Facts About New England's Drug Regulatry System

Transcription

1 New England Cuncil Healthcare Cmmittee Survey Title I: Putting Patients First by Incrprating their Perspectives int the Regulatry Prcess and Addressing Unmet Needs Drug Regulatry Prcesses and Prmting Drug Research & Discvery Subtitle A: Patient Fcused Drug Develpment: Require the FDA t establish a structured framewrk fr the meaningful incrpratin f patient experience data int the regulatry decisinmaking prcess, including the assessment f desired benefits and tlerable risks assciated with new treatments. Subtitle B: Surrgate Endpint Qualificatin and Utilizatin: Establish a predictable, transparent prcess fr FDA s cnsideratin, and pssible qualificatin, f surrgate endpints. Als allw FDA t use private-public partnerships t qualify ther types f bimarkers. Subtitle C: Apprval f Breakthrugh Therapies: Clarify that the FDA may apprve a drug that has received breakthrugh designatin when early stage clinical data prvides sufficient evidence under current safety and efficacy standards, cnsidering risks and benefits f the drug and the risks assciated with the disease r cnditin fr which unmet needs exist. Subtitle J: Streamlined Data Review: Streamline review prcess fr adding indicatins t a drug label by allwing FDA t accept and review data summaries rather than full data packages. Subtitle G: Expanded Access: Place transparency requirements n certain drug cmpanies regarding expanded access prgrams (prgrams fr patients t access drugs befre they are apprved). Create an expanded access task frce t prvide recmmendatins t Cngress fr further refrms f the prgram. Prmting Drug Research & Discvery Subtitle D: Antibitic Drug Develpment: Antibitic Develpment t Advance Patient Treatment Act t help fster develpment f new antibitics Create a new transferable exclusivity prgram Develping an Innvative Strategy fr Antimicrbial Resistance Micrrganisms Act t incentivize new drug develpment by imprving prcess f hspital payments Subtitle K: Cures Acceleratin Netwrk: Authrize additinal funds fr research n repurpsing drugs fr new uses. Subtitle L: Drmant Therapies: Rewards investment in treatments and cures fr diseases with unmet medical needs. Allws innvatrs t chse pathway and receive fixed year prtectin perid fr these therapies upn FDA apprval. Subtitle M: New Therapeutic Entities: Extend exclusivity fr tw years fr significant imprvements t existing mlecules (e.g., new delivery systems, new cmbinatins, new frmulatins that lead t les adverse events and increase patient benefits and adherence) Subtitle N: Orphan Prduct Extensins Nw: Prvides six mnths f additinal market exclusivity r drugs if cmpany establishes that drug treats a rare disease and receives rare disease indicatin frm FDA n its label.

2 New England Cuncil Healthcare Cmmittee Survey Breakthrugh Devices Subtitle E: Pririty Review fr Breakthrugh Devices: Establishes prcess fr designatin expedited review f devices that represent breakthrugh technlgies with ptential t address unmet medical needs. FDA designatin wuld translate int Medicare and Medicaid transitinal cverage benefits [currently placehlder]. Subtitle F: Accelerated Apprval fr Breakthrugh Devices: Establish accelerated apprval pathway fr medical devices. Manufacturer & FDA Cmmunicatins Subtitle H: Facilitating Respnsible Cmmunicatin f Scientific and Medical Develpments: Prpsal t clarify and ratinalize rules gverning cmmunicatin and infrmatin sharing frm manufacturers t physicians, insurers, researchers, etc [currently placehlder]. Subtitle I: Mdernizing the Regulatin f Scial Media: Regulatins f cmmunicatins n scial media by FDA. Title II: Building the Fundatin fr 21 st Century Medicine, Including Helping Yung Scientists New Cuncils/Cmmissins Subtitle A: 21 st Century Cures Cnsrtium Act: Establishes public private partnership ( cnsrtium ) based n the EU s Innvative Medicines Initiative t accelerate the discvery, develpment, and delivery f innvative cures, treatments, and preventive measures. Subtitle B: Medical Prduct Innvatin Advisry Cmmissin: Creates Medical Prduct Innvatin Advisry Cmmissin (based n MedPAC) t advise Cngress n issues related t discverydevelpment-delivery cycle. FDA Regulatry Issues Subtitle C: Regenerative Medicine: Requires FDA t update guidance n surrgate and intermediate endpints fr accelerated apprval f regenerative medicine prducts. Subtitle D: Genetically Targeted Platfrm Technlgies fr Rare Diseases: Clarifies accelerated apprval pathway t enable FDA t rely n data frm prducts that utilize similar genetically targeted platfrm technlgy. Subtitle E: Sensible Oversight fr Technlgy Which Advances Regulatry Efficiency: Helps prvide regulatry certainty fr thse develping apps and health infrmatin technlgy. Subtitle I: Cmbinatin Prducts: Requires FDA t issue guidance n review prcess fr prducts that include bth drugs and devices. Subtitle J: Mdernizing Regulatin f Diagnstics: [Placehlder] Evidence, Data, and Infrmatin Technlgy Subtitle F: Building a 21 st Century Data Sharing Framewrk: Establish data sharing framewrk t: Enable physicians and patients t better identify nging clinical trials

3 New England Cuncil Healthcare Cmmittee Survey Enable researchers and develpers t use Medicare data t imprve patient care Establish a prcess fr Cngress t address ther issues identified by the President s Cuncil f Advisrs n Science and Technlgy Subtitle G: Utilizing Real Wrld Evidence: Authrize FDA t utilize real wrld evidence and require FDA t issue guidance n cllecting such evidence. Subtitle H: Cverage with Evidence Develpment: Addresses Medicare s cverage prcess and allws Medicare beneficiaries t secure cverage fr prducts that are the subject f the clinical trial in which they participate. Subtitle K: Interperability: Natinal interperable health infrmatin infrastructure [placehlder] Subtitle L: NIH Federal Data Sharing: Requires thse receiving NIH grants t share data. Subtitle M: Accessing, Sharing, and Using Health Data fr Research Purpses: Enabling research ptential f health facility data. Other Subtitle N: 21 st Century Chrnic Disease Initiative Act: Requires HHS t carry ut lngitudinal study designed t imprve utcmes f patients with chrnic diseases. Subtitle O: Helping Yung Emerging Scientists: Establishes prgram at NIH t help yung emerging scientists. Subtitle P: Fstering High-Risk, High-Reward Science: Requires NIH t supprt high-risk, high-reward (ptentially breakthrugh) prjects. Subtitle Q: Precisin Medicine: [Placehlder] Title III: Mdernizing Clinical Trials Subtitle A: Clinical Research Mdernizatin Act: Streamline institutinal review bard prcess, particularly fr clinical trials cnducted at multiple sites, by minimizing regulatry duplicatin and unnecessary delays. Subtitle B: Brader Applicatin f Bayesian Statistics and Adaptive Trial Design: Encurage brader applicatin f Bayesian statistics and adaptive trial designs. Subtitle C: Pst-Apprval Studies and Clinical Trials: Ensure FDA and spnsrs peridically evaluate whether pst-apprval studies remain scientifically warranted. Subtitle D: Pediatric Research Imprvement Netwrk: Require NIH t implement Natinal Pediatric Research Netwrk Act Subtitle E: Glbal Pediatric Clinical Trial: Sets Sense f Cngress that NIH and FDA shuld wrk with the EU, industry, and thers t establish a glbal pediatric clinical trial netwrk. Title IV: Accelerating the Discvery, Develpment, and Delivery Cycle and Cntinuing 21 st Century Innvatin at NIH, FDA, CDC, and CMS NIH, CDC, and FDA Prvisins Subtitle A: Natinal Institutes f Health: NIH Research Strategic Investment Plan Bimedical Research Wrking Grup t Reduce Administrative Burden n Researchers

4 New England Cuncil Healthcare Cmmittee Survey NIH Travel [Placehlder] Increasing Accuntability at NIH GAO Reprt n Cmmn Fund Exemptin fr NIH frm Paperwrk Reductin Act Requirements Additinal Funding fr NIH Cmmn Fund Additinal Funding fr NIH Brain Research NCATS Phase IIB Restrictin Subtitle B: Advancing Research fr Neurlgical Diseases: Require CDC t set up a surveillance system fr neurlgical diseases. Subtitle C: Vaccine Access, Certainty, and Innvatin: Prvide certainty and transparency with regard t the regulatin f vaccines, including with respect t CMS and CDC. Subtitle D: Reagan-Udall Imprvements: Imprve Reagan-Udall Fundatin. Subtitle E: FDA Hiring, Travel, and Training: [Placehlder] Subtitle F: FDA Successin Planning: Ensure FDA staff has ability t cntinue t imprve expertise and that FDA develps successin plan fr management psitins. Medicare Prvisins: Cverage Subtitle G: Dispsable Medical Technlgies: Refrm cverage requirements fr certain dispsable medical technlgies. Subtitle H: Lcal and Natinal Cverage Decisin Refrms: Refrms Medicare LCD prcess. Subtitle P: Medicare Pharmaceutical and Technlgy Ombudsman: Establish mbudsman at CMS t allw medical device and pharmaceutical cmpanies t appeal decisins and better understand ratinale behind cverage decisins. Subtitle Q: Ensure Lcal MACs Evaluate Data Related t Categry III Cdes: Ensure that lcal Medicare Administrative Cntractrs (MACs) review all data befre making cverage decisins n Categry III cdes. Medicare Prvisins: Payment Subtitle J: Revise IPPS New Technlgy Add n Payment Reimbursement Amunts: Prvides mre transparency regarding new technlgy add n payment reimbursement prcess. Subtitle K: Lwing Medicare Patients OOP Csts: Allws senirs t better identify the OOP csts they might face fr a given treatment r service. Subtitle L: Glbal Surgery Services Rule: Prevent implementatin f glbal surgery services rule, which des away with bundled payments fr surgens. Subtitle M: Prviders Cnslidatin and Medicare Payments Examined Thrugh Evaluatin: Requires CMS t analyze and seek public input n hw prpsed Medicare payment plicies wuld affect the cnslidatin f prviders and payers. Miscellaneus Medicare Prvisins Subtitle I: Telemedicine: Advance pprtunities fr telemedicine and new technlgies t imprve the delivery f quality health care services t Medicare beneficiaries.

5 New England Cuncil Healthcare Cmmittee Survey Subtitle N: Medicare Part D Patient Safety and Drug Abuse Preventin: Help prevent high-risk Medicare beneficiaries frm abusing cntrlled substances. Subtitle O: Accelerating Innvatin in Medicine: Establish a prgram that allws fr patients t access medical device treatments sner than therwise wuld be available. Other Subtitle R: Advancing Care fr Exceptinal Kids: Establish Medicaid and CHIP Care Crdinatin prgram fr children with medically cmplex cnditins. Subtitle S: Cntinuing Medical Educatin Sunshine Exemptin: Clarify that peer reviewed jurnals, jurnal reprints, jurnal supplements, and medical textbks are excluded frm the reprting requirement under the Sunshine Act. Subtitle T: Medical Testing Availability: Clarify law regarding Research Use Only (RUO) labeled prducts. Title V: Mdernizing Medical Prduct Regulatin Manufacturing Subtitle A: Manufacturing Incentives: Prvide incentives fr manufacturing generic drugs here in the US. Subtitle B: 21 st Century Manufacturing: Require FDA t update guidance regarding nvel manufacturing techniques. Subtitle C: Cntrlled Substance Manufacturing and Exprts: Prvide US pharmaceutical cmpanies with a level playing field regarding cntrlled substances exprts. Medical Device Refrms Subtitle D: Medical Device Refrms: Third Party Quality System Assessment Clarify Valid Scientific Evidence as Well-Dcumented, Real Wrld Evidence Gathered frm Clinical Registries and Studies Published in Peer-Reviewed Jurnals Ensure FDA Reviewers are Trained n Least Burdensme Cncept Imprve Prcess f Gvernment Recgnitin f Apprpriate Standards Set by Medical Cmmunity Streamline Prcess f Marketing Class I Medical Devices Streamline 510(k) Prcess fr Medical Devices Allw FDA t Apply the Humanitarian Device Exemptin (HDE) t Areas that Impact >4,000 Patients Where Public Health Requires Greater Availability t Treat r Diagnse and there is n Alternative Streamline FDA Cmmittee Advisry Prcess Supply Chain Security fr Devices