Effectiveness Marlene Haffner, MD MPH Haffner Associates, LLC

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Presentation transcript:

Regulatory Considerations for the Development of Substantial Evidence of Effectiveness Marlene Haffner, MD MPH Haffner Associates, LLC World Orphan Drug Congress 2014 April 24, 2014

History of regulation in the drug industry Sulfanilamide - 1938 - safety Thalidomide - 1962 - safety and efficacy PDUFA - 1987 - post marketing requirements REMS - 2007 - stricter post marketing controls for safety and efficacy evaluation Safety and Efficacy - risk/benefit - intertwined

Need for a path to drug development What is the disease and what is known about the disease - Natural History Is the disease life altering/shortening? Is the disease of genetic origin or not? Is the gene known? Are you treating symptoms or affecting the gene? Is the disease monogenic or heterogeneous? Is there a support group for the disease? And what is their interest? What is the drug and how will it affect the disease? Do we know the mechanism of action of the drug? What animal studies have already been done? Is the drug approved for another use? - repurposing

ProActive engagement with patients/advocates and FDA What does FDA need; What do patients want Design the trial to show efficacy - work proactively with FDA Listen to the patients/advocates Codified in FDASIA Know the disease and the natural history of the disease Ongoing activities between FDA and patient groups - PPMD and development of guidance for endpoints in DMD Increasing transparency of all involved - FDA and industry

New potential methodologies for preclinical and early clinical work BioPontis Foundation Other similar models No need to continually reinvent the wheel

Development of Clinical Trials Whenever feasible - placebo controlled. Fastest way to get answers Build in a follow-on with active drug - if appropriate Involve the patient community they know the disease and where patients can be found Small populations have their own difficulties no chance to perform multiple trials Multicenter/multi country trials may be needed with very small populations. Always more than one center. Always hard to find the patients

Endpoint and BioMarker Development Constructive plans for endpoint and biomarker development Validation of surrogate endpoints Involve the patients Expect long term follow-up FDA becoming more involved and interactive May/may not be necessary to utilize surrogates

Reaching the Goal - an Effective Product Goal -to develop an effective and safe product as quickly as possible Many orphan drugs will qualify for “Breakthrough status” Keep the patients involved Continual dialogue with FDA Closely monitor the trials. No opportunity for errors

Orphan Drugs must be Safe and Effective for their intended use FDA is flexible but risk/benefit is the golden rule Orphan disease patients do not want to be exposed to unnecessary risk or lack of efficacy Statistics must be clean - Figures don’t lie; but liars figure! May need to evaluate previously unknown subgroups Work with the patients

Marlene E. Haffner, MD, MPH Questions Marlene E. Haffner, MD, MPH President & CEO 11616 Danville Drive Rockville, Maryland 20852 http://www.mhaffner.com mhaffner3@verizon.net 301 984 5729 (office) 301 641 4268 (cell)