#2014GGSummit - Global Genes · 2019. 12. 20. · Emerging Adulthood Transition overlaps with...

September 11 – 12, 2014 Huntington Beach, California

#2014GGSummit

Transmutation, Transition & Transformation Maya Doyle, LCSW-R, PhD

Pediatric Nephrology, Children’s Hospital at Montefiore Quinnipiac University Dept of Social Work

Session 6

Transmutation is defined as: “a change or alteration in form, appearance, or nature…especially to a higher form” (Merriam-Webster, 2011)

Medieval & Enlightenment Thought • Universal life forces and processes active in the

macrocosm of the natural world and the microcosm of the human body.

• Relationship between God, Man, and Nature

• Transmutation: “changing base metals to noble metals” (lead into gold)

• Commonly thought of as spiritual, magical, and even irrational

• Influences from Greece, China, India

Revered thinkers of the Scientific Revolution -

Robert Boyle,

Isaac Newton -

avidly pursued alchemical questions

“Attempts at transmutation may have failed,

… the outcomes of such attempts lead to new

analysis of the natural world” Moran, B. T. (2005). Distilling knowledge: Alchemy, chemistry, and the scientific revolution: Harvard Univ Pr.

Forerunners of modern chemists, they originated many chemical processing methods… still in use

today. (http://www.greekmedicine.net/history/Alchemy_and_Medicine.html)

In the history of medicine,

wrongheaded ideas,

disparate discoveries,

and imaginative leaps

often fuel

Magic and Miracles?

When medicine is able to alter the course of disease, improve the health of the body, and preserve and extend life…

We know it’s not magic…it’s dedicated and collaborative work!

(But there are definitely miracles)

Transmutation of Disease

“Medical, technological, and social interventions can all change the course of disease, from its natural history to a transmuted course, altering its processes and dynamics of care, its complications, its outcomes, and its timeframe.”

“Disease refers to a malfunctioning of biological and/or psychological processes, while the term illness refers to the psychosocial experience and meaning of perceived disease”.

– Kleinman, A. (1981). Patients and Healers in the Context of Culture.

“Disease” and “Illness”

Transmutation of Illness

As the disease is transmuted, so too is the experience and meaning of the disease. Advances in the ability to understand, treat, and prevent disease alter the experience of illness for individuals and for their families.

Treatment Contributes to Transmutation

• Treatments that – Manage symptoms or pain – Slow progression – Support survival – Change prognosticated outcome – Extend life

• 7000 rare disease, 200 with approved treatment – Some conditions have treatment, some are in the pipeline, others are

in need of research and/or funding – that’s why many of you are here

• How does illness change as treatment changes?

Transitioning • Children with congenital, chronic, rare conditions are surviving to adulthood

• Term, in the medical context, first came into use in the late 1980’s by Surgeon-General Koop

• Children whose lives he had saved through surgery as neonates were now surviving into childhood and adolescence.

• In 1989, at a medical conference titled “Growing up and Getting Medical Care: Youth with Special Health Care Needs,” Koop described transition as the “one major issue” for chronically ill adolescents that had not been adequately addressed by the healthcare system

Transitioning - Definition

The goal of transitioning for young adults with special health care needs is:

“to maximize lifelong functioning and potential through the provision of high-quality, developmentally appropriate health care services that continue uninterrupted as the individual moves from adolescence to adulthood”

(from 2002 Consensus)

A Time of Many Changes

Transitioning entails changes in – Healthcare system

– Education system

– Legal status

– Insurance

– Developmental changes

– Increasing autonomy and self-management

Emerging Adulthood

Transition overlaps with timeframe of “emerging adulthood” • 19-25 or 18-30 • characterized by five main features:

– Identity exploration, as individuals try out possibilities, particularly in love and work

– a sense of instability and constant revision of life’s plan – a powerful focus on self – a feeling of being “in-between” – a sense of possibility, being filled with hope and opportunities for

transformation

Arnett, J. (2004). Emerging adulthood: The winding road from the late teens through the twenties: Oxford University Press, USA.

Progress…

• 20+ years have passed

• Transition is a hot topic at pediatric and peds subspecialty meetings

• Transition research, programs in many centers, many readiness assessments tools

…and Problems

• Inconsistency across institutions

• Inconsistency within institutions

• Disconnects between pediatrics and

adult-oriented services

Particular Challenges for Rare Disease

• Inconsistencies across institutions • Illness- specific provider (in)experience • Illness-specific impact on needs and development • Availability, experience, and interest of adult providers • Long, trusted, hard-fought relationships with ped providers • Peds team/family trust in new adult team • Family and family knowledge/literacy about condition • Communication between peds and adult teams

HCT “CEO” Model Healthcare Transition Model

Stage Professional

Parent Child

Adult Resource Consultant CEO and HR EA Mentor Source of

Support Emerging CEO

Mid Adol

Consultant Supervisor Staff/executive trainee (primary do-er)

Earlier CTO CEO Mailroom! Moving the info around.

Adapted from: Reiss, J. G. (2011). Emerging Adults with Childhood-Onset Chronic Conditions and Disabilities: Using the Theory of Emerging Adulthood to Improve Health Care Transitions. Presented at the 5th Conference on Emerging Adulthood, Providence, RI, Oct 2011.

11 Essential Steps of Healthcare Transitioning (physician-oriented)

adapted from the 2002 Consensus Statement’s 6 Critical Steps, and recent transitioning literature 1,2

Suggestions for Patients & Parents

• Start early – age 12 or 14 – or sooner!

• Find out your institutions’, state’s policies and programs

• Find out when/if insurance changes happen

• Investigate your options (with or without your team – pts/parents must b pro-active)

• Use readiness tools (pts, fams, providers)

Suggestions for Advocates • Consider specific needs for transition for your community

• Be attentive to emerging adult and adult members of your disease community

• Create connections with adult-oriented providers, database

• Become patient-focused and patient-led if not already (as well as child/caretaker focused)

www.gottransition.org

Taking Control: Gaining Independence as a Young Adult with a Rare Disease

New Resource!!

• AAP, AAFP, ACP,ASIM. (2002). A consensus statement on health care transitions for young adults with special health care needs. Pediatrics, 110(6), 1304-1306

• Transitions Clinical Report Authoring Group, Cooley, W. C., & Sagerman, P. J. (2011). Supporting the health care transition from adolescence to adulthood in the medical home. Pediatrics, 128(1), 182-200. doi: 10.1542/peds.2011-0969

• Doyle, M., & Werner-Lin, A. (2014). That eagle covering me: transitioning and connected autonomy for emerging adults with cystinosis. Pediatric Nephrology, 1-11. doi: 10.1007/s00467-014-2921-5

• Arnett, J. J. (2004). Emerging adulthood: The winding road from the late teens through the twenties: Oxford University Press, USA.

Recommended Reading

Preparing for Success – Lobbying at the State and Federal Level

Panelists: • Jennifer Bernstein, Horizon Government Affairs • Julia Jenkins, Rare Disease Legislative Advocates • James Romano, Patient Services, Inc.

Session 7

“In this country it is not only permissible to

question our leaders, it is our responsibility.” Michael J. Fox, The American President

James Romano

Questions to Think About

• How many know who their State Representative is? • How many know who their Congressman/woman? • How many Senators do you have in Congress? • Do you know if your representative sit on key committees? • How many have ever written comments on a

rule/regulations? • Do your representatives (state or federal) know you and

your family?

Federalism

• Powers are constitutionally shared between the State and the Federal Government

• Ideological divide on how much power each should hold

• Healthcare is regulated on a state and federal basis

3 Branches of Government

• Executive Branch (President/Governor)

– Also includes Federal and State Agencies

• Legislative Branch (Congress/State Legislatures)

– Bi-Cameral (2 Houses)

– Upper House/Lower House

– Nebraska is the only state with only one house in their legislature

• Judiciary (Supreme Court/State Supreme Courts)

Legislative Branch (Federal)

• Develops and Passes Budget/Appropriations

• Develops and Passes Laws (or they Should)

• Provides Constituent Services – Getting Social Security Checks – Veterans Benefits – Tours of the Capitol

• Committee System – Important Committees

• House Energy and Commerce Committee--regulates health policy • House Ways and Means--regulates Taxation, Social Security and Medicare • House/Senate Appropriations—How health dollars are spent • Senate Finance Committee--regulates Taxation, Social Security, Medicare and other health policy • Senate Health, Education, Labor and Pensions (HELP)—regulates health policy

Legislative Branch (State)

• Bi-Cameral—House of Representatives/Senate

• Led by Speaker of the House/President of the Senate

• Power of the Purse

• State Budgets must be balanced—so health/education programs are always a focus

• Committee Structure: – Health and Human Services Committee – Ways and Means Committee – Appropriations Committee – Insurance Committee

Executive Branch (State/Federal)

• Executive Staff vs. Agency Staff

• Governor’s Health Staff vs. Secretary of Health

• Directs Policy Priorities

• Implements Laws – Develops the Regulations in implementation

– Review Public Comments

Judicial Branch

• Federal Courts – Judicial Review – Appointed Judges – Court of Last Resort (the Supreme Court) – Federal Court Districts

• State Courts – Review state/local laws – Appointed or Elected judges – State Supreme Courts appealed to Supreme Court

• Supreme Court & The Affordable Care Act

Healthcare Policy (State vs. Federal) • Disease Research Funding—NIH (Federal)

• Drug/Treatment Trials—FDA (Federal)

• Medicare (Federal)

• Medicaid—State and Federal Partnership

• Health Insurance—Both

– Most insurance is directly regulated by states

– Self Insured plans and ACA plans regulated by feds

Building Relationships with Members of Congress

Julia Jenkins, Executive Director

EveryLife Foundation for Rare Diseases

Empowering the Patient to be an Advocate!

• By growing the patient advocacy community and working together, we can amplify our collective voices to ensure rare disease patients are heard in state and federal government.

RDLA is a program of the EveryLife Foundation for Rare Diseases, a 501(c)(3) nonprofit, dedicated to spurring biotech innovation for rare disease treatments. EveryLifeFoundation.org

www.RareAdvocates.org

Rare Disease Legislative Advocates

• Monthly conference calls & webinars to learn about rare disease legislation

• Online advocacy tools to contact Members of Congress • RareAdvocates.org – Online clearinghouse for rare disease

legislation & news • Coordination of Rare Disease Congressional Caucus Briefings • Coordination of Legislative Conference & Lobby Days • Office space for organizations visiting DC • Consulting on legislative & grassroots strategies

Free Resources & Advocacy Tools

Find your member: http://www.congressweb.com/kaki/legislators

Getting to Know your Member of Congress

Important things to consider: – Biography: Previous profession & Children, Grandchildren

– Has she co-sponsored important legislation?

– What Committees does she sit on?

– Party affiliation – is her in power?

– What is her relationship to Leadership?

– Are there major medical research institutions or universities in her district?

– Is her seat safe? Or does she have a tough race?

– What are the demographics in her district?

– Who are her major donors? Or Major supporters?

Coming October 2014 • The most powerful tool a legislator has is their ability to author, co-sponsor and vote on

legislation. • We believe legislators should be both held accountable and given credit for taking

these actions. • Poor scores are more a reflection of the work we as a community need to do to

educate Congress on the issues that are important to us than a reflection of Congress not supporting rare disease issues.

• We see this document as a tool to help the rare disease community reach out to their Members of Congress and educate them on the bills that are important to patients.

Rare Disease Legislative Report Card

Why Relationships Matter

Rare Disease Congressional Caucus

• Gives the rare disease community a permanent voice to on Capitol Hill

• Brings public and Congressional awareness to the unique needs of the rare disease community- patients, physicians, scientists, and industry

• Creates opportunities to address roadblocks in access to and development of crucial treatments

• Check to see if your member is part of the Caucus at: www.rareadvocates.org/rarecaucus

• Members of Congress can join the Rare Disease Caucus by contacting the offices of co-chairs Rep. Joseph Crowley (NY-7) or Rep. Leonard Lance (NJ-7)

Rare Disease Congressional Caucus

• RDLA hosted two Caucus Briefings earlier this year – February 27th “Science Behind Rare Disease Policy” with

Special Guest Jonny Lee Miller, who stars as Sherlock Holmes in the hit TV show Elementary

– May 7th - “Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective”

• Two additional Caucus Briefings scheduled for this fall – September 17th - FDASIA Implementation – November 13th – Incentives for Drug Development

Congressional Caucus Briefings

– In-District Lobby Days

– Town Halls

– Campaign Events

– Washington DC Lobby Days • During this campaign season, many lawmakers are holding events in their

local districts, including Town Hall meetings.

• A perfect opportunity to interact directly with Members of Congress and urge them to support the Rare Disease community.

Opportunities to Meet your Member of Congress

Bringing Congress to You!

February 2015 Monday the 23rd – Friday the 27th

Annual Legislative Conference & Lobby Days!

Meeting With Capitol Hill Staff

How to be Most Effective in the Least Amount of Time

Know your Audience • Do you have a Member-level meeting or a staff-only meeting

Prepare Staff Ahead of Time (If Possible) • Send staffer a summary of your issue, which will allow them time to

familiarize themselves with your issue prior to the meeting • Allows more time to discuss your “ask” during your meeting instead

of spending the majority of your time on background info • Meetings will be very brief—generally no longer than 20 minutes.

Therefore, it is essential to make every minute count

Before Your Meeting

Do your homework

• Is the Member/office you are meeting with part of the Rare Disease Congressional Caucus? If not—ask them to join

• Often times, a Member/office will have a connection to a rare disease issue • The Member might be immediately impacted • Staff might be impacted • The Member might chair a Caucus within the rare space • The Member might be a medical professional

Before Your Meeting (cont.)

Robert Andrews (D-NJ) Spencer Bachus (R-AL) Lou Barletta (R-PA) Marsha Blackburn (R-TN) Suzanne Bonamici (D-OR) Bruce Braley (D-IA) Mo Brooks (R-AL) Julia Brownley (D-CA) Michael Burgess (R-TX) G.K. Butterfield (D-NC) Shelley Moore Capito (R-WV) Mike Capuano (D-MA) Andre Carson (D-IN) John Carter (R-TX) Gerald Connolly (D-VA) Joe Crowley (D-NY) Rodney L. Davis (R-IL) Charlie Dent (R-PA) Eliot Engel (D-NY)

Rare Disease Caucus Membership

Be on Time • Especially during session days, staff are over-

scheduled with hearings, markups and meetings

During Your Meeting

Be Prepared • Gauge the staffer’s level of knowledge about your issue. You will likely need to

spend some time on the “basics” • Have a well-defined “ask” and perfect your five-minute “elevator pitch” • Have “leave behind” documents ready for the staffer at the conclusion of your

meeting • If you are meeting with a Member, be sure to get a photo for your follow-up

homework

Send a “thank you” email to the staff/Member

Promote your meeting via social media • Facebook/Twitter • Post that meeting picture!

Send any additional useful information to staff • Be a resource/position yourself as an “expert” for

staff to rely on

Periodically check in with staff

After Your Meeting

If you were unable to meet with your Member of Congress while you were in DC, consider utilizing the district office • Less hectic • More undivided attention • Minimal travel required

Consider the District Office

Jennifer Bernstein Executive Vice President, Horizon Government Affairs

Contact information:

[email protected] www.horizondc.com

Questions?

Innovations in Science Brief

Immunosoft Matthew Scholz, PhD

Founder and CEO

Immusoft Corporation | [email protected]

Turning patients’ cells into drug factories

September 12th, 2014 Immusoft Corporation | [email protected]


• Immusoft turns patients’ cells into drug factories

- Ex vivo cell therapy

- Genomic modification of cells

- Autologous long-lived plasma cells engraft and secrete therapeutic proteins in vivo for many years

60

IMMUSOFT: OVERVIEW

Immusoft Corporation | [email protected] 61

IMMUSOFT: PROCESS OVERVIEW

Artwork adapted from New Scientist article about Immusoft Issue 2935, 20 September, 2013; Mini drug factory churns out drugs from inside bone

Immusoft’s Immune System Programming (ISP™) platform

turns patients’ cells into drug factories

Transpos

on PROTEINS

Your own drug factory Engineered immune cells lodged in bone marrow could replace medication

B cells extracted From patients’ blood

Genetic code that instructs the B cells to produce a new protein is inserted using a transposon

Modified B cells are injected into the bloodstream where they migrate to bone marrow and churn out the proteins they were programmed to make

BLOODSTREAM


62

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Day 7 Day 0

CD20

CD38

Day 10

Differentiating peripheral blood B cells into plasmablasts ex vivo

CULTURE SYSTEM


IN VITRO

B cells modified to produce VRC01, a broadly neutralizing antibody against HIV

B cells modified to produce the lysosomal enzyme α-L-iduronidase (IDUA) as detected by fluorometric assay in cell lysate

* The IDUA studies were performed in collaboration with Discovery Genomics

PROOF-OF-CONCEPT STUDIES A clinically viable technology for programing patients’ own immune cells to continually produce therapeutic proteins

0

0.5

1

1.5

2

2.5

pg/cell/day

AverageVRC01producingfromISP™-modified Bcells

B cells modified to secrete the lysosomal enzyme α-L-iduronidase (IDUA) as detected by fluorometric assay in cell media


In vivo imaging system (IVIS) data – signal from DiR dyed human B cells in mice

64

IN VIVO PROOF OF

0.00E+00

5.00E+09

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10/22/2012

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DateNSGmicewereinjectedwith3.6E+6DiR-dyedhumanBon10/26/12

JacksonLabsStudy1(~100days)totalflux(photons/sec)-ventralside

MemorySystem(Mouse3)




CD19System(Mouse2)

CD19System(Mouse5)

CD19System(Mouse8)

CD19System(Mouse11)

Control(FixedCells)(Mouse1)




CONCEPT STUDIES


BUSINESS STRATEGY BENCH TO CLINIC FOR < $10M

Commercial Focus Rare diseases •MPS I, Hemophilia A, LCAT deficiency

Pompe, PKU •Accelerated regulatory approval •Will advance multiple indications at once • $127B market by 2018

Heart disease •Novel class of therapeutic in a > $100B

market

First Human Trials MPS I (Hurler-Scheie syndrome) •Rare genetic disease •Very experienced collaborators •Only a small amount of enzyme is

needed

HIV •Great patient advocacy groups •Quick and clear efficacy data

Immusoft is a platform company, our immediate goal and primary value inflection point is to show the platform works in humans. Our business model is to license out our platform to partners for later stage clinical trials and commercialization.


• To be performed at University of Minnesota (Investigator Sponsored Trial)

• Key collaborators

- Paul Orchard, M.D.

- R. Scott McIvor, Ph.D.

- Perry Hackett, Ph.D.

• Treatment

- Autologous plasmablasts modified to secrete IDUA (the missing or deficient enzyme in MPS I patients).

66

CLINICAL TRIAL MPS I


• First informal interaction with FDA next week • Pre-pre-IND meeting in ~3 months • Proposed trial design

- Multi-dose Phase I/II study - Rapid dose escalation (3 patient cohort) - Estimated therapeutic dose (6 patient cohort) - Four doses 2-4 weeks apart

• Proposed patient inclusion criteria - Prior allogeneic transplant >2 years previously - Age at enrollment <14 years - Willing to commit to traveling to the University of Minnesota every 6 months for the duration

of the study

• Estimated start date Q1 2016

67

CLINICAL TRIAL MPS I


Immusoft

Clinical scale-up

and production

Fred Hutchinson Cancer Research

Center

Shelly Heimfeld Hans-Peter Kiem

Clinical Collaborato

rs (MPS I)

U Minn

Chet Whitley Paul Orchard

Clinical Collaborato

rs (HIV)

UCSF / SFGH

Joseph McCune

Steven Deeks

Vector Production

U Indiana Aldevron Waisman

Pre-clinical studies

Discovery Genomics

R. Scott McIvor Perry Hackett

Gene therapy dream team • Immusoft core team

- Developed ISP™ platform

• R. Scott McIvor - 25 years of gene therapy experience - Renown MPS I expert

• Perry Hackett - Creator of the Sleeping Beauty Transposon system - 50 years experience in molecular genetics

• FHCRC - Dedicated division for scaling up cell therapies

• Steven Deeks - Helped design Sangamo’s recent HIV gene therapy

trial

• University of Minnesota - Leading center for treatment of MPS I and other rare

genetic diseases - Performs clinical trials for Genzyme and other large

pharmaceutical companies

68

CLINICAL TEAM AND COLLABORATORS


BUDGET AND TIMELINE PRE-CLINICAL TO PHASE I

DealsinkedPre-pre-IND

Pre-IND

Animalstudiescomplete

GMPscale-upcomplete FileIND

Readytobeginhumantrials

0

500

1000

1500

2000

2500

3000

3500

4000

4500

0 1 2 3 4 5 6 7 8 9 101112131415161718192021222324

USD(in

thou

sand

s)sp

ent

Months

Today


TURNING PATIENTS’ CELLS INTO

DRUG FACTORIES

[email protected] 70

IMMUSOFT: A NEW MEDICAL PARADIGM


SUPPORTING

SLIDES

71

IMMUSOFT: A NEW MEDICAL PARADIGM


CORE TEAM AND COLLABORATORS

Core Team

Matthew Scholz (CEO)

• Seasoned startup executive; founded and exited Point B Telematics.

Zach Hall (Operations Dir.)

• Operations director at two previous biomedical startups.

Eric Herbig, PhD, MBA

(CSO)

• 15 years of research experience; commercialization and management expertise

Mei Xu, PhD (Research Dir.)

• Molecular biologist with extensive research experience

Key Advisors & Collaborators

Steven G. Deeks, MD • HIV clinical expert

Joyce Frey-Vasconcells, PhD • Regulatory expert (Fmr. FDA OCTGT)

Shelly Heimfeld, PhD • GMP scale-up

Daniel F. Hoth, MD • Regulatory expert

Hans-Peter Kiem, MD • GMP scale-up

R. Scott McIvor, PhD • Clinical gene therapy expert

Board of Directors

Mark Ahn, PhD • CEO, Galena BioPharma

(Nasdaq: GALE)

• Former VP & Corporate Officer at Genentech

• Former Senior Director of Immunology at Bristol-Myers Squibb

Matthew Scholz


Incorporated

First funding

2018 2017 2016 2015 2014 2013 2011 2010 2009 2012

•Licensed core technology

•First proof of concept studies

•Breakout Labs,

NIH, private

investors

•First mouse studies

Scale up for human

trials

•GMP production

• IND-enabling animal

studies Phase I

human

trials

TIMELINE OVERVIEW PARTNER EARLY

Raising $8M for scale up and Phase I clinical trial First expected exit or licensing opportunity.

Continued clinical

development – partner

and license


• Original IP from the Lab of David Baltimore at Caltech - Culture system for differentiating CD34+ HSCs into plasma cells - Synthetic plasma cell-specific promoter (based on the kappa light chain promoter)

drives protein expression

74

SCIENTIFIC BACKGROUND

(Luo et al. 2009)


• Terminally differentiated cells instead of progenitor cells

• Inducible caspase 9-based suicide gene

- Allows the rapid elimination of ISP™-modified cells with a chemical inducer of dimerization

- Already used in humans • Killed more than 90% of the transduced

cells within 30 minutes

75

SAFETY FEATURES

(Di Stasi et al. 2011)


B CELL DEVELOPMENT AND MIGRATION

(Radbruch et al. 2006)


BONE MARROW SURVIVAL NICHE

(Van T. Chu Claudia Berek 2013)

Putting into Practice – 21st Century Cures

Presented by: Jennifer Bernstein, Horizon Government Affairs

Session 8

All About Accelerating Cures • Congress is examining how to accelerate the

pace of cures in America • Spearheaded by Chairman Fred Upton and

Rep. Diana DeGette • Bipartisan! Hooray! • Examining the full arc from investment to

discovery to development

What is the 21st Century Cures Initiative?

“We have to make sure there’s not a major gap between the science of cures and

the way we regulate those therapies.” –Chairman Upton (R-MI)

–Rep. DeGette (D-CO)

Why?

Who is Involved?

This is a cycle that ultimately touches every single American, whether they are a patient, loved one,

caregiver, researcher, advocate, innovator, or government official.

A Huge Spectrum of Participants

The Committee Knows it Doesn’t Know Everything

• Staff are listening to all ideas—no idea is too big or too small

• Looking for specific ideas

• Send comments directly to the committee today

• Attend in-district meetings

• Hearings are live-streamed on the Committee’s website

• Follow the initiative on social media

How to Become Involved

BE HEARD! Fill out and return to summit registration desk today!

Follow the Initiative:

• Twitter: @ECcures, #Path2Cures

• Facebook: Facebook.com/EnergyCommerceCures

• Web: energycommerce.house.gov/cures

• You Tube: youtube.com/user/energyandcommerce

• RSS: energycommerce.house.gov/rss.aspx

Resources

Jennifer Bernstein Executive Vice President, Horizon Government Affairs

Contact information:

[email protected] www.horizondc.com

Questions?

The Must Have Collaboration for Successful Drug Development

Who Needs a Seat at the Table?

Session 9

Session Chairwomen

• Wendy White

• Barbara Wuebbels

Panelists

• Steve Groft, PharmD

• Hudson Freeze, PhD

• Matt Wilsey

• Jayne Gershkowitz

• Jacob Jacoby

• Kate Rauen, MD

The Changing Role in Orphan Drugs

Pharma

– Small number of patients

– High cost of drug development

– Regulatory approval in multiple countries

– Sales force/distribution challenges

Patients

– Better educated

– Well networked

– Social media

– Fund raising


Academia

– Universities want to license intellectual property

– Researchers looking for funding

– Source of KOLs

– Clinics


Regulatory and Government Agencies – Recruit “patient experts” to serve on panels

– Public hearings on high interest topics

– Sponsor scientific research projects and collaborative networks

– Expanded access

– Expedited approval


The Table

• Key players all need a seat

• Clarification of roles is important

• Communication is critical

• Success is possible

Format for Today

• Brief overview of everyone’s role

• Case study: expanded access/compassionate use

– Role of participants

– Challenges in each role

• Impact of “right to try” legislation

• Successful collaborations

End of Day 2

Thank you!

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