October RDLA Lunch Meeting/Webinar Oct. 16th, 2014

1. October RDLA Lunch Meeting/WebinarOct. 16th, 2014

2. Agenda • 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

3. Joel White, Horizon Government Affairs The National Association of Insurance Commissioners – Model Insurance regulations

4. Mid-Term Elections 2014:The Forecast Less Than 3 Weeks Out Rare Disease Legislative Advocates Meeting October 16, 2014 Nick Manetto Faegre BD Consulting

5. What We Know

6. 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?

7. 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?

8. The National Map Today

9. 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.

10. The Senate Today

11. 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.

12. 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.

13. 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.

14. 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?

15. 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.

16. 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.

17. 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).

18. 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).

19. Ginny Sessions, National Down Syndrome Society Achieving a Better Life Experience (ABLE) Act

20. Dr. Dana Kuh, Patient Services Incorporated The OIG and Patient Assistance Programs Possible Upcoming Changes

21. MODDERN Cures: update • 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.  

22. Going Beyond the Current Regulatory and Incentives Frameworks A new proposal to foster greater orphan drug development

23. The OPEN ACTOrphanProductExtensionsNowAcceleratingCures&Treatmentswww.CureTheProcess.orgwww.EveryLifeFoundation.org Be Sure to Follow us • http://www.facebook.com/EveryLife4RareDiseases • http://twitter.com/#!/curetheprocess

24. 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

25. 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

26. 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

27. 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?

28. 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

29. 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

30. 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

31. 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

32. 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

33. 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

34. 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

35. Impact of LegislationSurge in Patented Drug Repurposing Investment in the next 15 years Small change in regulation: Large effect in innovation Small delay in patented drugs becoming generic New label indications granted for Rare Diseases Sponsors invest 100 + new repurposing programs New Orphan Exclusivity Extension Legislation Enacted 2015 2017 2019 2021 2022 2024 2026 2028 2030 • Immediate surge in research investment • New high paying biotech Jobs • Increased tax revenue • Rare Disease patients access to clinical trials 100’s of drugs available for rare disease patients

36. 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

37. 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

38. 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

39. Rare Disease Legislative Scorecard:rareadvocates.org/scorecard/ • 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

40. 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: 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 Patient Organization: FightSMA, National Pediatric Research Network Act National Down Syndrome Society, Achieving a Better Life Experience (ABLE) Act March of Dimes, Newborn Screening Saves Lives Re-authorization Act Patient Advocate: Federal 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 Patient Advocate: State Level Advocacy 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

41. November RDLA Legislative Meeting • November 12th • In-person and conference call/webinar Email invite coming soon