Presentation on theme: "BME-IDEA Workshop, September 28, 2005"— Presentation transcript:
1 BME-IDEA Workshop, September 28, 2005 Fostering Innovation on the Critical Path to Medical Device DevelopmentBME-IDEA Workshop, September 28, 2005Donna-Bea Tillman, Ph.DDirector, Office of Device EvaluationCenter for Devices and Radiological Health
2 FDA Mission: Historical Basis Protect the public from unsafe productsI’d like to start my talk by looking back at the historical roots of FDA, which trace back to a public health protection problem.107 people, mostly children, died when sulfanilamide, the first “wonder drug” was formulated into an elixr containing the toxin ethylene oxide. Exisiting laws did not require the drugs manufacturer to test the formulation for safety before it was sold.Congress corrected this weakness in the law the next year when it passed the Federal Food, Drug, and Cosmetic Act. This law, for the first time, required companies to prove the safety of new drugs before putting them on the market.
3 Over the next hundred years, Congress continued to give FDA new responsibilities , including the requirement that drugs and medical devices be proven effective as well as safe before they can be sold.This is the only painting in the series wherein Perez caricatures a recognizable, well-known doctor-former U.S. Surgeon General, C. Everett Koop. Dr. Koop receives this honor because Jose Perez has been so impressed by Koop's leadership in national and global public health. God bless our unheralded public-health doctors.
4 FDA Mission Today Promote Protect Today, FDA’s mission has broadened beyond public health protection to also include public health promotion – ensuring the availability of new products that promote the health of the American people.When most people think of FDA, they think of our public health protection role:-Historical roots of FDA-Methods well understood-Good business – basis of public confidence in medical productsHowever, many of FDA’s most difficult challenges lay in the public health promotion arena.
5 The world today… Technological changes occur at lightning speed Medical devices are more complex than everThe Internet is the new “snake oil” salesmanThe American public is demanding great control over healthcare decisionsThe global marketplace means global regulation
6 Technology trends Miniaturization Smart devices Minimally invasive Biotechnology revolutionCombination ProductsHome useSpecial PopulationsNew technology is smaller, uses computer intelligence, and allows less invasive procedures. New materials, sometimes tissue derived and combined with pharmaceuticals create new combination products with features of both a drug and a device.Some of these innovations are referred to as “disruptive technologies” because they change the way we do business and they change how medical devices deliver value.Consider if a hand held ultrasound could be added to the stethoscope as a tool for your doctor. No longer would your doctor thump your back, or poke at your liver, or listen to your neck. Your doctor could easily see if you have gall stones, carotid artery narrowing, a bicuspid aortic valve, a pleural effusion, ascities, a renal cyst, or fibroid uterus. All this in about the same time as a normal exam, and only requiring a referral to the radiologist for more detailed or specialized images.
7 Disruptive Technologies That change the nature of medical devicesThat change how medical care is deliveredThat change health outcomes, hopefully for the better…that change how the industry and FDA do business.
8 Challenges to Innovation Basic biomedical science investment & progress has surpassed medical product development investment and progress
9 Challenges to Innovation We are using the evaluation tools and infrastructure of the last century…to develop this century’s advances.
10 Challenges to Innovation This has resulted in a bottleneck at the “critical path” for delivering new products to patients
11 Critical Path Research Leverages basic science knowledgeLeverages cumulative research experiencesDoes not compromisesafety and effectivenessevaluations
12 Critical Path for Medical Devices MarketApplicationApprovalBasicResearchPrototypeDesign orDiscoveryPreclinicalDevelopmentClinicalDevelopmentFDA Filing/Approval &LaunchPreparationCritical PathThe journey from medical product candidate to full-scale production and marketing
13 Why is FDA interested?Because of the significant benefit of bringing innovative products to the public fasterBecause of our unique perspective on product development -- we see success, failure and missed opportunitiesBecause it will help us to develop guidance and standards that foster innovation and improve chances of success
14 What does FDA want to accomplish? Work together with industry, academia and patient care advocatestomodernize, develop and disseminate solutions (tools) to address scientific hurdles impacting industry-wide product development.
15 What are the Critical Path tools? The methods and techniques used for:Assessment of Safety – how to predict if a potential product will be harmful?Proof of Efficacy - how to determine if a potential product will have medical benefit?Industrialization – how to manufacture a product at commercial scale with consistent quality?
19 Devices are Different: Development Complex componentsDesignedDrugsPure moleculesDiscoveredHow do devices differ from drugs? What are the challenges that must be met to assure that such products are safe and effective?Drugs are chemically synthesize pure molecules while devices complex products assembled out of many components.Drug toxicology establishes the pharmacologic problems that occur from metabolites or in special situations such as pediatrics or pregnancy, while we examine devices from an engineering perspective, looking at issues ranging from mechanical integrity, to electrical safety and biocompatibility.
20 Devices are Different: Life-cycle Short product life-cycleDurable equipmentDrugsLong market lifeShort half-lifeHow do devices differ from drugs? What are the challenges that must be met to assure that such products are safe and effective?Drugs have long market lives, protected by patents which can be extended by FDA approval, while Devices have rapid product cycles with continuous incremental improvement.Drugs have short half lives while devices are durable equipment, often still in use long after manufacturing has ceased.
21 Devices are Different: Adverse Events DrugsDrug interactionsWrong drug/wrong doseDevicesMalfunctionUser errorHow do devices differ from drugs? What are the challenges that must be met to assure that such products are safe and effective?With drugs we worry about drug interactions, name confusion, dosage errors, while with devices we worry about mechanical failure and malfunction and problems with user errors.
22 Devices are Different: Regulatory Requirements DrugsClinical trialClinical endpointsDevicesRisk-basedSurrogate endpointsHow do devices differ from drugs? What are the challenges that must be met to assure that such products are safe and effective?Drugs are almost always studied clinically while over 90% of devices are studied at the bench for performance characteristics. Clinical trials for drugs almost always involved clinical endpoints. Devices frequently have well-understood mechanisms of actions, so surrogate endpoints are frequently used.
23 Risk-Based Classification of Medical Devices Class I: simple, low risk devicesGeneral controlsMost exempt from premarket submission8% class III, 46% class I, 46% class II-substantial equivalence to a legally marketed device-decision usually based on descriptive information, sometimes bench or animal, rarely clinical data.Determination of risk based on technology and intended use. These two aspects must be assessed in concert.
24 Risk-Based Classification of Medical Devices Class II: more complex, higher riskSpecial controlsPremarket Notification [510(k)]Substantial equivalence10-15% require clinical dataPerformance testing8% class III, 46% class I, 46% class II-substantial equivalence to a legally marketed device-decision usually based on descriptive information, sometimes bench or animal, rarely clinical data.Determination of risk based on technology and intended use. These two aspects must be assessed in concert.
25 Risk-Based Classification of Medical Devices Class III: most complex, highest riskData “soup to nuts”Premarket Application [PMA]Establish safety and effectivenessBench - Animal - HumanMay include post-approval study requirements8% class III, 46% class I, 46% class II-substantial equivalence to a legally marketed device-decision usually based on descriptive information, sometimes bench or animal, rarely clinical data.Determination of risk based on technology and intended use. These two aspects must be assessed in concert.
26 Critical Path is Different for Devices Device RegulationLeast Burdensome Provision of FDAMAQuality Systems and Design ControlsDevice Innovation ProcessBiocompatibilityIterative ProcessUser learning curvePerformance and durabilityDevice Industry is Represented by Small Manufacturers
28 Medical Device Critical Path Projects of Interest Validation of biomarkersBlood panel to assess sensitivity and specificityPeripheral vascular stentsComputer models of human physiology to test and predict failure (before animal and human studies)Intrapartum fetal diagnostic devicesClear “Regulatory Path” -- with consensus from the Obstetrics community)
29 Medical Device Critical Path Projects of Interest Permanently implanted devicesPractice guidelines for appropriate monitoring -- with medical specialty organizationsNeural tissue contacting materialsExtent of neurotoxicity testing
30 Are you interested? Web Address Open DocketDocket # 2004N-0181CDRH webpage (under news and events) provides links to the critical path white paper and docket
31 Questions? One does what one is; one becomes what one does. Robert von Musil