Presentation on theme: "Don’t take my word for it"— Presentation transcript:
0 Is FDA failing us Or are we failing FDA? Jonathan C. Javitt, M.D.,M.P.H Senior Fellow, Potomac Institute for Policy Studies Former Chair, Health Subcommittee, President’s Information Technology Advisory Committee,
1 Don’t take my word for it Our [FDA’s] approach to risk management includes finding ways to help make sure that drugs we review are used safely once they’re approved.Too often, the drugs, devices, and other products we regulate are involved in preventable adverse events.As many as 20% of Americans have experienced some kind of medical error.Preventable errors and complications involving prescription drugs alone are responsible for thousands of deaths, millions of hospitalizations, and billions of dollars in additional health care costs each year, not to mention the unnecessary suffering.Mark McClellan, M.D., Ph.D.
2 A seemingly vicious circle Too few NME’s will be approved this yearThe cost of bringing new drugs to market is far too high and takes too longCongress and the public have labeled FDA’s drug safety process as fundamentally flawedThe more pressure that is brought to bear on FDA to improve drug safety, the more risk averse the agency becomesNobody ever got fired from FDA for witholding approval on safety grounds
3 The other side of the coin The real threat to American lives is not dangerous drugs, but rather errors in the use of generally safe and effective drugsMore Americans are harmed by their failure to receive a needed drug than will ever be harmed by administration of a harmful drugThe problem lies in the disconnect between postmarketing surveillance, pharmaceutical risk management, and clinical practice
4 Yesterday’s answer anecdotal reporting Today’s questionWhat is the best way to ensure public safety once drugs are introduced to the marketplace?Yesterday’s answer anecdotal reportingToday’s answer database analysisTomorrow’s answer sentinel systems
5 A Unified Sentinel System: transforming a vicious circle into a virtuous one A Sentinel SystemContinuously monitors all available sources of medical information in order to identify:Common medical errorsDrug adverse events and interactionsFailure to comply with careThreatening clinical parametersOpportunities to improve care
6 GAO Recognizes the problem However the proffered solutionwill add enormous cost to the regulatory process and provide far less benefit than might otherwise be achievedGAO does realize, however, that anecdotal reporting databases for drug complications are inherently flawed and that population surveillance is needed
7 Drug Safety Health Plan Records Seen as Cost-EffectiveW ay to Identify Risks of Drugs on Market Analyzing large health plan databases to detect drug side effects would be a more cost-effective way to monitor drug safety than the risk-minimization plans proposed in a Senate bill (S. 484), a former head of the Food and Drug Administration said recently.Mark McClellan, the former commissioner of FDA, said that he supports S. 484, the drug-safety legislation sponsored by the chairman and ranking member of the Senate Health, Education, Labor, and Pensions Committee, calling it "the right foundation to build on." However, he and others who spoke March 21 at a Capitol Hill briefing cast doubt on whether the legislation makes use of the best type of postmarket drug-safety surveillance
8 As Safety concerns mount, sponsors have three basic alternatives 10,000 – 100,000 person safety trials prior to market approval Cost prohibitiveConstruction of ad-hoc registry programs for each newly launched drug Extremely expensiveDeploy Sentinel Database systems in order to monitor drug safety and utilization $.50 pmpm
9 The drug safety paradigm must change While reporting databases can, and should be used to report rare, life-threatening events, moving to active surveillance systems enables us to:Actively keep drugs safe in the marketplaceImprove patient safety by identifying those who would benefit from therapies they are not receivingPotentially launch new drugs much more quickly into a monitored environment
10 The Same Framework that addresses the most critical problems confronting patient care, also addresses the challenges facing postmarket surveillance and drug approval
11 The Active Health Care Engine® Hospital ClaimsLab ResultsPharmacyClaimsProvider ClaimsRN Notes16 millioncovered livesLettersMD / RN ReviewCClinical RulesCommunication PortalClinical Review System7. Action by Doctor and PatientAutomated4. Apply Medical Intelligence5. Create Recommendations6. Communicate Recommendations
12 Can deployed Sentinel Systems save lives and save cost? 100,000 member midwestern managed care planSelected all members with at least one physician visit and one pharmacy claim in preceeding 12 months20,000 members selected per groupRandom allocation to Sentinel system with mailed clinical prompts or to watchful waitingStudy period of 12 months
14 Randomized Prospective Study: Approximately 1,000 interventions issued in the intervention group and a similar number observed in the control groupControlStudyDifferenceTotal Admissions105293611%*Admits/100047.5542.1Hospital Bed-Days total4089348315%*ALOS3.89 days3.72 days4.5%*Days per 1000184.8156.915%**Total paid$31.3 MM$29.6 MM5.5%**Inpatient paid$4.6 MM$4.1 MM11%**Overall cost saving of 6% - 8%* p<.03 ** p<.005
15 Sentinel Systems in Postmarketing Surveillance Reinventing the concept of drug safetyKeeping drugs safe in the marketplace through early detection of adverse eventsEnables early interventionEnables early implementation of monitoringEnables early labeling changesKeeping drugs safe in the marketplace by avoiding interactions and proactive notifying caregiversKeeping drugs safe by ensuring clinical monitoringKeeping patients safe by increasing compliance with therapyKeeping patients safe by identifying those in need of unprescribed therapy
16 Sentinel Systems will actually Increase appropriate sales of lifesaving drugs Half of those who would benefit from beta blockers post MI are not receiving themHalf of those who qualify for ACE inhibitors under the HOPE trial criteria do not receive themAnd so on…
17 Keeping Drugs Safe in the Marketplace When Rezulin was approved for the treatment of diabetes, the possibility of liver failure was well recognized and all involved were confident that with proper laboratory monitoring patient safety could be assuredWhen the first (predictable) cases of liver failure were reported, FDA determined that only 9% of physicians were following the rezulin label and obtaining LFT’s. Therefore the drug was withdrawn on the grounds that safety could not be maintained in the marketplace
18 Frequency of pharmaceutical risk management recommendations: Clinical IssuePatients (N)Triggering (%)Need for LFT’s on Rezulin15513%Need renal monitoring on Metformin32919%Need for INR’s on Coumadin1537%Need for LFT’s on Statins93110%In other words, 87% of physicians in this sample were monitoring their Rezulin patients (not 9% as FDA thought) and those who were not monitoring frequently complied with a single reminder letter.
19 Proven Results Sentinel systems are currently workable and deployable Sentinel systems can play a direct role in pharmaceutical risk managementSentinel systems suggest action that Physicians will accept and implementThe costs of sentinel systems are more than offset by savings in health care
20 Are Sentinel Systems merely a Dream? 25 Million Americans already say NO!However, these systems were engineered only for the purpose of patient safety and care improvementThe next phase is the RHIO – Regional Health Information ExchangeFirst Gateways: 20 million lives this yearIndiana: 5+ million livesNew England: 5+ million livesRHIOs are being engineered primarily for transportability of health information, driven by health systems economy, with patient safety as a secondary opportunity and postmarket surveillance not even a major topic of conversation.
21 The RHIO environmentDraws together all known diagnoses, procedures, admissions, labs, and increasingly other findings under a common data umbrella.Privacy and security issues have been legislated and ironed out.Provides the ideal environment for post-market surveillance, in addition to patient safetyThe problem is the business model to support RHIOs – right now they are grant funded
22 Business Case for RHIOS Today’s case1 in 5 diagnostic tests is performed simply because the results of previous tests are not available during the time frame of the medical decision1 in 8 hospital admissions is triggered by events resulting from failure of information flow in the outpatient environmentTomorrow’s caseNew drugs will come to the market soonerDrugs will be less likely to be withdrawn from the market because of unexpected and unmanageable toxicitiesPatients will be more likely to be offered critical, lifesaving drugs
23 The future opportunity Reallocate current resources spent on episodic reporting to supporting RHIO initiativesCreate a structure for earlier monitored drug launches in a RHIO environment with user fees paid by the sponsorsInstead of requesting a 10,000 person premarketing safety study, the safety results from 100,000 patients can be obtained in a RHIO environment at far lower costReplace single product risk management programs with population based programs that work through and simultaneously support the evolving RHIO structure
24 Cost EffectivenessSentinel systems cost approximately $10 per person / year to implementCost represents < 0.5% of overall health care costsDirect savings exceed costs at least 10-fold, not including the benefits to industry and FDA of safer drugs, maintained in the marketplaceExtrapolation of these implications to the nation suggest that a very modest level of IT investment (i.e. not universal EHR’s) could readily save the country in excess of $70 Billion/yr
25 Mark McClellan, MD, PhD Testimony to the Think about the potential for doing better, more real-world clinical studies at a much more affordable cost. For example, post-approval studies could be much cheaper and easier to do, using clinical protocols administered over the internet via electronic records, with patients selected and results recorded directly from electronic medical records. Low-cost clinical trials to be performed more quickly and easily in real-world medical settings. And we can use the signals we obtain from our better surveillance system to work with health care providers, funding agencies, and health care payers to target these studies more effectively.Mark McClellan, MD, PhD Testimony to thePresident’s Information Technology Advisory Committee
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