Transcript Slides - National Health Council

Patient Engagement in Drug Development:
Experiences, Good Practices, Lessons Learned
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About Duchenne muscular dystrophy
X-linked, pediatric neuromuscular disease, with onset in early childhood
Incidence rate: 1:4600 boys (30% spontaneous)
Diagnosis: 3-5 years of age
Predictable course
Progressive loss of function
100% lethal
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Why are we here?
A drug development ecosystem is a community of stakeholders (universities,
companies, patient organizations, patients, government organizations) living in
conjunction with the nonliving components of their environment (regulations,
economic factors, reimbursement potential), interacting as a system. These
components are linked together through clinical research cycles and funding flows.
Companies
Economy
Government
Regulations
Patients
Universities
Patient organizations
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Reimbursement
Importance of the Patient Voice
GOVERNMENT
PROVIDERS
INDUSTRY
PATIENT
VOICE IS
INMPORTANT
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An organized patient voice
is necessary and important
for change.
Regulatory Journey
•
•
•
•
•
Policy
Partnership
Include the Patient Voice
BUT –
Request Transparency from
Regulatory Agencies
• Understand What was Included
into the Decision Making Process
•
– Patient Reported Outcomes (PRO)
– Preference Data
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PPMD has been advocating for
methods to assess the benefitsrisks of treatments for rare
disease.
We have also:
• Validated this work
• Studied caregiver worries
• Published a white paper on
benefit risk
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7
“When it comes to terminal illnesses [the
FDA’s] job should be to make sure a product is
safe and that the risks and benefits presented
by the producer are accurate. Our job should
be to determine, given all that information,
whether to give it to our children. It is an
intensely personal decision that involves the
parents and the child with Duchenne.”
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WORKING with BIO
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9
FDA Engagement – A collaborative
Community
•
•
•
•
Meetings with Division of Neurology
Duchenne Policy Forum (December 2013)
PPMD submits Draft Guidance (June, 2014)
NIH/FDA/PPMD meeting –dystrophin quantification
methodologies (February, 2015)
• FDA releases Draft Guidance (June, 2015)
Setting the stage for the draft guidance
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Clinical Trial, NDA, AdComm
• flawed study
– Outcomes, dystrophin, open
label, small number
– Scientific community support
– 1000 people participated in
the Advisory Committee
– 52 speakers only 1 negative
– Advisory Committee Split Vote
– PDUFA Date ignored
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The Decision
• September 19 2016
– Eteplirsen Approved (Exondys 51)
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The Fallout
• 162 page FDA
document
describes agency
turmoil
• NORD meeting Dr.
Jenkins states
‘eteplirsen should
not be approved’
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Sarepta Therapeutics Announces Third
Quarter 2016 Financial Results and …
Business Wire15h
Humana spells out its conditional Exondys 51
coverage policy — …
endpts.com1d
Sarepta Therapeutics (SRPT) Stock: They Can
Shake And Rattle, But Will …
cnafinance.com1d
After 'Female Viagra,' Muscular Dystrophy
Drug, Will FDA Stand …
Forbes1d
The FDA’s Controversial Duchenne Drug
Approval And The Moral …
Health Affairs2d
FDA expert lashes out at 'worrisome' Sarepta
approval in JAMA
Fierce Biotech2d
Learnings
• Rare Disease is hard
• Deserves the greatest degree of flexibility when making decisions
• Once a drug is approved, utilizing the established process – ALL
STAKEHOLDERS MUST MAKE PEACE WITH THE DECISION
• Disruption encourages conversation.
• No one wants their child to receive weekly infusions, injections or
for that matter oral drugs if they have no efficacy
• Given the trajectory of the illness, the limited life span, once safety
is established and a trend toward benefit, consider adaptive
licensing.
• Without this conversation, patients will wait and wait and wait….
• Eteplirsen is approved – the Community is interested in a real-world
experience to fully understand both benefit and risk.
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The most important piece of the puzzle for developing
therapies is the question of MEANINGFUL BENEFIT.
THANK YOU!
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