

Venture capitalist

Are you ready to enter

The shark tank is a platform that brings successful drug developers, patient organisations or industry associations together with venture capitalists - sharks.

Whetheryouhaveabreakthroughresearch,afaradvancedclinicaltrialoralreadyoperatingsuccessfullyandlookingtoexpandandcouldusefinancialbacking,thesharktankistheplatformforyou.

Wearelookingfordrug developers, patient organisations or industry associations who can pitch their break-through business concepts, products, properties and services to our sharks in the hopes of receiving investment funds. If selected, our sharks could be willing to part with their resources to give you the funding you need.

You can apply soon for your chance to enter the shark tank and see if your business is ready for a shark investment. For more information send an email to [email protected]

Nanna LüneborgInvestment Director

Novo A/S

Rob Hopfner Managing Director, BayCityCapital

John Doux Analyst/Investor

Palo Alto Investors

Book 30 minutes with one of our selected consultants and get your

burning questions answered!

What do you do when you get sick? It is highly likely you go to a doctor and ask for advice. Why don’t we do the same for our business?

That is why we are organising a consultation hour for your business, where expert industry consultants will act as the doctor to help cure your business. You get the possibility to book 30minuteswithoneofourhighlyselecteddoctorstoaskthemyourburningquestions.

How healthy is your organisation?

Consultation Corner

I could use some advice...

Now it is time for answers!

Henrik Balle Boysen, Executive Director, HAEiHenrikBalleBoysenistheExecutiveDirectorofHAEi–InternationalPatientOrganizationforC1InhibitorDeficiencies(www.haei.org)–andPresidentofthePatientAssociationforHAEinDenmark,NorwayandSweden(HAEScandinavia).HeassumedhispositionastheHAEi’sExecutiveDirectoron1September2009afterspending5yearsonitsExecutiveCommittee.Henrikhasabackgroundinsalesand marketing and has held management positions in several global corporations. Since 2000, most of Henrik’s spare time has involved hereditaryangioedema.HewasdiagnosedwithHAE(typeI)attheageof17–morethan10yearsafterhissymptomsfirstappeared.

Ana Mingorance Le Meur, Scientific Director, Dravet FoundationDrAnaMingoranceistheScientificDirectoroftheDravetSyndromeFoundationinSpain,anon-profitpatientorganizationfocusedon running and promoting research on Dravet syndrome and related neurological rare diseases. In this role, she is responsible for long-term research strategy and for building and managing a portfolio of research collaborations ranging from diagnostics to clinical research. Ana has prior experience in neuroscience and drug discovery from both industry and academia, as a lab head and discoveryprojectleaderattheglobalpharmaceuticalcompanyUCBandtherecipientofmultipleawardsforherresearchattheUniversitiesofBritishColumbiainCanadaandBarcelonainSpain,wheresheobtainedherPhD.Sheisalsoanindependentconsultanttouniversities,companiesandpatientorganizationsintheareasofneuroscienceandrarediseasesviahercompanyDracaenaConsulting.

Jean Nordstrom, CEO, Sixera PharmaIndustrialist and seasoned manager with 40 years of International Management positions in several Industry sectors. Last 25 years in Pharmaceutical and Biotech. Thorough experience from managing companies with substantial context in development and R&D. HaveworkedwithlargecompaniesaswellasSMEs.FormerExecutivePresidentofPharmacíaSpainandmemberoftheCorporateManagementgroup,VPCorporateandBusinessDevelopmentatArexisAB,SwedishBiotechCompany.CEOofLabianaGroup,SpanishCMOandproducerofownproductsforhumanandanimalhealth.Accustomedtoworkwithcompanieswithfinancialownersaswellas industrial partners. Mr. Nordstrom has in parallel to his employments as executive also carried out various assignments as advisor in strategy and development issues. Since 10 years special interest in developing Orphan Drugs. Sixera Pharma is now main focus and Jean Nordstrom is also co-founder and shareholder of the company.

Segolene Ayme, Emeritus Director of Research, INSERMSégolèneAyméisEmeritusDirectorofResearchattheFrenchInstituteofHealthandMedicalResearch(INSERM).Shededicatedher professional life to the development of tools and innovative practices to improve the diagnosis and management of rare diseases, theaccesstoexpertservicesandthedevelopmentofappropriatepoliciesatnationalandinternationallevel.Shewasthefirstpresident of the International Federation of Human Genetics Societies in 1996. She chaired from 1998 until 2007 the Public and ProfessionalPolicyCommitteeoftheEuropeanSocietyofHumanGenetics.ShewasthefounderofOrphanetin1997andExecutiveManagerupto2011.ShechairedtheEUCommitteeofExpertsonrareDiseasesfrom2010to2013andisnowamemberoftheCommissionExpertGrouponRarediseases.ShealsochairstheWHOTopicAdvisoryGroupforRareDiseasesandservesasEditor-in-ChiefoftheOrphanetJournalofRareDiseases(www.ojrd.com).Sheistheprojectleaderof“SupportIRDiRC”,whichprovidestheservicesofascientificsecretariattotheInternationalRareDiseasesResearchConsortium(www.irdirc.org).

Josie Godfrey, Associate Director – Highly Specialised Technologies, NICEJosieGodfreyisAssociateDirectoratNICEwithresponsibilityforthenewHighlySpecialisedTechnologiesprogramme.Sheisleadingwork to establish programme which will make recommendation to the NHS in England about the use of new highly specialised technologies. BeforejoiningNICE,JosieledworktoestablishthenewAdvisoryGroupforNationalSpecialisedServices(AGNSS).Shedevelopedadecision-making framework to support AGNSS in making these recommendations and developed the process for considering highly specialised services,drugs and technologies. Josie has worked in health policy development and implementation for the NHS in England and national and local government.

Nanna Lüneborg, Investment Director, Novo A/S Nanna is Investment Director of Novo Seeds, the early stage investment arm of Novo A/S. Novo Seeds is a leading early stage investor, actively creating new biotech companies primarily in Scandinavia through seed and venture investments. Nanna joined Novo A/S in 2012.Priortothis,sheworkedforAppositeCapital,asuccessfulLondon-basedhealthcareventurefund,whereshewasresponsiblefordiligence of new investment opportunities and involved in all aspects of investment activities, deal structuring and portfolio management of companiesindrugdevelopment,medicaldevicesandhealthcareservices.Earlierinhercareer,sheworkedatCancerResearchUKasaresearchmanager.NannacompletedundergraduatestudiesatUniversityofOxford,aPhDinNeuroscienceatUniversityCollegeLondon,andMBAatUniversityofCambridge.ShecurrentlyservesontheboardofDirectorsofAffinicon,Glionova,IOBiotech,MinervaxandPcovery,andsheisanobserver on the board of Galecto.

SIXERA

Speakers:

Meriem Bouslouk, Desk Officer Pharmaceuticals Department, Federal Joint Committee (G-ba) GermanyMeriemBouslouk(PhDindentistry,MScInternationalHealth)joinedthePharmaceuticalsdepartmentoftheFederalJointCommittee(G-BA)in2011,andhasworkedontheimplementationofAMNOG,theGermanActontheReformoftheMarketforMedicinalProducts,from the start. Her main responsibilities are conducting consultations with industry representatives on clinical study design and appropriate comparators,assessingreportsbytheInstituteforQualityandEfficiencyinHealthCare(IQWiG),anddraftingG-BAresolutionsunderAMNOG(withanemphasisonorphandrugs)whichbecomeamendmentstotheGermanPharmaceuticalDirective.Meriemworkscloselywith the voting members of the G-BA and numerous other stakeholders.

Lise Aagaard, Professor,Institute of Public Health, University of Southern DenmarkDr. Aagaard is professor of clinical pharmacy at the University of Southern Denmark. She has been working with different aspects of medicine use and health policy, and conducted several studies on medicine use in children and adolescents. She graduated as a pharmacist (Mscpharm)in2001,andin2008sheearnedherPhDdegreeforanalysesofhowknowledgeaboutadversedrugreactionscanbeimproved.From2004to2008shewasemployedasapharmacovigilanceofficerattheDanishMedicinesAgency.DuringtheperiodintheDanish Medicines she also attended monthly meetings in the European Medicines Agency representing Denmark in the Pharmacovigilance WorkingParty.

Flóra Raffai, Head of Development, FindacureFlóraRaffaiistheHeadofDevelopmentatFindacure,aUKcharitybuildingtherarediseasecommunitytodriveresearchanddeveloptreatments.ShewasthefirsthireatFindacure,developingthecharity’sprojects,funding,andcommunity.FlóraorganisesFindacure’spatientgroupempowermentprogrammes,runsscientificcommunityengagementprojects,developsthecharitystrategy,andoverseesmajor funding applications and online communications. She also line manages other members of staff. Flóra graduated from the London SchoolofEconomicsandPoliticalSciencewithaBSc(hons)inInternationalRelations.SheisafoundingmemberoftheCambridgeRareDiseasesNetwork,anot-for-profitorganizationbringingtogetheractivestakeholdersinrarediseaseresearchanddevelopment.FlóraisalsoamemberoftheCambridgeWomen’sResourceCentre’sfundraisingcommitteeandvolunteersasCo-OrganiserfortheCambridgeChapterofGoodforNothing.

Speakers:

Tim Kanters, Researcher, Erasmus University Rotterdam Institute for Medical Technology Assessment (iMTA)TimKantershasbeeninvolvedinorphandrugresearchsince2008.Heperformedcost-effectivenessstudiesforabroadrangeoforphandrugs,mostnotablyalglucosidasealfainPompedisease,forsubmissiontotheHealthCareInsuranceBoardintheNetherlands.Furthermore, he is performing a methodological study regarding the appropriateness of guidelines for health technology assessment to support decision making on orphan drugs.

Rob Hopfner RPh, PhD, MBA, Managing Director, Bay City Capital RobHopfner,RPh,PhD,MBA,isaManagingDirectorofBayCityCapitalandhasbeenwiththefirmsince2002.Dr.Hopfner’sworkatBayCityhasincludeddiscoveryscience,productdevelopment,andcommercialandbusinessdevelopmentprojects.BeforejoiningBayCityCapital,Dr.HopfnerworkedinDuPontPharmaceuticals’BusinessDevelopment&StrategicPlanninggroupandatAg-WestBiotech,aWesternCanadianseed-stagebiotechventurecapitalfirm.Dr.HopfnerholdsaPhDinPharmacologyandadegreeinPharmacyfromtheUniversityofSaskatchewan,andanMBAfromtheUniversityofChicagoBoothSchoolofBusiness.His biomedical research focused on endocrine pharmacology and he completed his post-doctoral work at Harvard Medical School. Dr.Hopfner’snumerousawardsincludetheGovernorGeneralofCanadaGoldMedalAward,andhehaspublishedseveralarticlesintop medical journals based on his work. Dr. Hopfner started his career as a pharmacist.

Dr Robert Ryan, President & CEO, Scioderm Dr.RobertRyanCo-FoundedScioderm,LLCwithmorethan27yearsofresearch,pharmaceuticalandbiotechexperience,spanning the global development process from preclinical through Phase IV. Dr. Ryan previously held senior regulatory positions atthreeofthetop5leadingCRO’s,PPD,INCResearch,andQuintileswherehealsoservedastheChiefRegulatoryOfficerforQuintile’s very successful PharmaBio division, leading the due diligence teams to a 100% track record in successful investments of approximately $800 million in various biotech/pharmaceutical companies. Dr. Ryan’s diverse pharmaceutical career covered seniorpreclinical,regulatoryandclinicalactivitieswithRoche,Bristol-MyersSquibb(BMS),UCB,Atherogenics,andPfizer.Dr.RyangraduatedwithadoctoratedegreeinToxicologyfromtheUniversityofNorthCarolinaatChapelHill,andisboardcertifiedin Toxicology. In addition, Dr. Ryan has advanced degrees in Genetics and Pharmacokinetics.

Jimmy Cheng-Ho Lin, MD, PhD, MHS, President, Rare Genomics InstituteJimmy Lin, MD, PhD, MHS, is a 2012 TED Fellow and Founder & President of Rare Genomics Institute, the world’s first platform to enable any community to leverage cutting- edge biotechnology to advance understanding of any rare disease. Partnering with top medical institutions, RGI helpscustomdesignpersonalizedresearchprojectsforrarediseases.Dr.LinisalsotheDirectorofClinicalGenomicsattheGeneticsBranchoftheNationalInstituteofHealth/NationalCancerInstitute(NIH/NCI).Priortothis,heledthecomputationalanalysisofthefirsteverexomesequencingstudiesforanyhumandiseaseatJohnsHopkinsandwasaresearchinstructoratWashingtonUniversityinSt.Louis.HehasnumerouspublicationsinScience,Nature,Cell,NatureGenetics,andNatureBiotechnology,andhasbeenfeaturedinForbes,Bloomberg,WallStreetJournal,WashingtonPost, BBC,TIME,andtheHuffingtonPost.

Blair van Brunt, Founder, Rare Disease Perspectives

Kate Bushby, Professor of Neuromuscular Genetics Newcastle University John Walton Centre for Muscular Dystrophy Research, MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, International Centre for LifeProf.KateBushbyMDFRCPisaProfessorofNeuromuscularGenetics.SheisaclinicalacademicwithjointappointmentsbetweenNewcastleUniversityandtheNHS.TheMuscleTeamwithintheInstituteofGeneticMedicineisapartnerintheMRCCentreforNeuromuscularDiseases,of which Prof Bushby is Deputy Director.Professor Bushby has a long-standing interest in inherited neuromuscular diseases, especially Duchenne muscular dystrophy and the molecular genetics of the limb-girdle muscular dystrophies and related disorders. She has also been leading on the best possible development, dissemination and implementation of care guidelines as well as clinical trials. The 70 strong muscle team at Newcastle has developed an extensive programme of research in NMD from basic molecular pathology to clinical studies.Along with Volker Straub, Professor Bushby was one of the founding co-ordinators of the TREAT-NMDNetworkofExcellenceandremainsamemberofitsscientificsecretariat.SheisamemberoftheEuropeanExpertGrouponRareDiseasesandleadstheJointActionsupportingthisgroup.SheisImpactworkpackageleaderinRDConnectandInternationalPIontheNIHfundedinternationalstudyofsteroiduseinDMDand the Jain foundation study of clinical outcome measures in dysferlinopathy as well as an investigator in several industry sponsored clinical trials in DMD.

Ghassan Karam, Technical Officer, International Clinical Trials Registry Platform - World Health OrganizationGhassanhasbeenmanagingtheInternationalClinicalTrialsRegistryPlatform(ICTRP)intheWorldHealthOrganizationsince2011. He is an expert in clinical trial design,, clinical trials registration and ethics committees. He has many years of professional experienceintheprivatesectorandwithindifferentareasofworkintheWorldHealthOrganization.GhassanhasaBSinComputerScience, PG Diploma in International Management and MSc candidate with the University of London.

Birthe Byskov Holm, President,Rare Diseases Denmark / EurordisBirthe has a long history of active participation in patient organizations. She became involved in the field of rare diseases because her now grown up son was born with the rare disease Osteogenesis Imperfecta. Birthe has been a member of the Danish OI Association since 1983 where she has acted aspresidentandsheisalsotheco-founderandcurrentlythepresidentoftheDanishrarediseasealliance(RareDiseasesDenmark).BirthehasuntilJune2015beenaEurordisnominatedpatientrepresentativefor12yearsatthecommitteeoforphanmedicinalproducts(COMP)oftheEuropeanMedicinesAgency(EMA)wheresheheldthepositionofvicechairuntil2012.SheiscurrentlymemberoftheEurordisboardofdirectorssince2012.

Alexander D. Crawford, CEO, Theracule

Speakers:

John Doux, Analyst/Investor, Palo Alto Investors John Doux, MD obtained a B.S. with distinction and an MD from Stanford University, where he was a Howard Hughes Medical Institute Fellow.AfteraninternshipatBrighamandWomen’sHospitalinBoston,hereturnedtoStanfordtocompletehisresidencytrainingindermatology.HealsocompletedanMBAattheWhartonSchoolofBusinesswherehewasaPalmerScholar.Heisboardcertifiedindermatology and maintains an active clinical practice. Since 2004 he has also served as an analyst at Palo Alto Investors, an investment fund specializing in healthcare with $2B in assets under management, with a historical focus in therapeutics for rare diseases.

Mark Corbett, Senior Vice President, Clinigen Global Access Programs (GAP) – Clinigen MarkCorbettjoinedClinigeninmid2010asVicePresidentClinigenGlobalAccessProgramswithoverallresponsibilityforClinigen’sGlobalAccessProgramsDivisionwhichspecialisesintheconsultancy,development,setupandimplementationofaccess programs on behalf of the Biotech and Pharmaceutical industries. Over the last 6 years Mark has specialised in the area ofNamedPatient/EarlyAccessandCompassionateUsePrograms.PriortojoiningClinigen,MarkwastheHeadofEuropeanBusinessDevelopmentandaGlobalAccountDirectoratthemarketleaderinthefieldofNamedPatientPrograms.Withinthis role Mark was responsible for overseeing all new business opportunities for the provision of services to the pharmaceutical industryoriginatingthroughtheglobalheadquarters.Markhasgainedextensivespecialistknowledgeandoperationalexpertisein implementation of over 100 Global Early Access / Named Patient Programs for a variety of companies ranging from niche Biotech to Large Top 10Pharma.MarkiswidelyrecognisedasathoughtleaderonthesubjectofEarlyAccess/CompassionateUse/NamedPatientProgramshavingspoken at a number of industry congresses and workshops over the last 6 years. Prior to specialising in the area of Early Access and Named Patient Programs, Mark spent 13 years within the pharmaceutical industry in a number of sales, training and marketing roles with Servier Laboratories Ltd. Latterly, he had overriding responsibility for the launch of an innovative novel product to the market during which he developed his interest in and experience in the complexities of market access to innovative medicines across Europe.

CallforpapersThe Orphan Drugs Summit organizing team aim to change the way conference agendas look. The goal for the 5th Annual Orphan Drugs Summit agenda is to provide great experience based on premium content. As such, we are currently looking for interesting papers on various topics in the orphan drugs field that will be published in the digital and printed agenda.

We consider five different types of articles for publication in the orphan drugs summit agenda:

• Futureinsights

• Opinionpieces

• Interviews

The author of the article agrees that the work he/she is submitting meets the appropriate criteria and does not infringe upon any copyright or intellectual property laws. All submitted articles are first screened by the editor-in-chief. Articles should not exceed 1000 words. Pictures and graphs are welcomed.

If interested please contact [email protected]

• Academic

• CaseStudies

Gülce Belgin, Founder & Director, ProceuticaMrsBelginholdsaB.ScdegreeinChemistryfromBogaziçiUniversityandanM.B.AfromKoçUniversity,Istanbul.SincecompletingherMBAshe worked as a consultant to pharmaceutical industry for the last 13 years in Turkey. During this period she developed special interest in Orphan Drugs. Gülce’s entrepreneurial experience already includes successfully founding and running a business consultancy company since 2008. She is the founder and Director of Proceutica, a company based in Istanbul providing market access services to global Orphan Drug companies.Gülce’sexperienceintheareaofNamedPatient/EarlyAccessandCompassionateUseProgramsenablespatientstohaveaccessto therapies as early as possible through existing and innovative early access mechanisms.

Eva Rye Rasmussen, Specialty registrar of Oto-Rhoni-Laryngology – Head and Neck Surgery, University hospital of CopenhagenDr.RasmussenisamemberoftheDanishresearchgrouponBradyKinin-mediatedAngioedema(DABKA)whichperformsepidemiologicalandbasalresearchwithinthefieldsofhereditaryandacquiredangioedemaincludingangiotensinconvertingenzyme-inhibitor and angiotensin II antagonist mediated angioedema. She has published a number of papers on the topic in various national and international medical journals. Furthermore she is currently taking part in an international multicentre study regardingantihypertensivedrugsandangioedemainco-operationwiththeEUsponsoredPREDICTION-ADRgroup.Furthermoresheiseducatingstaff at the emergency departments, internal medicine, intensive care units and otolaryngology department in the assessment and management of angioedema including hereditary angioedema.

Speakers:

n 12:00 Registration opens

n 12:45Chairmanopenstheconference

Program:

Day 1

Consultingcorner:

Meet the industry’s best consultants and book

your 30 minutes of qualityadvice!

Theagendaiscodedwiththeuseofsymbolstoshowyouwhatsessionsarespecificallydesignedforyou.Thebigsymbolsshowwhowillbespeaking during a certain session. The small symbols show who should be attending a certain session. So remember your symbol and see what this agenda has in store for you!

PAYERS HOSPITAL REPRESENTATIVES

PATIENT ORGANISATIONS

VENTURE CAPITALISTS

REGULATORY BODY

DRUG DEVELOPERS

RESEARCHERS INDUSTRY ASSOCIATIONS

n 13:00 Regulatory market update: what are the major changes and differences worldwide?The regulatory landscape of orphan drugs imposes a lot of challenges and has shown a great deal of complexity. Not only are the regulations different across borders, making it extremely difficult to market your drugs in different countries, they are also changing quite rapidly. That is why we will start this conference with a regulatory market update, providing you with insights in the recent changes and differences across borders.

Learning points:

•Regulatorychanges

•Differencesinregulations across borders

+

Marlene E. Haffner MD, MPH, CEO, Haffner Associates, LLC (former Director of OOPD at US FDA)

n 13:45Casestudy:Anapprovedorsoon-to-beapprovedorphandrug-Howdidtheydoit?The road to an approved orphan drug is long, challenging and costly. There are many challenges concerning regulations, clinical trial design, finding and keeping patients, and let’s not forget the financing. In this session we will present a case study of a success story and share the best practices and lessons learned from the road to drug designation.

Learning points:

•Bestpracticesofanorphan drug designation success story

•Lessonslearnedontheroadto designation

+


Program:

Consultingcorner:



n 14:30 Debate: regulation on cross border healthcare: how to overcome this? Different countries, different regulations, but the same disease. Rightnowthecountryyouliveinmight decide whether or not you have access to a drug that saves, or improve your life. The access to drugs suffers from inequality due to the different regulations across borders. It makes it very difficult for drug developers to market their drugs and reach all potential patients.Butitisnotthatsimpletoovercome this issue, since there are many parties involved. The question is: how can we overcome this?

n 14:30 Debate: Affordability & maintainability: socially re-sponsible pricing and ensuring patient access to treatmentsEstablishing a foundation for price that is balanced and sustainable while satisfying multiple parties with different interests is a big challenge. Drug development is very costly, but is the patient the one that has to pay the price with the risk of them not being able to access the medication they need?

n 14:30 Panel: Is global colla-boration the answer to reaching all potential patients? Some diseases are so rare that they only affect a handful of people in a certain country. With the lack of a central database designed specifically to list patient registries it is very hard to detect patients that suffer from the same disease. The questions is if global collabora-tion is the right answer to reaching patients and developing and identifying new treatments?

Learning points:

•Howcanwedealwithdifferent regulations across borders?

•Howdothesedifferences influence patient’s access

+ Learning points:

•Foundationmechanismsfor pricing

•Ensuringpatientaccessto treatments

+

Learning points:

•Isglobalcollaborationthe right answer?

•Developing&identifyingnew treatments through global collaboration

+

n 15:30 Debate: Problems with reimbursement andeconomicburdenofACEinhibitorinducedangioedemaThis session will be about reimbursement in patients suffering from bradykinin mediated angioedema due to antihypertensive (ACE inhibitors) and anti-diabetic drugs (DPP-IV inhibitors). These patients are “shared” between emergency medicine, oto-rhino-laryngology, internal medicine and intensive care units, but who are to pay for the expensive medications?

n 15:30Casestudy:Buildingapatientregistry - lessons learned Buildingapatientregistryisabigchallenge.Astheadvocate for a rare disease I realised this and learned the hard way. In this case study I will share my story on building a patient registry and share my best practices and lessons learned.

Blair van Brunt, Founder, Rare Disease Perspectives

Learning points:

•IcatibantinACEinhibitorinducedangioedema

•“Shared”care:whoispaying?

•Howarereimbursementmanagedinother counties?

+

Learning points:

•Bestpracticesonbuildingapatientregistry

•Lessonslearnedfrombuildingapatientregistry

+

Gülce Belgin, Founder & Director, Proceutica

Eva Rye Rasmussen, Specialty registrar of Oto-Rhoni-Laryngology – Head and Neck Surgery, University hospital of Copenhagen

Program:

Consultingcorner:



n 16:00Roundtable:Changingregulatory frameworks for better reimbursement strategiesSetting the right reimbursement strategy that satisfies all parties is easier said than done. With the complex regulatory framework on orphan drug designation it is the question if we can make some changes to improve reimbursement strategy.

n 16:00 Masterclass: International clinical trials registry platformThe International Clinical Trials RegistryPlatform(ICTRP)isaglobalinitiative that aims to make informa-tion about all clinical trials involving human beings publicly available. It was established in 2006 in response to demand from countries through theWorldHealthAssemblyresolutionthat called for “a voluntary platform to link clinical trials registers in order to ensure a single point of access and the unambiguous identification of trials with a view to enhancing access to information by patients, families, patient groups and others”. In this session Ghassan will explain more about the International Clinical Trials RegistryPlatform.

n 16:00Roundtable:Collabora-tivelyfightingforpatientrights:equalaccessforeveryoneAs a patient organisation you are constantly trying to fight for patient rights. The country you are living in, the type of insurance you have, the doctor you are meeting etc should not be factors preventing you from getting the best care and accesstotreatments.Buthowcan we establish equal access for everyone?

Learning points:

•Regulatoryframeworks influencing reimbursement

•settingreimbursement strategies

+

Learning points:

•TheInternationalClinical TrialsRegistryPlatform

•Linkingclinicaltrialsregisters to have a single point of access

+

Learning points:

•Patientrights

•Establishingequalaccess

+

Henrik Balle Boysen, Executive Director, HAEi

n 16:30 Pricing of orphan drugs: Establishing a reimbursement foundationPricing of orphan drugs is a delicate question, since it needs to be balanced and sustainable. Establishing the right reimbursement foundation that satisfies the developer whilst enables equal access for patients is easier said than done. It asks for close collaboration between different stakeholders.

Learning points:

•Howtoestablishafoundation for reimbursement

•Howtopriceorphandrugsina way that is balanced and sustainable

+

n 17:15 Early Access to Orphan Drugs: The evolving changing environment and additional benefitsofEarlyAccess

Learning points:

•Understandthechangesintheregulatoryenvironment,includingUKEAMSandCureActintheUSA

•Learnaboutthepracticalconsiderationsforprovidingearlyaccess

•Patientsandpatientsgroupsroleinearlyaccess

•CananEarlyaccessprogramprovideadditionalRealWorldEvidenceforOrphanDrugs

+

Ghassan Karam, Technical Officer, International Clinical Trials Registry Platform - World Health Organization

Mark Corbett, Senior Vice President, Clinigen Global Access Programs (GAP) – Clinigen

18:00NetworkingCocktail

19.00 Networking Dinner

Big announcement coming soon:

Stay tuned for our amazing venue!

September 17th 2015

WELCOMETO THESOCIALCLUB

18:00Copenhagen

Program:

Consulting corner:

Meet the industry’s

best consultants and book your 30

minutes of quality advice!

Shark tank: Apply to

pitch venture

capitalists!

n 08:45Chairmanopensday2oftheconference

Day 2

n 09:00TheCathedralandtheBazaar:OrphanDrugsandtheGenomicRevolutionBottom-upcollaborativeinfrastractureshaverevoluntionizedinformation technology; the same is happening in genomics and rare diseases. Parterning with patient advocacy groups, biotechnology companies,pharmaceuticalcompanies,andacademic,weatRareGenomics Institute are building systems for crowdsourcing knowledge, funding, genomic information, biospecimen and other and to contribute to the bigger revolution that is underway for orphan drugs.

Learning points:

•Rarediseasesfollowtheclassic long tail distribution and thus require an alternative paradigm from the traditional blockbuster drug approach

•Thesharpdeclineingenome sequencing prices has demo- cratized the technology and has allowed a distributed collaborative mechanism of data generation and sharing

•AtRareGenomicsInstitute, we empowering patients by connecting them with researchers, doctors, industry, and funders and provide them with all the tools and connections to advance understanding for their research. New disease are being discovered using this approach

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Jimmy Cheng-Ho Lin, MD, PhD, MHS, President, Rare Genomics Institute

n 09:45 The voice of patients living with rare diseases in Europe In Europe alone there are about 30 million people living with a rare disease. Therefore there is the need to build a European community of patient organisations and people living with rare diseases at a European level.

Learning points:

•BuildingastrongEuropean community

•Unitingpatientorganisations and people living with rare diseases

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Birthe Holm, President, Rare Diseases Denmark, Board member EURORDIS


Program:


pitch venture

capitalists!

n 11:00 Research on diagnostics: How to make diagnostics easier, more correct and faster? The earlier you correctly diagnose people, the earlier you can link them to the right specialists and treatmentpossibilities.Butmakingthe right diagnosis, especially on a rare disease can be very hard since the symptoms can vary from patient to patient. Can research on diagnostics help us to make diagnostics easier, more correct and faster?

n 11:00 Round table: Aligning regulations across borders: minimizing regulatory gaps & differencesRegulationsdifferacrossborders.In order to achieve equal access for patients we must minimize regulatory gaps and differences betweencountries.Butthisasksfor very close collaboration and political involvement. The questions is how we can achieve this?

n 11:00 Research discussion: budget impact of orphan drugs in the NetherlandsIn this study the number of orphan drugs, number of patients using them and the annual budget impact of orphan drugs in the Netherlands over a six-year period is researched. What is found is that the budget impact has grown both in absolute terms as well as relative to total pharmaceutical spending. TimKanterswhoconductedthisstudy, will discuss the research and its results.

Learning points:

•Researchondiagnostics

•Enablingfaster,morecorrect and easier diagnostics

+ Learning points:

•Minimizingregulatorygaps

•Achievingcollaboration

+ Learning points:

•Budgetimpactoforphan drugs in the Netherlands

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Tim Kanters, Researcher, Erasmus University Institute for Medical Technology Assessment (iMTA)

n 11:30Masterclass:Crowdfunding: NewstrategyforfinancingclinicalresearchThis master class will introduce case studies where crowdfunding has successfully funded clinical research, such as observational studies and clinical trials. The interactive session will then delve into the key steps you will need to run a successful campaign, ranging from building a supporter base to active donation generation.

n 11:30 Masterclass: Establishing comprehensive pricing schemes to be accepted by payers Establishing a pricing scheme for orphan drugs is hard. It has to satisfy many different requirements, but mostimportantlyithastobeacceptedbypayers.Butwhatisitthatpayersfindacceptable?Howcanyouset up a pricing scheme they will accept?

Learning points:

•Crowdfundingasaformofsocialfinancing

•Thebenefitsassociatedwithrunningacrowd- funding campaign to support clinical research

•Thestrategicstepsinvolvedindevelopinga successful campaign

+ Learning points:

•Settinguppricingschemes

•Whataretherequirementsforpayers?

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Flóra Raffai, Head of Development, Findacure: The Fundamental Diseases Partnership

Consulting corner:




TBA, MME

Program:

Consulting corner:





pitch venture

capitalists!

n 13:30 Round table: Orphan drugs in emerging markets: how to ensure market access? Demand for access to orphan drugs which are not yet com-mercially available is often high, from both patients and physicians. What are the practical consider-ations for a manufacturer? This round table will focus on the prac-ticalities, challenges and potential benefits of early access. Different perspectives from the group will be discussed and some practical examples will be shared.

n 13:30Casestudy:Thepatientas a partner: lessons learnt from an impatient patient organizationFrombasicdiseaseunderstandingtopreclinical models and clinical trials, working with rare diseases poses par-ticular challenges for drug developers. Patient organizations can play a major role in identifying these challenges and partnering with academia and industry to solve them. This session covers some of the misconceptions around the role of patient organizations in promoting drug development for their disease, structured around the lessons learnt by a young patient organization.

n 13:30 Panel Discussion: How do they judge at the EMA? - Advocacy for approvalIn this panel discussion we will have expert panelists representing different countries to discuss a cross country view on drug ap-proval. There are many questions abouthowtheyjudgeattheEMAand how we can advocate for drug approval.

Learning points:

•Demandinemergingmarkets

•Regulatoryandpricing considerations

•Wheredopatient organisations fit

+Learning points:

•Whataresomeofthemain misconceptions around the role of patient organizations in drug development.

•Theconceptofimpatient patients: why patient organizations can and should play a major role in orphan drug development

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Learning points:

•Howdotheyjudgeatthe EMA?

•Howcanweadvocate approval?

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Meriem Bouslouk, Desk Officer Pharmaceuticals Department, Federal Joint Committee (G-ba)

Josie Godfrey, Associate Director - Highly Specialised Technologies, NICE

Jean Nordstrom, CEO, Sixera Pharma

SIXERA

Ana Mingorance-Le Meur, PhD, Scientific Director, Dravet Syndrome Foundation Spain

n 12:00 Debate: Strategies for regulatory fast tracks and global market access Mostrarediseasesareseriouscondi-tions with a high unmet medical need. In order to speed up development there is a demand for regulatory fast tracks.Butthisdoesnotcomewithoutastrategy.Butwhatarethesestrate-gies?Howcanyouworktogetherwiththe regulatory bodies to secure global market access?

n 12:00 Round table: Research design: can we optimise resources by new methodologies?Researchdesignhasahugeimpact on it’s costs and outcome. The question is whether we can optimize our resources by changing our research design. Are certain methodologies more resource effective?

n 12:00 Round table: Patient diagnostics & patient care: choosing the right treatment more timelyAchieving an earlier correct diag-noses and empowering patients will enable a higher quality of life. The question is how we can achieve earlier correct diagnosis and to what extent patients can be empowered?

Learning points:

•Whatarestrategiesfor regulatory fast tracks?

•Howcanyouworktogether with regulatory bodies?

+ Learning points:

•Researchdesignasawayof cutting costs

•Otherresourcestobe optimized by research design

+ Learning points:

•Thelimitsofpatient empowerment

•Howtoachieveearliercorrect diagnosis?

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12:30 Networking lunch

Mark Corbett, Senior Vice President, Clinigen Global Access Programs (GAP) – Clinigen

Program:n 14:00 Masterclass: Trial de-sign: how to make it regulatory acceptable and cost effective? Clinical trial design is subject to many different requirements. It should follow the rules that are established by the regulatory bodies, but it should also be cost effective. The question is how you can save costs by optimising your clinical trial design and still make it regulatory acceptable?

n 14:00 Research discussion: SafetyissuesfromuseofC1-inhibitors and bradykinin receptor antagonist in treat-mentofangioedema”Long-termsafetyaspectsfromuseof orphan drugs (C1-inibitors and bradykinin receptor antagonists) in treatment of hereditary angioedema are scarce. The lack of sufficient knowledge about adverse drug reac-tions(ADRs)atthetimeoflicensingmakesspontaneousADRreportsan important source of information. Since 2012 researchers were allowed toaccesstheEUADRdatabase,EudraVigilance (EV) and this has opened for cross-sectional analysis based on standardized reporting format.InthissessionADRsreportedto the EV database by healthcare professionals,ADRreportsfromcon-sumersandADRinformationfoundin the literature are being presented.

n 14:00 Debate: how to keep research costs down? Researchcostscansignificantlyinfluence development costs of orphan drugs, leading to higher costs for the developers, payers and patients. The question is how you can cut research costs and manage them. In this session we will debate the ways to keep research costs down.

Learning points:

•Optimizingcostsofclinical trial design

•Understandingwhatis regulatory acceptable

+ Learning points:

•Managingresearchcosts

•Findingwaystokeepthecosts down

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Lise Aagaard, Professor, Institute of Public Health, University of Southern Denmark


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capitalists!

Consulting corner:




n 14:30 Masterclass: The role of registries and networks in post marketing surveillance programmes for rare disease drugs InthisMasterclassKateBushby,whowontheEURORDISScientific Award 2015, will discuss the role of registries and networks in post marketing surveillance programmes for rare disease drugs. Active postmarketing surveillance of drug adverse effects is essential because side effects of drugs can’t be anticipated based on pre-approval studies.

n 14:30 Debate: Engaging policy makers & driving clinical trial development The huge amount of policies slow down clinical trial development.Byengagingpolicymakers,clinicaldevelopmentcanbestimulatedandspeededup.Buthow can we engage policy makers? In this debate we will discuss the question of how to engage policy makers and come up with ways to drive clinical development.

Learning points:

•Theroleofregistriesandnetworksinpost marketing surveillance programmes

•Importanceofpostmarketingsurveillance programmes

+ Learning points:

•Howtoengagepolicymakers?

•Findingwaystodriveclinicaldevelopment

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Kate Bushby, Professor of Neuromuscular Genetics, Newcastle University

Program:

17:00Chairmanclosestheconference


n 15:30 Patient registries: enabling database access for all stakeholdersRegistriesarenecessarytospeeduptheacquisitionofknowledgeonrare diseases, the development of clinical research and to meet the post-marketing obligations. Patient registries need to be internationally interoperable to allow pooling of data to reach sufficient numbers for clinical research and public health purposes. Communication technology evolves very quickly and offers solutions to set up real data warehouses but obstacles remain, mainly legal and behavioural ones.

Learning points:

•Up-to-datepresentationof theEUropeanRDregistries landscape

•Reviewoftechnologicaloptions and new innovative projects

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Segolene AYME, Emeritus Director of Research, INSERM

n 16:15Groundbreakingresearch:PersonalizedmedicinebyusingzebrafishmodelsTheracule is a young start-up that is using genetically modified zebrafish models to do personalized medicine in rare diseases. The technology has a huge potential for all genetic rare diseases. In this session the CEO of Theracule will discuss the possibilities of personalized medicine by using zebrafish models.

Learning points:

•Personalizedmedicine

•Newpotentialforgeneticrare diseases

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Alexander D Crawford, CEO, Theracule

17:15Onefortheroad-Closetheconferenceinarelaxedatmospherewithadrinkinyour hand. You can discuss your key take-aways with your new contacts and strengthen your relationships. Because we believe that with the right relations and the right discussions we can collaboratively shape the future of orphan drugs!

Consulting corner:





pitch venture

capitalists!

Program:Whatisthenumberonereasonwhyshould you become a partner?Your brand will experience a surge in positive recognitionFollowingtheconferenceweconductedabriefsurveywithourdelegatesonhowtheyviewedourpartnersbeforeand after the conference. The overwhelming trend was an increase in the positive light delegates saw our partners.

Partners who took part in plenary, stream, or round table sessions experienced the most significant gains.

Need more reasons?

For more information on becoming a partner please contact:

Associate your brand with the annual Orphan Drugs Summit 2015

Limited Partner Opportunities;We

are inviting a limited number of partners, all well evaluated for

the best match- making effect

BenefitfromtheEuropean Marketing

coverage through our extensive industry

database

Be a part of business critical conversations

that take this industry forward

Show your solutions to an audience that

really counts

By invitation only; All attendees are

carefully screened for business critical

issues

Lawrence Hyde Head of Life Science Division

Tel: +46 8 120 505 52 Mob: +46 720 137 154

E-mail: [email protected] 1 2 3 4 5

After

Before

Partner 5

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Delegate awareness of partners before and after Orphan Drugs 2014

n TERMS & CONDITIONSYour booking is binding. You may substitute a delegate at any time. Please note that substitutions are not permitted unless approved by the organizers. For all cancellations (without an approved substituted delegate) received in writing more than 5 business days prior to the event and, a €120 (+VAT) administrative fee will be charged and a credit voucher for the remaining amount will be issued. Credit vouchers may be used at any Midfield Media conference within one year of issuance. For cancellations less than 5 business days prior to the event, the full amount of the delegate pass is non-refundable. Full payment is due 10 days upon invoice and no later than 5 business days prior to the event. Delegates that have NOT submitted payment prior to the event will not be admitted to the event. Admittance is then only granted upon approval of credit card payment directly onsite.

3 Easy ways to register:Phone: +46 8 651 10 90

Email: [email protected]

Website: www.orphandrugssummit.com

Register - 2015

Team Send Program Our agenda is the perfect opportunity for you to bring a cross-functional team. When attending as a team of five or more you’ll receive additional benefits, including complimentary registrations or discounts.

Group Booking Offer! Book 3 get 1 free = 4 delegates

Book 5 get 2 free = 7 delegates

Book 8 get 3 free = 11 delegates Use code BOOK3, BOOK5 or BOOK8 in the message field and we will contact you for the additional attendees


or call hotline: + 46 8 651 10 90

Early Bird Standard Rate2 day event incl dinner (€) Ends 23 March

Academic/Government 790 Euro 1090 EuroPharma/Biotech 1390 Euro 1890 EuroSolution Provider/Consultancy 1390 Euro 3490 Euro

Please use the chart below to determine your registration fee

We are pleased to announce the venue for this Years Orphan Drugs Summit is the beautiful Carlsberg Museum & Business Centre in Copenhagen.

This beautifully preseved building offers a rich sense of history combined with modern conveniences, making the venue an integral part of a memorable conference experience.

More details about the venue will soon be available, along with information about accomodations.

Venue - 2015

All prices are excluding tax

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Transcript of Orphan Drugs Summit 2015