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March 18-20, 2025
Pre-Conference Workshops on March 17, 2025Sheraton Philadelphia Downtown |
Philadelphia, PA
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March 18-20, 2025
Pre-Conference Workshops on March 17, 2025Sheraton Philadelphia Downtown |
Philadelphia, PA
UNITE WITH THE RARE DISEASE COMMUNITY TO CREATE POWERFUL PARTNERSHIPS, ADVANCE
ORPHAN THERAPEUTICS AND DELIVER LIFE-CHANGING BREAKTHROUGHS
THE RARE DISEASE SUMMIT CONNECTS KEY STAKEHOLDERS TO DRIVE THERAPEUTIC PROGRESS,
PROPEL COMMERCIAL STRATEGIES AND INSPIRE IMPACTFUL ADVOCACY. YOU’LL GENERATE
SOLUTION-FOCUSED APPROACHES SURROUNDING PATIENT ACCESS, REIMBURSEMENT,
COMMERCIALIZATION, LAUNCH, PARTNERING AND MORE, AS YOU UNIFY IN AREAS OF UNMET
MEDICAL NEED.
Request the 2025 Agenda
SAY HELLO TO YOUR RARE DISEASE SUMMIT LEADERS COUNCIL
Who can you expect meet at the Rare Disease Summit? Our Leaders Council is just
the tip of the iceberg when it comes to the vast expertise our speaker lineup
will bring. Prepare to meet these industry experts and so many more!
LARRY BRESSLER
HEAD, GLOBAL RARE DISEASE VALUE (MARKETING) AND MARKET ACCESS
CHIESI
ALYCIA SHILTON LLOYD
EXECUTIVE DIRECTOR, HEMATOLOGY-ONCOLOGY, PATIENT INNOVATION & ENGAGEMENT, GLOBAL
MEDICAL & SCIENTIFIC AFFAIRS
MERCK
WHAT'S ON TAP? CHECK OUT YOUR RARE DISEASE 2025 HOT TOPIC SNEAK PEEK
We're hard at work developing the robust 2025 agenda – What can you expect? In
this constantly evolving landscape, our industry experts will sharing their
intel and expertise on the biggest challenges facing the rare disease industry.
Your sneak peek includes:
FDA UPDATES ON RARE DISEASE PRODUCT DEVELOPMENT
NAVIGATING THE COMPLEXITIES – THE EVOLVING PRICING LANDSCAPE IN RARE DISEASES
MANUFACTURER CONSIDERATIONS FOR BUILDING A STRATEGIC COMMERCIALIZATION PLAN
CRACKING THE CODE – INVESTOR INSIGHTS INTO RARE DISEASE FUNDING OPPORTUNITIES
UTILIZING AI TECHNOLOGY IN A WORLD OF ZEBRAS, NOT HORSES
Request the 2025 Agenda
HAVE A PROFOUND IMPACT ON THE RARE DISEASE COMMUNITY
While the industry has seen growth and heightened focus around the pursuit of
orphan products and rare therapies, we’ve only scratched the surface. 2024
Attendees benefitted from high-impact content from experts from across the rare
disease industry:
INNOVATIVE APPROACHES TO AMPLIFY COLLABORATION AND ADVANCEMENT
Uncover innovative solutions to issues alongside the top industry experts
committed to shaping the future of Rare Disease
* Navigate FDA Updates on Rare Disease Product Development
* Helpful or Harmful – Using AI in the Rare Disease Space
* Unlocking Rare Disease Investment Success through Valuation, Risk and
Innovation
* Innovation Called and Gene Therapy Answered
RIVETING KEYNOTES AND GROUND-BREAKING PANELS TO DELIVER FRESH PERSPECTIVES
Join market leaders setting the stage for game-changing dialogue
* The Importance of Funding and Partnering from the Perspective of an Early
Stage BioPharma Company
* Exploring the Terrain - Rare Disease Landscape from a Payer Perspective
* Thinking Outside the Box – Forming Unique Partnerships within Rare Disease
* Rare Caregivers Report—Experiences Supporting Loved Ones Living with Rare
Diseases
A COLLABORATIVE THINK TANK TO BENCHMARK AND STRATEGIZE ON THE TOP CHALLENGES IN
RARE DISEASE
The all-plenary design of this event enables for more meaningful interaction,
higher level networking and closer up learning
* Hear multiple keynote addresses featuring key stakeholder perspectives,
including Industry, Advocacy and Regulatory
* Have dedicated time to brainstorm with your peers on innovative ideas
surrounding the different phases of Rare Disease Drug Development (ie.
diagnosis, preclinical, drug development, regulatory)
* Engage in our Innovation Station to hear rapid fire presentations from the
key innovators within the Rare Disease space
* Be able to take home with you new connections, new ideas and new best
practices
Request the 2025 Agenda
Global Medical Lead
Patient Engagement & Rare Diseases
Pfizer Inc
The rare Innovation summit is an intimate engaging meeting facilitating
discussion amongst speakers and attendees – nice mix of industry and patient
groups especially stakeholders I haven’t met before at other meetings.
Vice President
Regulatory Affairs, North America
Sobi, Inc.
I thought this was one of the best conferences I’ve attended over the years. The
focus was very practical and each speaker shared real world examples instead of
theory. It was well organized and the passion each participant has for helping
people with rare disease was very evident.
President & CEO
Tuberous Sclerosis Alliance
The Rare Disease Innovation Summit provided the opportunity for cross
communication between industry, nonprofits and vendors who bridge the gap
between the two. From presentations to panels, every attendee walked away with
new information and resources to help improve the quality of life for the rare
disease community. Thank you for making this possible CBI!
Senior Vice President
Global Business Operations & External Affairs
Horizon Pharma
The inaugural Rare Disease Innovation Conference brought together
multidisciplinary stakeholders to discuss important issues challenging the rare
disease ecosystem. The contributions by the audience accelerated thinking how to
address and eventually resolve key access related issues impacting people in
desperate need of therapeutic options.
President & CEO
Canadian Organization of Rare Disorders
Most amazing were the voices from the patient community who are driving drug
discovery and access. What some doing for a few benefits us all in this rare
space. CBI’s Summit was a prime example of how the patients are critical to
informing the research and development dialogue but also vice versa. We need to
know the innovations planned and in progress.
Global Medical Lead
Patient Engagement & Rare Diseases
Pfizer Inc
The rare Innovation summit is an intimate engaging meeting facilitating
discussion amongst speakers and attendees – nice mix of industry and patient
groups especially stakeholders I haven’t met before at other meetings.
Vice President
Regulatory Affairs, North America
Sobi, Inc.
I thought this was one of the best conferences I’ve attended over the years. The
focus was very practical and each speaker shared real world examples instead of
theory. It was well organized and the passion each participant has for helping
people with rare disease was very evident.
President & CEO
Tuberous Sclerosis Alliance
The Rare Disease Innovation Summit provided the opportunity for cross
communication between industry, nonprofits and vendors who bridge the gap
between the two. From presentations to panels, every attendee walked away with
new information and resources to help improve the quality of life for the rare
disease community. Thank you for making this possible CBI!
Senior Vice President
Global Business Operations & External Affairs
Horizon Pharma
The inaugural Rare Disease Innovation Conference brought together
multidisciplinary stakeholders to discuss important issues challenging the rare
disease ecosystem. The contributions by the audience accelerated thinking how to
address and eventually resolve key access related issues impacting people in
desperate need of therapeutic options.
President & CEO
Canadian Organization of Rare Disorders
Most amazing were the voices from the patient community who are driving drug
discovery and access. What some doing for a few benefits us all in this rare
space. CBI’s Summit was a prime example of how the patients are critical to
informing the research and development dialogue but also vice versa. We need to
know the innovations planned and in progress.
Global Medical Lead
Patient Engagement & Rare Diseases
Pfizer Inc
The rare Innovation summit is an intimate engaging meeting facilitating
discussion amongst speakers and attendees – nice mix of industry and patient
groups especially stakeholders I haven’t met before at other meetings.
* 1
* 2
* 3
* 4
* 5
RARE DISEASE INNOVATION & PARTNERSHIP SUMMIT: PART OF ACCESS USA
Hundreds from the access community came together as three industry-leading
events united under one roof for compelling discussions, industry benchmarking
and extended networking. What happened? Click play to see event highlights and
why you can't miss this pivotal access forum!
SEE WHO YOU'LL MEET AT THE RARE DISEASE SUMMIT
You will benefit from attending this event if you work for a company that
focuses on rare disease and orphan drugs and have responsibilities or
involvement in the following areas:
* RARE DISEASE
* ORPHAN DRUGS
* MARKET ACCESS
* COMMERCIAL STRATEGY/OPERATIONS
* REIMBURSEMENT
* EARLY/EXPANDED ACCESS
* LAUNCH EXCELLENCE
* PRODUCT LAUNCH
* PORTFOLIO MANAGEMENT/STRATEGY
* MARKETING
* DEVELOPMENT
* PATIENT ADVOCACY
* PAYER STRATEGY
* PATIENT SUPPORT/SERVICES
This event will also benefit consultants and solution providers who provide
services in the rare disease space.
GET THE LATEST EVENT UPDATES
Sign up to get the latest event updates and information.
Country *United KingdomUnited StatesAfghanistanAlbaniaAlgeriaAmerican
SamoaAndorraAngolaAnguillaAntarcticaAntigua And
BarbudaArgentinaArmeniaArubaAustraliaAustriaAzerbaijanBahamasBahrainBangladeshBarbadosBelarusBelgiumBelizeBeninBermudaBhutanBoliviaBosnia
And HerzegovinaBotswanaBouvet IslandBrazilBritish Indian Ocean TerritoryBrunei
DarussalamBulgariaBurkina FasoBurundiCambodiaCameroonCanadaCape VerdeCayman
IslandsCentral African RepublicChadChileChinaChristmas IslandCocos (Keeling)
IslandsColombiaComorosCongoCook IslandsCosta RicaCroatiaCubaCyprusCzech
RepublicDenmarkDjiboutiDominicaDominican RepublicEast TimorEcuadorEgyptEl
SalvadorEquatorial GuineaEritreaEstoniaEthiopiaFalkland IslandsFaroe
IslandsFijiFinlandFranceFrench GuianaFrench PolynesiaFrench Southern
TerritoriesGabonGambiaGeorgiaGermanyGhanaGibraltarGreeceGreenlandGrenadaGuadeloupeGuamGuatemalaGuineaGuinea-BissauGuyanaHaitiHeard
And McDonald IslandsHondurasHong Kong,
S.A.R.HungaryIcelandIndiaIndonesiaIranIraqIrelandIsraelItalyIvory
CoastJamaicaJapanJordanKazakhstanKenyaKiribatiKuwaitKyrgyzstanLaoLatviaLebanonLesothoLiberiaLibyan
Arab JamahiriyaLiechtensteinLithuaniaLuxembourgMacau,
S.A.R.MacedoniaMadagascarMalawiMalaysiaMaldivesMaliMaltaMarshall
IslandsMartiniqueMauritaniaMauritiusMayotteMetropolitan
FranceMexicoMicronesiaMoldovaMonacoMongoliaMontenegroMontserratMoroccoMozambiqueMyanmarNamibiaNauruNepalNetherlandsNetherlands
AntillesNew CaledoniaNew ZealandNicaraguaNigerNigeriaNiueNorfolk IslandNorth
KoreaNorthern Mariana IslandsNorwayOmanPakistanPalauPalestinian
TerritoryPanamaPapua New
GuineaParaguayPeruPhilippinesPitcairnPolandPortugalPuerto
RicoQatarReunionRomaniaRussian FederationRwandaSaint Kitts And NevisSaint
LuciaSaint Vincent And The GrenadinesSamoaSan MarinoSao Tome And PrincipeSaudi
ArabiaSenegalSerbiaSeychellesSierra LeoneSingaporeSlovakiaSloveniaSolomon
IslandsSomaliaSouth AfricaSouth GeorgiaSouth KoreaSpainSri LankaSt. HelenaSt.
Pierre And MiquelonSudanSurinameSvalbard And Jan Mayen
IslandsSwazilandSwedenSwitzerlandSyrian Arab RepublicTaiwan,
ChinaTajikistanTanzaniaThailandTogoTokelauTongaTrinidad And
TobagoTunisiaTurkeyTurkmenistanTurks And Caicos IslandsTuvaluUgandaUkraineUnited
Arab EmiratesUnited States Minor Outlying IslandsUruguayUzbekistanVanuatuVatican
CityVenezuelaVietnamVirgin Islands (British)Virgin Islands (U.S.)Wallis And
Futuna IslandsWestern SaharaYemenYugoslaviaZambiaZimbabwe
Sign Up
CONNECT AND NETWORK WITH KEY DECISION-MAKERS AT THE RARE DISEASE SUMMIT
Whether you’re looking to generate quality leads, showcase thought leaders, or
reinforce your brand, collaborate with us to identify custom sponsorship
opportunities to help you reach your business objectives.
For more information about custom opportunities to reach a qualified audience,
please contact us here.
DOWNLOAD SPONSOR PROSPECTUS
RELATED EVENTS
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17 - 20 Mar 2025
20 - 21 Nov 2024
17 - 20 Mar 2025
12 - 13 Sep 2024
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