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Thursday, October 29
4:00 PM
Politics and Prognostication with The Cook Political Report's David Wasserman

It's time for some "political insider baseball" – and the last session of the summit.

David Wasserman, Senior Election Analyst at The Cook Political Report, is here to give us some insight on what could happen next Tuesday. Here are a few takeaways.

So, can we still trust the polls?

In 2016, the final polling average was pretty on target – after all, Hillary Clinton won the popular vote.  

A lot of the polls in swing states were “chronically undersampling Trump’s best demographic: white voters without college degrees.” This year, pollsters have corrected this issue, to some degree.

Polls are a lot like GPS in that they can get you into the right neighborhood, but not exactly the right address, he said.

Take a look at the averages and keep a long view of the race.

What about the “October surprise”?

The first “October surprise” was the nomination of Amy Coney Barrett to the Supreme Court. This issue drove a wedge in Trump’s coalition by shifting the focus to the Affordable Care Act (ACA) and Roe v. Wade; around 22% of Trump’s voters are pro-choice, and as many as 16% of Republicans disapprove of the Trump administration asking the Supreme Court to overturn the ACA.

How did we get to a point of such extreme polarization between the two parties?

It’s not just that we’ve siloed ourselves into closed information ecosystems—it’s also the fact that, increasingly, Americans are choosing to live in communities where the vast majority of their neighbors agree with them.

Many Americans just can’t believe that the other side could win.

The implications of COVID-19

Expect “massive” turnout, he said.

The challenge? "We’re headed for historic turnout in the midst of a pandemic that’s made it harder for election administrators to grant access to the ballot in a safe and secure way," he said. Many states and counties have struggled to meet the historic demand for absentee ballots and ensure everyone has a fair chance at voting.

In addition, with concerns about health and safety, many poll workers have dropped out. 

Worth noting: Mail-in voting is not new; in 2018, about a quarter of voters nationwide voted by mail. However, this year brings a lot of challenges.

"If you're holding an absentee ballot right now that you haven't sent in, take it to an absentee dropbox or make sure you vote in person to make sure your vote is counted," he advised.

3:20 PM
Lessons from the Pandemic: Clinical Trials and Access to Them

Cartier Esham, EVP, ECS and SVP, Science and Regulatory at BIO, moderates a discussion on clinical trials during COVID-19, with:

  • Jeff Allen, PhD, Executive Director, Friends of Cancer Research
  • Ryan Fischer, Senior Vice President, Community Engagement, Parent Project Muscular Dystrophy
  • Annie Kennedy, Chief of Policy and Advocacy, Everylife Foundation For Rare Diseases
  • Sung Poblete, PhD, RN, Chief Executive Officer, Stand Up to Cancer

The Role of Data and Real-World Evidence (RWE)

“Real-World Evidence is based on the idea that information can be collected outside of a formally run clinical trial that can be informative to how the patients are benefitting or how the treatment is working – using all the data that is available,” explained BIO’s Cartier Esham.

Friends of Cancer Research’s Dr. Jeff Allen has been working with the Reagan-Udall Foundation on the COVID-19 Evidence Accelerator, which brings together different experts and data sources related to the use of RWE.

“The evidence accelerator, itself, is not a traditional clinical trial, but the information generated from this will hopefully inform the clinical trials about things such as treatment patterns and different populations and how they were affected by the first waves of COVID 19 and how treatment modalities have changed over time,” he said.

“In a pandemic, it’s important to catch data as it is coming in, analyze it across the country and across the globe as quickly as possible.”

Parent Project Muscular Dystrophy conducted a survey with the muscular dystrophy community to assess the impact of COVID-19. 

“Like many disease communities, we were worried about COVID-19 impact on our patients including access to care, access to clinical trials, and access to newly approved therapies,” said Ryan Fischer. 

“We’re almost getting to a true understanding of how we can bring the biotech revolution and merge that with the data technology revolution that has been occurring and start to think about building a paradigm that is more patient centric, more effective, and more informative,” said Esham.

Lessons Learned

“This is evolving. Some of the lessons remain to be seen. We are going to have to take time to evaluate from the patient’s perspective, from the physician’s perspective, and from industry about what worked and what didn’t,” said Dr. Allen.

“People’s risk tolerance plays a key role here. Not everybody is comfortable with the risks associated with going into a clinic vs. doing telemedicine. We still have a lot of work to do.”

“Our patient communities have learned to navigate technology in a whole new way,” said Everylife Foundation for Rare Diseases’ Annie Kennedy. For example, telehealth has allowed people to participate in clinical trials who otherwise would not have been able to. 

Decentralized clinical trials allow “for greater enrollment and increased access for our patients, which addressed one of the biggest barriers to health equity,” said Stand Up to Cancer’s CEO Dr. Sung Poblete.

“We found that patient satisfaction went through the roof and physicians appreciated extra communication with their oncologists and internists.”

“COVID has revealed many of the deficiencies of the healthcare systems and the inequities that remain,” concluded Allen.

“It has also revealed what can be done with some of these unnatural partnerships and collaborations. If nothing else, we’ve learned we’re all in this together and the best way to try and get out of it is to leverage each other’s strengths, mutual interests, and shared capabilities to find data from different sources and motivate each other to accelerate everything that we’ve been doing.”

2:40 PM
Fireside Chat: Impact of Innovative Treatments for Rare Pediatric Diseases

Dr. Jeremy Levin, Chairman of BIO and CEO of Ovid Therapeutics led a conversation with Terry Jo Bichell, Executive Director of Combined Brain, and Rebecca Burdine, Professor of Molecular Biology at Princeton University, about the unique experience and lessons learned on forming a patient advocacy organization focusing on a rare disease.

Both women have children living with Angelman syndrome, a rare neuro-genetic disorder, and advocate for patients living with this disease and other neurological disorders. They work to stimulate discovery and drug development. 

"This is the year that Angelman's changed forever," said Dr. Levin.

Ovid is developing a potential therapeutic for Angelman's, the first to undergo a phase 3 trial.

What is Angelman syndrome?

Early signs of Angelman syndrome in babies often include difficulty sleeping and feeding. As they get older, patients will have difficulty walking and communicating. Many will suffer from seizures, and later in life, anxiety.

However: "If you've met one kid with Angelman syndrome, you've met one kid with Angelman syndrome," said Burdine. 

"This is a big issue when you start thinking about doing clinical trials, because how do you measure progress in kids that are so different from one another even though they have the same disorder?" she continued.

What advice would they give to individuals interested in starting an advocacy organization in a new disease area?

Here are a few tips:

1. First, collect as many families as you can. Then, collect data from the very beginning and ensure patients own the data. 

2. Raise money.

3. Put together a scientific advisory board as fast as you can, and put them to work.

4. Don't underestimate help from your community – and don't turn help away. Be inclusive. Then, when pharmaceutical companies come forward to work with you, they'll see people who want to work together.

5. “The natural history is really important,” she added. Get experts on natural history data, and get a company involved with you from the beginning to tell you what questions they need to know.

And a piece of advice for the biopharmaceutical industry: Talk to families and patients from the beginning. Don’t wait until you have a compound. That way, you can be sure you have something that matters.

1:30 PM
Challenges in the States: The Patient Perspective

Ben Chandhok, State Government Affairs Director, Eastern Region at BIO, moderates a conversation on the 2021 state legislative sessions with:

  • Lisa Albany, JD, Director, State Policy, American Academy of Dermatology
  • Anna Hyde, Vice President of Advocacy and Access, Arthritis Foundation
  • Jake Johnson, State Advocacy Manager, Crohn’s and Colitis Foundation-SAIM Coalition
  • Heidi Ross, Director of Policy, National Organization for Rare Disorders (NORD)

Here are a few issues of focus for the 2021 state legislative session:

1. Step therapy. Step therapy is a protocol used by health insurance companies that requires patients to try and fail on one or more lower cost medications before they will provide coverage for the medication originally prescribed by the patient’s provider, explains Johnson. It leads to a delay in optimal patient care and could result in worsening health outcomes, he adds. 

Learn more from Chron’s and Colitis Foundation on Step Therapy Reform.

2. Prior Authorization. Prior authorization is a cost-control process requiring health care providers to qualify for payment by obtaining approval from health insurers before performing a service, explains Albany. It can result in patients getting less effective treatment or abandoning treatment altogether, she says. 

Learn more from American Academy of Dermatology on prior authorization and drug pricing.

3. Accumulator Adjustment Programs. These programs prevent any co-payment assistance that may be available for high cost specialty drugs from counting towards a patient’s deductible or maximum out-of-pocket expenses, explains Hyde. 

Patients are often unaware they are enrolled in one of these programs until they go to the pharmacy counter and realize they must pay the full cost of their medication, which can lead them to abandon or delay their prescription, she continues. 

Learn more from Arthritis Foundation’s Your Coverage, Your Care toolkit

Rare Disease Advisory Councils (RDACs) have helped reduce these barriers, says Ross. RDACs are advisory bodies to state governments and decision-makers that represent the rare disease community and advise on common obstacles. 

Advocating in a virtual environment to reduce these barriers and more will continue to be a unique and powerful opportunity, agree the panelists.

There’s a major opportunity to bring in and cultivate new advocates to participate that ordinarily couldn’t, says Hyde. 

It’s more important than ever to elevate patient stories and have voices heard and the virtual environment gives us the opportunity to do that, says Johnson. 

1:00 PM
Outlook 2021 State Legislative Sessions

A lot of people are concerned with congress and federal health care legislation, but states are very involved in health policy, said Patrick Plues, BIO’s Vice President of State Government Affairs and External Relations. 

He moderated a discussion with:

  • Kate Blackman, Health Program Group Director, National Conference of State Legislatures
  • Laura Blake, Outreach and Development Manager, Women In Government

The pandemic is a great example of that, Kate Blackman pointed out. “States really led the way in the response and it started a lot of activity in the executive branch.” 

As the states look ahead to the distribution of a vaccine, they’re preparing their plans in concert with the CDC to refine considerations such as priority age groups, who can distribute the vaccine, insurance coverage, and even tracking who has received a vaccine. 

The pandemic has had a disproportionate burden on women. Between February and May of 2020, “11.5 million women lost their jobs in comparison to 9 million men. While some of these jobs did not have benefits, there are a lot of people who are losing their private health insurance when they lose their job,” Laura Blake said. There’s also been immense pressure on working mothers with remote schooling and closed daycares. 

Women in Government have been working with local policymakers to think through programs that can support women and children in this difficult time.

“I find that having a patient voice really puts a face to an issue and I know I can say from personal experience that once you get a woman state legislator to really care about your issue, there’s going to be a very determined effort to see it through,” Laura Blake said.

12:50 PM
Bayh-Dole’s lasting impact on innovation

Eighteen years ago, Betsy de Parry was diagnosed with Non-Hodgkin’s lymphoma. At the time, she believed her options were limited and that she was facing a death sentence.

Today, she is cancer free. She attributes this to two things: a new radioimmunotherapy therapy that had just come on the market, and the

Bayh-Dole Act. “Bayh-Dole laid the foundation for my life to be saved. Of course, my doctor saved my life, but he had made this amazing discovery that led to the development of a drug that saved me and millions of others,” she said.

How does an act of Congress save a life?

“What Bayh-Dole did was it changed 40 years of federal policy,” explained Joe Allen, the architect of the 1980 legislation. 

“Before Bayh-Dole, if the government funded an invention, even for a small percentage of the invention, the invention was taken from the inventor by the government and placed in the public domain which seemed like a nice idea after WWII but the fact was, it destroyed any incentive to commercialize it.”

Happy 40th, Bayh-Dole!

This year is the 40th anniversary of Bayh-Dole. Joe Allen heads the Bay-Dole 40 coalition which has been celebrating all year. Although there is hope the bill’s success speaks for itself, Allen claims it is under attack.

“We really want to celebrate this year and let people know how much Bayh-Dole has done but also let them know that it is under attack. Bayh-Dole could be unraveled if it is misapplied,” Allen said. “Bayh Dole relies on the private sector to take on significant risk with very little chance it is going to pay off.

“If the government micromanages the system, it would kill the whole thing.”

12:00 PM
From Passion to Practice: Building a Patient Advocacy Organization From the Ground Up

To kick off Day 2, Paul Hastings, President and CEO of Nkarta Therapeutics, led a conversation with Ashley Valentine, Co-Founder of Sick Cells, a patient advocacy organization dedicated to the sickle cell disease (SCD) community.

Ashley's brother Marqus was born with SCD, a genetic disease that changes the shape of the blood cells and often causes extreme pain crises and complications. 

At the time, SCD was an unknown disease with a lot of stigmas, she said. "It was just easier growing up to not talk about it." 

But Marqus talked about it – because he understood that he could receive better care if he told people about his experience and brought awareness to the disease. They founded Sick Cells to empower other patients to tell their stories and encourage drug development for the disease.

How does an advocacy organization maintain independence and serve patients?

Sick Cells hires a diverse staff so they can bring in different perspectives and focus on what will serve and benefit the SCD community, said Valentine.

How do you change the narrative about a disease like SCD?

The narrative has historically been that SCD is a "poor Black disease," said Valentine. Every single person with SCD or around SCD has experienced this type of stigma when trying to receive care. 

Sick Cells has changed the narrative by empowering the patients to know that they deserve good care, and then encouraging patients to tell their stories. Sick Cells carries the story to decision makers to change laws to ensure there are repercussions if patients are mistreated.

"You don't make change by talking to each other," said Valentine, echoing civil rights leaders. "We need other people. We need the support and the help from other entities in order to make meaningful change for sickle cell disease." 

"It's important to understand history to change the problem." For example, drug companies developing SCD treatments must understand what happened in the Tuskegee experiments.

How do you get patient stories in front of policymakers?

“Lots of phone calls.”

Sick Cells looks at what policies impact sickle cell patients, then they work to ensure patients’ views and needs are included in policies.

Want to know more about sickle cell disease? Visit www.sickcells.org.

P.S. Paul Hastings runs a patient advocacy organization, too. The Youth Rally advocates for kids and teens with conditions of the bowel and bladder. To learn more, visit www.youthrally.org