EXPANDED ACCESS: RECENT LEGISLATION AND POLICY UPDATE

Transcription

1 EXPANDED ACCESS: RECENT LEGISLATION AND POLICY UPDATE 21 st Century Cures and Beyond Policy and Regulatory Frameworks Shaping Early Access February 23, 2016 David J. Farber Partner King & Spalding LLP Preeya Noronha Pinto Partner King & Spalding LLP 1

2 Today s Agenda Expanded Access The Basics What s Driving Policy Change Policy Proposals and Solutions FDA and the Congress Where Do We Go From Here? 2

3 What is Expanded Access? Program established by Congress and regulated by FDA Goal is to improve access to investigational drugs by certain patients for treatment purposes Differs from a clinical investigation where the primary purpose is research (i.e., systematic collection of data to determine safety and/or effectiveness) 3

4 Authority for Expanded Access 1997 FDA Modernization Act (amended Section 561 of the FDCA) 21 C.F.R Part 312, Subpart I (FDA regulations have permitted Expanded Access since 1987) Guidance for Industry, Expanded Access to Investigational Drugs for Treatment Use Qs and As. FDA Draft Guidance, May 2013 Charging for Investigational Drugs Under an IND Qs and As. FDA Draft Guidance, May

5 General Criteria for FDA Authorization Patient must have a serious or immediately life-threatening disease or condition No comparable or satisfactory alternative therapy exists FDA must determine that: Potential patient benefit justifies the potential risks, and those risks are not unreasonable in the context of the disease or condition Providing the investigational drug will not interfere with or compromise clinical investigations that could support marketing approval 5

6 Three Categories of Expanded Access Individual Patients (including for emergency use) 21 C.F.R Large Patient Populations (Treatment IND or Treatment Protocol) 21 C.F.R Intermediate-Size Patient Populations 21 C.F.R

7 Individual Patient Expanded Access Physician must determine that the probable risk is not greater than that of the disease Manufacturer must agree to provide the investigational drug FDA must determine that (1) the general criteria are met and (2) the patient cannot obtain the drug under another type of IND or protocol IRB must review and approve EXPANDED ACCESS 7

8 Individual Patient Emergency Use All applicable criteria must be met IRB must be notified within 5 working days of initiation of treatment Emergency use verbally approved by FDA Physician or sponsor must agree to provide a written submission to FDA within 15 working days of FDA authorization 8

9 FDA Authorization of Expanded Access During the fiscal year ending October 2013, FDA authorized 863 requests or 99% of all cases reviewed (1/3 on emergency basis) FDA has authorized 99% of requests since October 2009 (on average, 932 requests annually) 99% authorization rate The FDA Says It s More Compassionate Than You Think, Wall Street Journal, May 5,

10 So What Is Driving Policy Change? 10

11 Josh Hardy Media Firestorm 11

12 Chimerix CEO Takes the Fall 12

13 Ongoing Media Focus CNN September 28, 2013 Andrea Sloan is dying of ovarian cancer. She's seeking "compassionate use" of a new drug that's not FDA-approved, but a drug maker says it's too risky. 13

14 Ebola Raises More Questions 14

15 Navigating the Issues Clinical Data Ethics Cost FDA Population vs. Individual Health Liability Protection & Other Business Considerations 15

16 Pending Policy Changes: Congress States FDA Private Sector 16

17 : Recent Federal Policy Efforts th Congress 111 th Congress 112 th Congress 113 th Congress H.R and S (June 2008) Bi-partisan, Democratic-led effort (Rep. Watson, D-CA) Required FDA to create new expanded access regime Permitted manufacturers, following Phase I, to apply for expanded access authority and to charge for drugs Provided manufacturer liability protection H.R Same as previous bill, but no Senate counterpart H.R Rep. Ron Paul (R- TX) Permitted expanded access upon informed consent of patient Prohibited FDA from regulating access, or even collecting information No Phase I requirement H.R Rep. Morgan Griffith (R- VA) introduces updated version of Rep. Paul s bill, with liability protection for manufacturers H.R Rep. Michael McCaul (R- TX) introduces Andrea Sloan CURE Act, requiring manufacturers to post contacts for requests and provide denials in writing, and requiring GAO and Task Force Reports to Congress 17

18 2016: Pending Policy Changes H.R. 790 Rep. Griffith Act re-introduced H.R. 909 Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act H.R. 6 ( 21 st Century Cures ) Section 2082 modified version of H.R. 909 H.R Right to Try Act of

19 Congress Is Actively Considering the Issues Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act (H.R. 909) Introduced in 113 th and 114 th Congresses, by Rep. Michael McCaul (R-TX) Requires manufacturers of breakthrough drugs to make expanded access policies publicly available, and provide written explanations for denials Commissions a GAO analysis of expanded access requests Establishes an Expanded Access Task Force at HHS to explore ways to improve patient access to investigational drugs Requires FDA to define how adverse drug event data reported through expanded access is used Sections of H.R st Century Cures Act Passed by House on July 10, 2015 (still needs to be passed by Senate to become law) Would require manufacturers to make publicly available a policy to evaluate and respond to expanded access requests Policy requires point of contact, procedures for making requests, general criteria for approval, and anticipated timeline for acknowledgement of receipt of requests Would require FDA to finalize Expanded Access Q&A Draft Guidance (May 2013) Final guidance shall define how FDA interprets and uses adverse event data reported from expanded access 19

20 Some in Congress Trying to Build on State Right to Try Laws Twenty-four States have adopted Right to Try laws as of August 2015 Approximately 14 other States have introduced similar legislation Congress is also joining in the effort H.R Right to Try Act of 2015 (Reps. Salmon, Gosar and Stutzman) would require FDA to respect RTT Laws 20

21 H.R Right to Try Act of 2015 Proposes to federalize state RTT laws by prohibiting FDA from blocking access if: Intended to treat a patient who has been diagnosed with a terminal illness Is authorized by, and in accordance with, State law Drug is past Phase I clinical trials With three co-sponsors to date, uncertain prospects in Congress 21

22 Status of State Right to Try Laws Source: 22

23 Do s and Don t s of Right to Try Laws What They DO: Generally, they aim to grant patient access to investigational treatments if: Patient is terminally ill Physician recommends treatment Patient provides informed consent, and Treatment has completed Phase I clinical safety/dose limitation trial They grant physicians and manufacturers liability protection against claims arising from adverse events caused by investigational treatments What They DON T DO: They don t create a right for terminally ill patients to access life-saving treatments exempted from FDA rules State laws do not permit manufacturers to provide patients with access to unapproved drugs, when the federal law mandates no person shall introduce or deliver for introduction into interstate commerce any new drug They don t address questions of patient welfare and public health They don t provide free access to experimental treatments They don t compel anyone or any company to fulfill a patient request 23

24 Is FDA Trying to Get Ahead of the Curve? FDA issued a Proposed Rule on Clinical Trials on November 19, 2014 If expanded access is authorized, Responsible Party would submit an Expanded Access Record with a unique ClinicalTrials.gov Identifier Applicable only to expanded access for Intermediate- Size Patient Populations and Large Patient Populations (not for Individual Patients) Responsible Party would submit various information, including eligibility criteria, status of drug availability via the program, contact information and the IND number Responsible Party would be obligated to update and correct information What will FDA do in the Final Rule, and will it preempt legislation? 24

25 FDA Reduces the Paperwork In February 2015, FDA issued Draft Guidance containing a new form (Form FDA 3926) for physicians to make expanded access requests for individual patients Form FDA 3926 streamlines the process for making such requests, which would otherwise need to be made on the general IND submission form 25

26 Policy Changes Being Driven By the Private Sector A Dutch startup, mytomorrows, has raised 4.5 million Euro to fund a business that navigates expanded access requests ( and has competition from Clinigen Group Nonprofits are also trying to establish a space for navigating requests, such as the ALS Emergency Treatment Fund ( IRBs and others are also entering into the space through nonprofits, such as the WCG Foundation, set up by WIRB-Copernicus Group ( 26

27 What s Next? Social media may overtake planning and procedure FDA will continue to authorize the vast majority of the Expanded Access requests it receives Congress is engaging, forcing policy change States, driven by the Goldwater Institute, will continue to enact Right to Try laws 27

28 Where Do We Go From Here? Should patient advocacy and rare disease groups engage, and if so, where? This is not a simple issue there is as much opportunity to do harm than to do good Is social media the best tool for such a nuanced issue? And how does one become a responsible voice in the debate? 28

29 QUESTIONS? THANK YOU! David J. Farber (202) Preeya Noronha Pinto (202)