orphan drugs – 2011 what’s happening?
DESCRIPTION
Orphan Drugs – 2011 What’s Happening?. Women in BIO Marlene E. Haffner , MD, MPH [email protected] 301 984 5729 - Office 301 641 4268 – cell November 1, 2011. LOTS!. INCENTIVES. 7 Years Exclusive Marketing Waiver of PDUFA Filing Fee - PowerPoint PPT PresentationTRANSCRIPT
Haffner Associates, LLC 1
Orphan Drugs – 2011What’s Happening?
Women in BIO
Marlene E. Haffner, MD, MPH
[email protected] 301 984 5729 - Office 301 641 4268 – cell November 1, 2011
Haffner Associates, LLC 2
LOTS!
Haffner Associates, LLC 3
INCENTIVES 7 Years Exclusive Marketing Waiver of PDUFA Filing Fee Assistance of OOPD Tax Credits – 50 % of clinical
development costs – one year fall back; 20 year carry
forward Grant Support Recommendations from Brownback
Brown Report Associate Director for Orphan Drugs
- CDER
Haffner Associates, LLC 4
Accomplishments ~ 390 products approved > 2000 designations > 15,000,000 patients
potentially treated > 45 products approved
through the grants program
Haffner Associates, LLC 5
2009 Emergence of Big PhRMA GSK Novartis Pfizer Genentech/Roche Sanofi
Haffner Associates, LLC 6
Biotech and Orphan Products
Began with Genentech – hGH - and has progressed
Now the emergence of “molecular medicine”
Personalized medicine – genetic knowledge fits well with orphan drug development
Haffner Associates, LLC 7
1992 Regulations 2011 NPRM 2011 NPRM released week of
October 21st
Codifies practices already in place
Medically plausible subsets vs the disease process
Better defines the designation process
Haffner Associates, LLC 8
Review and Approval of Orphan
ProductsOrphan Products must be Safe and Effective
for their intended use Is that possible? FDA has demonstrated flexibility 90 of 135 non-oncology approvals approved
with non standard approaches – NORD study Smaller clinical trials Historical controls Single arm/non-randomized Case-by-case
Haffner Associates, LLC 9
Current Issues and Activities Safety concerns REMS Cost Reimbursement Large # of orphan programs
and activities globally Increasing coordination with
the EU
Haffner Associates, LLC 10
Safety Orphan Products approved based on small
clinical trials Infrequent adverse events will not be seen Until the last 3 – 4 years “there had been no
safety concerns with orphan products” That has changed – e.g. EPO – 25 years
following initial approval; Botulinum toxin - black box warning
Virtually all new orphan approvals carry a REMS
Haffner Associates, LLC 11
REMS – Risk Evaluation and Mitigation Strategy Orphan Products with REMS –
Nplate – no safety signals in clinical trials
Botulinum toxin – new findings FDA (and Congress) erring on
the side of safety Allows FDA to approve some
products which would not previously have been approved
Haffner Associates, LLC 12
Cost of Orphan Drugs US expenditure for drugs - 2.2% of GDP in
2000; will be 5.6% in 2030 Newest products in the health care arena
are Biotech Biotech products are expensive – whether
they are orphan products or not Because of some high profile examples,
orphan products are often sighted as expensive Some orphan products are
expensive; most are not Economies of scale for a small
population is not present – need to explain better to the public
Haffner Associates, LLC 13
Reimbursement
Must figure reimbursement strategies as drug development proceeds. This involves working with CMS and individual insurance companies
Must explain to patient groups the rationale for cost of the product if high. Cost may by necessity be high
Some EU member states very conservative in reimbursement
Recent unfortunate examples
Haffner Associates, LLC 14
Personalized Medicine Human genome allows more
targeted therapies Many of these will subdivide
larger diseases into smaller ones which may be orphan diseases – gene mutations
NIH TRND program Repurposing of old drugs
Haffner Associates, LLC 15
What does this mean for my company? What is your strategy? Orphan Products generally treat life
threatening diseases – may mean faster approval
Common application format in EU and US – saves money
Tax incentives Exclusivity Waiver of PDUFA Filing Fee – $1.8
mil in 2011
Haffner Associates, LLC 16
Examples of Office of Orphan Products
Assistance
Haffner Associates, LLC 17
Envenomation Envenomation – OOPD
convinced review division to broaden thinking re indication – approved product in 2011
Other envenomation products being developed
Haffner Associates, LLC 18
Narcolepsy How should the product be
DEA scheduled? Without the intervention of
OOPD the product may not have been developed
Haffner Associates, LLC 19
Prevalence and # of clinical trials A disease with < 100 patients
in the US Hard, if not impossible, to do
TWO placebo controlled trials When to use historical controls How to develop phase 1,2,3
trials Inviting the OOPD to meetings
with the division Need for Natural History
studies
Haffner Associates, LLC 20
Toxicity testing The disease is lethal at a very
young age What are the considerations Can toxicity testing be done
following clinical trials in humans
Need new methodologies for preclinical drug development
Haffner Associates, LLC 21
Roll of the Associate Director for Orphan Products in CDER Primarily policy/consistency Evaluating new science with
regards to Orphan Product development
Biomarkers Close collaboration with the
OOPD Hopefully will grow in size
Haffner Associates, LLC 22
A New Day in OOPD Appointment of new Acting
OOPD Director – Gayatri Rao, MD, JD
NPRM issues 19 years following initial regulations
Increasing # of companies join the drive toward rare disease therapies
NIH efforts Commitment of FDA with
Associate Director for Orphan Products
Haffner Associates, LLC 23
?Marlene E. Haffner, MD, MPH
Haffner Associates, LLC [email protected]
www.mhaffner.com 301-984-5729 301-641 4268