On Day 4 – the final episode of Good Day BIO Live – we have interviews with BIO leadership and policy experts on Accelerated Approval, antimicrobial resistance, and the patient experience, plus clips of the great Venus Williams.
Transcript BIO Convention Day 4 – Thursday
Welcome to day four of Good Day BIO Live, coming to you from the 2022 BIO International Convention in San Diego, California. I’m Tetiana Anderson.
The ultimate goal this week: better serving patients and the planet. Whether it’s building bridges for business or boosting diversity and inclusion in the industry, experts say the stakes for creating equitable global health are higher than ever.
Part of that depends on expedited access to medicine. With Accelerated Approval Pathways making headlines recently, BIO’s Chief Policy Officer, John Murphy, and several patient advocates talked about why the pathway is key to new cures.
“The Accelerated Approval Pathway has been really instrumental in bringing new therapies to the market. We just released a study where we looked at the Accelerated Approval Pathway for the past 15 years and we saw 203 new therapies brought to the market.
The vast majority of those therapies were in ultra-rare and very rare conditions; in other words, therapies that very unlikely would have come to market had there not been the ability to bring the product to the approval process fast. And, interestingly enough, in looking at that study, we also found that despite being approved on surrogate endpoints versus primary endpoints, the vast majority of those products remain on the market after converting to a traditional approval between three and four years later.”
Michael Ward, Alliance for Aging Research
“What the Accelerated Approval Pathway allows the FDA to do is to look at rigorous scientific evidence, the same level of rigor as they would for a standard FDA approval, but they’re looking at an endpoint that is likely to show clinical benefit.
So, based on everything that we know about the disease, if we use this medicine and it has the results in that secondary endpoint, patients are likely to benefit in their lives.”
Pam Traxel, American Cancer Society, Cancer Action Network
“Threats to the Accelerated Approval Pathway could mean that patients do not receive access to the therapies they need to save their lives. So, in fact, patients could die if the Accelerated Approval Pathway is really interfered with.”
Linda Goler Blount, Black Women’s Health Imperative
“There’s a saying in the field that effective therapeutics are always disparities-producing because people with access, people with resources always get access to those therapeutics first. Low-income people, people living in rural areas, Black and Brown populations tend not to get these therapeutics as quickly. So, we have got to make sure that we reduce those barriers and make sure that everybody has access to lifesaving drugs and therapeutics.”
BIO’s Chief of Staff, Khushboo Sharma, says it’s a hot topic in Congress right now.
“Over the last few months, we’ve seen a lot of attacks on Accelerated Approval Pathway. There are policies out there, there are groups out there that are thinking that this pathway is not actually helping the system, which is purely false. And we, as an industry, need to ensure that this pathway works and it’s actually helping patients get the drugs they need faster.
So that has become one of the biggest challenges with the Prescription Drug User Fee Act, because we’re seeing some policy changes that are hindering this pathway and we want to make sure that we take those bad policies and avoid them becoming law.”
Venus Williams, Tennis Champion
Speaking of patient advocacy, tennis champion Venus Williams sat down with Emmy-nominated sports reporter Erin Andrews. She talked to Williams about life, tennis, and how sports prepared her to deal with a debilitating condition.
“I was diagnosed with an autoimmune disease called Sjögren Syndrome. Basically, it is like a box of chocolates. You never know what you’re going to get. You’re going to wake up and you never know what it’s going to be. But I had to just take what I had and start going with it.
And definitely, every time that you get knocked down, which in this sense is all the time, you just get right back up. You know, you get a bruise, and you say, ‘Give me some more’. And I think that’s what I got used to. But I think also sports prepared me for that. It definitely did.
I have an illness where you look like you’re healthy, but you aren’t. So, what I go through on a daily basis when I wake up, before I go to bed, how I feel when I’m on the court, I have to be, or I feel very stoic about it. No one knows what I’m going through. So no one really knows what each person is going through, but it’s wonderful to know that everyone in this audience today, there’s so many people who want to help.”
Diane Shader Smith, Mom
When microbes evolve, so does their ability to survive. The UN estimates that 10 million people could die of antimicrobial resistance, or AMR, by 2050 if new ways to fight microbes aren’t developed soon. Diane Shader Smith knows what that’s like. She shared a powerful story about what happened to her daughter.
TA: So, super personal issue for you. Tell us a little bit about your daughter’s story.
DSS: “So, my daughter Mallory was diagnosed with Cystic Fibrosis when she was three, and when she was 12, she was colonized by resistant bacteria called Cepacia. That’s the short term. It’s not as common as some of the other resistant bacteria people know, like Pseudomonas or Staph or MRSA, but it is deadly and it proves to be deadly over time because as it grows, it develops resistance to the antibiotics.
My daughter, in 2016 when she was 24, had a double lung transplant at the UPMC Transplant Center. It was an amazing nine-hour surgery, the lungs were pristine, everything was perfect, and everybody was so excited. She did a lot of press around it and then, a couple of months later, she developed necrotizing pneumonia because the superbug was resistant to any of the treatments that they were throwing at her at that point. She died very shortly after from resistant bacteria.”
The interactive Innovation Zone showcased nearly 30 cutting-edge biotech companies. They all got seed money from the National Institutes of Health. Marisa Briones, PhD, CEO/Co-Founder of BDH Pharma, runs one of those businesses. She told us about her mission and the science behind it.
“Our lead product is a combination therapeutic targeting chronic pain. It’s combining a partial opioid and a cannabinoid that we believe, based on our preclinical data and what we know in the literature, is a safer analgesic and has the potential to help a lot of patients that are currently suffering.
I think one of the benefits of our combination therapeutic is that we’re combining two already FDA-approved medications, so we’re able to leverage the 505 (b) (2) regulatory pathway, which is a shortened time to market. Not only are we combining two medications that have already been deemed safe and appropriate by the FDA for use in humans, we’re doing so at a lower dose to reduce side effects, so we don’t have to do as much pre-clinical research and we’re able to get to clinical trials much faster, and overall, that’s a lower cost and an accelerated timeline.
So that’s something that I think our company is very fortunate with the regulatory pathway that we can use.”
We’ve talked a lot this week about diversity and inclusion. BIO board members agree that CEOs need to take the lead and close the gaps.
And as a leader of Nkarta, you guys are doing a lot inside that company. What are some of the initiatives when it comes to the DEI that you’re leading?
Paul Hastings, Nkarta Therapeutics
“Thank you for asking that question. I would refer you to our website. When you go to Nkartatx.com, you will see Board of Directors, Leadership Team, Senior Vice Presidents, different categories of members of our organization in these pie-charts that will show you male, female, people of color, all the different diverse populations. You’ll see that we take a concerted effort to make sure that we have a balanced workforce from the top, all the way to all the levels in our company. That’s one initiative we have.”
It’s been a busy week for everyone, including BIO CEO, Michelle McMurry-Heath. I caught up with her earlier and asked her what’s on tap for the 2023 convention and what struck her most about this year’s gathering.
“What we’ve seen this week is that our partnering platform is just the most essential part to doing biotech business right now. You walk the floor of the exhibit halls and it’s clear that, whether or not you have a country or a state setting up their own partnering booth, you have a million small biotechs that are meeting one-on-one with investors. This is a primary place for our leaders to do business.
And so when we get to Boston in 2023, we’re going to blow that up even more and give even more opportunities for those small companies to shout out about the great ideas they have and the progress that they’re making.”
Before we get a look at what we’re watching Thursday, let’s take a quick look at how conferencegoers cut loose last night aboard the USS Midway.
“It’s great to be able to just relax a little bit with our colleagues, get to meet the people outside the network that we’ve been meeting today, and just, be in this location, it’s amazing and the weather is perfect.”
“BIO has done so much in these past two years and we haven’t been able to really celebrate it. To be able to come together and still celebrate and learn. It’s just like the family is back together.”
