Transcript presentation slides - Life Science Tennessee

Afternoon Breakout Session:
FDA and Regulatory Environment As We Enter the User
Fee Reauthorization Period
Mike Druckman, Partner, Hogan Lovells
FDA Update: Regulatory
Environment and PDUFA
Reauthorization
Mike Druckman
November 2016
Topics
1.
PDUFA VI highlights, including innovative trial design, MIDD, and RWE
initiatives
2.
Impact of Sorrell, Caronia, and Amarin on ability of sponsors to make claims
beyond approved labeling
3.
CBER Reorganization
4.
FDA’s orphan drug policy and strategies to consider
5.
Other issues of interest to the audience
Topic 1
PDUFA VI Highlights
PDUFA VI Reauthorization Timeline
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Topic 2
Impact of Sorrell, Caronia, and Amarin on ability of sponsors to make claims
beyond approved labeling
Topic 3
CBER Reorganization
Topic 4
FDA’s orphan drug policy and strategies to consider
Scope of Exclusivity
Blocks Approval of the “Same Drug”
• Same Drug analysis has two components
– Chemical comparison
– Clinical comparison
• 316.3(b)(14): Same drug means:
– (i) small molecule drug: the “same active moiety as a previously approved drug
… for the same use … except that if the subsequent drug can be shown to be
clinically superior to the first drug…”
– (ii) If it is a drug composed of large molecules (macromolecules), a drug that
“contains the same principal molecular structural features (but not
necessarily all of the same structural features)…”
– Proteins, polysaccharides, polynucleotides, “closely related, complex partly
definable drugs…”
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Scope of Exclusivity
Clinical Superiority
• 316.3(b)(3): Clinically superior means “shown to provide a significant
therapeutic advantage over and above that provided by an approved
orphan drug (that is otherwise the same drug)” in one of three ways:
– Greater effectiveness as assessed by effect on a clinically meaningful endpoint in
adequate and well-controlled trials;
– Greater safety in a substantial portion of the target population; or
– In unusual cases, a demonstration that the drug makes a major contribution to
patient care
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Clinical Superiority as Prerequisite for Exclusivity
• OOPD has long taken the position, now codified in the regulations, that a
clinical superiority showing is required whenever there is a previously
approved “same drug”
– Where first drug has ongoing exclusivity: needed to “break” exclusivity and gain approval
– This is the basic operation of the clinical superiority regime as originally conceived
– Where first drug has expired exclusivity: needed to obtain exclusivity
– Where first drug never had orphan exclusivity: needed to obtain exclusivity
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Clinical Superiority as Prerequisite for Exclusivity
Depomed and Eagle cases
• Depomed case
– Depomed’s Gralise (gabapentin) for PHN; Pfizer’s Neurontin never had orphan
exclusivity
– OOPD designated Gralise on a plausible hypothesis of clinical superiority under old
regulations
– DDC held that Orphan Drug Act unambiguously prohibited FDA from imposing
additional requirements for exclusivity beyond designation and approval; Chevron Step 1
• FDA announced that it will not apply the Depomed holding. Policy on
Orphan-Drug Exclusivity; Clarification, 79 FR 76888 (Dec. 23, 2014)
• Eagle v. Burwell: pending in DDC
– Eagle’s Bendeka (bendamustine HCl) for CLL/iNHL; Cephalon’s Treanda has expired
orphan exclusivity = second scenario
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Fixed Combination Drug Products
• OOPD considers fixed-combination drug products to be “different drugs”
than single ingredients
– We are aware of a handful of examples where OOPD has designated the combination as
well as the single ingredients as orphan drugs for the same disease
– Separate designation requests
– Scope of exclusivity is similarly limited
– For recombinant plasma therapeutics, FDA has considered addition of fusion protein a
factor in rendering a product to be not the “same drug”
• What about co-administration use?
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Strategies to consider in being first to approval
• FDA’s Expedited Programs:
– Priority review (shortens FDA PDUFA deadline to 6 months from acceptance for filing)
– Fast Track
– Breakthrough therapy designation
– Accelerated approval (surrogate endpoints)
• Priority Review Vouchers (Tropical Disease; Rare Pediatric Disease)
– Entitles holder to priority review (can be purchased)
• Adaptive and innovative trial designs
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Topic 5
Other issues of interest to the audience
Other potential issues of interest
• FDA’s increasingly aggressive position on international inspections –
– Including citations based on processes used to manufacture products outside the U.S.
and shipped wholly outside the U.S., based on U.S. standards, when the manufacturer
makes identical or similar products that are imported into the U.S.
• Priority review vouchers – for tropical diseases and rare pediatric
diseases
– Sold for as high as $350 million
• Resurgence of gene therapy clinical trials
– Challenges of genetic indications and genotyping programs
• Human tissue draft guidance documents and recent public hearings
• Final rule on clinicaltrials.gov results submission – now results must be
posted even if the drug has not been, and never will be, approved
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