November 15, 2010 Washington, DC Welcome & Introductions Thank - - PowerPoint PPT Presentation

November 15, 2010 Washington, DC

 Welcome & Introductions

• Thank you to our host FasterCures
• See slides 23 & 24 for list of attendees

 Overview of Planning Session  Discussion: Policy Agenda Items

• NIH/Academic Grants, Training Programs
• Incentives to Spur Innovation
• FDA and Regulatory Issues
• Access & Reimbursement

 PDUFA Reauthorization  Update on Current Legislation and Appropriations  Next Steps: Path Forward Toward Building an

Effective Coalition, Actions Items and Rare Disease Day

 Gain Consensus on Congressional Caucus

Purpose & Membership Drive for New Caucus Members

 Discussion and Consensus on Legislative Policy

Agenda Items

 Agreement on Next Steps: Caucus

Briefings/Hearings, Policy Development, Rare Disease Day

 Agreement on Need for Bold and Transformative

Action!

 The Orphan Drug Act was enacted in 1983 to

encourage pharmaceutical companies to develop drugs for diseases that have a small patient populations

 Since the Act’s passage almost 1,800

investigational therapies have entered the research pipeline, but only about 17% have been approved for marketing by the FDA in 25 years

All Orphan Designations and Approvals Ultra-Rare Designations and Approvals

20 40 60 80 100 120 140 160 180 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 Orphan Designations Orphan Approvals

# of Designa natio ions ns and Appro provals vals

5 10 15 20 25 30 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 Ultra Orphan Designations Ultra Orphan Approvals

# of Designati ations ns and Approval als

* Taken from the FDA.gov website table on accelerated approvals

Therapeu peuti tic Area Number of Acceler erated ted Approv

• vals

Surroga

• gate

te endpoi

• int

Other

Cancer 26

Tumor load/PFS

Most pivotal studies without a control group

HIV 29

CD4 or viral load

Combination therapies also approved

Other 18

Variety

PAH, MS, hormones, iron, Crohns, antibiotics

Genetic 1

Renal pathology

Fabrazyme

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedand Approved/DrugandBiologicApprovalReports/ucm121597.htm

Only 1 genetic disease treatment approved via Accel. Approv. in 16

16 years

6

1990 1992 1994 1996 1998 2000 2004 2008

New Accelerated Approval Regulations put into Effect

29 drugs in a 16 year period

All accelerated approvals

7

 Both are often overlooked by drug developers or by

• thers instrumental in drug access, such as government
• fficials, public health programs and the news media

 Private pharmaceutical companies cannot recover the

cost of developing and producing treatments for these diseases

 Affects Millions of People:

• Estimated 7000 Rare Diseases, affect 30 Million Americans
• Neglected Diseases affect more than 1 billion people, 1/6th of the

world's population

 Most of these disease remain untreated  The science exists to treat many of these diseases  New science is needed for many more treatments

 National Institute of Medicine (IOM) Committee for

Accelerating Rare Disease Research and Orphan Product Development report recommends the Creation of a National Taskforce

 NIH’s Therapeutics for Rare and Neglected Diseases (TRND)

Program, budget doubles to $50 Million

 Cures Acceleration Network, new NIH Grant Program, created

in Healthcare Reform Package

 FDA creates new position, Associate Director of Rare

Diseases in the Office of New Drugs

 NIH Global Rare Disease Registry  Brownback/Brown Amendment for Rare & Neglected Diseases

passed FY 2010 FDA Appropriations, H.R. 2997, Section 740

• Calls for the creation of internal FDA committees to evaluate

issues for rare & neglected diseases and issue a report to Congress, Report will be due March 2011, Recommendations for guidance and policy changes will be due Sept. 2011

 GOP gained 6 seats in the Senate

• 53 Democrats, 47 Republicans

 GOP gained 60 seats in the House

• 239 Republicans, 176 Democrats

 8 races still undecided

• 5 of these leaning Republican

 Changes in Committee/Subcommittee Chairs and

Members

 GOP will take a fiscally conservative approach to

budget

• FDA and NIH likely to face increased resource constraints

 Bring all stakeholders together to seek creative

solutions to problems faced by patients, doctors, academia and industry

 Give a permanent voice to the rare & neglected

disease community in Congress

 Raise Public and Congressional Awareness

• Congressional Briefings
• Press Conferences
• One on One meetings with Members

 Help Drive a Legislative Agenda

 Goal 110 MOC’s & 20 Senators participating

 Bi-Partisan/Bi-Cameral

• Paperwork filed & accepted in the House
• House Co-Chairs: Crowley (D-NY) & Upton (R-MI)

 Caucus Co-Chair Upton could potentially become Chairman of the House Energy & Commerce Committee

• Brown (D-OH) & Barrasso (R-WY) Confirmed for the

Senate

 Senator Brown may be moving to the Senate Appropriations Committee

 Other Confirmed House Members: Baldwin, King, Melancon,

Holt, Rothman, Tim Johnson, Gary Peters, Schauer

 Refine Member target list, begin reaching out current MCs

now and develop relationships with new MCs in early 2011.

 The goal of the planning session is to receive input from

the whole rare and neglected disease community

 Build consensus on a broad and inclusive federal

legislative agenda

 To advance multiple initiatives to accelerate the

development of new treatments and cures for rare and neglected diseases.

 Agenda items should be legislative in nature, politically

reasonable and achievable

 Inclusive of multiple diseases or non disease specific  All suggestions received will be compiled for further

discussion and not necessarily be a part of the final agenda

 Items can be submitted after the meeting

• Incentives to Spur Innovation
• FDA and Regulatory Issues
• NIH/Academic Grants, Training

Programs

• Access & Reimbursement

 Priority Review Vouchers (Kids V Cancer’s Creating

Hope Act of 2010)

 Angel and VC Tax Credit for Investments in

Programs/Companies

 Improve TPDC when reauthorized so that grants & tax

credits are for new projects & are more $$

 Create an incentive to prevent new company from

shelving a Rare Disease Project when acquiring smaller company

 Create incentives for Universities to create centers of

excellence/become “research triangles” for rare diseases & actually begin drug development

 Increase the OOPD Grant Program  PDUFA V Reauthorization  New Office of Drug Evaluation for Biochemical and Genetic

Diseases/additional funding for Rare Disease Drug Review and reviewers who are specialized in the diseases/increased NIH & academic connections (Kakkis EveryLife Foundation)

 Ensuring the Brownback Brown Amendment is fulfilled to include

guidances on the use of alternative clinical trial designs and surrogate endpoints (Kakkis EveryLife Foundation)

 Regulatory reforms that can help minimize unnecessary costs and

time related to bringing new drugs to the market and expanding their approved indications/uses once they are initially approved (International Myeloma Foundation)

 Accelerate consideration of or Fast Track IND’s to get into clinical

trials

 Uniform IRB process  More Resources for the FDA to hire more staff  Improved Regulatory Science - specific grant for rare disease

regulatory Science

 Continued funding for CAN & TRND Grant Programs  Career development programs, research fellowship programs,

funded training programs and incentives for pediatric genetic researchers to expand the pool of investigators, both in basic research programs and in clinical genetics (National MPS Society)

 Expanded resources and role of the NIH’s Office of Rare Diseases

(Pediatric OMS Research Fund)

 Give Grant Making Authority like office of AIDS research  Training programs  Expand Consortia  Increase funding for Conferences/Workshops  Include Report Language in NIH Funding Bill

 Ensure Rare Diseases have a fair opportunity in current NIH grant

process

• New applications have less space to explain the importance – hurts R.D.
• Ensure Study Sections have specializations in RD

 Patient Registries – Registry of Registries (American Autoimmune

Related Diseases Association)

 Access to insurance coverage for all high risk

individuals must be a priority as Health Care Reform is implemented (National MPS Society)

 The cost of premiums in the PCIP (Pre-existing Condition Insurance Plan) should not be a barrier to coverage  Remove the requirement that applicants be uninsured for the prior six months to qualify should be removed

 Revamp Medicaid requirements and require private

insurance companies to ensure access to enteral feeding formulas and equipment (Russell Silver Syndrome Advocacy and Support )

 Medical Foods Equity Act to ensure insurance covers

medical foods (National PKU Alliance)

 Educational Programs on Rare Diseases for

Kids

 Education on Newborn Screening for Parents  $10 fee on Newborn Screening Tests to fund

research

 Caregiver Support (compensation)

 Prescription Drug User Fee Act (PDUFA) Reauthorization Process

• Congress must pass PDUFA V by Sept 2012 to ensure funding for the FDA
• A group of Patient Organization Stakeholders have been attending regular

meetings

 Rare Disease has been included in the FDA’s "Proposals for Potential

Enhancements in PDUFA V."

 Item #13, of the 17 proposals, suggests the development of a rare

disease program that will:

• Develop rare disease specific training programs for internal and external

stakeholders

• Develop regulatory policy, procedures, and guidances specific to rare diseases
• Communicate, outreach and support of external rare disease stakeholders

to support and accelerate rare disease drug development

• Conduct workshops for complex issues relating to rare disease drug

development

• Website on FDA.gov to serve as repository for information related to rare

disease drug development.

 How do we make item #13 a priority for the FDA?  Next Stakeholder Meeting: Wednesday Nov. 17th

 PDUFAreauthorization@fda.hhs.gov

 Clinical Trials Bill Passed  Hope Act Introduced to Extend Priority Review

Vouchers to Rare Diseases

 Congress unlikely to move on non essential

legislation, all bills will need to be reintroduced next session

 FY 2011 FDA/NIH Appropriations

• Senate & House passed a Continuing Resolution for all fiscal

year 2011 appropriations. It continues government funding through December 3, 2010. All government agencies are being funded at their FY 10 level

• An Omnibus appropriations bill is currently being negotiated,

we do not have updates on NIH (CAN/TRND funding) or FDA (Section 741 & OOPD grant increase)

 Summary of Meeting Takeaways from Today  Finalize Legislative Agenda, Items, Prioritization,

Positioning & Actions

• Send additional suggestions to patientadvocate@kakkis.org
• Discuss potential items with Caucus Co-Chairs
• Create a one sheet for Meetings

 Target & Recruit Members of Congress to join  Hold 1st Caucus Briefing with Staff and then Members

• Educational briefing/staff roundtable in December
• Plan event centered around Rare Disease Day

 Events on Rare Disease Day

 American Autoimmune Related Diseases  Assoc for Frontotemporal Dementias.  Bio Marin  Cavarocchi Ruscio Dennis Associates  Chevy Chase Bio Partners  Crowley Family Foundation  CURE--Research in Epilepsy  Cystic Fibrosis Foundation  Faster Cures  Friedreich's Ataxia Research Alliance  Genzyme Corporation  Global Health Technology Coalition  Health & Medicine Counsel of Washington  Huntington Disease Research Association  International Myeloma Foundation  JC White Consulting  Kakkis EveryLife Foundation

Kids V Cancer Men's Health Network Muscular Dystrophy Association National Adrenal Diseases Foundation National MPS Society National PKU Alliance NephCure Foundation Newborn Coalition NIH Office of Rare Diseases Pancreatic Cancer Action Network Parent Project Muscular Dystrophy Pediatric Hydrocephalus Foundation Prader Willi Syndrome Association Sarcoma Foundation of America Solace Nutrition The ALS Association The Sarah Jane Brain Foundation Tuberous Sclerosis Alliance



Cystinosis Foundation



Encocrine Society



Engage Health



Epidermolysis Bullosa (EB)



Hemophilia Federation



JF Campbell Consultants LLC



Life Raft Group



Lymphatic Research Foundation



Marble Road



Noah's Hope - Batten Disease



PC (Pachyonychia Congenita) Project



Pediatric OMS Research Fund



Russell Silver Syndrome Advocacy



Senator Boxer’s Office



The Children's Rare Disease Network



Wall Street Journal

Adult Congenital Heart Association/Children's Heart Foundation Celiac Spruce Association Cushing's Support & Research Foundation Desmoid Tumor Research Foundation Emma's Gift (Newborn Screening) Fibrodysplasia Ossificans Progressiva Foundation for PSP Heartland Cooperative Holland & Knight IDEA League PhRMA PMD Foundation RE Children’s Project Thyroid Cancer Survivors' Association