1 / 23

Orphan Drugs – 2011 What’s Happening?

Orphan Drugs – 2011 What’s Happening?. Women in BIO Marlene E. Haffner , MD, MPH Mhaffner3@verizon.net 301 984 5729 - Office 301 641 4268 – cell November 1, 2011. LOTS!. INCENTIVES. 7 Years Exclusive Marketing Waiver of PDUFA Filing Fee

tallys
Download Presentation

Orphan Drugs – 2011 What’s Happening?

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript


  1. Orphan Drugs – 2011What’s Happening? Women in BIO Marlene E. Haffner, MD, MPH Mhaffner3@verizon.net 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 Products Orphan 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 – GayatriRao, 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 mhaffner3@verizon.net www.mhaffner.com 301-984-5729 301-641 4268 Haffner Associates, LLC

More Related