C URE T HE P ROCESS 2: Improving the Development Process for Rare - - PowerPoint PPT Presentation

CURE THE PROCESS 2:

Improving the Development Process for Rare Disease Treatments

Emil D. Kakkis, M.D., Ph.D.

President and Founder

October 29, 2014

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About the Foundation:

• Dedicated to Accelerating Biotechnology

Innovation for Rare Disease Treatments

• Advocating practical and scientifically sound

change in policy and law to increase the predictability of the regulatory process through scientific analysis and dialogue, grassroots support & expert-led workshops.

• Foundation Mission:

– No disease is too rare to deserve treatment – All treatments should be safe & effective – We could be doing more with the science we already have

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Roadblocks in the Development

• f Rare Disease Treatments
• More challenges for rare diseases than common diseases

– Due to their rarity, there is often limited understanding of the clinical disease, even if the science underlying the disease is clear

• Lack of a defined regulatory pathway based on:

– Limited prior clinical study experience and no natural history information – Challenges defining new clinical endpoints to assess efficacy – Lack of acceptable biomarkers indicating clinical improvement – Fewer variable patients to participate in clinical trials, thus requiring alternative clinical trial designs and analysis.

• Potential treatment development cost requirements are

too great and too uncertain for many rare diseases, making them less attractive to investors

• Launched May, 2009 at the Rare Disease Summit

in Washington, DC

• Tasked with improving the clinical development

and regulatory process for rare diseases through science-driven changes in public policy

First Cure The Process Campaign

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Original Goals of the CURETHEPROCESS Campaign

• Specialize Review: Establish a new Office of

Drug Evaluation specifically for Biochemical and Genetic Diseases

• Increase Accelerated Approvals :Create new

qualification criteria for surrogate endpoints to allow Subpart H Accelerated Approvals

• Improve Clinical Designs and Analyses: Devise

acceptable optimal statistical analyses and alternative designs for clinical endpoint driven pivotal studies

Moving a Vision to Reality

• Goal: Science-driven public policy changes
• Initiated Workshop Series

– Science-based discussion – Rational strategies – Development solutions – Drive policy ideas

• Organized & educated patient groups

– A unified force to compel change

• Published White Papers
• Worked with Congress to move Legislation

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Success in Moving Legislation Through Congress: ULTRA/FAST

New law expands access to Accelerated Approval process

• Drafted legislation with Reps.

Stearns & Towns –ULTRA/FAST Acts

• Vigilant communication and

cooperation with key stakeholders

• FAST language inside the PDUFA V bill (FDASIA, S. 3187)
• Organized the patient community as a political force
• 300+ patient organizations signed on to support ULTRA/FAST
• 3,675 emails were sent to Congress to support ULTRA/FAST

FDASIA signed by President Obama July 9, 2012

• Working with Stakeholders to Implement Legislation

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Thank You to Our 181 Partners

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FDASIA is a Great Start but More Needs to be Done

• Lack of expertise and specialization on

complex rare diseases in drug review divisions

• Early stage clinical trials start first oversees,

delaying access to treatments for US patients

• Companies are not repurposing drugs for

rare diseases, forcing patients to use off label drugs with no safety or efficacy data

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Introducing Cure The Process 2

• Rationalize: Encourage the FDA to accommodate a

more scientifically rational and flexible application of safety data to allow US patients to have access to early stage clinical trials.

• Incentivize: Enact legislation encouraging industry to

repurpose major market drugs for rare disease patents or “rare-purpose” drugs to ensure patients have access to safe, effective and affordable treatments.

• Specialize: Encourage the FDA to create more

specialized drug review divisions and allow reviewers access to the latest science to facilitate a better understanding of the diseases they are reviewing.

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Specialization for Review Decisions on Rare Diseases

• Specialized experience and training
• Allowing for concurrent academic activities will let

reviewers keep informed on the latest science

• Allow sufficient therapeutic focus to be an expert
• Allow sufficient time to research complex target

indications and to hone skills The needs of the increasingly complex rare disease therapeutic areas are creating additional challenges for FDA

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No Disease Is Too Rare to Deserve Treatment

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Rationalizing Safety Requirements

• The FDA’s requirements are causing early stage clinical trials

to be moved outside the US

• US patients are being denied access to these potentially

lifesaving trials

• To avoid delays, many rare disease programs have left

the US

• FDA should follow the same ICH guidelines and risk-based

flexibility that are applied by other competent authorities

• Rationalizing treatment safety:
• Refine current toxicology requirements to a model using

better risk-based scientific justification

• Most importantly, always ensure treatments are safe &

effective

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Rare Disease Scientific Workshop

• Hosted Scientific Workshop in 2014 on “Rationalizing

Safety Testing to Enable Clinical Studies and Approval in the US for Rare Disease Treatments”

• Developing paper based on Workshop outcomes

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Incentivize the Investment in Rare Disease Drug Development

Introducing Orphan Product Extensions Now Accelerating Cures & Treatments (OPEN ACT) before Congress in November

• Hundreds of large market patented drugs exist with

novel technologies and diverse possible uses

• Companies will not invest to test these drugs in rare

diseases

– No financial incentive – Increased risk that adverse events might taint the product when treating very sick orphan diseases

• Can we create an incentive to study patented drugs

in rare diseases as soon as they are approved?

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How a New Orphan Product Repurposing Exclusivity Could Work:

Qualifying for OPEN-Act Extensions

• Sponsor receives FDA approval for their major market

drug or has a current approved drug still under patent protection

• Sponsor could seek rare disease indications to extend

its patent life & protect revenue from market competition for 6 months

• Must be a Rare Disease – under 200,000 patients in

the US

• Must qualify for Fast Track Designation: serious or

life-threatening disease

• Must obtain data to place the new rare disease

indication on the label

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How a New Orphan Product Repurposing Exclusivity Could Work: The Development Process

• Sponsor begins rare disease indication development:
• multiple trials for multiple rare diseases
• Establish the dose, the safety and the efficacy
• High quality rigorous studies conducted
• Company receives new rare disease indication on the

label

• Safety, efficacy and dosing demonstrated
• Obtains 6 months additional market exclusivity at the

end of the product’s patent life or NCE exclusivity period

• Would stack on top of BPCA to provide an additional

incentive to develop pediatric cancer & rare pediatric disease treatments

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Key Benefits of Rare-Purposing* in the OPEN-ACT to Speed Development

• Sponsor already exists for the program
• Leverages existing expertise of clinical development

staff and scientists

• Manufacturing and toxicology work complete
• Safety is known in humans
• Reduced time for development trials & approval
• Focus on science, and rare disease clinical studies
• Rare-purposed Orphan Drugs will likely cost less than

typical orphan products: Drug price will be set by the large market indication; less than typical orphan drugs

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Outcomes – Win Win Win Patients, Companies, Economy

• Public Health: Patients have access to safe & effective

treatments more quickly

• Reduced Drug Prices: Repurposed drugs will be priced

at major-market rates

• FDA: Improved academic excellence, increased

specialization, and the best science will ensure the US maintains the best regulatory standards

• Economy: Increased biotech investment, renewed

innovation and more high quality US jobs in biotechnology

21st Century Cures Initiative

• Implementation of the Cure The Process 2 goals

has already begun with the 21st Century Cures Initiative

• Submitted White Paper comments on first series of

21st Century Cures Roundtables

• PDF of comments available on EveryLife Foundation Website
• Working closely with Energy & Commerce Committee

Leadership

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Help Us Reach Our Goal of 200 Partner Organizations

• Endorsement packet included in webinar

confirmation email, or visit www.CureTheProcess.org to download

• Review the provided materials, and have the

endorsement letter signed by the organization’s most-senior executive

• Submit your endorsement letter to

CureTheProcess@everylifefoundation.org

• Promote the campaign to your members & partner
• rganizations

How to get involved:

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Questions?

Be Sure to Follow us

• www.CureTheProcess.org
• @CureTheProcess on Twitter

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