The FDA Landscape. TOPRA discussion Florence Houn MD MPH VP, Regulatory Policy and Strategy Celgene Corporation May

Transcription

1 The FDA Landscape TOPRA discussion Florence Houn MD MPH VP, Regulatory Policy and Strategy Celgene Corporation May

2 Headliner Trends at FDA Decreasing the burden of REMS Eliminating Med Guide-only REMS Standardization of REMS Changing the accelerated approval paradigm Single arm versus randomized studies Confirmatory studies (2) ongoing at submission Increasing the arsenal for compliance Park doctrine, stream-lining civil monies penalties, coordination with DOJ-OCI, HHS exclusions 2

3 The Bottom Line 1. Is the bar moving higher? Yes. 2. Is FDA more risk adverse? Yes. 3. Is CE at the FDA? Not yet but coming. Is there any good news? Yes. 3

4 1 2 3 PDUFA 3 approval % is lower than pre-pdufa % 4

5 1,2 3 John Jenkins FDA CMS Summit Dec

6 Statistics from the Pink Sheet In 2010, FDA s CDER approved 21 novel drugs. 15 new molecular entities (NMEs) & 6 biologics license applications (BLAs) 48% (10/21) granted priority review. The average time to approval was 15.1 months The average time for NME approval was approximately 13.6 months. CDER met the PDUFA goal date for action for 86% of its novel approvals. Pinksheet: the overall CDER first-cycle approval rate comes to a very healthy 76%. January 24,

7 Statistics from FDA For FY2010, 8 priority 14 standard applications filed by FDA (22 NMEs) FDA did not give out approval rate or numbers of NMEs for FY 2010 cohort. In FY 2009, there were 8 priority NME/BLA and 22 standard NME/BLAs filed. (In FY 2008, 10 p and 19 s NME.) Not 48% granted priority review, but 24%-27%. eportsmanualsforms/reports/userfeerep orts/performancereports/pdufa/ucm pdf 7

8 Approval Times/% Per FDA 13 m. 9 m % first cycle approval rate for FY 09 cohort vs. PinkSheet s 76% Pinksheet: of those that are approved in 2010, how many are 1 st cycle FDA: of those that are submitted in FY 2010, how many are 1 st cycle 8 35% 33%

9 2. Risk Aversion: Safety First premarket comparative safety The comparative exists for QT prolongation Hepatoxicity CV Suicide ideation and acts Safety studies prior to approval in certain therapeutic fields of unmet medical need DM Weight Loss Pain Neuro-psychiatric drugs In oncology, more emphasis on dose-finding 2011 vandetanib and QTc and 2009 AP ofatumamab 9

10 3. Comparative effectiveness in US "We can't pay for crappy stuff I think this CER thing is hugely important to this." UnitedHealth Group Chief of Medical Affairs Reed Tuckson 1/12/11 Sentinel Public Meeting 10

11 Personalized Medicine Diagnosis and Risk Prediction Pharmacogenomics New Therapies Identify rare variant responsible for childhood illness Begin colonoscopy at age 40 (HNPCC) Rx by drug metabolism genetic profile Clopidogrel v- CPY2C19 Acambavir HLA B5701 Gene-based drug therapy for cancer BCR-ABL T315I CML (omacetaxine?) KRAS WT (erbitux/vectibix) 11

12 Expertise in devices, IVDs CDRH changes 510k redesign underway LDT enforcement discretion being pulled back Co-development guidance to be issued Warning Letters and public hearing on OTC genetic testing 12

13 US comparative effectiveness and the FDA Relative safety has been used for non-approval. Relative effectiveness, while not a statutory requirement, can also be a safety issue. Safe and effective marries reasonable and necessary Payors can mine their own data Medco and warfarin/genetic testing hospitalization; Kaiser and REMS pregnancy prevention burdens WellPoint and Avandia outcomes Provide data stream of value of the product at time of approval; waiting for phase 4 studies may be too late. 13

14 PCORI -March 2010 PPACA Patient-Centered Outcomes Research Institute s (PCORI) NIH and AHRQ, not FDA, on BoG Registries To assist patients, clinicians, purchasers, and policymakers in making informed health decisions by advancing the quality and relevance of evidence Findings can be used by government if through public process Private, non-profit umbrella organization outside of government paid by public Research agenda Methodology standing committee Medicare data open for PCORI use 14

15 At CDER, major focus has been on FDAAA and BPCIA not CER, but 15

16 FoodandDrugAdministration/ucm htm 16

17 Parting Thoughts We are operating in a highly competitive business and within a difficult regulatory environment The expectations of consumers, providers and payors are steadily increasing. The regulatory agencies to whom we submit data have staff who are usually very good at what they do. To be successful we must meet the expectations of all our customers including the regulators to whom we submit data. Take advantage of all available resources (internally and externally) to make this happen. 17

18 There is a well-traveled road map for success in regulatory affairs Successful models Obey the law Quality is Job 1 Treat Employees Well Be expert in what you do Have passion for the job Without our 20 smartest people, we re just another software company Have products that anticipate desire 18