A Discussion Focused on Right to Try

Transcription

1 Abigail Alliance for Better Access to Developmental Drugs A Discussion Focused on Right to Try CBI Expanded Access Programs 2017 March 9-10, 2017 Alexandria, VA Steven Walker Co-Founder, Abigail Alliance

2 The Origins of Right to Try FDA s Access Programs 1987 creation by FDA of the Treatment IND in Parallel Track policy issued by FDA in 1992 in response to HIV/AIDS community activism 1997 Food and Drug Administration Modernization Act (FDAMA) codifies the Treatment IND and Single-Patient IND 2009 FDA promulgates a rule setting forth its policies regarding access to and charging for investigational drugs. The 2009 regulations effectively codified and clarified existing agency policy and practice, but made no material improvements FDA Commissioner s office begins internal program to track FDA s performance on its access programs 2016 FDA issues revised application form and final guidance on expanded access and charging for investigational drugs st Century Cures Act requires sponsors to develop and make available to the public their policies and contact information for patients and physicians

3 Performance of FDA s Access Programs FDA now tracks how many access applications it receives and approves each fiscal year Emergency INDs (single patient for each approval) Single-Patient INDs Intermediate Treatment INDs (up to about 100 patients) Treatment INDS (more than 100 patients) Single Patient Protocols (61 in 2015) Single Patient Emergency Protocols (20) Intermediate Treatment Protocols (9) Treatment Protocols (10) Treatment Protocols are clinical trial extensions generally started near the end of a Phase III registration trial to allow trial participants to continue, or through cross-over to start, receiving the investigational treatment. May or may not allow patients not in the trial to enroll.

4 Performance of FDA s Access Programs

5 Patient Advocacy Efforts to Improve Performance Abigail Alliance 2002 Informally proposed a tiered approval model to FDA designed to recognize the unique risk/benefit conditions of seriously- and terminally-ill patients who had run out of approved treatment options FDA rejected the proposal in writing Early 2003 Abigail Alliance and Washington Legal Foundation filed a Citizen s Petition with FDA proposing the tiered approval system FDA has never responded to the Citizen s Petition 2003 to Abigail Alliance and Washington Legal Foundation filed a constitutional lawsuit Abigail Alliance v von Eschenbach - asserting that the right to try to save one s own life by accessing investigational drugs under a responsibly limited set of conditions is a fundamental right. Lost in circuit court won on appeal to DC court of appeals lost in en banc rehearing denied certiorari by SCOTUS DC appeals court opinion stated that nothing in the ruling prohibited addressing the issues in legislation 2007 to 2010 Worked with Senators and Representatives to introduce the Access Act in the 110 th and 111 th Congresses proposing a tiered approval system, revisions to FDA s access programs, scientific modernization of FDA and other specific reforms Bills did not advance to a floor vote but kept the subject before the public and Congress and proposed ideas that continue to be discussed and included in proposed and passed legislation

6 The Result Availability of Investigational Drugs Falls Far Short of the Legitimate Need The 2015 Math (an estimate) Single Patient INDs Single Patient Emergency INDs Intermediate Size INDs (100 patients x 46) Treatment INDs (500 patients x 2) 692 patients 494 patients 4,600 patients 1,000 patients Total (estimated) 6,786 patients

7 Right to Try General Facts The Right to Try (RTT) campaign is being lead by the Goldwater Institute a conservative and libertarian think tank but the underlying concepts have been in discussion for decades RTT was first adopted in Arizona in a state-wide election with 78.5% voting for the law RTT has now been passed and enacted in 33 states with overwhelming bipartisan support in fact near unanimous support Federal RTT laws written to prohibit federal pre-emption of State RTT laws have been introduced in the US House and Senate

8 State Right to Try Laws Eligibility - Patients with terminal diseases as determined by qualified physicians Patient has exhausted approved treatments and has considered or been denied access to an appropriate clinical trial Patient must provide written informed consent Investigational drug has completed at least a Phase I clinical trial under an FDA IND and is in continuing development in clinical trials under an FDA IND Provides certain legal and regulatory protections for physicians, drug companies and others participating in treating the patient Allows but does not require sponsors, physicians and certain others treating the patient to receive compensation Sponsors, physicians and others not required to provide treatment under RTT Insurance coverage not required or prohibited FDA and IRB approvals not required

9 Right to Try State Voting Data 33 States Have Enacted RTT More to Come

10 Federal Right to Try H.R Compassionate Freedom of Choice Act of 2017 Prohibits the Federal Government from preventing or restricting, the manufacture, importation, distribution, or sale of an investigational drug or device intended for use by a terminally ill patient; prohibits requiring the collection and submission of information regarding the treatment but allows the sponsor to voluntarily collect and submit information; and, provides certain liability protections for the sponsor, physician and others who participate in providing the treatment. H.R 878 & S. 204 Right to Try Act of 2017 For patients receiving an experimental drug, device of biological product authorized and in accordance with State law, prohibits the Federal Government from taking any action to prohibit or restrict the production, manufacture, distribution, prescribing, or dispensing of the experimental drug, biological product, or device to a terminal patient, and the possession or use of an experimental drug, biological product, or device, for which the patient has received a certification from a physician, who is in good standing with the physician s certifying organization or board, that the patient has exhausted, or otherwise does not meet qualifying criteria to receive, any other available treatment options. Provides liability protections at the federal level similar to State law protections. Prohibits use of outcome information from the treatment by a Federal agency reviewing the experimental drug, biological product, or device to delay or otherwise adversely impact review or approval of such experimental drug, biological product, or device. Experimental drug is defined as a drug, device or biological product that has successfully completed a Phase I trial, remains under investigation in an FDA approved clinical trial, and is not approved for commercial distribution by the FDA.

11 Some Additional Perspective In 2015 the American Cancer Society estimated that 589,430 people died from cancer and are projecting deaths at about 600,000 for In 2015, PhRMA reported that 836 medicines were in development for the treatment of cancer In 2014 (most recent available), the CDC estimated that more than a million people died from other common and rare diseases (e.g., heart disease, strokes, Alzheimer s, Parkinson s, etc.) Many medicines are in development for a wide range of common and rare diseases. The support for Right to Try is based on a broadly-held and rational perception among much of the patient community and the public that our system is not working in the best interests of terminally-ill patients.

12 Closing Thoughts Whether you agree or disagree that Right to Try is the Appropriate Solution Don t Miss the Message Thirty-three states have passed Right to Try laws and prospects for passage of a federal law authorizing them to function without federal interference appears likely Right to Try is a movement with momentum and staying power The pressure of accelerating medical progress will only increase the pressure for investigational drug access The new administration has signaled its support for fundamentally changing our health care and medical innovation systems, and it has signaled clear support for Right to Try If you think there is a better way to very substantially improve access to investigational drugs for seriously- and terminally-ill patients start doing it!