New Drug Application (NDA) Webinar December 6, 2016

Transcription

1 New Drug Application (NDA) Webinar December 6, 2016

2 Introduction Presenter: Jill Jarecki Chief Scientific Officer, Cure SMA

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4 New Drug Application Process

5 NDA Not yet an approved therapy: ~90% chance of success. Expanded Access: Still very limited access: May not be possible for all drug types Criteria, Capacity, Cost. More than trials, less than approval. Not the main goal All about the strength of the data: Better = Broader.

6 The Closer We Get

7 The Drug Discovery Process and Clinical Issues Presenter: Doug Kerr Global Development Team Lead and Vice President for Neurodegeneration, Shire

8 The Drug Development Process

9 Late Stage Drug Development Evaluating data and results Determining when to file an NDA What goes into an NDA package Preparing for a possible approval

10 Important FDA Considerations Presenter: Jeanne Ireland Principal, Ireland Strategies, and Former Senior Advisor to the FDA Commissioner

11 FDA Expedited Programs for Drugs Priority Review shortens review time from 10 to 6 months for drugs for serious conditions that provide a significant improvement over existing therapies Fast-Track allows for early and frequent interaction with FDA and rolling review for drugs for serious conditions that meet an unmet need Accelerated Approval for drugs to treat a serious condition AND meaningful advantage over existing therapies AND demonstrate an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit. Requires post-market studies; FDA can withdraw the product if effect not confirmed. Not a faster review. Breakthrough Therapy -- for drugs to treat serious condition AND preliminary clinical evidence that the drug may demonstrate substantial improvement on a clinically significant endpoint over available therapies, i.e., game-changer. Intensive guidance and support during development stage, commitment from senior review staff, rolling review. Orphan Drugs Benefits of designation Flexibility in applying approval standards

12 Other FDA Considerations Benefit-Risk Assessment Evidence of safety and effectiveness Nature and severity of condition Benefits and risks of other therapies for the condition Risk management tools Labeling Impact of Recent Developments on Drug Review Sarepta decision Transition in Administration

13 Post-Approval Issues Presenter: Kenneth Hobby President, Cure SMA

14 PROVE KNOW BELIEVE HOPE THINK Averages across all the data, not just the few best, to Prove.

15 After Approval Proving it the first step to broad access Make it. Deliver it. Pay for it. Expand it. More Than 1 Therapy: Slow to stop to reverse. Different therapies for ages and types. Multiple therapies over time. Combinations to work better.

16 Steps From Approval to Administration

17 Incentives and Alignment Goal: Families/Patients Companies A safe drug A drug that works For as many as possible* As soon as possible *vs Individual Need

18 Our Priorities for the FDA Obtaining broad labels for drugs across SMA types, ages, and stages of disease progression Educating regulators on the severity of SMA on patients and families daily lives Preventing decline or small incremental changes is critical Improving independence and activities of daily living are key Impact of the first approved drug on the design of future clinical trials Multiple drug options and drug combination therapies Evaluating the use of historical controls versus placebo in SMA type I

19 Q & A Session

20 How Our Community Can Participate Presenter: Megan Lenz Senior Communications Manager, Cure SMA

21 Current Cure SMA Activities Industry Collaboration Group Seven different companies are collaborating with Cure SMA on regulatory topics, education, and clinical trial issues sharing their data and their ideas to benefit our community. Insurance Coverage & Payment Policy Project The goal of this project is to help facilitate a favorable public and commercial coverage and payment environment. Launched in anticipation of FDA approval of new SMA treatments, the project will cover the full range of care and treatment for all types, ages, and stages of SMA. Newborn Screening Initiative Working in partnership with MDA, this project will focus on getting SMA added to the federal Recommended Uniform Screening Panel, and implementing that recommendation in each state.

22 How You Can Participate Patient Focused Drug Development Meeting At the Patient Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to testify directly to the FDA. The testimony will also be used to create a Voice of the Patient report. This report will become part of the review process for all SMA treatments that are submitted to the FDA. Community Update Survey We will soon be launching a new community survey. This survey will cover a number of areas that are relevant to the real-world experiences of living with SMA. Through this survey, we will collect and distribute data to support the testimony presented at the PFDD meeting. The data from this survey will also be used to support the insurance and payment project.

23 Next Steps The Patient Focused Drug Development Meeting is scheduled for Tuesday, April 18, The Community Update Survey will launch within the next several weeks. Stay informed: Visit and click on Advocacy on the right-hand side of the page. Sign up to receive Cure SMA s by entering your address at the bottom of any page on our website. Or, just send an to and say, Please add me to your list!

24 Thank You!