US Medical Devices Innovation - the “21st Century Cures Act” and Streamlined Medical Devices

Approval美国医疗器械创新 -《21世纪治愈法案》

与医疗器械简化审批

July 20172017年7月

Patricia ShraderVice President

Global Regulatory AffairsMedtronic, plc

美敦力公司全球法规事务副总裁

Americas美洲

Europe, Middle East,

& Africa欧洲、中东以及非洲

GreaterChina

大中华区

Asia Pacific亚太地区

MEDTRONIC- WORLD’S LARGEST MEDICAL TECHNOLOGY COMPANY美敦力——全球 大的医疗技术公司

OUR MISSION: ALLEVIATE PAIN, RESTORE HEALTH, AND EXTEND LIFE我们的使命：减轻病痛、恢复健康、延长寿命

Medtronic is a multi-national company structured into four regions.作为一家跨国公司，美敦力业务覆盖全球四大区域

53,000+Patents超过53,000项专利

$2B Annual R&D Spend每年研发投入20亿美元

56Researchcenters56个研究中心

160+Number of countries operating in业务覆盖全球160多个国家

3

WE USE THE BREADTH OF OUR BUSINESS美敦力广泛的业务范围TO IMPROVE HEALTHCARE用以改善全球医疗状况

CARDIAC AND VASCULAR GROUP心血管业务集团 Aortic and Peripheral Vascular 主动脉及周围血管介入业务

Cardiac Rhythm andHeart Failure

心脏节律与心脏衰竭业务

Coronary and Structural Heart 冠脉及结构性心脏病业务

RESTORATIVE THERAPIES GROUP恢复性疗法业务集团 Spine 脊柱业务

Brain Therapies 神经调控

Pain Therapies 疼痛治疗

Specialty Therapies 专业疗法

DIABETES GROUP糖尿病业务集团 Intensive Insulin Management 强化胰岛素治疗

Non-Intensive Diabetes Therapies 非强化糖尿病治疗

Diabetes Services and Solutions 糖尿病服务与解决方案

MINIMALLY INVASIVE THERAPIES GROUP微创治疗业务集团 Early Technologies 早期诊疗新兴技术

Patient Monitoring and Recovery 患者监护与康复

Surgical Innovations 外科手术创新

4

WE IMPROVE LIVES THROUGHOUR MEDICAL TECHNOLOGIES AND SOLUTIONS

Clinical Care Optimization

Patient Management

Operational Performance

Financing Training &

Education Value-Based

Healthcare

AIRWAY & LUNG Acute Respiratory Distress

Syndrome (ARDS) Asthma Chronic Obstructive

Pulmonary Disease (COPD) Lung Cancer Pulmonary Valve Disease Respiratory Compromise Sleep ApneaBRAIN Acute Ischemic Stroke Blood Clots Brain Aneurysms Brain Tumors and Lesions Cranial Trauma Dystonia Essential Tremor Hydrocephalus Idiopathic Intracranial

Hypertension Obsessive-Compulsive

Disorder (OCD) Parkinson’s Disease Subdural HematomasDIABETES Gestational Type 1 Type 2

DIGESTIVE, GASTROINTESTINAL, UROGYNECOLOGIAL, AND UROLOGICAL Acid Reflux Barrett’s Esophagus Bowel Incontinence Chronic Benign Liver

Disease Colon Disease Crohn’s Disease Gallstones Gastroparesis GI Bleeding Hemorrhoids Hernia Nutrition Insufficiency Obesity Ovarian Tumors and

Ruptured Ectopic Pregnancy Overactive Bladder Pancreatic Cancer Polyps Reflux/GERD Urinary Retention Uterine Tumors And FibroidsEAR, NOSE & THROAT Ear Infections Hearing Loss Otologic and Neurotologic

Disorders Sinus Infections & Disease Thyroid Conditions

HEART & VASCULAR Abdominal Aortic Aneurysm

(AAA) Atrial Fibrillation Bradycardia (Slow Heartbeat) Congenital Heart Disease Coronary Artery Disease (CAD) Deep Vein Thrombosis (DVT) Fainting (Unexplained) Heart Failure Heart Valve Disease Peripheral Arterial Disease Pulmonary Embolisms Pulmonary Valve Disease Stroke Sudden Cardiac Arrest (SCA) Tachycardia (Fast Heartbeat) Thoracic Aortic Aneurysm

(TAA) Venous InsufficiencyPAIN Cancer Pain Chronic Pain Severe Spasticity Spinal FracturesSPINE AND ORTHOPEDIC Cervical Herniated Discs Degenerative Disc Disease Lumbar Spinal Stenosis Orthopedic Trauma Scoliosis Spinal Fractures Tibial Fractures

OUR HEALTHCARE SOLUTIONS

5

通过医疗技术和解决方案改善患者健康

临床护理优化

患者管理

方案运行性能管理

融资

培训和教育

基于价值的医疗保健

呼吸道与肺部疾病 急性呼吸窘迫综合征(ARDS) 哮喘 慢性阻塞性肺病(COPD) 肺癌 肺动脉瓣疾病 肺功能损伤 睡眠呼吸暂停

脑部疾病 急性缺血性脑卒中 血栓 脑动脉瘤 脑肿瘤及脑损伤 颅脑损伤 肌张力障碍 特发性震颤 脑积水 特发性颅内压增高 强迫症(OCD) 帕金森病 硬脑膜下血肿

糖尿病 妊娠期糖尿病 I型糖尿病 II型糖尿病

消化道、胃肠道、生殖系统以及泌尿系统疾病 胃酸反流 巴雷斯特食道症 大便失禁 慢性良性肝脏疾病 结肠疾病 克罗恩病 胆结石 胃瘫 胃肠道出血 痔疮 疝气 营养不良 肥胖 卵巢肿瘤及异位妊娠破裂 膀胱过度活动症 胰腺癌 息肉 反流/胃食管反流病 尿潴留 子宫瘤、子宫纤维瘤

耳鼻喉部疾病 耳部感染 听觉缺失 耳部以及耳神经疾病 鼻窦炎以及鼻窦疾病 甲状腺疾病

心脏与血管疾病 腹主动脉瘤(AAA) 房颤 心动过缓(心跳慢) 先天性心脏病 冠状动脉疾病(CAD) 深静脉血栓(DVT) 昏厥(原因不明) 心力衰竭 心脏瓣膜疾病 外周动脉疾病 肺栓塞 肺动脉瓣疾病 卒中 心脏骤停(SCA) 心动过速(心跳快) 胸主动脉瘤(TAA) 静脉功能不全

疼痛 癌痛 慢性疼痛 重度痉挛 脊柱骨折

脊柱与骨科疾病 颈椎键盘突出 退行性椎间盘疾病 腰椎椎管狭窄 骨科创伤 脊柱侧弯 脊柱骨折 胫骨骨折

我们的医疗解决方案

PRESENTATION TOPICS主题

• CDRH Mission• CDRH的使命

• Priority and Interactive Review• 优先审评和互动审评

• Food and Drug Administration Safety and Innovation Act (FDASIA)• 《美国食品药品监督管理局安全与创新法案》（FDASIA）

• Smart regulation• 智能监管

• Expedited access• 快速通道

• 21st Century Cures Act / Breakthrough Pathway Provision• 《21世纪治愈法案》／突破性途径条款

• Medical Device User Fee Act (MDUFA)• 《医疗器械用户费用修正案》（MDUFA）

• MDUFA Performance Goals• MDUFA绩效目标

DEVELOPMENT TIME LINE发展时间表

Priority Review

Guidance2008

2008年优先

审评

指南

FDASIAAct2012

2012年《美

国食品药品

监督管理局

安全与创新

法案》

（FDASIA）

SMART Regulation

20142014年智能

监管

Expedited access

Guidance2015

2015年快速

通道指南

21st Century Cures ActDecember

20162016年12月《21世纪治

愈法案》

MDUFAAct October

20172017年10月《医疗器械

用户费用修

正案》

（MDUFA）

CDRH MISSION AND VALUESCDRH的使命和价值观

Protect and promote public health 保护并改善公共卫生

Timely and continued access to safe, effective, high-quality devices

及时且持续地获取安全、有效、优质的医疗器械

Provide accessible science-based information

提供以科学为基础的信息

Facilitate innovation 推动创新

- Public Health Focus- 公共卫生重点- Science based decisions- 基于科学的决策- Our people- 我们的人员- Innovation- 创新- Transparency- 透明度- Honesty and Integrity- 诚实守信- Accountability- 问责

Priority Reviews优先审评Scope: all premarket submissions, not only PMA applications范围：所有上市前申请，而不仅仅是PMA申请Devices appropriate for Priority Review适用于优先审评的器械 Treat or diagnose a life-threatening disease or condition 用于治疗或诊断危及生命的疾病或病症 Meets at least one of the following: 至少符合下列任意一项： Breakthrough technology 突破性技术 No approved alternative treatment 尚无已批准的替代疗法 Device offers significant advantages over approved alternatives 与已批准的替代疗法相比具有明显优势 Availability of the device is in the best interest of patients 将器械推向市场符合广大患者的 佳利益

Impact: Placed at the beginning of the review queue影响：置于审评队列的 前面

FOOD AND DRUG ADMINISTRATION SAFETY AND INNOVATION ACT (FDASIA)《美国食品药品监督管理局安全与创新法案》（FDASIA）

July 20122012年7月One of the key intents of the law: Promoting innovation法案的重点之一：促进创新Notable provisions contained in FDASIAFDASIA中的重要条款 Promote innovation 促进创新 Priority review 优先审评 Novel technologies addressing an unmet patient need 解决尚未满足的患者需求的创新技术 Interactive reviews 互动审评 Performance objectives 绩效目标

SMART REGULATION智能监管

Attain the goal of protecting the public health while encouraging innovation实现保护公共卫生和鼓励创新的目标 Smart, sound, science-based regulation 智能、可靠、以科学为基础的监管 Imposes appropriate regulatory framework 采用适当的监管框架 Minimizes unnecessary burden 尽量减少不必要的负担

Regulation done correctly can通过正确的监管，可以： Provide pathway toward meaningful innovation 提供有意义的创新途径 Instill consumer confidence 树立消费者的信心 Level the playing field for business 提高业务竞争水平 Decrease litigation (time and money in court) 减少诉讼（花费在法庭上的时间和金钱） Prevent recalls 避免产品召回

“We are committed to flexible, smart regulation, and to working with companies and the clinical community to ensure that innovative new medical devices that demonstrate a reasonable assurance of safety and effectiveness are available for patients in a timely manner.““我们致力于通过灵活的智能监管，并与企业和临床机构合作，确保及时为患者提供已证明具有合理安全性和有效性保证的创新医疗器械。”

- Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health- Jeffrey Shuren, M.D., J.D.，FDA器械和辐射健康中心主任

EXPEDITED ACCESS FOR PREMARKET APPROVAL MEDICALDEVICES INTENDED FOR UNMET MEDICAL NEED FOR LIFETHREATENING OR IRREVERSIBLY DEBILITATING DISEASES OR

CONDITIONS预期用于治疗目前缺乏治疗方法的危及生命的或不可逆衰弱性疾病或病症的医疗器械的快速上市前批准

Background背景

Innovation Pathway 创新途径

Role of Postmarket Data 上市后数据的作用

Expedited Access Premarket Approval (PMA)上市前批准（PMA）的快速通道

21ST CENTURY CURES ACT《21世纪治愈法案》

The 21st Century Cures Act is a bipartisan approved Act to accelerate the discovery, development, and delivery of 21st century cures.《21世纪治愈法案》是经由两党联合批准的法案，旨在加速21世纪疗法的发现、研发与应用。

An innovation game-changer, a once-in-a-generation, transformational opportunity to change the way we treat disease.一次前所未有的创新、改革机遇，将会改变我们治疗疾病的方式。

Key Device Provisions:关于器械的主要规定：-Breakthrough Pathway-突破性途径-Single IRB-单一IRB-Least Burdensome- 小负担

MEDICAL DEVICE INNOVATIONS医疗器械创新BREAKTHROUGH PATHWAY突破性途径

Section 3051: Breakthrough Pathway – Establishes a new priority review program for breakthrough devices modeled on the breakthrough pathway for drugs. 第3051条：突破性途径——针对突破性器械，建立了新的优先审评项目（基于药品的突破性途径）。

This program builds on FDA’s existing Expedited Access Pathway Program. 该项目建立在FDA现有快速通道（EAP）项目基础之上。

The EAP Program is currently for unmet medical needs for life-threatening or irreversibly debilitating diseases or conditions that are subject to PMAs or de novo review.目前，EAP项目旨在解决需要经过PMA或重新分类审评的、目前缺乏治疗方法的危及生命的或不可逆衰弱性疾病或病症。

Medical Device User Fee Act《医疗器械用户费用修正案》（MDUFA）MDUFA IVMDUFA第四章Under the Medical Device User Fee Act reauthorization, the FDA is given authority to collect user fees and obliged to meet respective performance goals. 根据《医疗器械用户费用修正案》，FDA有权收取注册费用，并应实现相应的绩效目标。

PMA’sAverage Total Time to Decision Goal达成PMA决策的平均总用时

320 days in 2018 (3-year cohort) 2018年为320天（3年队列） Ramping down to 290 days in 2020-2022 2020年至2022年，将缩短至290天

510(k)’s针对510(k)

124 days in 2018 2018年为124天 Ramping down to 108 days in 2022 2022年，将缩短至108天

Transparency: Published reports to Congress透明度：向国会提交报告

Answering the question: “How is FDA performing?” 回答问题：“FDA的执行情况如何？”

MDUFA IV PERFORMANCE GOALS MDUFA第四章绩效目标

- Infrastructure- 基础设施

- Quality management function- 质量管理职能- Expanding review capacity- 扩展审评能力- Transparency in fee setting- 费用设定的透明度

- Process Improvement - 流程改进

- Improving third party review- 改进第三方审评- Statements of deficiency for submission reviews- 申报资料审评的发补声明- Software, Digital health- 软件以及数字健康- Use of patient input- 采用患者意见- Use of real world evidence- 利用真实世界证据- Guidance document development- 制定指南文件