Transcript September RDLA Lunch Meeting/Webinar Sept. 19th, 2014

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The National Association of Insurance Commissioners – Model
Insurance regulations – Joel White, Horizon Government Affairs
2014 Midterm Elections Forecasting - Nick Manetto, FaegreBD
ABLE Act update, Ginny Sessions - National Down Syndrome
Society
The OIG and Patient Assistance Programs Possible Upcoming
Changes – Dr. Dana Kuh, Patient Services Incorporated
MODDERN Cures update
OPEN Act - Julia Jenkins, EveryLife Foundation for Rare Diseases
Rare Disease Legislative Scorecard - Andy Russell, Rare Disease
Legislative Advocates
Updates From RDLA:
 2014 RareVoice Awards Gala
 November RDLA Meeting: Nov. 12th 12:00 noon – 1 :00 pm EST
The National Association of Insurance Commissioners – Model
Insurance regulations
Mid-Term Elections 2014:
The Forecast Less Than 3 Weeks Out
Rare Disease Legislative Advocates Meeting
October 16, 2014
Nick Manetto
Faegre BD Consulting
What We Know
Some (relatively) Safe Bets
► We
will continue to have divided government. The question is how divided will the Congress
be.
► Republicans
► Democrats
► There
will gain House seats. The question is how many.
will lose seats in the Senate. The question is how many.
will be very limited opportunity for legislating in 2015 before everyone is focused on the
2016 Presidential cycle, and the final two years of Presidential terms are not known for major
actions. Will this cycle remain or will leaders find a way to address issues of national
significance?
Some Key Questions & What to Watch For
► Watch
the polls closely including sample size, type of voter, etc. Pay
attention to trends and don’t put too much stock in an outlier.
► Watch
where the national parties and outside groups are putting and
pulling their money. Candidates will try to spin in their favor, but pull
outs often are bad news.
► Consider
the larger climate and factors, such as the impact of other
races, voter turnout, etc.
► Will
a national climate in favor of Republicans be realized at the state
level, or will Democrats be successful in keeping their distance from
the Administration and Senate leaders?
The National Map Today
Where Have We Been Since Late February?
► ~10
or so Senate races remain pivotal and the list has remained pretty consistent with a few
real surprises (Kansas, maybe South Dakota).
► Republicans
have been able to avoid self-inflicted wounds that plagued them in 2010 and
2012. No Republican incumbent has been defeated, but some, notably Sen. Thad Cochran
and Sen. Pat Roberts, have been bruised.
► While
the math simply is against the Democrats this cycle, many of the key races remain close
a little more than two weeks out.
The Senate Today
53-45-2
Democrat
Republican
Independent
A Safe Bet
►2
seats now held by Democrats – West Virginia and Montana – very likely to flip to
Republicans.
► Brings
us to 53 Democrats (Counting King and Sanders) and 47 Republicans.
Battlegrounds or Bust?
► South
Dakota has long appeared likely to go Republican, but two most recent polls trend
against former Gov. Mike Rounds, and a unique three-way race involving former Sen. Larry
Pressler makes it quite interesting.
► Michigan
looked like more of a Republican pick-up possibility earlier in the year, but Democrat
Rep. Gary Peters seems to have a stable lead.
Republican Seats In Question
► Kansas
certainly a surprise to Republicans. Big questions – will voters in a deep red state cast
votes to potentially keep the Senate in Democratic hands, and how will the gubernatorial race
and schism in the state GOP impact this race? GOP rescue effort appears to be working with
Greg Orman’s lead going away.
► Georgia polls have David Perdue lead of Michelle Nunn narrowing. Are polls underrepresenting traditionally Democratic constituencies, and could this race head to a post-New
Year’s run-off?
► In Kentucky, Minority Leader McConnell has led most polls. Will he pull it out like Harry Reid in
2010, or will Democrats “Daschle” him? DSCC pull out bodes well for McConnell.
Democrat Seats in Question – Slide 1
► New
Hampshire, Colorado and Iowa are all examples of Republican’s success in widening the
map.
► New
Hampshire: Recent polls show Scott Brown gaining, but a big question is whether or not
he will have enough time with a just a little more than two weeks to go.
► Iowa:
Join Ernst continues to lead but by very narrow margins. Will it be enough to turn a longblue seat red?
► Colorado:
Very similar to Iowa – can Rep. Cory Gardner maintain a lead in a state that has
been increasingly purple?
Democratic Seats in Question – Slide 2
► North
Carolina: Sen. Hagan has held leads between 1 to 4 percent in polls over the past month. In the
end, will this be enough, and will voters mobilize to turn out for her?
► Alaska: Dan Sullivan has led all polls since winning the nomination in early August and recent polls
have him up between 3 to 6 percent. How accurate is the polling?
► Arkansas: Recent polls show Rep. Tom Cotton maintaining his lead. Given the red hues of the state,
will this be too much for Sen. Pryor this go-around?
► Louisiana: With Sen. Landrieu firing her campaign manager, the big question appears to be run-off or
no run-off?
► Pryor and Landrieu may end up like Lincoln Chafee, Nancy Johnson, and Chris Shays – generally wellregarded lawmakers defeated because their party label just did not fit their state and region.
So What Do I think Happens?
► We
start at 55-45.
► Republicans pick up West Virginia and Montana (53-47).
► Republicans hold Georgia, Kansas & Kentucky and Democrats hold Michigan, New Hampshire
and North Carolina (53-47).
► Republicans pick up Alaska, Arkansas, & Louisiana (50-50).
► Parties split Colorado and Iowa.
► 114th Senate: 51 Republicans, 49 Democrats.
What Might We See in 2015?
► Certainly
expect an early dose of ACA/Obamacare related votes.
► A new
“Majority Leader” McConnell will likely face continued challenges managing a very
diverse caucus.
► At
the same time, in 2016 the tables will be turned on Republicans as ~6 or so Republican
Senators from blue or purple states elected in the 2010 ACA backlash year will be up for reelection (Toomey, Johnson, Kirk, Rubio, Portman, Ayotte).
What Does It Mean for Our Community?
► Rare
disease and patient advocacy groups more broadly are fortunate
to have strong champions and supporters on all sides. This goodwill
should continue going forward.
► Fiscal challenges, notably the scheduled return of sequestration,
continue to have a significant impact on the sector. One big question –
will or how will sequestration be addressed going forward?
► Will the energy and momentum behind 21st Century Cures remain or
will it fizzle? Can something happen near-term with thornier issues
positioned for PDUFA VI?
► Enactment of targeted and narrowly focused bills remains a real
possibility, such as modest updates or reauthorizations (muscular
dystrophy, autism, etc.) or modest new programs (pediatric research
network).
Achieving a Better Life Experience (ABLE) Act
The OIG and Patient Assistance Programs Possible Upcoming
Changes
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The bill currently has 76 House cosponsors (39 D and 37 R).
Senators Bennet and Hatch are finalizing Senate language that
contains the dormant therapies provisions but does not
include diagnostics provisions, since most of them were
included in the enacted SGR bill.
They plan to either introduce in the lame duck or first thing in
2015.
The NHC will be circulating a sign-on letter next week to
support inclusion of MODDERN (or something like MODDERN)
in the 21st Century Cures package.
Going Beyond the Current Regulatory
and Incentives Frameworks
A new proposal to foster greater orphan
drug development
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THE OPEN ACT
ORPHAN PRODUCT EXTENSIONS NOW
ACCELERATING CURES & TREATMENTS
WWW.CURETHEPROCESS.ORG
WWW.EVERYLIFEFOUNDATION.ORG
Be Sure to Follow us
• http://www.facebook.com/EveryLife4RareDiseases
• http://twitter.com/#!/curetheprocess
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Successes in FDASIA showed
there is momentum for more
rare diseases legislation
• Patients are motivated & ready to take action
• Rep. Upton is actively seeking proposals to improve
FDA, spur drug development & innovation
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CureTheProcess – 2
Small policy changes that will dramatically increase the
availability rare disease treatments in the next 5-10 years
• Specialize: Create more specialized FDA New Drug
Review Divisions; give reviewers sufficient time and
opportunity to stay connected to the scientific and
academic community
• Rationalize: Allow for a more scientific rationalized
application of the ICH guidelines for safety studies
• Incentivize: Create an additional market incentive
to encourage industry drug sponsors to repurpose
major market drugs for rare diseases
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Current Market Incentives
that Foster Drug Development
• Orphan Drug (ODE) - 7 years
– Extremely successful for incentivizing drug development
for orphan products
– Does not include major market drugs that would be
repurposed for rare diseases
• New Chemical (NCE) - 5 years
• Pediatric Exclusivity (PED) - 6 months added to
existing Patents/Exclusivity
– Requires a study only - not a new label indication
• Antibiotic Exclusivity - 5 years
– GAIN Act passed in FDASIA in 2012
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We Can Do More with
the Science We Already Have
The Potential of Drug Repurposing for Rare Diseases
• Many patented drugs already developed and
approved for common conditions which might
effectively treat rare diseases
• Quality drugs with high potency and selectivity
• A single targeted drug is likely to have multiple
therapeutic uses
• But rare disease indications will not be developed
for patented drugs: Why not?
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Roadblocks for Repurposing
Large Market Drugs for Rare Diseases
• The perception of RISK to a billion dollar product is
too great to allow any rare disease development
―RISKS: Fear that potential adverse effects in clinical trials
on very sick patients would risk the product’s market
―NO BENEFIT: Adding a few hundred or few thousand
rare diseases patients does not increase market revenue
enough to justify the costs of repurposing or the
potential risk
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Need for an incentive for repurposing
patented drugs
• Can be developed for less usual 10 to 15 yrs
• Sponsor already exists
• Much lower investment than traditional drug
discovery process
• Drugs have already been proven to be safe
• Pricing for product based on a larger markets, so
drug prices should be lower relative to usual
orphan pricing
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Learning From Policy That Has Worked:
Best Pharmaceuticals for Children Act
• BPCA provides 6 months of market exclusivity on top of patent
life if studies for pediatric use are conducted
• Prior to BPCA, drugs were infrequently tested in children
• Off-label use in the pediatric population was over 70% which
has since dropped to about 50%
• Since 1998, over 400
labeling changes have
occurred, indicating
whether the drug is safe
for children
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Building on BPCA
An additional incentive can help fulfill
the goals to ensure children have access to
safe & effective medicines
• BPCA is still not enough incentive for sponsors to
develop new pediatric cancer treatments
– In the past 20 years, only one drug has been expressly
developed for any form of pediatric cancer
• BPCA only requires a study & does not require a
new labeled indication be achieved
• A new Orphan Product Market Exclusivity Extension is
needed to incentivize repurposing drugs for rare
and pediatric diseases & cancers
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How a New Orphan Product
Repurposing Exclusivity Could Work
• 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 :life-threatening disease
– Must obtain data to place the new rare disease indication on the label
• Sponsor might begins rare disease indication development:
multiple trials for multiple rare diseases
• 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*
that would 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 set by
large market indication
* Nickname courtesy of Kay Holcombe, BIO
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Finding the right balance
• An estimated 120 drugs go off patent each year
– Once a drug is off patent there is no sponsor support
– No financial incentive to study a drug for a rare disease
– Complete loss of opportunity
• An economic incentive will allow companies to
– Recoup the clinical trial & FDA regulatory costs of
multiple repurposing trials
– Provide sufficient financial benefit that a company might
be willing to risk their current product market
• Still allows for timely generic competition
• Maximizes the number of drugs in development NOW
for rare disease patients
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Impact of Legislation
Surge in Patented Drug Repurposing
Investment in the next 15 years
Small change in regulation: Large effect in innovation
2015
•
•
•
•
2017
2019
2021
2022
Immediate surge in research investment
New high paying biotech Jobs
Increased tax revenue
Rare Disease patients access to clinical trials
2024
2026
2028
2030
100’s of drugs available for
rare disease patients
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Benefits to the Economy
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Macro/Economic
Surge in Biotech Investment from development
New high paying Biotech Jobs
Lower cost orphan drugs
– Potential of hundreds of new rare disease treatments
on the market priced at major market drug prices
– Lower healthcare costs for government, private
insurance and out of pocket costs for patients
– Helps solve the problem more quickly with so
many rare diseases and so few treatments
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We must ACT now to
Accelerate Cures & Treatments
• A new Repurposing Exclusivity Incentive could more
than double the current number of rare disease
products approved each year in the next 5-10 Years
• More Patients would have access to safe, effective
and affordable treatments
• Off Label Usage Would Decline
– Correct drug choice and dosage would ensure patients
would have access to safer & more effective treatments
– Reimbursable treatment options for patients
We Want Your Feedback !
Where we are now:
• Solicited feedback from BIO, PhRMA, & NORD
• Open to additional input from Community
• Proposal submitted to Legislative Council to provide a
discussion draft for circulation
• Seeking Examples of patented drugs that could be
repurposed for rare disease
• Seeking a Dem Co-Sponsor in the House
• Our Goal is to introduce a bi-partisan stand alone bill in
the Fall with significant support for the patient
community
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The most powerful tools a
legislator has is their ability to
author, co-sponsor, vote, and
influence legislation.
We believe legislators should
be both held accountable and
given credit for taking these
actions.
We see this document as a tool
to help the rare disease
community reach out to their
Members of Congress and
educate them on the bills that
are important to patients.
Most importantly, our goal for
this Report Card is to thank
those Members of Congress
who received an A and truly
have been champions for rare
disease patients.
All scorecards will be available on rareadvocates.org by 10/23
2014 will mark the 3rd year of the RareVoice Awards hosted by Rare Disease
Legislative Advocates (RDLA). The purpose of the Gala is to acknowledge and
honor those who give rare disease patients a voice on Capitol Hill.
Nominees:
Congressional Staff:
Patient Organization:
Taylor Gilliam, Office of Representative
Carter, CAL Diagnosed Diseases
Research & Network Act
Josh Teitelbaum, Office of Senator
Hagan, Newborn Screening Saves Lives
Re-authorization Act
Cheryl Jaeger, Former Office
of Representative Cantor, Gabriella Miller
Kids First Research Act
James Paluskiewicz, Office of
Representative Burgess, MD Care Act
Jennifer McCloskey, Office of Senator
Casey, Achieving a Better Life Experience
(ABLE) Act
FightSMA, National Pediatric
Research Network Act
National Down Syndrome Society,
Achieving a Better Life Experience
(ABLE) Act
March of Dimes, Newborn
Screening Saves Lives Reauthorization Act
Patient Advocate:
Federal Level Advocacy
Patient Advocate:
State Level Advocacy
Julie Flygare, Project Sleep,
Narcolepsy: Not Alone
Kim & Mike Illions, Pediatric
Hydrocephalus Foundation
Gabriella Miller, Smashing
Walnuts Foundation
Marc Yale, International
Pemphigus Pemphigoid
Foundation
Nicole Dreyer Gavin, PKU Parent
Advocate, Rhode Island
Jennifer Knapp & Kristen Norgaard,
Adrenal Insufficiency United, Oregon
Jeff Leider, Let Them Be Little x2, New
Jersey
RSVP today at: RareVoiceAwards.org/rsvp
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November 12th
In-person and conference call/webinar
Email invite coming soon