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Right to Try, Tier One Approval, and Other Approaches to Access to Investigational Therapies Impact on Individuals and Drug Development NCCS Cancer Policy.

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Presentation on theme: "Right to Try, Tier One Approval, and Other Approaches to Access to Investigational Therapies Impact on Individuals and Drug Development NCCS Cancer Policy."— Presentation transcript:

1 Right to Try, Tier One Approval, and Other Approaches to Access to Investigational Therapies Impact on Individuals and Drug Development NCCS Cancer Policy Advocate Training Washington D.C. November 13, 2014 Robert Erwin

2 Why Look Beyond the “Standard of Care?” The standard of care in oncology is not good enough Science is yielding progress Evidence varies in quality and utility and there is no minimum standard for “standard of care” Statistics do not apply to individuals Every oncologist should read The Median Isn’t The Message by Stephen Jay Gould

3 After Standard Treatment Failure – What are a Patient’s Options?  Selection of a “thought leading” physician (early adopter of newly approved drugs/individualized care)  Off-label use of approved drugs  Enrollment in clinical trials  Expanded access programs for investigational agents  Quality of life care (also called hospice or palliative care)

4 Optimism About Experimental Drugs With currently approved drugs Hope is for high probability of benefit with possibility of harm, but... Reality is certainty of harm with possibility of benefit. An opportunity for a positive outcome has value Failure does not cancel the importance of the possibility of benefit Should the risk/benefit judgment be an individual or a societal decision?

5 Clinical Trials – What Is Important For a Patient To Know?  Understand and evaluate possible conflicts and combinations of interest  What is the potential for therapeutic benefit?  Entry & exclusion criteria; endpoints & monitoring  Options for subsequent treatment, if needed  Informed consent - Not one signed document, but rather an ongoing process

6 Evaluation of Potential for Therapeutic Benefit from a Clinical Trial  Begin with patient’s medical record and preferences  Eliminate trials for which patient is not qualified  Review medical literature on similar trials of possible interest  Eliminate trials that are not practical for the patient (geography, for example)  Tap a network of advisors

7 Narrowing Choices Important questions about first choice trials  Number of patients treated so far with this agent and/or protocol?  Responses and types & duration of responses in prior studies?  Side effects?

8 Narrowing Choices Further Sources of information about first choice trials  Informed consent documents  Protocol document  Informal information from other patients  Abstracts and interim analyses from scientific meetings  Company press releases

9 Why Clinical Trials Are Not Enough  Poorly resolved conflicts between research and treatment  Equipoise and community equipoise  Placebos and ineffective “standard of care”  Leads to slow accrual and delay in gaining knowledge  Restrictive enrollment criteria  Small patient populations are “underserved”  Conclusions may not be generally applicable to “real” patients

10 Do Expanded Access Programs Offer Realistic Hope?  YES, If the experimental drug works!  One example, Gleevec - Ann Oncol (2008) 19 (7): 1320-1326  Novartis provided early access to 7,380 patients in 34 countries prior to FDA marketing approval  TTP, OS, and safety were similar to positive Phase 2 data used for drug approval  Additional EAPs have yielded positive indications of benefit  Lapatinib – Ann Oncol (2010) 21(3): 474-480  Sunitinib – Oncologie (2010) 33 Suppl 1:12-14  Ipilumumab – Cancer Immunol Immunother (2011) 60(4) 467-477

11 So, What is the Problem?  A...  B...  C...  D...  E... But let’s start here: Notice that the FDA is not on this list !

12 Anti-FDA Propaganda “There’s every reason to believe that the FDA approval process is killing as many or more people than it saves, especially as the FDA doesn’t allow approvals from Europe and elsewhere to stand in for trials here.” The FDA prevents access to “life-saving” drugs The FDA approves hundreds of individual compassionate access requests each year and virtually never turns down a request. There are no cases of a drug approval being delayed due to an adverse event or even death in a compassionate use situation

13 Tier 1 Approval and Right to Try Petition by the Abigail Alliance and subsequent lawsuit against the FDA 2003 Petition District Court defeat Court of Appeals 3-judge panel victory 2007 Court of Appeals en banc defeat (8-2) Supreme Court denied petition to review This was valuable work despite defeat State Right-to-Try Laws Dallas Buyers’ Club movie Goldwater Foundation Symbolic politics without tangible effect This may contribute to policy refinement

14 FDA’s Role in Early Access to Investigational New Drugs  1987 – Revision of IND regulations to provide for treatment protocols  1997 – FDAMA included expanded access provisions  2006 – Draft Guidance to address inconsistent application of access policies and inequities in access  2009 – Final rule on expanded access to investigational drugs for treatment use & final rule on charging for investigational drugs  2013 – EAP Q&As – Draft Guidance

15 FDA Rules on Expanded Access to New Investigational Drugs A Fair Balance  Individual patients’ desires to make their own decisions  Society’s interest in efficient development of new therapies  Need to protect vulnerable patients from unnecessary & unacceptable risks

16 So, What is the Problem?  Opposition to access by corporations and their legal and “ethics” advisors  Ignorance  Logistical complexities including manufacturing limitations and inefficiency of local IRBs  Costs  Greed, and other human frailties

17 Anti-EAP Propaganda  Academic terms created to justify action and non-action  “Community Equipoise” – A handy concept enabling the “ethical” enrollment in a randomized clinical trial with an inferior treatment arm  “Therapeutic Misconception” – A handy description helpful in denying patients access to experimental drugs  Trade organization (BIO and PhRMA) sugar-coated justifications for avoiding decisions  “Patient safety is our first priority”  “FDA approval is the best way to make promising drugs available to larger numbers of patients”

18 Corporate Communications "This is a challenging request since it involves complex regulatory, political, manufacturing, and fiscal issues that need to be addressed before a compassionate access program can be developed." This statement might not have been so bad if the company that made it had actually “addressed” any of the issues

19 Corporate Communications “Use of experimental medications outside of clinical trials unnecessarily delays the ultimate availability of conclusive evidence on the safe, effective and appropriate use; hampers the health authorities’ consideration for approval; and can obstruct access outside of clinical trials. This position is well supported by most constituencies who work in drug development, including patient advocacy groups.” Is any part of this statement true?

20 Expanded Access Myths and Realities  Properly designed Expanded Access Programs DO NOT conflict with clinical trials  Exclude patients who are eligible for clinical trials of the experimental agent  Begin Expanded Access for trial-eligible patients only when trial accrual is complete  Expanded Access Protocols can provide useful data about a drug’s impact in the “real” target market  EAPs can often be designed to “ask a question”

21 The Ethics of EAPs  EAPs should be designed with reasonable safety and efficacy data in hand  While companies receive “profile” and “pre-approval awareness” benefits, promotion should not be the driving force  EAPs are generally better than individual compassionate access (single patient INDs)  Fairness matters – However, too few life boats doesn’t mean the right thing is for everyone to drown!

22 There is Good Corporate News!  As of yesterday, there were over 40 open cancer expanded access protocols listed on, most sponsored by companies: Bayer (2) Merck Boehringer Ingelheim (2) Miltenyl Biotec SynCore Biotechnology BTG International Eisai Inc (2) Novartis Pharmceuticals (2) Pfizer (2) Cold Genesys Regeneron Pharmaceuticals Taiho Oncology Northwest Biotherapeutics Eli Lilly BTG International ERYtech Pharma Bristol-Myers Squibb (2) Amgen (2) Catalyst Pharmaceutical Jacobus Pharmaceutical

23 Final Thoughts  Policy is designed for groups but is experienced by individuals, and individuals are more than “anecdotes”  Creating the circumstances under which an individual’s life might be extended requires activism, while creating effective and broadly useful policy requires advocacy  Activism and Advocacy are both most effective when backed by facts and driven by passion

24 Thank you I also thank and acknowledge the many good and wise physicians and patients who have guided my work as a patient advocate Marti Nelson Cancer Foundation Email:

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