October RDLA Lunch Meeting/Webinar

Oct. 16th, 2014

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

Agenda

Joel White, Horizon Government Affairs

The National Association of Insurance Commissioners – Model Insurance regulations

Mid-Term Elections 2014:

The Forecast Less Than 3 Weeks Out

Rare Disease Legislative Advocates MeetingOctober 16, 2014

Nick ManettoFaegre 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 will gain House seats. The question is how many.

►Democrats will lose seats in the Senate. The question is how many.

►There 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

DemocratRepublicanIndependent

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 under-representing 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 long-blue 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 well-regarded 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 re-election (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).

Ginny Sessions, National Down Syndrome Society

Achieving a Better Life Experience (ABLE) Act

Dr. Dana Kuh, Patient Services Incorporated

The OIG and Patient Assistance Programs Possible Upcoming Changes

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.  

MODDERN Cures: update

Going Beyond the Current Regulatory and Incentives Frameworks

A new proposal to foster greater orphan drug development

22

THE OPEN ACTORPHAN PRODUCT EXTENSIONS

NOWACCELERATING CURES &

TREATMENTS

WWW.CURETHEPROCESS.ORGWWW.EVERYLIFEFOUNDATION.ORG

Be Sure to Follow us • http://www.facebook.com/EveryLife4RareDiseases• http://twitter.com/#!/curetheprocess

23

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

24

CureTheProcess – 2Small 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

25

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

26

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?

27

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

28

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

29

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%

30

• Since 1998, over 400 labeling changes have occurred, indicating whether the drug is safe for children

Building on BPCAAn 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

31

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 32

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

33* Nickname courtesy of Kay Holcombe, BIO

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

34

35

Impact of LegislationSurge in Patented Drug Repurposing Investment in the next 15 years

2015 2017 2019 2021 2022 2024 2026 2028 2030

New O

rphan

Exc

lusi

vity

Ext

ensi

on Leg

isla

tion E

nacte

d

Spons

ors

inve

st 1

00 +

new

repu

rpos

ing

prog

ram

s Small delay i

n patented drugs

becoming generic

New la

bel in

dica

tions

gran

ted

for R

are

Diseas

es

Small change in regulation: Large effect in innovation

100’s of drugs available for rare disease patients

• Immediate surge in research investment• New high paying biotech Jobs • Increased tax revenue• Rare Disease patients access to clinical trials

Benefits to the Economy• 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

36

We must ACT now toAccelerate 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

38

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.

Rare Disease Legislative Scorecard:rareadvocates.org/scorecard/

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.

RSVP today at: RareVoiceAwards.org/rsvp

Congressional Staff:

Taylor Gilliam, Office of Representative Carter, CAL Diagnosed Diseases Research & Network ActJosh Teitelbaum, Office of Senator Hagan, Newborn Screening Saves Lives Re-authorization ActCheryl Jaeger, Former Office of Representative Cantor, Gabriella Miller Kids First Research ActJames Paluskiewicz, Office of Representative Burgess, MD Care ActJennifer McCloskey, Office of Senator Casey, Achieving a Better Life Experience (ABLE) Act

Patient Organization:

FightSMA, National Pediatric Research Network ActNational Down Syndrome Society, Achieving a Better Life Experience (ABLE) ActMarch of Dimes, Newborn Screening Saves Lives Re-authorization Act

Patient Advocate:Federal Level Advocacy

Julie Flygare, Project Sleep, Narcolepsy: Not AloneKim & Mike Illions, Pediatric Hydrocephalus FoundationGabriella Miller, Smashing Walnuts FoundationMarc Yale, International Pemphigus Pemphigoid Foundation

Patient Advocate:State Level Advocacy

Nicole Dreyer Gavin, PKU Parent Advocate, Rhode IslandJennifer Knapp & Kristen Norgaard, Adrenal Insufficiency United, OregonJeff Leider, Let Them Be Little x2, New Jersey

Nominees:

November 12th In-person and conference call/webinar

Email invite coming soon

November RDLA Legislative Meeting