REMS: the long tail can sting you

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  • REMS: the long tail can sting you

    1. 1. REMS: the long tail can sting you Sponsored By: Cole Werble is Editor-in-Chief of The RPM Report . Prior to joining Windhover in 2005, Werble was an independent consultant to the pharmaceutical and health products industries. For 26 years, Werble was owner and editorial director of FDC Reports, Inc., the publisher of “The Pink Sheet,” “The Gray Sheet,” and other leading news publications for the pharmaceutical industry. Arnold I. Friede is counsel to the law firm of McDermott Will & Emery LLP based in its Washington, D.C. office. Arnie is a member of the Firm’s Health Law Department. Arnold I. Friede is a widely respected food and drug law counselor and advocate with significant advertising law, health care law, First Amendment, environmental, and commercial and transactional experience. REMS: the long tail can sting you
    2. 2. Clinipace REGISTRIES CLINICAL TRIALS GRANT MANAGEMENT On-Demand Clinical Research Solutions CLINICAL SERVICES Sponsored By:
    3. 3. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) www.mwe.com Sponsored By:
    4. 4. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><ul><li>ESTIMATED SIZE OF THE POPULATION LIKELY TO USE THE DRUG INVOLVED. </li></ul></ul><ul><ul><li>SERIOUSNESS OF THE DISEASE OR CONDITION THAT IS TO BE TREATED WITH THE DRUG. </li></ul></ul><ul><ul><li>EXPECTED BENEFIT OF THE DRUG WITH RESPECT TO SUCH DISEASE OR CONDITION. </li></ul></ul><ul><ul><li>EXPECTED OR ACTUAL DURATION OF TREATMENT WITH THE DRUG. </li></ul></ul><ul><ul><li>SERIOUSNESS OF ANY KNOWN OR POTENTIAL ADVERSE EVENTS THAT MAY BE RELATED TO THE DRUG AND THE BACKGROUND INCIDENCE OF SUCH EVENTS IN THE POPULATION LIKELY TO USE THE DRUG. </li></ul></ul><ul><ul><li>WHETHER THE DRUG IS A NEW MOLECULAR ENTITY. </li></ul></ul>www.mwe.com <ul><li>FACTORS TO CONSIDER IN DECIDING WHETHER TO IMPOSE REMS BECAUSE BENEFITS OUTWEIGH RISKS BUT ONLY WITH REMS </li></ul>Sponsored By:
    5. 5. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><li>POST-APPROVAL REMS BASED ON </li></ul><ul><ul><li>NEW SAFETY INFORMATION; REMS NECESSARY TO ENSURE THAT BENEFITS OUTWEIGH RISKS </li></ul></ul><ul><ul><li>NEW SAFETY INFORMATION: INFORMATION DERIVED FROM A CLINICAL TRIAL, AN ADVERSE EVENT REPORT, A POST-APPROVAL STUDY OR PEER-REVIEWED BIOMEDICAL LITERATURE; DATA DERIVED FROM THE POST-MARKET RISK IDENTIFICATION AND ANALYSIS SYSTEM; OR OTHER SCIENTIFIC DATA DEEMED APPROPRIATE BY FDA ABOUT </li></ul></ul><ul><ul><ul><li>A SERIOUS RISK OR AN UNEXPECTED SERIOUS RISK ASSOCIATED WITH USE OF THE DRUG THAT FDA HAS BECOME AWARE OF (THAT MAY BE BASED ON A NEW ANALYSIS OF EXISTING INFORMATION) </li></ul></ul></ul><ul><ul><ul><li>THE EFFECTIVENESS OF THE APPROVED RISK EVALUATION AND MITIGATION STRATEGY FOR THE DRUG OBTAINED SINCE THE REMS. </li></ul></ul></ul>www.mwe.com Sponsored By:
    6. 6. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><li>CONTENT OF REMS </li></ul><ul><ul><li>TIMETABLE FOR ASSESSMENT SPECIFIED IN THE STATUTE AND/OR IN THE REMS ITSELF. </li></ul></ul><ul><ul><li>REMS ASSESSMENTS MAY BE ELIMINATED IF FDA DETERMINES THAT THAT SERIOUS RISKS OF THE DRUG HAVE BEEN ADEQUATELY IDENTIFIED AND ASSESSED AND ARE BEING ADEQUATELY MANAGED. </li></ul></ul><ul><ul><li>MINIMAL REMS </li></ul></ul><ul><ul><li>MINIMAL REMS ELEMENTS IF FDA DETERMINES THESE HELP MITIGATE SERIOUS RISK OF THE DRUG </li></ul></ul><ul><ul><ul><li>MEDGUIDE </li></ul></ul></ul><ul><ul><ul><li>PPI </li></ul></ul></ul><ul><ul><ul><li>COMMUNICATION PLAN </li></ul></ul></ul>www.mwe.com Sponsored By:
    7. 7. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><li>ADDITIONAL REMS ELEMENTS TO PROVIDE SAFE ACCESS TO DRUGS WITH KNOWN SERIOUS RISKS THAT WOULD OTHERWISE BE UNAVAILABLE. </li></ul><ul><ul><li>DRUG HAS BEEN SHOWN TO BE EFFECTIVE, BUT IS ASSOCIATED WITH A SERIOUS ADVERSE DRUG EXPERIENCE   </li></ul></ul><ul><ul><li>CAN BE APPROVED ONLY IF, OR WOULD BE WITHDRAWN UNLESS, SUCH REMS ELEMENTS ARE REQUIRED AS PART OF SUCH STRATEGY TO MITIGATE A SPECIFIC SERIOUS RISK LISTED IN THE LABELING OF THE DRUG; AND </li></ul></ul><ul><ul><li>ASSURE ACCESS AND MINIMIZE BURDEN </li></ul></ul>www.mwe.com Sponsored By:
    8. 8. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><ul><ul><li>SPECIAL TRAINING AND EXPERIENCE OR SPECIAL CERTIFICATION. </li></ul></ul></ul><ul><ul><ul><li>AVAILABILITY ONLY IN CERTAIN HEALTH CARE SETTINGS, E.G. HOSPITALS </li></ul></ul></ul><ul><ul><ul><li>EVIDENCE OR OTHER DOCUMENTATION OF SAFE-USE CONDITIONS, SUCH AS LABORATORY TEST RESULTS; </li></ul></ul></ul><ul><ul><ul><li>PATIENT MONITORING; OR </li></ul></ul></ul><ul><ul><ul><li>REGISTRY </li></ul></ul></ul>www.mwe.com <ul><ul><li>ELEMENTS TO ASSURE SAFE USE </li></ul></ul>Sponsored By:
    9. 9. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><ul><li>IMPLEMENTATION SYSTEM </li></ul></ul><ul><ul><ul><li>MONITOR AND EVALUATE IMPLEMENTATION OF SUCH ELEMENTS BY HEALTH CARE PROVIDERS, PHARMACISTS, AND OTHER PARTIES IN THE HEALTH CARE SYSTEM WHO ARE RESPONSIBLE FOR IMPLEMENTING SUCH ELEMENTS; AND </li></ul></ul></ul><ul><ul><ul><li>WORK TO IMPROVE IMPLEMENTATION OF SUCH ELEMENTS BY SUCH PERSONS. </li></ul></ul></ul>www.mwe.com Sponsored By:
    10. 10. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><ul><li>EVALUATION OF REMS ELEMENTS TO ASSURE SAFE USE </li></ul></ul><ul><ul><li>CRITERIA </li></ul></ul><ul><ul><ul><li>UNDUE BURDEN ON ACCESS </li></ul></ul></ul><ul><ul><ul><li>MINIMIZE BURDEN ON THE HEALTH CARE DELIVERY SYSTEM </li></ul></ul></ul><ul><ul><ul><li>FDA ONGOING EVALUATION OF REMS IN GENERAL </li></ul></ul></ul>www.mwe.com Sponsored By:
    11. 11. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><ul><li>ADDITIONAL MECHANISMS TO ASSURE EXPANDED ACCESS TO UNAPPROVED DRUGS UNDER SECTION 561 </li></ul></ul><ul><ul><li>WAIVER OF REMS IN PUBLIC HEALTH EMERGENCIES </li></ul></ul><ul><ul><li>REMS CANNOT BE USED TO BLOCK GENERIC DRUG APPROVALS </li></ul></ul>www.mwe.com Sponsored By:
    12. 12. FDAAA RISK EVALUATION AND MITIGATION STRATEGY (REMS) (cont’d) <ul><li>PERIODIC ASSESSMENT AND MODIFICATION OF APPROVED REMS </li></ul><ul><li>DISCUSSIONS, ACTION, DISPUTE RESOLUTION PROCESS </li></ul><ul><li>DRUG SAFETY OVERSIGHT BOARD ROLE IN DISPUTE RESOLUTION. </li></ul><ul><li>USE OF ADVISORY COMMITTEES </li></ul><ul><li>INTERNATIONAL COORDINATION </li></ul><ul><li>REMS IN ANDA’s </li></ul><ul><li>DRUG SAFETY OVERSIGHT BOARD CODIFIED </li></ul>www.mwe.com Sponsored By:
    13. 13. ENFORCEMENT <ul><li>MISBRANDING </li></ul><ul><ul><li>FAILURE TO COMPLY WITH POST-MARKET STUDY OR POST-MARKET CLINICAL TRIAL REQUIREMENT </li></ul></ul><ul><ul><li>VIOLATION OF REMS </li></ul></ul><ul><li>CIVIL PENALTIES </li></ul>www.mwe.com Sponsored By:
    14. 14. REMS: The New FDA Post-Market Safety Restrictions Cole Werble Senior Executive Editor FDC-Windhover Biopharma Group Dec. 10, 2008 Sponsored By:
    15. 15. Table of Contents <ul><li>REMS Intro In Five Sections </li></ul><ul><li>Background and impact (3-7) </li></ul><ul><li>Decision-makers (8-11) </li></ul><ul><li>Pyramid builders (12-15) </li></ul><ul><li>Metrics (16-18) </li></ul><ul><li>Lessons from first REMS (19-26) </li></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    16. 16. Milestone Legislation FDA Amendments Act, September 2007 Kefauver Amendments August 1962 Efficacy amendments were designed to prevent approval of drugs like thalidomide. Safety amendments are designed to permit controlled marketing of thalidomide. Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    17. 17. Policy in Progress <ul><li>&quot;We are writing the history of drug regulation with what we are doing every day in response to FDAAA &quot; </li></ul>-- John Jenkins, Office of New Drugs Director “ Institutionalized inside FDA as part of the regulatory process. I don’t think it has yet been elevated to the level of regulatory science.” -- Scott Gottlieb, former Deputy Commissioner FDA Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    18. 18. <ul><li>FDA has been experimenting with risk minimization programs for 35 years </li></ul><ul><li>Thalomid – STEPS, registered MDs only, one-month supply, pregnancy tests </li></ul><ul><li>Clozaril – “no blood, no drug,” registered pharmacies, National Registry </li></ul><ul><li>Accutane – Pregnancy Prevention Program, monthly tests, one-month supply, patient and MD surveys </li></ul><ul><li>Atromid-S – too many Rxs </li></ul><ul><li>Methadone – special clinics </li></ul>1972 1978 1988 1989 1998 Risk Evaluation & Mitigation Strategies REMS: New Acronym, Not a New Concept Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    19. 19. Still A Profound Change <ul><li>Risk Management has become foundation of FDA regulatory system: pre- and post-market </li></ul><ul><ul><li>REMS authority gives FDA the upper hand </li></ul></ul><ul><ul><li>Power To Mandate = Final Say In Negotiations </li></ul></ul><ul><li>Spirit of REMS everywhere </li></ul><ul><ul><li>Post-Marketing Trials </li></ul></ul><ul><ul><li>Class Labeling Authority </li></ul></ul><ul><ul><li>DTC Pre-Review Authority </li></ul></ul>FDA Has Power To Dictate The Patient Population That Can Access A Medicine Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    20. 20. Dramatic shift in landscape <ul><li>FDA’s premarket expectations </li></ul><ul><ul><li>Specific patient population data </li></ul></ul><ul><ul><li>Key issue: How will companies limit to select populations </li></ul></ul><ul><ul><li>Be prepared for questions about intended markets </li></ul></ul><ul><ul><li>More “comfort”; no change in risk-benefit </li></ul></ul><ul><li>Control of the post-market </li></ul><ul><ul><li>Refrain : A change in locus of post-market expertise – sponsor no longer dominates </li></ul></ul><ul><ul><li>FDA expects to get more info from outside sources: “We will engage the outside world,” OSE Director Dal Pan. </li></ul></ul><ul><ul><li>Sponsor should be able to match those outside sources </li></ul></ul>Gone are the days when someone within a drug company can say: “ Don’t introduce the issue; if we don’t study it, we aren’t going to have to deal with the problem.” A quote that former FDA Com. Kessler is fond of repeating. Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    21. 21. REMS Decision-makers <ul><li>More lawyers in process – especially on FDA’s side during the first REMS years </li></ul><ul><ul><li>Lawyers are keen to watch, control and set precedents </li></ul></ul><ul><ul><li>One of the reasons that REMS products are missing review deadlines; the lawyers come in last and are being careful </li></ul></ul><ul><ul><li>Effect of presidential election on precedental struggle </li></ul></ul><ul><ul><ul><li>Expect further slowdown and change of course on REMS when FDA gets an Obama general counsel –one of the most under-appreciated but influential appointments in health care </li></ul></ul></ul><ul><li>Larger, Broader Constituency </li></ul><ul><ul><li>Patients, distribution chain </li></ul></ul><ul><ul><li>Positive support for product </li></ul></ul><ul><ul><li>Sponsors cede control </li></ul></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    22. 22. Who and When at FDA <ul><li>Office of New Drugs </li></ul><ul><ul><li>Experienced sponsors say that the review divisions have been leading the REMS effort </li></ul></ul><ul><ul><li>Shows how the post-market tools are quickly morphing into part of the premarket review process </li></ul></ul><ul><ul><li>One division will generally take the lead </li></ul></ul><ul><ul><ul><li>Neurology and GI among divisions with experience </li></ul></ul></ul><ul><ul><ul><li>Opportunity for Division-search </li></ul></ul></ul><ul><li>Office of Surveillance & Epidemiology </li></ul><ul><ul><li>Has acting director of risk management, Claudia Karwoski </li></ul></ul><ul><ul><li>Staffing up but cannot reach authorized levels: 156 people total at OSE (211 authorized) </li></ul></ul><ul><ul><ul><li>Tough competing for “pharmacoepi” people </li></ul></ul></ul><ul><ul><li>OSE has been participating in pre-NDA meetings for 6 years </li></ul></ul><ul><ul><li>Starting to join end-of-Phase II meetings </li></ul></ul>REMS are frequent issues at “Regulatory Briefings” – will establish frequent, uniform use Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    23. 23. Who and When at FDA (II) <ul><li>Drug Safety & Risk Management Advisory Committee </li></ul><ul><li>The Fentora lesson: </li></ul><ul><ul><li>If you are scheduled to take a product before this advisory committee, you better be prepared to present a good risk management plan. </li></ul></ul><ul><li>Eight members </li></ul><ul><ul><li>Including an institutionalized industry gadfly with 30+ years of experience attacking drug products and a MacArthur genius award </li></ul></ul>Sidney M. Wolfe, MD   Expertise: Internal Medicine Term: 08/08/2008 - 05/31/2012 Director Health Research Group of Public Citizen 1600 20th Street NW Washington, D.C. 20009 Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    24. 24. Who and When at Sponsors <ul><li>Celgene’s REMS Teams for </li></ul><ul><li>STEPS and RevAssist </li></ul><ul><li>Risk Management Committee </li></ul><ul><li>Customer Care </li></ul><ul><li>Risk Intervention </li></ul><ul><li>Compliance </li></ul><ul><li>Field Force </li></ul><ul><li>Technical Operations </li></ul><ul><li>Drug Safety </li></ul><ul><li>Senior Management </li></ul><ul><li>Registration </li></ul><ul><li>Prescriber </li></ul><ul><li>Patient </li></ul><ul><li>Pharmacy </li></ul><ul><li>Patient Counseling </li></ul>Surveys Rx Authorization “ Get the right parties in the room, internally, so that you are prepared to have a good discussion externally” Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    25. 25. Tiers of Regulation MedGuides, Follow-up messages for docs, DTC rules Specialties, limited pharmacies, hospitals only Patient-specific-monitoring, lab tests, registries Control is “necessary to ensure that the benefits of the drug outweigh the risks.” Timelines for re-review; Safety labeling authority ( 1) “known serious risk; (2) “signals of serious risk”; (3) “identify an unexpected serious risk.” Copyright © 2007 Windhover. All rights reserved. PM Safe Access Enhanced Communications Labeling & Assessment Mandatory Trials Sponsored By:
    26. 26. Monumental Control Patient Population Few Patients, Tight Control More Patients, Less Control Tysabri, Thalomid, NPlate Entereg, Fentora, Onsolis Xenazine, Remoxy, Embeda Cimzia, Banzel, Vimpat 7 of 11 drugs approved in first six months of REMS era Copyright © 2007 Windhover. All rights reserved. PM Safe Access Enhanced Communications Labeling & Assessment Mandatory Trials Regulatory Control Sponsored By:
    27. 27. REMS Classes <ul><li>Flouroquinolones </li></ul><ul><ul><li>First class of products to get REMS labeling </li></ul></ul><ul><ul><li>Tendon rupture </li></ul></ul><ul><ul><li>Medguides and surveys – surveys of physicians and patients </li></ul></ul><ul><li>Four More Likely </li></ul><ul><ul><li>ESAs – will cut prescribing/volume by a further 10-20% </li></ul></ul><ul><ul><li>Anti-epileptics – better than a black box (?) </li></ul></ul><ul><ul><li>TNF – Enbrel , Raptiva , Cimzia </li></ul></ul><ul><ul><li>Opioids – Fentora and Onsolis leading the way </li></ul></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    28. 28. REMS: The Pioneers New original approvals this year Entereg Cimzia Nplate Promacta Treximet Aplenzin Xenazine PegIntron Rebetol Combo Pack Vimpat Banzel Tapentadol REMS for previously approved drugs Enbrel Advair Diskus Advair HFA Intron A Venlafaxine ER Viramune Ziagen Fluoroquinilones Raptiva Exubera See “The Impact of FDAAA on Drug Approvals” The RPM Report, November 2008 Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    29. 29. By The Numbers FDAAA Impact: NMEs-Novel Biologics March 25, 2008 - Sept. 25, 2008 Total = 11 FDA’s Scorecard: CDER Director Jenkins, Dec. 5, 2008 An Even Distribution Copyright © 2007 Windhover. All rights reserved. Total REMS Approved 21 REMS with Medication Guides only 16 REMS updates with MG only 6 REMS: Communication/safe use 5 Post-Market Requirements 34 PMR as part of approval 24 Safety labeling changes 7 Safety labeling orders 1 Sponsored By:
    30. 30. REMS and an Approval Rebound NME/Novel Biologic Approvals SOURCE: FDC-Windhover, FDA 17 Novel Approvals Since March 25 Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    31. 31. The REMS Ramp Up Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    32. 32. REMS: Tough But Necessary Trade Tougher Regulation More Approvals Entereg and telavancin are examples of products that were stalled. 20 NMEs approved this year, during Safety First. Exceeds 2007. 10%-20% further drop in ESA use Control Fentora off-label use Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    33. 33. <ul><li>Drug Development Nightmare </li></ul><ul><ul><li>Constipation Drug Shows CV Safety Signal </li></ul></ul><ul><ul><li>Off-Target Effect Can’t Be Ruled Out </li></ul></ul><ul><li>Approved With REMS </li></ul><ul><ul><li>Short-Term Hospital Use Only </li></ul></ul><ul><ul><li>$200 Million Market Max </li></ul></ul><ul><li>Not What GSK Wanted… </li></ul><ul><li>… Better Than Nothing At All </li></ul>Entereg: REMS Revives Product Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    34. 34. Entereg : FDA’s Market Control <ul><li>Customer control </li></ul><ul><ul><li>Drop shipment only to registered acute care hospitals </li></ul></ul><ul><ul><li>Sponsor is responsible – GSK/Adolor wanted to make wholesalers responsible </li></ul></ul><ul><li>Market Definition </li></ul><ul><ul><li>Sponsors had to identify full extent of hospital market and trained surgeons to meet FDA’s demands for metrics </li></ul></ul><ul><ul><li>FDA had one motive: statistical accuracy </li></ul></ul><ul><ul><li>But sponsors’ portrait of market gives agency (and investment community) a baseline for judging future use of the product. </li></ul></ul><ul><li>Reports on Off-label Use </li></ul><ul><ul><li>“ Extent of use in non-bowel resection procedures” </li></ul></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    35. 35. Tysabri : PML Publicity Protection <ul><li>PML Cases Roil Wall Street </li></ul><ul><li>Non-Event For FDA </li></ul><ul><li>Agency Confident Program Appropriately Warns Patient of Risk </li></ul><ul><li>And It Has ALL Reports </li></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    36. 36. <ul><li>Survives ‘05 market withdrawal </li></ul><ul><li>Re-enters market with restrictive TOUCH RiskMAP (mid-’06) </li></ul><ul><li>Adds new indication in different specialty (GI) at the nadir of FDA’s approval policy (late ’07) </li></ul><ul><li>Survives three subsequent cases of PML: two in Europe; one in US. (’08) </li></ul><ul><li>Still growing (37,000 patients worldwide) towards 100,000 target in 2010. </li></ul>Tysabri: The Midas TOUCH Initial Marketing: 7,500 Return to Market: 8,313 MS and Crohn’s: 19,500 NEW DEFINITION OF “MIRACLE DRUG” Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    37. 37. Information or Control: EU vs. US <ul><li>Biogen believes Tysabri offers real-world test of risk management techniques </li></ul><ul><ul><li>In EU, Biogen risk management is primarily “education-based” </li></ul></ul><ul><ul><li>In US, the company is working on a tight registration system </li></ul></ul><ul><li>Mandated close tracking allows Biogen to compare incidence and outcomes more precisely and convincingly </li></ul><ul><ul><li>If outcomes are similar, EU might be an argument to step back from current high-level of control </li></ul></ul><ul><ul><li>Will help define which elements are most important </li></ul></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    38. 38. Signs of Things to Come: Threats <ul><li>Marketed Under a Risk Management Plan </li></ul><ul><ul><li>Extensive off-label use (80%) </li></ul></ul><ul><ul><li>Application rejected to expand label </li></ul></ul><ul><li>FDA Asks Cephalon to Demonstrate Effectiveness of REMS </li></ul><ul><ul><li>Stop off-label use as a precondition to regaining current market </li></ul></ul><ul><li>Opening for competitors? </li></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    39. 39. Signs: Opportunities <ul><li>Nplate Requirements </li></ul><ul><li>Three registry studies </li></ul><ul><li>One post-market clinical trial </li></ul><ul><li>Three levels of registration </li></ul><ul><ul><li>Institutions – must have protocols for Nplate use </li></ul></ul><ul><ul><li>Physicians – patient monitoring schedule for weekly complete blood counts </li></ul></ul><ul><ul><li>Patients – enrolled patients must attest to understanding Medguide, carry a Patient ID card and a dosing tracker </li></ul></ul><ul><li>Protection from CMS </li></ul><ul><li>CMS proposes a national coverage decision for the new drug </li></ul><ul><li>CMS ready to act prior to FDA approval </li></ul><ul><li>Amgen has felt the impact of CMS limitations on Aranesp and Epogen </li></ul><ul><li>Amgen says: Safety is not an issue. We have a REMS as part of FDA approval. </li></ul>Copyright © 2007 Windhover. All rights reserved. Sponsored By:
    40. 40. Q & A <ul><li>Arnold Friede - [email_address] McDermott Will & Emery LLP http://www.mwe.com </li></ul><ul><li>Cole Werble - [email_address] The RPM Report http://therpmreport.com </li></ul>www.clinipace.com Sponsored By:

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