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 Ultra-Rare Designations and Approvals and Approvals vals 180 30 provals als ns and Approval 160 ns and Appro 25 140 120 20 ations ions 100 15 natio # of Designati 80 # of Designa 60 10 40 5 20 0 0 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 Orphan Designations Ultra Orphan Designations Orphan Approvals Ultra Orphan Approvals
Only 1 genetic disease treatment approved via Accel. Approv. in 16 16 years Number of Surroga ogate te Therapeu peuti tic Area Acceler erated ted Other endpoi oint Approv ovals Most pivotal studies Tumor Cancer 26 without a control load/PFS group Combination CD4 or viral HIV 29 therapies also load approved PAH, MS, hormones, Other 18 Variety iron, Crohns, antibiotics Renal Genetic 1 Fabrazyme pathology * Taken from the FDA.gov website table on accelerated approvals http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedand Approved/DrugandBiologicApprovalReports/ucm121597.htm 6
1990 1992 1994 1996 1998 2000 2004 2008 New Accelerated Approval Regulations put into Effect 29 drugs in a 16 year period 7 All accelerated approvals
Both are often overlooked by drug developers or by others instrumental in drug access, such as government officials, 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, 1/6 th 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)
Recommend
More recommend
