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Pink Sheet – Opioid, NME, And Generic Drugs Changes Plotted In US FDA's 2018 Policy Roadmap

Pink Sheet – Opioid, NME, And Generic Drugs Changes Plotted In US FDA's 2018 Policy Roadmap

Executive Summary

‘Better informed patients,’ ‘drug competition,’  ‘biosimilar innovation’ – as well as nutrition and tobacco goals will be achieved through guidances, promotional plans and other items FDA will develop over the next year.

FDA’s 2018 Strategic Policy Roadmap

Opioid Policy Work Plan

Re-examine and modernize FDA’s framework for evaluating pre- and post-market safety of opioid products based on risk and benefit considerations, including an evaluation of a product’s potential risks for illicit use or its potential for abuse

  • Scientists and FDA officials debated study designs for assessing abuse-deterrent formulations in real world settings during a 2017 workshop. (Also see “Abuse Deterrent Opioids: Which Studies Will Show Real World Effect?” – Pink Sheet, 19 Jul, 2017.)


Address inappropriate prescribing of prescription opioids with new modifications to REMS programs, such as through mandating provider training

  • Training was made available to prescribers of immediate-release formulations as part of an expansion of the opioid REMS to include those products. (Also see “FDA’s Opioid Programs: After Training, What’s The Next Step?” – Pink Sheet, 5 Oct, 2017.)


Support development of abuse-deterrent opioid formulations by exploring new methods for analyzing and evaluating abuse-deterrent features and the nomenclature used to describe them, facilitate development of science for generics development, and encourage the conversion of the market to effective, abuse-deterrent formulations

  • FDA also is studying whether the term abuse-deterrent conveys the correct description of the products. (Also see “Opioid Hearing: Senators Want Speed, But Agency Heads Stress Time, Effort” – Pink Sheet, 5 Oct, 2017.)


Finalize policy to enable OTC availability of naloxone

  • The non-profit Harm Reduction Therapeutics has targeted the OTC switch and wants to make the product available nationwide. (Also see “Nonprofit Pharma Targets Making Naloxone Available OTC, Trimming Price” – Pink Sheet, 13 Dec, 2017.)


Gather stakeholder input through public hearings and workshops on opioid issues while FDA explores innovations in packaging, regulation, and prescribing to reduce opioid exposure and address misuse and abuse

  • FDA held a public hearing in December on potential changes to opioid packaging, which could be linked to prescribing guidelines and potentially labeling. (Also see “Opioid Packaging Standards Could Emerge From Dosage Guidelines in Labeling” – Pink Sheet, 11 Dec, 2017.)


Advance policy to enable more innovation of non-addictive pain remedies and development of medication-assisted treatment

  • FDA Commissioner Scott Gottlieb announced guidance on establishing novel endpoints and other actions for new opioid addiction treatments in November 2017. (Also see “Opioid Treatments Could Get Survival Claim As US FDA Develops Protocol” – Pink Sheet, 1 Dec, 2017.)


Implement a new and more robust approach to the oversight of illicit controlled substances and foreign unapproved drugs imported into the US through international mail facilities. FDA will expand its efforts through the formation of an Enforcement Task Force

  • Gottlieb said in 2017 that he would increase personnel at international mail facilities, where many drug shipments enter the US. (Also see “US FDA’s China Staff Increase Takes Hold As Gottlieb Boosts Border Security” – Pink Sheet, 14 Aug, 2017.)


Launch public campaign to drive more adoption of medication-assisted treatment and take new steps to address the stigma and other obstacles to widespread availability.

  • Gottlieb has said cultural, as well as opioid labeling, changes are needed to handle the crisis. (Also see ” Addiction Recovery Medications’ ‘Stigma’ Needs To Be Eliminated, Gottlieb Says” – Pink Sheet, 25 Oct, 2017.)

Drug Competition Action Plan and Biosimilar Innovation Plan

Identify and address where brand firms are inappropriately impeding generic competition, including guidance on how FDA determines whether to waive the requirements of a single-shared Risk Evaluation and Mitigation Strategy and proper design of shared-system REMS

  • New HHS Secretary Alex Azar said during his confirmation hearing that one option may be determining whether a REMS is still necessary when generic competition enters the market. (Also see “Azar’s HHS: Generic Certainty, REMS Fixes Can Lower Drug Prices” – Pink Sheet, 29 Nov, 2017.)


Issue final guidance on product developers’ communication with payers about the economic consequences of their products, which may promote value-based contracting

  • The House Energy and Commerce Subcommittee on Health recently advanced a bill that would allow discussions between payers and manufacturers about clinical and economic information about off-label uses of approved drugs, as well unapproved drugs. (Also see “Off-Label Communications Bill Allowing Discussion Of Unapproved Drugs Advances” – Pink Sheet, 18 Jan, 2018.)


Continue enhancing the development and review of complex generics, including implementation of the new pre-ANDA program to expedite access.

  • FDA allowed pre-submission meetings for complex generic product sponsors as part of the 2017 generic drug user fee reauthorization. (Also see “Complex ANDAs: Early Meetings With FDA Can Generate Bonus Communication” – Pink Sheet, 2 Oct, 2017.)


Maximize the efficiency of the generic drug program to reduce cycling and lower the time required to reach the market

  • FDA recently issued an internal policy document outlining how ANDA assessments should be handled, in part to streamline them and increase consistency. (Also see “In Switching Generic ‘Reviews’ To ‘Assessments’ Is US FDA Changing More Than Just A Word?” – Pink Sheet, 10 Jan, 2018.)


Establish an inter-agency working group to improve collaboration with FTC and issue guidance on FDA’s handling of 505(q) citizen’s petitions

  • The Center for Drug Evaluation and Research included a citizen petition guidance in its 2018 guidance agenda. (Also see “Disease-Specific Drug Development Guidances Coming From US FDA In 2018” – Pink Sheet, 19 Jan, 2018.)


Advance policies to promote the more efficient entry of generic drug-device combinations. Develop new guidance to address and modernize FDA’s current interpretation and adoption of Hatch-Waxman

  • Gottlieb has signaled that generics of products with drug and device constituent parts could be allowed some degree of difference in labeling and design, an issue that seems to have stymied copies of Mylan NV’s epinephrine auto-injector EpiPen and GlaxoSmithKline PLC’s asthma inhaler Advair. (Also see “Gottlieb’s Complex Generics Remarks Could Signal (Some) Increased Flexibility” – Pink Sheet, 28 Nov, 2017.)


Launch program to promote development and adoption of safe, high-quality biosimilars as part of a new Biosimilar Innovation Plan

  • FDA has indicated a willingness to educate the relevant providers and patients about biosimilars once the products began entering the market. (Also see “FDA Biosimilar Education Campaign Will Need To Be Targeted” – Pink Sheet, 17 Sep, 2015.)

Advanced Manufacturing Strategy Roadmap

Establish an Emerging Technology Team to encourage and assist companies implementing new and innovative manufacturing technology, such as continuous manufacturing

  • FDA finalized guidance in 2017 outlining how firms can talk with the Emerging Technology Team, but the generics industry is concerned that continuous manufacturing could be used to protect brand products. (Also see “Will Continuous Manufacturing Mean Continuous Generic Delay? FDA Hears AAM’s Warning” – Pink Sheet, 4 Dec, 2017.)


Develop clear regulatory standards to promote wider use of continuous manufacturing

  • FDA hopes a standardized continuous manufacturing platform will emerge and push generic manufacturers to invest in the technology. (Also see “FDA Making A Generic Case For Continuous Manufacturing” – Pink Sheet, 2 May, 2016.)


Write guidance to help lower the cost for compounders to become registered outsourcing facilities and promote development of a robust outsourcing sector

  • FDA intends to place outsourcing facilities in a lower risk category for GMP oversight, according to the agency’s 2018 compounding policy priorities. (Also see “FDA Commissioner Outlines Priorities For Tightening Oversight Of Drug Compounding Facilities” – Pink Sheet, 19 Jan, 2018.)

Medical Innovation Access Plan

Implement streamlined orphan designation review template and expand orphan opportunities to new areas

  • FDA is not very interested in establishing an ultra-orphan designation for diseases much smaller than the traditional 200,000 patient threshold to encourage more development. (Also see “US FDA Doesn’t Support Creating Ultra-Orphan Designation” – Pink Sheet, 19 Dec, 2017.)


Modernize the Office of New Drugs to ensure policy is rooted in the best science and management principles and staff have the support and tools to achieve their mission

  • OND’s deputy director said recently that all practices are under review as part of the restructuring. (Also see “US FDA Drug Office Reform: ‘Everything’ Is On The Table” – Pink Sheet, 15 Nov, 2017.)


Develop new disease-focused medical product guidances to update and modernize approaches to clinical trial design and other aspects of drug development, including guidances targeting serious neurodegenerative diseases

  • Gottlieb said one of the guidances will cover Alzheimer’s disease drug development. (Also see “Alzheimer’s Guidance Coming From US FDA, Part Of Broader OND Reform” – Pink Sheet, 14 Sep, 2017.)


Update and modernize FDA’s approach to applying evidentiary standards for establishing safety and effectiveness for new drugs to better define the role of real-world evidence, especially for postmarket safety.

  • FDA has sponsored demonstration projects to study the use of real-world evidence in the regulatory and patient care settings. (Also see “Real World Evidence Benefits, Limits Explored In US FDA Demonstrations” – Pink Sheet, 29 Oct, 2017.)


Develop patient-focused drug development guidance to facilitate a more systematic approach to gathering and using patient perspectives for regulatory decision-making

  • FDA recently incorporated a section in its drug review documents devoted to patient experience data. (Also see ” ‘Patient Experience Data’ Section Added To US FDA Drug Reviews” – Pink Sheet, 8 Jan, 2018.)


Advance the use of in silico techniques to develop novel methods for creating models of virtual patient outcomes and modernizing FDA benefit-risk evaluation

  • In silico evidence could be used in development of targeted therapies, according to FDA guidance. (Also see “US FDA Outlines Streamlined Development Path For Targeted Therapies” – Pink Sheet, 16 Dec, 2017.)


Explore development of a framework using accelerated approval to support marketing of a drug demonstrating a survival benefit early in clinical development and expedite availability while the magnitude of benefit is confirmed

  • Gottlieb has said that products showing a substantial, early effect on a clinical endpoint could potentially qualify for accelerated approval, which could expedite the review. (Also see “‘Progressive Approval’ Coming? US FDA Considers Accelerated Approval Without Surrogates” – Pink Sheet, 30 Nov, 2017.)


Advance the use of new tools and mobile technology for better capturing clinical trial data and measurement of safety and benefit in pre- and post-market settings

  • Mobile health app use is expected to grow substantially and research suggests they could lower health care costs. (Also see “2018 An Inflection Point For mHealth After An Active Year” – Medtech Insight, 8 Jan, 2018.)


Advance a comprehensive framework to facilitate efficient development of safe and effective gene therapy products

  • The Center for Biologics Evaluation and Research is working on gene therapy guidances pertaining to safety and CMC issues for some of the products. (Also see “Gene Therapies: Safety, CMC Draft Guidances Coming From US FDA In 2018” – Pink Sheet, 23 Jan, 2018.)


Advance new framework for expediting development and approval of safe and effective cell-based regenerative medicine products

  • CBER released guidances on regenerative medicine and launched the Regenerative Medicines Advanced Therapies pathway in 2017. (Also see “RMAT Designation, Gene Therapies Highlight 2017 CBER Transformation” – Pink Sheet, 17 Jan, 2018.)


Expand oversight and enforcement of cell-based regenerative medicine and address products putting patients at risk and making false health claims

  • CBER’s director has said that enforcement in the regenerative medicine area will increase in 2018. (Also see “CBER Director Marks: Regenerative Medicine Enforcement Will Be ‘Notched Up’ In 2018” – Pink Sheet, 17 Jan, 2018.)


Advance a new program to foster more efficient development of products intended for use in austere environments that can benefit military personnel

  • Amid Pentagon issues with delays in approving some battlefield medical products, FDA said it would offer a breakthrough-like designation to expedite their review. (Also see “US FDA Creates Expedited Review For Military-Related Products” – Pink Sheet, 16 Jan, 2018.)

Better Informed Patients

Establish an agency-wide patient affairs staff to implement best practices for patient engagement

  • FDA is not creating a separate office for it to avoid the potentially lengthy official reorganization process. (Also see “US FDA’s Patient Affairs Office Is An Office … In Spirit” – Pink Sheet, 17 Nov, 2017.)


Establish policies to make more information available from the product review process to better inform consumers and providers

  • FDA announced in mid-January that it would begin releasing clinical study reports to improve transparency and review efficiency. (Also see “Clinical Study Disclosures Will Make Drug Reviews More Efficient – Gottlieb” – Pink Sheet, 17 Jan, 2018.)


Improve the usability of the searchable database of adverse event data reported to FDA

  • FDA unveiled an upgraded adverse event search portal in 2017, but Gottlieb had to issue a statement a short-time later reminding stakeholders of the system’s limitations. (Also see “Gottlieb Issues Statement On Adverse Event Limitations – An Attempt To Quell Fears?” – Pink Sheet, 2 Oct, 2017.)


Launch a new pilot project to create a structure enabling FDA to have its own capacity to more routinely update old generic drug labels with new safety and efficacy information

  • The Friends of Cancer Research released a “straw man” paper in 2017 describing a potential system allowing FDA to update old generic labels with information for current clinical use. (Also see “Labeling Updates For Old Drugs Could Be Faster Under Streamlining Proposal” – Pink Sheet, 28 Nov, 2017.)…