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FDA’s Access Mechanisms When They Work – When They Don’t And Why

FDA’s Access Mechanisms When They Work – When They Don’t And Why. Steven Walker Co-Founder, Volunteer. Abigail Alliance for Better Access to Developmental Drugs.

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FDA’s Access Mechanisms When They Work – When They Don’t And Why

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  1. FDA’s Access MechanismsWhen They Work – When They Don’t And Why Steven Walker Co-Founder, Volunteer Abigail Alliance for Better Access to Developmental Drugs KakkisEverylife Foundation Panel – March 27, 2014How Patients Can Work With FDA and Industry on Expanded Access, Compassionate Use and Emergency INDs

  2. FDA PERSPECTIVE • FDA – Access Sometimes OK If: • Drug is Being Developed (e.g. ongoing clinical trial development program – preferably in same or very similar indication) • No Significant Interference with Trial Enrollment • Risks to Patient Deemed Acceptable or Controlled • All Required Applications are Submitted/Reviewed/Approved by FDA • Informed Consent is Obtained • Institutional Review Board Approval Obtained • FDA Flexibility? • Some Flexibility for Emergency and Single-Patient INDs (Compassionate Use) • Much Less Flexibility for Treatment INDs (Expanded Access Programs) • FDA Legal Authority and Transparency • Authority is Absolute – No FDA Approval – No Access – Period! • FDA is Opaque (in part required by law) – Patients/Public Learn Backstory Only if Sponsor Provides Insight

  3. INDUSTRY PERSPECTIVE • Industry –Sometimes Agree to Provide Access If: • Consistent with Company Policy/Philosophy/Investor Wishes • Think or Strongly Think Drug May Be Safe/Effective for Proposed Use • Have Ability/Willingness to Manufacture Needed Quantities of Investigational Drug • Have Needed Financial and Personnel Resources to Administer Program • Willing to Accept Uncertainties Associated with Allowing Access • Must Decide Whether to Provide Access to All? - Some? - Only a Few? - Only One? • Must Decide Whether to Provide for Free? - or to Charge? • Company Can’t/Won’t Provide Access if : • FDA Won’t Approve - or is Strongly Discouraging - Access • Clinical Development Program Requirements Make Access Program Infeasible (e,g., If FDA is requiring large randomized controlled study(s), need to enroll/complete studies first) • Level of Confidence that Development/Regulatory Pathway to Approval Is Defined • Industry Legal Authority • No Legal Authority – Can Only Provide Access After Complying with FDA Requirements and Receiving FDA Approval – Even for a Single Patient

  4. PATIENT PERSPECTIVE • Patient Situation • Dealing with Serious/Life-Threatening Diseases with Unmet Medical Need And: • No Approved Treatment Available • No Clinical Trial or Trial Eligibility • Usually - No Existing Access Program • Waiting is Not a Reasonable Medical Option • Patient Rationale • Obtaining Access to a Promising Investigational Treatment Represents: • Only Reasonable Medical Treatment Option Other Than Palliative Care • Best Potentially Available Medical Care • Rational and Well-Considered Objective • Precisely the Same Objective Sought by a Patient/Family Who Succeeds in Entering a Clinical Trial for an Investigational Drug • Same Risks – Same Potential for Benefit • Risk of Disease Substantially Outweighs Risk of Treatment With an Investigational Drug • Eyes Wide Open! Patient/Family in Best Position to Weigh Risk/Benefit

  5. Single-Patient IND • It’s all on the patient/family • Try to find a trial – fail • Identify the investigational drug(s) that pose the most potential for benefit • There is no mechanism for a patient/family to request access on own behalf • Find a physician willing to be the sponsor, and request and administer the drug • Convince the company to provide the drug and assist the physician in applying for FDA approval • Most Attempts End Here – FDA Does Not Count as a Request for Access • If successful – wait - often for weeks or months • Wait for application to be prepared and submitted • Wait for FDA review/approval • Seek and obtain IRB approval • If Approved – Wait another 30 days to receive treatment unless FDA waives waiting period

  6. Treatment IND • It’s all on the company • Regulatory Submissions for Approval Approach Requirements of Application for Marketing Approval • Usually Not Considered Feasible Until All FDA Required Clinical Trials are Completed and Approval Application Process is Underway • If FDA Approval of Drug is Uncertain – Starting Program Can Carry Substantial Financial, Procedural and Ethical Risks for Sponsor • Takes Time to Set Up – May Not Actually Launch Before FDA Decides on Application for Marketing Approval • $$ Expensive $$ - All costs usually borne by company • Treatment INDs (aka Expanded Access Programs) are: • Rare • Usually Started Very Late in the Development Program • Usually Small • Have Restricted Eligibility • Generally Underserve the Patient Community with a Legitimate Need for Access

  7. Why So Seemingly Dysfunctional? • Investigational Drug Access Programs are Designed to be Exceptions to the FDA’s Authority to Prevent Marketing and Use of Unapproved Drugs • FDA Insists on Directly Regulating the Provision of Any Investigational Drug to Any Patient – Even for a Single Terminal Patient with No Other Options • FDA Considers Enrolling and Completing Clinical Trials for Investigational Drugs to Be Its Core Functi0n in Establishing Safety and Efficacy for Purposes of a Marketing Approval • The FDA believes that its preferred Phase III clinical trial design – the randomized controlled trial – often blinded and placebo-controlled – might be difficult to enroll and complete if an investigational drug is available by other means

  8. The Blame Game Why Can’t I Get the Investigational Drug I Need? FDA Blames the Drug Company – Says/Implies Agency Would Approve It • The Drug Drug Company Blames the FDA – Says/Implies FDA Won’t Approve It The Half Truth – And Nothing But the Half Truth!

  9. Bottom Line The FDA’S Investigational Drug Access Mechanisms Seem Dysfunctional to Patients Because They are Designed and Intended to Be Relatively Rare Exceptions FDA’s Authority to Regulate Medicines is Based Primarily on Its Power to Say No FDA Believes That If It Allows Broad Access to Investigational Drugs or Approves Drugs Before All the Clinical Trials it Wants Are Completed Some of the Data the Agency Believes Should Be Collected May Be Unobtainable This Tension is Created Primarily by the FDA’s Still Rigid Requirements for Completion of Randomized Controlled Trials (Often Blinded and Placebo-Controlled) Prior to Considering an Application for Approval of Most New Drugs Understandably – Patients with Serious and Terminal Diseases Would Rather Get the Drug Than a Sugar Pill The Solution is Access Mechanisms Designed to be Real Medical Delivery Options Coupled With Smarter, More Scientific, More Ethical Trial Designs – a Related and Extremely Important Subject for Discussion

  10. Working With FDA and Industry On Access • The tough news – in most cases – is that you are on your own • There are presently no financially-resourced, non-profit or for-profit organizations providing services to patients seeking access to investigational drugs – because there is usually no functional pathway for them to follow • If there is no Treatment IND, and initial attempts to gain access through a Single-Patient IND fail – try to work directly with the drug company and the FDA • Request face-to-face meetings, and keep requesting them until you get them. Start at the top – drug company CEO’s and the Commissioner’s office at the FDA • Network and organize with fellow patients and families, and use the media • Remember that the FDA has no drug to give you - but may very well be the reason – either directly or indirectly – behind the drug company’s denial of access. Focus your efforts on understanding the position of the drug company and working with them to make provision of the drug you need a workable option for them

  11. The Hard Reality • If you are seeking access to an investigational drug outside a clinical trial for a serious or life-threatening condition – then you have fallen into a severe gap in our health care system • FDA’s access mechanisms mostly don’t work by design and intent • The more likely outcome is that if initial efforts fail, you will not gain access to the investigational drug you seek, and if you do – it may take a long time – sometimes too long - for that access to become available • Make contingency plans and pursue them in parallel • One of those contingency plans should be for all patients and families facing this dilemma to get involved in solving it for everyone

  12. Abigail Alliance for Better Access to Developmental Drugs www.abigail-alliance.org See the Documentary Fight to Live Directed by the Academy Award Winning Film-Maker Barbara Kopple www.fighttolive.org Available on iTunes, Google Play and Most Cable On-Demand Movie Channels

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