Current Trends at FDA: Implications for Data Requirements

Save this PDF as:
 WORD  PNG  TXT  JPG

Size: px
Start display at page:

Download "Current Trends at FDA: Implications for Data Requirements"

Transcription

1 Introduction The environment surrounding medical device regulation in the United States has always been rigorous, but recent events including well-publicized quality issues associated with implantable medical devices have raised the scrutiny to which devices are examined prior to product approval and monitored for performance post approval. European requirements are undergoing a similar change in rigor as evidenced by the change from Directive to Regulation. The safety of medical products should be paramount in any determination regarding whether a product should be marketed. The US FDA is charged with evaluating safety and effectiveness as part of its mission to ensure the safety of public health as well as fostering access to new and innovative therapies. The current FDA climate requires significant data and scientific evidence to support approval for commercialization of new device on the US market. This article is intended to describe the types of data required by FDA and provide recent examples of how FDA is interpreting the regulations differently than in the past. Clinical Data to Support FDA Submissions FDA is requiring supportive clinical evidence for many more submissions, including both PMAs and 510(k)s. The PMA (premarket approval) route to market is typically reserved for the highest risk medical devices and requires a product be proven safe and effective on its own merit, whereas the 510(k) route to market allows moderate risk products to leverage experience from a product that has been on the US market. A 510(k) submission must demonstrate that the new product is substantially equivalent to another product that has been on the market since before 1976, when the medical device regulations were implemented. The need for supportive clinical evidence is a relatively new trend for 510(k)s that appears to have been triggered by heightened public scrutiny of this route to market. As a result, FDA reviewers are less willing to accept bench testing or logic to demonstrate substantial equivalence of the new device to the predicate. In addition, the amount of clinical evidence required to support US FDA submissions is increasing. FDA expects well-controlled clinical trials that minimize typical sources of bias and compliance with good clinical practice (GCP) standards, including a formal protocol defining the study specifics, ensuring adequate protection of human subjects, and establishing standard operating procedures governing processes to control all aspects of study performance, data integrity, and data reporting. Hierarchy of Clinical Data When considering the need for clinical data to support FDA submissions, the randomized controlled trial is the gold standard. This type of trial offers the most credible data because the design ensures that most sources of bias are minimized. In this type of design, all subjects meet the same inclusion and exclusion criteria, are treated and followed at the same centers by the same health care professionals, during the same time period. Therefore, the only real difference between the standard of care group and the investigational therapy group is likely the treatment itself or statistical chance. 1

2 While the randomized controlled trial results in the most credible data, there are times when it might not be feasible, or ethical to randomize subjects. For example, when companies were developing endocardial leads for use with implantable cardioverter defibrillators (ICDs), there were physicians who felt it was not ethical to perform a thoracotomy to implant subjects with epicardial leads (which are placed on the outside of the heart through an open heart procedure) when those subjects could otherwise be treated with the endocardial lead (which is placed via an intravascular approach). Although the performance of the endocardial lead was unproven at the time of the study, there was sufficient experience with implantation of this type of lead with pacemaker devices and furthermore, testing of the lead was performed with each ICD procedure to ensure its effectiveness for its intended use. In this situation, credible data could be collected at clinical sites using the investigational endocardial leads with comparison to clinical sites using the standard of care epicardial leads. All subjects would meet the same inclusion and exclusion criteria as defined by the protocol, the same type of data would be collected from all subjects, and all subjects would be followed in the same manner. This type of study design is considered a prospective controlled trial. The sources of bias introduced in this type of study include the potential for site treatment or follow-up differences, statistical chance and, of course, differences due to the treatment. In the hierarchy of clinical evidence, the next most credible data would come from prospectively collected data from a single arm study compared to retrospectively collected data from the same subject, or matched control data selected from an available historical data set. In this type of study design, the control group data would be available in line listing form so FDA could reproduce the data analyses. The sources of bias introduced in this type of study design include differences in time and treatment standards, differences in site treatment or follow-up, statistical chance and of course differences due to the treatment. Moving lower on the clinical data hierarchy, then, would be single arm studies compared to historical data from literature reports. Literature represents some difficulties for comparison purposes because non-devicerelated and non-procedure-related safety issues are typically not reported in a manner similar to current clinical practices. Therefore, it is likely that differences will be seen in safety measures. Also, bias could potentially be introduced from the same sources identified previously. Note that although these data have the potential for bias, the data still may represent valid scientific evidence, and may be very useful for publication or for the advancement of scientific knowledge. Similar clinical evidence can be collected from data registries and meta-analyses from literature reports. These sources of clinical data again offer scientific evidence, but because sources of bias have not proactively been controlled, the level of credibility is less than what is typically required by FDA. To address FDA s need for higher levels of valid scientific evidence, we recommend that Sponsors design a clinical study to provide the highest level of credible clinical data as possible, balancing the need to be practical for clinical investigators. At study completion, highly credible data is a very valuable tool for regulatory purposes, for reimbursement purposes as well as for marketing purposes. 2

3 Statistical Data to Support FDA Submissions Another emerging trend in FDA review of submissions is the need to demonstrate statistical significance to prove clinical effectiveness of a new treatment. The reviewing clinicians at FDA rely on the statisticians to interpret the data and justify statistically whether the stated claims and intended use can be supported by the endpoints and analyses of the resulting data. During the clinical study design phase, objectives, performance endpoints, and the statistical sample size are determined a priori based on expected assumptions. If after the study is completed a statistical endpoint is not met, it may be because there was not enough subjects studied, or the assumptions used to develop the statistics might have been flawed or the product might not perform better than the standard of care. In these cases if a statistical justification cannot be supported the FDA and their medical reviewers will try to assess if the data support a clinical justification. The current trend in FDA reviews is that clinical justifications are generally insufficient in the absence of the statistical support. FDA and FDA s Advisory Committees have requested additional clinical studies be conducted, often with a new design or new assumptions in cases such as these. Even when FDA is involved in providing input into the clinical study design before the study is initiated, there is, of course, no guarantee that the study will yield statistically significant results upon its completion. There have also been cases where a study did meet its defined endpoints, but in the final analysis FDA and its medical reviewers felt that the defined endpoint was not sufficiently clinically relevant and required further confirmatory studies. Therefore, it is important to work with knowledgeable clinicians to understand the expected performance of the new therapy, determine conservative statistical assumptions, and ensure FDA is supportive of the study design. A pilot study is often helpful in providing early evidence upon which to base performance and statistical assumptions. These efforts will be helpful in determining the best possibility of success for the study for all parties. The Importance of Intended Use The key element when developing the FDA strategy is the Intended Use, or the statements made in the labeling regarding how the product is intended to be used by the healthcare professional. It is this language that will drive the evidence FDA will need to support an approval/clearance decision for commercialization of the product in the US. For example, a device filter on a cardiovascular catheter intended to prevent thrombus from embolizing to prevent stroke would require a significant clinical trial to demonstrate a difference in stroke events in a population of subjects with the filter versus a population of subjects treated without the filter. The same device filter intended for use in preventing embolization to the extremities and limbs would require a different study. The same device filter used with a cardiovascular catheter for general infusion use might only require bench data. The Intended Use statement is also a critical element in determining the regulatory route to market, particularly whether a product can be authorized for market via a PMA or a 510(k) route. Where a similar product is already on the market and is classified as Class II (falling into a moderate risk category), a new product can follow the 510(k) path by using an identical Intended Use statement as that of the predicate device. New Intended Use statements will automatically eliminate the option to pursue a 510(k), requiring a PMA path for high risk devices or a new De Novo process for moderate and low risk devices. 3

4 De Novo Process Until earlier this year, FDA law required that any new devices that did not fall into the 510(k) category were automatically classified as Class III requiring a PMA. US regulations, however, acknowledged that some of these devices would be low to moderate risk and as such did not need to be subject to the high level of scrutiny and resource requirements for a PMA submission. FDA issued a guidance document that allowed an alternative to the automatic Class III designation which was called a de novo application. In these cases, sponsors submitted a 510(k), received a not substantially equivalent decision, and within 30 days submitted a de novo application providing information to demonstrate that the product presents only a low to moderate risk to public health. The de novo process also required FDA to issue a guidance document for future devices that will use the subject device as a predicate. When FDA granted a clearance decision, the product was placed in Class II. Earlier this year, US Congress passed a new law that codified the de novo process and made it a single step submission no longer requiring the first step of a standard 510(k) application. It remains to be seen what FDA will expect to see as part of the submission to incorporate the information typically included in a 510(k) as well as the risk/benefit assessment. But it is clear that FDA is being very strict about 510(k) predicates and intended use language, thus sending more devices through the de novo process. FDA does prefer that companies who plan to pursue the de novo pathway request a pre-submission meeting to discuss the available data and allow FDA an opportunity to comment on information they would expect to see in a de novo application. Companies should be prepared to present their plans rather than expecting FDA to make recommendations. This pre-submission process can require 2-3 months to schedule and conduct the meeting, but the feedback can be valuable in understanding FDA s expectations when topics are controversial or it is unknown whether FDA will accept a proposed plan. FDA will often expect clinical data in support of de novo applications, particularly in situations where the intended use has been expanded beyond that of a predicate product. For example, a product that has a general indication, but for which a specific indication is being sought, would need clinical data support for the specific indication. Also, if a product has significant differences in technological characteristics from a predicate product, clinical data may also be required to demonstrate the safety profile of the product with those differences. The de novo submission process, although new, offers a faster alternative route to the US market compared to the PMA submission. Companies need to be aware of the requirements and solicit feedback from FDA to meet their expectations, in order to find the shortest path through the regulatory hurdles. Leveraging Other Sources of Data In the product development process overall, many types of data are generated including bench and mechanical testing, biocompatibility testing, pre-clinical animal testing, pilot clinical data, clinical data that is collected in specific countries to meet their regulatory or reimbursement needs, health economics data, post-market clinical follow-up and post-approval studies, etc. When developing a US regulatory plan, all types of data should be considered as to what will be useful in demonstrating safety and effectiveness, substantial equivalence or a positive risk/benefit analysis. For a typical 510(k), mechanical and bench data often are sufficient. For new and 4

5 novel products, FDA typically requires a small pilot study before allowing progression to a large scale pivotal trial. In many cases, companies can leverage clinical experience from outside the United States in lieu of a pilot study. The EU and FDA are now requiring post-market surveillance monitoring which usually consist of passive data collection on reported adverse events (e.g., MDR and vigilance reporting) and active data collection in the form of post-market studies and clinical follow-up. Additionally, third-party payors are requesting post-approval data to demonstrate that the product continues to perform as expected when used in all patients, in all healthcare settings. Companies should assess whether a single study design can be used to collect data to serve all the post-market clinical needs for US, EU and reimbursement purposes. Significant cost savings can be realized by setting up and conducting one study meeting many needs instead of managing multiple studies. Conclusions FDA s interpretation of the regulations is changing and has resulted in heightened requirements for clinical data and statistical relevance. Where FDA previously exercised some flexibility in evaluating Intended Use language and determining the regulatory route to market, they are now taking a strict view and holding firm to a narrow interpretation of the regulations. As such, companies wishing to enter the market in the US should develop a regulatory plan that includes sufficient data support and specifically, clinical evidence to support stated claims for the product. These requirements are not just new for the US market, similar requirements are being mandated for European regulatory needs, but are also useful for reimbursement and marketing as well. A well-designed, well-conducted clinical study can be leveraged for multiple purposes, but will particularly facilitate the submission process in the US when the data are supportive. Investment in clinical evidence to support regulatory applications in general and US applications in particular is funding well-spent, especially in light of FDA s current environment. 5

Planning for Successful Medical Device Reimbursement:

Planning for Successful Medical Device Reimbursement: Planning for Successful Medical Device Reimbursement: So Your Device Is Cleared, By Tiffini Diage, MPH Health Economics NAMSA White Paper Key Considerations for Targeting Success of Medical Device Sales

More information

PHARMA CONGRESS. Pharmacovigilance and Drug Safety: Assessing Future Regulatory and Compliance Developments. October 28, 2008

PHARMA CONGRESS. Pharmacovigilance and Drug Safety: Assessing Future Regulatory and Compliance Developments. October 28, 2008 PHARMA CONGRESS October 28, 2008 Pharmacovigilance and Drug Safety: Assessing Future Regulatory and Compliance Developments Beverly H. Lorell, MD Senior Medical & Policy Advisor King & Spalding LLP Assessing

More information

Medidée Services SA. Nano-Tera.ch. 05 February 2015 part 8. PMA, 510k, IDE. Pierre-Alain Sommer

Medidée Services SA. Nano-Tera.ch. 05 February 2015 part 8. PMA, 510k, IDE. Pierre-Alain Sommer Nano-Tera.ch 05 February 2015 part 8 PMA, 510k, IDE Pierre-Alain Sommer Pierre-alain.sommer@medidee.com www.medidee.com Nano-Tera 2015 05.02.2015 USA/FDA Pre Market Approval System - PMA, Pre Market Notifcation

More information

Role of the WHO IVD Prequalification Programme in Light of National Regulatory Authority Approval

Role of the WHO IVD Prequalification Programme in Light of National Regulatory Authority Approval Role of the WHO IVD Prequalification Programme in Light of National Regulatory Authority Approval UN Prequalification of Medicines, Diagnostics and Vaccines 6 th Consultative Stakeholder Meeting 4 April

More information

Murray Sheldon, MD Associate Director for Technology and Innovation Center for Devices and Radiological Health (CDRH) Office of the Center Director

Murray Sheldon, MD Associate Director for Technology and Innovation Center for Devices and Radiological Health (CDRH) Office of the Center Director Murray Sheldon, MD Associate Director for Technology and Innovation Center for Devices and Radiological Health (CDRH) Office of the Center Director The National Academies Innovation Policy Forum September

More information

Medical Device Development Tools. Draft Guidance for Industry, Tool Developers, and Food and Drug Administration Staff

Medical Device Development Tools. Draft Guidance for Industry, Tool Developers, and Food and Drug Administration Staff Reprinted from FDA s website by Medical Device Development Tools Draft Guidance for Industry, Tool Developers, and Food and Drug Administration Staff DRAFT GUIDANCE This guidance document is being distributed

More information

De Novo Classification Process (Evaluation of Automatic Class III Designation) Guidance for Industry and Food and Drug Administration Staff

De Novo Classification Process (Evaluation of Automatic Class III Designation) Guidance for Industry and Food and Drug Administration Staff De Novo Classification Process (Evaluation of Automatic Class III Designation) Guidance for Industry and Food and Drug Administration Staff Document issued on October 30, 2017. The draft of this document

More information

Regulatory Pathways. Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB

Regulatory Pathways. Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB Regulatory Pathways Devices vs. Drugs Are there roles for registries? John Laschinger, MD CDRH/ODE/DCD/SHDB johnlaschinger@fda.hhs.gov 1 Disclosures and Disclaimer John C. Laschinger, M.D. I am a full

More information

Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015

Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015 Pediatric-Specific Provisions Summary of Provisions in 21 st Century Cures Act (H.R. 6) as passed by full House of Representatives, July 10, 2015 Requires the NIH to complete a strategic plan, and in the

More information

Post Market Surveillance

Post Market Surveillance Post Market Surveillance December 6 th, 2017 Michael Maier, Medidee Services Slide 1 Agenda Postmarket surveillance Postmarket clinical follow up (PMCF) Incident reporting What changes with MDR Slide 2

More information

An Introduction to the Worldwide Regulatory Framework for Medical Devices. Elizabeth Malo M.S., Director, Regulatory Affairs

An Introduction to the Worldwide Regulatory Framework for Medical Devices. Elizabeth Malo M.S., Director, Regulatory Affairs An Introduction to the Worldwide Regulatory Framework for Medical Devices Elizabeth Malo M.S., Director, Regulatory Affairs Course Outline / Agenda» Overview Device Regulatory Framework» US/EU/Canada Premarket

More information

CDRH Pilot Activities

CDRH Pilot Activities CDRH Pilot Activities Cisco Vicenty Program Manager, Case for Quality 1 Office of Compliance, CDRH November 15, 2017 VCIP Voluntary Compliance Improvement Program Pilot Status Description: Assessment:

More information

Your complimentary Clinica Medtech Intelligence content

Your complimentary Clinica Medtech Intelligence content Your complimentary Clinica Medtech Intelligence content Clinica provides you with up to the minute coverage and opinion on company, product, market and regulatory developments as well as market size, growth

More information

Reimbursement Strategy for Companion Diagnostics:

Reimbursement Strategy for Companion Diagnostics: Reimbursement Strategy for Companion Diagnostics: Emerging Models and Requirements Edward E. Berger, Ph.D. Larchmont Strategic Advisors Definition Companion diagnostic A diagnostic test used to predict

More information

Ethical Issues in Clinical. Forum for Ethical Review Committees in Asia and the Western Pacific. Trials Cristina E. Torres, Ph.D.

Ethical Issues in Clinical. Forum for Ethical Review Committees in Asia and the Western Pacific. Trials Cristina E. Torres, Ph.D. Ethical Issues in Clinical Forum for Ethical Review Committees in Asia and the Western Pacific Trials Cristina E. Torres, Ph.D. Useful Definitions Research means a systematic investigation to include research

More information

INVESTIGATIONAL DEVICE EXEMPTIONS AND EARLY FEASIBILITY STUDIES TO SUPPORT DEVICE INNOVATION

INVESTIGATIONAL DEVICE EXEMPTIONS AND EARLY FEASIBILITY STUDIES TO SUPPORT DEVICE INNOVATION INVESTIGATIONAL DEVICE EXEMPTIONS AND EARLY FEASIBILITY STUDIES TO SUPPORT DEVICE INNOVATION Maureen L. Dreher, PhD Policy Analyst, Clinical Trials Program Office of Device Evaluation/ CDRH www.fda.gov

More information

Technical Guidance on Clinical Evaluation of Medical Devices

Technical Guidance on Clinical Evaluation of Medical Devices Annex Technical Guidance on Clinical Evaluation of Medical Devices I. Purpose The clinical evaluation of medical devices is the assessment procedure conducted by registration applicants to validate whether

More information

Good Clinical Practice (GCP) & Clinical Trial Registries

Good Clinical Practice (GCP) & Clinical Trial Registries Good Clinical Practice (GCP) & Clinical Trial Registries The Fifth Annual Pharmaceutical Regulatory and Compliance Congress and Best Practice Forum November 14-17, 2004 Kate Maloney, RN, MS, CPHQ Manager,

More information

Re: Docket No. FDA-2000-D-0067: Medical Device Patient Labeling; Request for Comments; Public Workshop

Re: Docket No. FDA-2000-D-0067: Medical Device Patient Labeling; Request for Comments; Public Workshop 701 Pennsylvania Avenue, NW Suite 800 Washington, D.C. 20004 2654 Tel: 202 783 8700 Fax: 202 783 8750 www.advamed.org October 30, 2015 Division of Dockets Management (HFA305) Food and Drug Administration

More information

SWOG ONCOLOGY RESEARCH PROFESSIONAL (ORP) MANUAL STUDY PROTOCOL CHAPTER 14 REVISED: OCTOBER 2015

SWOG ONCOLOGY RESEARCH PROFESSIONAL (ORP) MANUAL STUDY PROTOCOL CHAPTER 14 REVISED: OCTOBER 2015 THE STUDY PROTOCOL The study protocol is a written document detailing how a clinical trial is conducted. The elements of a protocol include: 1. Trial design and organization; 2. Study objectives; 3. Background

More information

FDA initiatives to facilitate evaluation of novel percutaneous valve technologies within the US

FDA initiatives to facilitate evaluation of novel percutaneous valve technologies within the US FDA initiatives to facilitate evaluation of novel percutaneous valve technologies within the US Nicole Ibrahim, PhD Chief, Structural Heart Devices Branch Division of Cardiovascular Devices ODE/CDRH/FDA

More information

DRAFT GUIDANCE. This guidance document is being distributed for comment purposes only. Document issued on: December 27, 2011

DRAFT GUIDANCE. This guidance document is being distributed for comment purposes only. Document issued on: December 27, 2011 Draft Guidance for Industry and Food and Drug Administration Staff The 510(k) Program: Evaluating Substantial Equivalence in Premarket Notifications [510(k)] DRAFT GUIDANCE This guidance document is being

More information

Recommendations for Strengthening the Investigator Site Community

Recommendations for Strengthening the Investigator Site Community Recommendations for Strengthening the Investigator Site Community October 2017 CTTI MISSION: To develop and drive adoption of practices that will increase the quality and efficiency of clinical trials

More information

Mark D. Kramer, RAC Regulatory Strategies, Inc.

Mark D. Kramer, RAC Regulatory Strategies, Inc. Mark D. Kramer, RAC Regulatory Strategies, Inc. 808 E. Fox Lane Fox Point, Wisconsin 53217 (414) 731-4257 kramer@regulatorystrategies.net Experience Regulatory Strategies, Inc. Fox Point, Wisconsin President,

More information

Guidance for Sponsors, Institutional Review Boards, Clinical Investigators and FDA Staff

Guidance for Sponsors, Institutional Review Boards, Clinical Investigators and FDA Staff Guidance for Sponsors, Institutional Review Boards, Clinical Investigators and FDA Staff Guidance on Informed Consent for In Vitro Diagnostic Device Studies Using Leftover Human Specimens that are Not

More information

Medical Products between

Medical Products between Proactive Communications about Medical Products between Manufacturers and Payors FDLI Advertising & Promotion Conference September 27, 2017 David J. Bloch Principal Legal Counsel, Corporate Legal Regulatory

More information

Post Market Surveillance (including PMCF): common non compliances

Post Market Surveillance (including PMCF): common non compliances Post Market Surveillance (including PMCF): common non compliances Jayanth Katta Ph.D Product Specialist & Certification Manager, General Devices Team, Healthcare 1 Overview EU PMS Requirements for Medical

More information

Breakthrough Devices Program Draft Guidance for Industry and Food and Drug Administration Staff

Breakthrough Devices Program Draft Guidance for Industry and Food and Drug Administration Staff 1 2 3 4 5 6 Breakthrough Devices Program Draft Guidance for Industry and Food and Drug Administration Staff DRAFT GUIDANCE This draft guidance document is being distributed for comment purposes only. Document

More information

RWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact.

RWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact. Real Real World World Data Data Across Across the the Product Product Lifecycle: Lifecycle: RWE from pre-clinical to launch RWE from pre-clinical to launch Standard of care Unmet needs Disease burden Budget

More information

Disclaimer This presentation expresses my personal views on this topic and must not be interpreted as the regulatory views or the policy of the FDA

Disclaimer This presentation expresses my personal views on this topic and must not be interpreted as the regulatory views or the policy of the FDA On multiplicity problems related to multiple endpoints of controlled clinical trials Mohammad F. Huque, Ph.D. Div of Biometrics IV, Office of Biostatistics OTS, CDER/FDA JSM, Vancouver, August 2010 Disclaimer

More information

The Role of the CRO in Effective Risk-Based Monitoring

The Role of the CRO in Effective Risk-Based Monitoring New Whitepaper The Role of the CRO in Effective Risk-Based Monitoring The clinical trial industry is evolving. In an effort to improve participant safety and data integrity, regulators are encouraging

More information

The In Vitro Diagnostic CRO

The In Vitro Diagnostic CRO The In Vitro Diagnostic CRO Choose Beaufort Because of Our People, Processes and Proven Experience The value of expertise cannot be overstated, especially when it comes to streamlining complicated in vitro

More information

Bayesian Statistics at the FDA: The Trailblazing Experience with Medical Devices

Bayesian Statistics at the FDA: The Trailblazing Experience with Medical Devices Bayesian Statistics at the FDA: The Trailblazing Experience with Medical Devices Greg Campbell, Ph.D. Director, Division of Biostatistics Center for Devices and Radiological Health Food and Drug Administration

More information

THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE

THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE THE FDA, THE DRUG APPROVAL PROCESS, AND THE PATIENT VOICE Ali Mohamadi, M.D. Senior Director, Global Regulatory Patient Engagement and Policy BioMarin Pharmaceutical 30-July-2016 Goals of Today s Presentation

More information

Patient Handbook on Stem Cell Therapies

Patient Handbook on Stem Cell Therapies Patient Handbook on Stem Cell Therapies WWW.ISSCR.ORG WWW.CLOSERLOOKATSTEMCELLS.ORG Patient Handbook on Stem Cell Therapies Introduction We have all heard about the extraordinary promise that stem cell

More information

Experience with Adaptive Dose-Ranging Studies in Early Clinical Development

Experience with Adaptive Dose-Ranging Studies in Early Clinical Development Experience with Adaptive Dose-Ranging Studies in Early Clinical Development Judith Quinlan MSc Vice President Adaptive Trials Cytel Inc. judith.quinlan@cytel.com Thanks to members of the PhRMA Adaptive

More information

Florida State University Policy 7-IRB-

Florida State University Policy 7-IRB- Florida State University Policy 7-IRB- Title of Policy: Institutional Review Board Jurisdiction/Applicability Responsible Executive: Gary K. Ostrander Approving Official: Gary K. Ostrander Effective Date:

More information

Testimony of Christopher Newton-Cheh, MD, MPH Volunteer for the American Heart Association

Testimony of Christopher Newton-Cheh, MD, MPH Volunteer for the American Heart Association Testimony of Christopher Newton-Cheh, MD, MPH Volunteer for the American Heart Association Before the House Energy and Commerce Subcommittee on Health 21st Century Cures: Examining the Regulation of Laboratory

More information

Medical Devices; Immunology and Microbiology Devices; Classification of the Nucleic Acid-

Medical Devices; Immunology and Microbiology Devices; Classification of the Nucleic Acid- This document is scheduled to be published in the Federal Register on 10/16/2017 and available online at https://federalregister.gov/d/2017-22287, and on FDsys.gov 4164-01-P DEPARTMENT OF HEALTH AND HUMAN

More information

Office for Human Subject Protection. University of Rochester

Office for Human Subject Protection. University of Rochester POLICY 1. Purpose Outline the responsibilities and regulatory requirements when conducting human subject research that involves the use of drugs, agents, biological products, or nutritional products (e.g.,

More information

Speed your time to market with FDA s expedited programs

Speed your time to market with FDA s expedited programs Regulatory Sciences Expediting drug approval Speed your time to market with FDA s expedited programs The faster way to marketing submission and drug approval for serious conditions and rare diseases In

More information

Streamlining IRB Procedures for Expanded Access

Streamlining IRB Procedures for Expanded Access Streamlining IRB Procedures for Expanded Access Marjorie A. Speers, Ph.D. Executive Director, WCG Foundation Richard Klein Director, FDA Patient Liaison Program Office of Health and Constituent Affairs

More information

FDA s Center for Devices and Radiological Health: Strategic Priorities for 2017 and Beyond

FDA s Center for Devices and Radiological Health: Strategic Priorities for 2017 and Beyond FDA s Center for Devices and Radiological Health: Strategic Priorities for 2017 and Beyond Jeff Shuren, MD, JD Center for Devices and Radiological Health U.S. Food and Drug Administration May 4, 2017 1

More information

Clinical Trials and Medical Device Innovation in the US New Policies and Trends

Clinical Trials and Medical Device Innovation in the US New Policies and Trends Clinical Trials and Medical Device Innovation in the US New Policies and Trends Laura Mauri, MD, MSc Brigham and Women s Hospital Harvard Clinical Research Institute Medical Technology Innovation Scorecard

More information

SUBJECT: INSTITUTIONAL REVIEW BOARD (IRB) - HUMANITARIAN USE DEVICES

SUBJECT: INSTITUTIONAL REVIEW BOARD (IRB) - HUMANITARIAN USE DEVICES This policy applies to the following entity(s): Children s Hospital of Wisconsin Children s Hospital and Health System, Inc. Administrative Policy and Procedure Children s Research Institute SUBJECT: INSTITUTIONAL

More information

Accelerating Pre-Market Approval for Medical Devices

Accelerating Pre-Market Approval for Medical Devices The National Academy of Sciences The Innovation Policy Forum Medical Devices Innovation: Opportunities, Threats, and Challenges Accelerating Pre-Market Approval for Medical Devices Michael J. Mack, MD

More information

RE: Docket No. FDA 2017 N 0041: Agency Information Collection Activities; Proposed Collection; Comment Request; Safety Assurance Case

RE: Docket No. FDA 2017 N 0041: Agency Information Collection Activities; Proposed Collection; Comment Request; Safety Assurance Case 701 Pennsylvania Avenue, NW Suite 800 Washington, D.C. 20004 2654 Tel: 202 783 8700 Fax: 202 783 8750 www.advamed.org Division of Dockets Management (HFA-305) Food and Drug Administration 5630 Fishers

More information

Guidance for Industry and FDA Staff Procedures for Handling Post-Approval Studies Imposed by PMA Order

Guidance for Industry and FDA Staff Procedures for Handling Post-Approval Studies Imposed by PMA Order Guidance for Industry and FDA Staff Procedures for Handling Post-Approval Studies Imposed by PMA Order Document issued on: [Level 2, June 15, 2009] This guidance supersedes the document issued under this

More information

Compliance Central: CDRH 2017 Trends and 2018 Priorities

Compliance Central: CDRH 2017 Trends and 2018 Priorities Compliance Central: CDRH 2017 Trends and 2018 Priorities CAPT Sean M. Boyd, MPH, USPHS Deputy Director for Regulatory Affairs CDRH Office of Compliance Assessing Benefit-Risk Benefit Risk Other Factors

More information

Policy Position. Pharmacy-mediated interchangeability for Similar Biotherapeutic Products (SBPs)

Policy Position. Pharmacy-mediated interchangeability for Similar Biotherapeutic Products (SBPs) Pharmacy-mediated interchangeability for Similar Biotherapeutic Products (SBPs) Geneva, April 2016 Appropriate use of biotherapeutics including SBPs - SBPs, also known as biosimilars, are developed to

More information

Medical Outcome Trials: Trends, Design Approaches and Operational Strategies for Success

Medical Outcome Trials: Trends, Design Approaches and Operational Strategies for Success Medical Outcome Trials: Trends, Design Approaches and Operational Strategies for Success Good Research Practice (GRP) Webinar Philip Galtry Patricia Steigerwald, MS, RN Copyright 2014 Quintiles Your Presenters

More information

The UDI Experience: From a US Perspective

The UDI Experience: From a US Perspective Simposio Nazionale Bergamo, Italy Saturday 6 May 2017 The UDI Experience: From a US Perspective Lena Cordie Qualitas Professional Services 2017 Qualitas Professional Services, LLC. All rights reserved.

More information

Human Research Protection Program Guidance for Human Research Determination

Human Research Protection Program Guidance for Human Research Determination Human Research Protection Program Guidance for Human Research Determination I.1.A The sole purpose of the Institutional Review Board (IRB), as defined in federal statutes, is the protection of human subjects

More information

A Risk-based Approach for In Vitro Companion Diagnostics Device FDA Approval Process Associated with Therapies that have Breakthrough Designation

A Risk-based Approach for In Vitro Companion Diagnostics Device FDA Approval Process Associated with Therapies that have Breakthrough Designation A Risk-based Approach for In Vitro Companion Diagnostics Device FDA Approval Process Associated with Therapies that have Breakthrough Designation A Risk-based Approach for In Vitro Companion Diagnostics

More information

STATEMENT SANDRA KWEDER, M.D DEPUTY DIRECTOR, OFFICE OF NEW DRUGS CENTER FOR DRUG EVALUATION AND RESEARCH U.S. FOOD AND DRUG ADMINISTRATION BEFORE THE

STATEMENT SANDRA KWEDER, M.D DEPUTY DIRECTOR, OFFICE OF NEW DRUGS CENTER FOR DRUG EVALUATION AND RESEARCH U.S. FOOD AND DRUG ADMINISTRATION BEFORE THE DEPARTMENT OF HEALTH & HUMAN SERVICES Public Health Service Food and Drug Administration Rockville MD 20857 STATEMENT OF SANDRA KWEDER, M.D DEPUTY DIRECTOR, OFFICE OF NEW DRUGS CENTER FOR DRUG EVALUATION

More information

Section I: Pharmaceuticals and Medical Devices

Section I: Pharmaceuticals and Medical Devices SUPPLEMENT on HEALTHCARE INNOVATION Visionary Goals and Recommendations 51th Japan-U.S. Business Conference Japan-U.S. Business Council / U.S.-Japan Business Council November 14, 2014 The R&D-based pharmaceutical

More information

UCI Medical Device Regulatory Awareness - Implications for Start-ups - Copyright 2017 Noblitt & Rueland. Contact Information.

UCI Medical Device Regulatory Awareness - Implications for Start-ups - Copyright 2017 Noblitt & Rueland. Contact Information. UCI Medical Device Regulatory Awareness - Implications for Start-ups - Copyright 2017 Contact Information Mailing Address: 5405 Alton Parkway 5A, #530 Irvine, CA 92604-3718 Phone: (1) 949-398 398-5222

More information

Adaptive Design for Medical Device Development

Adaptive Design for Medical Device Development Adaptive Design for Medical Device Development A guide to accelerate clinical development and enhance portfolio value Executive Summary In May 2015, the FDA released a draft guidance document regarding

More information

For personal use only

For personal use only Australia (ASX: AVH) U.S. (OTCQX: AVMXY) Galatéa de las esferas Salvador Dalí, 1952 Annual General Meeting 22 November 2013 Perth, Australia 2 This presentation may include forward looking statements.

More information

FDA Experience with the Sentinel Common Data Model: Addressing Data Sufficiency

FDA Experience with the Sentinel Common Data Model: Addressing Data Sufficiency FDA Experience with the Sentinel Common Data Model: Addressing Data Sufficiency Michael D. Nguyen, MD Office of Surveillance and Epidemiology Center for Drug Evaluation and Research US Food and Drug Administration

More information

GUIDELINES ON MEDICAL DEVICES

GUIDELINES ON MEDICAL DEVICES EUROPEAN COMMISSION DIRECTORATE GENERAL for HEALTH and CONSUMERS Consumer Affairs Cosmetics and Medical Devices MEDDEV 2.7/4 December 2010 GUIDELINES ON MEDICAL DEVICES GUIDELINES ON CLINICAL INVESTIGATION:

More information

April 13, Background

April 13, Background Pfizer Inc 235 East 42nd Street New York, NY 10017-5755 Tel 212 733 4210 Fax 646 383 9249 Email: marc.wilenzick@pfizer.com April 13, 2009 http://www.regulations.gov Christine Ireland Committee management

More information

Value of Industry Pharmacists (VIP) Case Competition ( ) Competition Guide Version 2.0. This year s competition is sponsored by:

Value of Industry Pharmacists (VIP) Case Competition ( ) Competition Guide Version 2.0. This year s competition is sponsored by: Value of Industry Pharmacists (VIP) Case Competition (2017-18) Competition Guide Version 2.0 This year s competition is sponsored by: 1 Preamble This guide shall serve as the main reference document for

More information

CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016

CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016 CORPORATE NEWS EARNINGS PAION AG PUBLISHES GROUP QUARTERLY STATEMENT FOR THE FIRST NINE MONTHS OF 2016 Positive remimazolam data in pivotal U.S. Phase III study for procedural sedation during colonoscopy

More information

Off-Label Use of FDA-Approved Drugs and Biologicals

Off-Label Use of FDA-Approved Drugs and Biologicals Off-Label Use of FDA-Approved Drugs and Biologicals Last Review Date: January 12, 2018 Number: MG.MM.AD.06cC2 Medical Guideline Disclaimer Property of EmblemHealth. All rights reserved. The treating physician

More information

By: Clay Anselmo COO, DLLS, Founder Reglera and Terry J. Dagnon M.S., Vice President Regulatory Affairs, DLSS

By: Clay Anselmo COO, DLLS, Founder Reglera and Terry J. Dagnon M.S., Vice President Regulatory Affairs, DLSS AN INTRODUCTION TO THE WORLDWIDE REGULATORY FRAMEWORK FOR MEDICAL DEVICES By: Clay Anselmo COO, DLLS, Founder Reglera and Terry J. Dagnon M.S., Vice President Regulatory Affairs, DLSS COURSE OUTLINE /

More information

MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155

MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155 MEDICAL DEVICE CLINICAL INVESTIGATIONS AND ISO 14155 EXECUTIVE SUMMARY Medical device regulations around the world generally require manufacturers of most types of medical devices to supply data as part

More information

Shaping the Future of Biomedical Device Design and Diagnostics. Kristian Debus

Shaping the Future of Biomedical Device Design and Diagnostics. Kristian Debus Shaping the Future of Biomedical Device Design and Diagnostics Kristian Debus Overview Biomedical Device Design: Benchtop and Clinical Trial Support Simulation and the regulatory system ASME V&V 40, MDDT,

More information

Technical Guidance on Clinical Evaluation of Medical Devices 1

Technical Guidance on Clinical Evaluation of Medical Devices 1 Technical Guidance on Clinical Evaluation of Medical Devices 1 I. Purpose The clinical evaluation of medical devices is the assessment procedure conducted by registration applicants to validate whether

More information

Expanded Access to Investigational Imaging Drugs

Expanded Access to Investigational Imaging Drugs Expanded Access to Investigational Imaging Drugs Phillip B. Davis, MD FDA/CDER/ODEIV/DMIP 6/09/2016 1 Overview Expanded Access (EA) Defined Requirements for all EA authorizations Types of EA Individual

More information

EU scientific regulatory support mechanisms and initiatives for innovation in drug development: the EMA perspective

EU scientific regulatory support mechanisms and initiatives for innovation in drug development: the EMA perspective EU scientific regulatory support mechanisms and initiatives for innovation in drug development: the EMA perspective FAMHP Workshop, Brussels, 2 nd May 2016 Presented by Zahra Hanaizi Product Development

More information

FDA Drug Approval Process Vicki Seyfert-Margolis, Ph.D.

FDA Drug Approval Process Vicki Seyfert-Margolis, Ph.D. Speaker Comparing The Effectiveness Of New Drugs: Should The FDA Be Asking 'Does It Work' Or 'Does It Work Better'? Vicki L. Seyfert-Margolis, PhD Senior Advisor, Science Innovation and Policy U.S. Food

More information

Emerging Trends in Regulatory Outsourcing WHITE PAPER.

Emerging Trends in Regulatory Outsourcing WHITE PAPER. WHITE PAPER www.makrocare.com/consulting You are not alone in the quest for bringing your products to market and improve the quality of the life for millions! MakroCare brings you the insight on how a

More information

Regulatory Overview of Proposed LDT Framework. FDA Concerns. Background. FDA Proposed Regulatory Approach. By Ben Berg, Meaghan Bailey, RAC

Regulatory Overview of Proposed LDT Framework. FDA Concerns. Background. FDA Proposed Regulatory Approach. By Ben Berg, Meaghan Bailey, RAC Regulatory Overview of Proposed LDT Framework By Ben Berg, Meaghan Bailey, RAC On July 31, 2014, the U.S. Food and Drug Administration (FDA or the Agency) notified both the Senate Committee on Health,

More information

EFPIA-PHRMA PRINCIPLES FOR RESPONSIBLE CLINICAL TRIAL DATA SHARING REPORT ON THE 2016 MEMBER COMPANY SURVEY

EFPIA-PHRMA PRINCIPLES FOR RESPONSIBLE CLINICAL TRIAL DATA SHARING REPORT ON THE 2016 MEMBER COMPANY SURVEY EFPIA-PHRMA PRINCIPLES FOR RESPONSIBLE CLINICAL TRIAL DATA SHARING REPORT ON THE 2016 MEMBER COMPANY SURVEY PREAMBLE The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the

More information

Short Course: Adaptive Clinical Trials

Short Course: Adaptive Clinical Trials Short Course: Adaptive Clinical Trials Presented at the 2 Annual Meeting of the Society for Clinical Trials Vancouver, Canada Roger J. Lewis, MD, PhD Department of Emergency Medicine Harbor-UCLA Medical

More information

Industry Experience: Early Collaboration with FDA on Combination Products. Kristi Kistner, Amgen Inc. CMC Strategy Form January 26, 2015

Industry Experience: Early Collaboration with FDA on Combination Products. Kristi Kistner, Amgen Inc. CMC Strategy Form January 26, 2015 Industry Experience: Early Collaboration with FDA on Combination Products Kristi Kistner, Amgen Inc. CMC Strategy Form January 26, 2015 Collaboration is working together to achieve shared goals Given the

More information

Expanded Access. to Investigational Drugs & Biologics. for Treatment Use

Expanded Access. to Investigational Drugs & Biologics. for Treatment Use SJMHS Research Compliance Office Guidance Document Expanded Access to Investigational Drugs & Biologics for Treatment Use May 2015 1 Expanded Access to Investigational Drugs & Biologics for Treatment Use

More information

Update on Regulatory Environment- Europe Experience with 2007/47/EC M5 & Discussions on Possible Recast of EU Medical Device Regulations

Update on Regulatory Environment- Europe Experience with 2007/47/EC M5 & Discussions on Possible Recast of EU Medical Device Regulations Update on Regulatory Environment- Europe Experience with 2007/47/EC M5 & Discussions on Possible Recast of EU Medical Device Regulations Paul Brooks Vice President Healthcare, Americas Presentation to:

More information

Early Feasibility Study (EFS) IDEs

Early Feasibility Study (EFS) IDEs Early Feasibility Study (EFS) IDEs A Valuable Regulatory Tool for Medical Device Development Carla M. Wiese Policy Analyst for the Early Feasibility Program Office of Device Evaluation Center for Devices

More information

Regulatory Requirements

Regulatory Requirements Regulatory Requirements CTTI Quality by Design Workshop 28-29 Jan 2013 Rockville, MD Fergus Sweeney, Head, Compliance and Inspections, European Medicines Agency An agency of the European Union Disclaimer

More information

Decoding Phase II Clinical Trial Terminations

Decoding Phase II Clinical Trial Terminations Decoding Phase II Clinical Trial Terminations Why Phase II trials are terminated and what can be done to improve Phase II success rates - the most critical inflection point for clinical development Subha

More information

Understanding Clinical Equivalence

Understanding Clinical Equivalence Understanding Clinical Equivalence BSI 2014 Medical Device Mini-Roadshow Ibim Tariah Ph.D Technical Director, Healthcare Solutions 1 Understanding Clinical Equivalence Review Requirements from Directives

More information

Understanding clinical research trials

Understanding clinical research trials Understanding clinical research trials Dr. Isabelle Fleury Medical Hemato-oncologist Hôpital Maisonneuve-Rosemont CIUSSS de l Est-de-l Île-de-Montréal Goals of this presentation Better understand clinical

More information

Weighing the Impact of FDA Regulatory Uncertainty on Clinical Decision Support Development

Weighing the Impact of FDA Regulatory Uncertainty on Clinical Decision Support Development Executive Summary Weighing the Impact of FDA Regulatory Uncertainty on Clinical Decision Support Development February 25, 2016 Clinical decision support ( CDS ) software is not getting the regulatory clarity

More information

Third Quarter 2009 Results. Analysts and Media Conference November 5, 2009 Zurich

Third Quarter 2009 Results. Analysts and Media Conference November 5, 2009 Zurich Third Quarter 2009 Results Analysts and Media Conference November 5, 2009 Zurich This presentation contains forward looking statements which involve risks and uncertainties. The actual performance, results

More information

Developing a European First-in-Human Study: Three Key Decisions

Developing a European First-in-Human Study: Three Key Decisions Developing a European First-in-Human Study: Three Key Decisions By Nicole Feist, BA Clinical A key step in the translational medicine benchtop to bedside process model is the move from research and preclinical

More information

March 6, Division of Dockets Management (HFA-305) Food and Drug Administration 5630 Fishers Lane, Room 1061 Rockville, MD 20852

March 6, Division of Dockets Management (HFA-305) Food and Drug Administration 5630 Fishers Lane, Room 1061 Rockville, MD 20852 701 Pennsylvania Avenue, NW Suite 800 Washington, D.C. 20004 2654 Tel: 202 783 8700 Fax: 202 783 8750 www.advamed.org March 6, 2017 Division of Dockets Management (HFA-305) Food and Drug Administration

More information

Introduction to Clinical Research

Introduction to Clinical Research Introduction to Clinical Research What is Clinical Research? Clinical research is medical research that involves people like you. People volunteer to participate in carefully conducted investigations that

More information

INVESTIGATIONAL DEVICE EXEMPTION APPLICATION. IDE Title (if title being used)

INVESTIGATIONAL DEVICE EXEMPTION APPLICATION. IDE Title (if title being used) INVESTIGATIONAL DEVICE EXEMPTION APPLICATION IDE Title (if title being used) Name of Sponsor Investigator, MD X Professor, Department Icahn School of Medicine at Mount Sinai Date of Submission Form version

More information

Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines

Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines Canadian Cancer Clinical Trials Network Portfolio Eligibility Criteria and Guidelines 1. Introduction The Canadian Cancer Clinical Trials Network (3CTN) will support a portfolio of academic clinical trials

More information

DEFINING BEST PRACTICE FOR ADVERSE EVENT REPORTING WITH INDUSTRY-SPONSORED CLINICAL TRIAL MANUSCRIPTS

DEFINING BEST PRACTICE FOR ADVERSE EVENT REPORTING WITH INDUSTRY-SPONSORED CLINICAL TRIAL MANUSCRIPTS 11TH ANNUAL MEETING OF ISMPP DEFINING BEST PRACTICE FOR ADVERSE EVENT REPORTING WITH INDUSTRY-SPONSORED CLINICAL TRIAL MANUSCRIPTS April 28, 2015 Hyatt Regency Crystal City Arlington, VA, USA 11TH ANNUAL

More information

Update on Current FDA Policies and Priorities

Update on Current FDA Policies and Priorities Update on Current FDA Policies and Priorities Vernessa Pollard MassMEDIC FDA Update December 12, 2017 Boston Brussels Chicago Dallas Düsseldorf Frankfurt Houston London Los Angeles Miami Milan Munich New

More information

The Path to Clinical Enterprise Maturity DEVELOPING A CLINICALLY INTEGRATED NETWORK

The Path to Clinical Enterprise Maturity DEVELOPING A CLINICALLY INTEGRATED NETWORK The Path to Clinical Enterprise Maturity DEVELOPING A CLINICALLY INTEGRATED NETWORK dhgllp.com/healthcare Kevin Locke PRINCIPAL Kevin.Locke@dhgllp.com 330.606.4699 Michael Strilesky SENIOR MANAGER Michael.Strilesky@dhgllp.com

More information

Draft agreed by Scientific Advice Working Party 5 September Adopted by CHMP for release for consultation 19 September

Draft agreed by Scientific Advice Working Party 5 September Adopted by CHMP for release for consultation 19 September 23 January 2014 EMA/CHMP/SAWP/757052/2013 Committee for Medicinal Products for Human Use (CHMP) Qualification Opinion of MCP-Mod as an efficient statistical methodology for model-based design and analysis

More information

CANCER CENTER SCIENTIFIC REVIEW COMMITTEE

CANCER CENTER SCIENTIFIC REVIEW COMMITTEE CANCER CENTER SCIENTIFIC REVIEW COMMITTEE The Clinical Scientific Review Committee (SRC) at The Medical College of Wisconsin Cancer Center plays a vital role in protocol review and monitoring to ensure

More information

Taking Control of Your Fuzzy

Taking Control of Your Fuzzy Taking Control of Your Fuzzy Front End MassMedic Presentation September 22, 2010 Sanjeev Wadhwani Project Management Consultant Integrated Project Management Company, Inc. Sanjeev Wadhwani Project Manager

More information