Navigating the challenge of covering breakthrough therapies at MedCity INVEST Precision Medicine

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Our virtual MedCity INVEST Precision Medicine conference, December 9-11, will highlight aspects of precision medicine from the biopharma companies developing cell and gene therapies to diagnostics and the data sharing initiatives to support precision and personalized medicine.

One vexing part of any conversation on precision medicine and breakthrough therapies that have been developed and are continuing to work their way through clinical trials for cancer to rare disease is how to address the practical challenge of their high price tags. Although there are several models to cover the cost of these therapies such as Cigna’s Embarc Benefit Protection program, what happens when someone changes insurers when they change their job? What is fair for patients and what’s fair for companies? When do these conversations even start?

The panel, Reimbursement Models for Cell and Gene Therapies, will highlight some of the pros and cons of different models that are coming to market. Panelists include Laura Okpala, Director, Reimbursement Policy, Gilead Sciences, and Mark Trusheim, Strategic Director, NEWDIGS initiative at the MIT Center for Biomedical Innovation.

Here’s a preview of some of the sessions. Click here to see the agenda.

Interoperability Progress Report

How far have we progressed with sharing patient medical records? Patient data is key in unlocking riddles of medical science but interoperability is necessary to facilitate this. What companies are making an impact on a regional and national scale? What milestones are on the horizon? What obstacles continue to vex further advancement? How can we make medical records more accessible to patients?

Moderator: Elise Reuter, Senior Reporter, MedCity News
Speakers:
Kevin Chaney, Senior Program Manager, Office of the National Coordinator for Health IT
Ida Sim, M.D., Ph.D., Professor of Medicine, University of California, San Francisco
Niko Skievaski, Co-Founder and President, Redox

What It Takes To Build A Successful, Regional BioInnovation Hub
Philadelphia is one of many cities seeking to support the continued growth of cell and gene therapy and connected health industries. What do cities need to do to address education, training and other needs to support these sectors? This session will be held as part of Venture Cafe Philadelphia.

(sponsored by IBX)

Lisa Dalton, Chief People Officer, Spark Therapeutics
Audrey Greenberg, Executive Managing Director, The Discovery Labs
Tiffany Wilson, President & CEO, University City Science Center

Moderator: Michelle Histand, Director of Innovation, Independence Blue Cross

Register now and be part of the conversation at INVEST Precision Medicine.

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YMCA offers free fitness challenge to Charlotte community

The STRONG challenge asks participants to commit to 20 minutes of activity per day five days a week.

CHARLOTTE, N.C. — The goal of the STRONG Challenge is to get people moving and active even during the pandemic.

 “I think we have over 130 Y’s across the country that are doing this challenge around the same time,”  YMCA of Greater Charlotte Regional Healthy Living Director, Amy Crane said.

“It’s really about reigniting our energy building community, taking care of ourselves and our family and getting back to developing healthy habits,” Crane said.

All you have you have to do is commit to 20 minutes of activity per day for five days a week for the duration of the six-week challenge.

“We actually have a theme every week, this week we’re in the play week, next week is connect week after that rest then serve and then balance is our final week,” Crane said.

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The challenge is free of charge and open to anyone regardless of a YMCA membership status.

In fact, challenge participants will receive regular encouragement and support messages through text or emails.

 “We do Facebook live group exercise classes that you can participate in,” Crane said.

Participants can also self-track their success through a downloadable calendar or the YMCA of Greater Charlotte mobile app.

It’s a way this pandemic to come together to start feeling good again and to have accountability in a fun way.

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Residents gear up for a safe, sanitised Dubai Fitness Challenge



a person on a court: Residents gear up for a safe, sanitised Dubai Fitness Challenge


© Provided by Khaleej Times
Residents gear up for a safe, sanitised Dubai Fitness Challenge

Armed with a packed calendar of over 200 virtual sessions and 2,000 classes across 150 locations, Dubai residents are all set to welcome a ‘safe and sanitised’ fourth edition of the Dubai Fitness Challenge (DFC).

Most of the residents have already made fitness a big part of their lifestyle this year, due to the pandemic. While several people have taken up running and yoga, there has also been a big boost people using YouTube and other video portals for at-home workout routines.

Tanya Zag, a Dubai-resident said being indoors amid the curfews took a toll on her mental well-being. “I began running a few months ago and it has become the best stress buster for me. It empowers me and even if I have had a tough day at work, running takes all my worries away.”

“I am really looking forward to the DFC this year. The entire city is buzzing with electrical energy, which is fantastic,” she added.

Jhona Meryl, a fitness influencer said: “It’s really exciting because it’s a collective effort, and the best part is that when people do something continuously for 30 days, it becomes a habit and enforces positivity, which is something people really need right now.”

Day 1 of DFC

Starting today, residents are all set with their athletic wear, yoga mats, personal exercise tools, and of course, their face masks for outdoor activities. To ensure social distancing, prior booking has been made essential for all events on www.dubaifitnesschallenge.com.

The annual event, launched in 2017 by Sheikh Hamdan bin Mohammed bin Rashid Al Maktoum, Crown Prince of Dubai and Chairman of the Dubai Executive Council, is one of the most sought events where families and individuals break a sweat, irrespective of their fitness levels.

The hybrid event has promised to make fitness easy and accessible to all – from young adults, teenagers, families, older residents and people of determination, to businesses and schools.

Saeed Hareb, Secretary-General of Dubai Sports Council, has promised the event will maintain strict safety and hygiene regulations, social distancing guidelines and sanitisation requirements.

“For us, sports are an essential activity at all times, but it has become even more important in this period of Covid-19. Exercising and staying fit strengthens your immune system and a strong immune system is your best defence against illness,” he said.

What to look forward to?

Residents can look forward to a packed calendar of free fitness events, sports activities, health and wellness programmes and virtual sessions, including three dedicated Fitness Villages, ten community-centric Fitness Hubs.

Participants can unlock free 30-day programmes from leading global apps – Fitbit Premium, NEOU, Steppi, Sweat, Sworkit, Daily Burn, FIIT and Les Mills on Demand.

This year, the challenge is placing an elevated focus on at-home sessions with the Find Your 30 virtual content hub. Free-to-access, the facility is being hosted on the DFC website, allowing participants

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Dubai Fitness Challenge: Nike launches free 30-day calendar of movement

Nike
The campaign features Zainab Al Eqabi an amputee and everyday athlete, as well as other successful Arab female athletes
Image Credit: supplied

Dubai: Nike launched the Middle East’s installment of “You Can’t Stop Us”, a campaign aimed at encouraging women across the Middle East to discover the world of movement in water, the film is a rallying cry for women across the region to go out and play.

The “You Can’t Stop Us” month-long campaign, features four remarkable women who have forged their very own success stories in the world of sport, starring Zainab Al Eqabi, amputee and everyday athlete, Sarra Lajnef, Olympic Swimmer, Nike Swim Coach, Rower and Triathlete, Maha Al Ameri, UAE National Rower and Manal Rostom, Founder of Surviving Hijab & Nike running coach and triathlete.

Despite concerns, criticism and limitations, these women have remained unstoppable and hope to inspire women in the region who want to pursue sport with their relentless spirit.

‘You Can’t Stop Us’ is meant to inspire and motivate athletes across the region to participate in Nike’s 30-day calendar of movement. This year’s calendar coincides with the fourth edition of the Dubai Fitness Challenge

The Nike 30-day calendar includes:

1. Virtual Coaching Hub: A WhatsApp coaching hub that provides members with expert tips and ongoing guidance on nutrition, mindset, recovery and movement from Nike Master Trainers to help athletes pursue a healthy lifestyle.

2. Livestreamed session led by Nike Master Trainers, tackling HIIT, yoga, dance and mediation. The free live-streamed community workouts run twice daily and are suitable for small spaces and all levels of fitness.

3. In partnership with Hamdan Sports Complex, Nike will host a swimming clinic for women. Including introductory and intermediate sessions held daily at limited capacity. The clinic is coached and led by Olympic swimmer and triathlete Sarra Lanjef.

4. Nike Training App: A free access to digital library of over 200 workouts available at the Nike Training apps.

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The next vaccine challenge: Nationalism

Instead of a coordinated global effort among governments to prepare the world to produce and distribute a vaccine, the pandemic has set off a wave of “me first” health care nationalism. Early in the pandemic, nations scrambled to bring production “home” and to secure enough doses of a still-hypothetical vaccine for their own citizens first. From export controls on critical health care supplies to closing borders to the movement of people, many measures governments took to protect their own citizens have also made it more difficult to orchestrate a global response, according to a new episode of POLITICO’s Global Translations podcast released Wednesday.

Governments have been looking for national solutions even though there is no “wholly domestic supply chain for any product” in the vaccine race, Jim Robinson, a former executive from the pharmaceutical giant Merck, told the podcast.

Robinson is now working with a handful of international organizations racing to fill in gaps in the global vaccine supply chain — and in global leadership — to ensure the world gets access to the vaccine. “Unfortunately, this is not a global effort to find a global solution,” he said.

Robinson leads the manufacturing strategy for Covid at the Coalition for Epidemic Preparedness Innovations, an international organization that works with the World Health Organization and GAVI, the Vaccine Alliance. Their partnership created COVAX, a voluntary multilateral effort to ensure equitable access to the vaccine around the world; 156 countries are now eligible to buy vaccines from COVAX. COVAX member countries get access to purchase vaccines from a portfolio of potential candidates and access to globally sourced supplies. The idea is to ensure it will be accessible to rich and poor countries alike.

Under the Trump administration, the United States, like Russia, has stayed out of COVAX. It is forging ahead on its own with Operation Warp Speed, a collaboration between the Department of Health and Human Services and the Pentagon to develop, manufacture and distribute a vaccine. It has partnered with several leading pharmaceutical companies, and a half dozen vaccine candidates are already in advanced clinical trials.

Anthony Fauci, the U.S. government’s top infectious disease expert, said the massive logistics and sourcing challenge is why the U.S. government turned to a four-star Army general, Gustave Perna, to serve as chief operating officer of the effort. “The purpose of getting someone of the experience and talent of Gen. Perna was precisely to assure that we can get [that] done,” Fauci said in an interview airing in the podcast.

Many countries around the world either don’t have the infrastructure to manufacture a vaccine in-house or don’t have access to necessary supplies, said Robinson. “Our goal is to really fill in the gaps for the global vaccine ecosystem to help create capacity for the rest of the world.”

The COVAX initiative is helping governments fumble toward cooperation. Initially, China, like the United States, stayed out of the coalition, preferring a nationalist approach to vaccine development. But earlier

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Low temps and high costs: Coronavirus vaccine distribution will face a “big challenge”

“CBS This Morning” explores whether America is ready for a coronavirus vaccine in a special three-part series, Road to a Vaccine. Part three airs Wednesday, October 28 on “CBS This Morning,” 7-9 a.m. on CBS. Watch part one here.


The earliest a coronavirus vaccine is expected to be ready for FDA authorization is the end of November. The CDC has already given states $200 million to prepare for distribution.

But shipping companies like DHL have a daunting task — preparing to transport a coronavirus vaccine without knowing where the vaccine will be manufactured, what the packaging will be or how cold it will need to be kept.

“There’s still a lot of things that are unknown. And we’ve been talking to the different manufacturers, who are in various phases of the clinical trials to get ready,” DHL’s CEO of Global Forwarding USA David Goldberg told CBS News senior medical correspondent Dr. Tara Narula. 

At the DHL cold-chain facility near Chicago’s O’Hare airport, vaccines are stored at various temperatures before they’re sent to doctors’ offices, pharmacies and hospitals.

“We’ve been moving the flu vaccine, the meningitis vaccine,” Goldberg said. “I think the challenge related to this vaccine is it’s, you know, a vaccine that the world needs as soon as possible, at once, which will make it very difficult in terms of logistics.”

The colder the vaccine, the more complicated the logistics. Pfizer’s coronavirus vaccine candidate needs to be kept at about minus 94 degrees Fahrenheit, while Moderna’s needs to be stored at minus 4 degrees Fahrenheit.

“A lot of providers don’t have that type of storage,” said North Dakota Immunization Program Manager Molly Howell.

The ultra-cold storage requirement will make it challenging for states to get the vaccine to their residents, Howell said. 

“Once a provider receives that vaccine, it really starts the clock that the vaccine needs to be administered within five days of when it’s put in the refrigerator,” she said. 

Pfizer’s vaccine is expected to ship in containers with almost 1,000 shots, which worries Howell.  

“The minimum increment of 1,000 doses and figuring out how we can get that to the rural areas is what’s keeping me up at night,” she said. “We’re thinking about the possibility of having to repackage and redistribute that vaccine into smaller quantities.”

While states like North Dakota gear up for mass distribution, the pandemic continues to batter state budgets. The trade associations that represent health officials across the country have asked Congress for $8.4 billion to help states distribute the vaccine.

States and their health departments are “tapped out financially,” said Georgetown professor Dr. Jesse Goodman. 

“Also, in terms of their human resources, they’ve been running at 100 miles an hour to do the contact tracing to make up for a not very efficient federal response,” he said. 

Goodman said there needs to be an effective national system for distributing and monitoring the vaccine.

“Otherwise, it’s going to be chaos,” Goodman said. “We may have multiple vaccines. We may

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Human Challenge Trials Will Deliberately Infect Dozens in the UK

Young, healthy people will be intentionally exposed to the virus responsible for COVID-19 in a first-of-its kind ‘human challenge trial’, the UK government and a company that runs such studies announced on 20 October. The experiment, set to begin in January in a London hospital if it receives final regulatory and ethical approval, aims to accelerate the development of vaccines that could end the pandemic.

Human challenge trials have a history of providing insight into diseases such as malaria and influenza. The UK trial will try to identify a suitable dose of the virus SARS-CoV-2 that could be used in future vaccine trials. But the prospect of deliberately infecting people—even those at low risk of severe disease—with SARS-CoV-2, a deadly pathogen that has few proven treatments, is uncharted medical and bioethical territory.

Proponents of COVID-19 challenge trials have argued that they can be run safely and ethically, and that their potential to quickly identify effective vaccines outweighs the low risks to participants. But others have raised questions about the safety and value of these studies, pointing out that large-scale efficacy trials involving tens of thousands of people are expected to deliver results on several COVID-19 vaccines soon.

“Deliberately infecting volunteers with a known human pathogen is never undertaken lightly. However, such studies are enormously informative about a disease,” said Peter Openshaw, an immunologist at Imperial College London and investigator on the study, in a press statement. “It is really vital that we move as fast as possible towards getting effective vaccines and other treatments for COVID-19, and challenge studies have the potential to accelerate and de-risk the development of novel drugs and vaccines.”

Dose testing

The planned COVID-19 challenge study will be led by a Dublin-based commercial clinical-research organization called Open Orphan and its subsidiary hVIVO, which runs challenge trials on respiratory pathogens. It will take place in the high-level isolation unit of the Royal Free Hospital in north London, says Open Orphan executive chair Cathal Friel.

The UK government’s COVID-19 Vaccine Taskforce has agreed to pay the company up to £10 million (US$13 million) to conduct the trial, with the possibility of contracting Open Orphan to run several more to test various vaccines. The UK Medicines and Healthcare Regulatory Agency (MHRA), which regulates clinical trials in the United Kingdom, and an ethical review committee, still need to approve the initial trial and its design, and that of future studies.

The initial trial will involve an estimated 30–50 participants, says Andrew Catchpole, a virologist and the chief scientific officer at Open Orphan who is leading the work. It is open only to healthy adults aged 18–30.

The precise design of the study has not been finalized. But it is likely that a small number of participants will receive a very low dose of a SARS-CoV-2 ‘challenge strain’ derived from a currently circulating virus and grown under stringent conditions. If none or few of the participants become infected, the researchers will seek permission from an independent safety monitoring board to expose

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Suicide Prevention Still a Daunting Challenge

Suicide screening protocols implemented in veteran and general populations reduced suicides, but making such screening tools more efficient remains a challenge, according to a pair of studies.

After the Veterans Health Administration implemented the Veterans Affairs Suicide Risk Identification Strategy (Risk ID) in 2018, results were positive in outpatient visits for 3.5% and 0.4% of primary and secondary screenings, respectively, and results were positive in emergency settings for 3.6% and 2.1% of primary and secondary screenings, respectively, reported Nazanin Bahraini, PhD, Rocky Mountain Regional VA Medical Center in Aurora, Colorado, and colleagues.

Compared to patients in ambulatory care, patients screened in the emergency department were more likely to endorse suicidal ideation with intent (odds ratio 4.55, 95% CI 4.37-4.74, P<0.001), have a specific plan (OR 3.16, 95% CI 3.04-3.29, P<0.001), and report recent suicidal behavior (OR 1.95, 95% CI 1.87-2.03, P<0.001) during secondary screening, Bahraini’s group wrote in JAMA Network Open.

Risk ID includes three tiers of screening starting with the nine-item Patient Health Questionnaire, followed by the Columbia Suicide Severity Rating Scale Screener and the VHA’s Comprehensive Suicide Risk Evaluation.

“The greater acuity of suicide risk among patients in the ED or [urgent care clinics] cohort compared with those in the [ambulatory care] cohort supports national implementation of evidence-based suicide prevention programs, such as Safety Planning in the ED,” Bahraini and co-authors wrote.

Suicide screening is recommended for all veterans, who have a 21% higher risk of dying by suicide than the general population.

However, close to two-thirds of veterans who die by suicide do not seek healthcare from the VA, indicating that many suicides will still be missed in VA screening initiatives, noted Roy H. Perlis, MD, MSc, of Massachusetts General Hospital and Harvard Medical School in Boston, and Stephan D. Fihn, MD, MPH, of the University of Washington in Seattle, in an accompanying editorial.

“Although the VA is not representative of general clinical practice, these numbers provide a useful reference for estimating the yield of routine screening in these settings,” Perlis and Fihn wrote.

Another study published in JAMA Network Open, from the Kaiser Permanente system in California and Washington, examined the practicality of a risk-based alert system to identify patients who may attempt suicide.

The tool was developed from electronic health records and included characteristics such as depression symptoms, mental health visits, and past suicide attempts to estimate suicide risk. When patients entered a certain adjustable risk threshold, the tool would deploy an alert to physicians.

Using the 95th percentile threshold, the tool yielded 162 daily alerts and demonstrated a positive predictive value of 6.4%. (That works out to a number-needed-to-screen of 17 to prevent one suicide attempt, Perlis and Fihn calculated.) In contrast, the system sent out four alerts per day at the 99.5th risk percentile.

“While this provides useful estimates for planning, many key effectiveness, clinical, operational, ethical and legal questions remain,” the study’s lead author Andrea H. Kline-Simon, MS, of Kaiser’s research division in Oakland, California, told MedPage Today

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Analysis: Lack of Antidotes Adds to Ethical Dilemmas for UK COVID-19 Challenge Trials | Top News

LONDON (Reuters) – UK scientists seeking approval to deliberately infect healthy people with COVID-19 in trials must first convince ethics specialists that, among other things, they have potential “rescue therapies” or antidotes to halt the disease.

The problem is that, for the novel coronavirus, there is still no effective treatment or cure.

That means, for now, that the best scientists planning the so-called human challenge studies can offer is Gilead’s remdesivir – an antiviral drug that was found in a large trial to have no impact on COVID-19 death rates.

Chris Chiu, a scientist at Imperial College London co-leading the COVID-19 challenge experiments, said the plan was to give remdesivir to infected volunteers based on a “strong belief” it will be effective if given in the very earliest stages of disease.

Some ethics and medicine experts said that posed problems.

“As an effective rescue therapy does not yet exist for SARS-CoV2, there is a serious ethical dilemma … to address here,” said Stephen Griffin, an associate professor in the school of medicine at Britain’s Leeds University.

Other specialists said the lack of antidotes or rescue therapies was only one of several risks the research team would have to minimise, and trial volunteers would have to accept, if the studies are to gain ethical approval.

Plans to reduce those risks include picking the most robust, healthy, young volunteers to be infected, and using the bare minimum amount of the coronavirus to infect them with COVID-19.

Dominic Wilkinson, a professor of medical ethics at Oxford university, said that, while desirable, having an effective treatment at the ready after deliberately infecting volunteers was “not essential for the ethics of such a trial”.

“The ethical necessity of these trials, and of any trial, is that you assess the risks, you minimise the risks, and you communicate the risks,” he told Reuters. “It’s not the case that you have a situation where there are no risks.”

Human challenge trials are not new. Scientists have used them for decades to learn more detail about – and develop treatments and vaccines against – several other diseases such as malaria, flu, typhoid and cholera.

“Generally with such trials in the past, they were done when you had a specific treatment,” said Margaret Harris, a spokeswoman for the World Health Organization (WHO), when asked about the planned UK trials using the novel coronavirus.

“You must ensure that everybody involved understands exactly what is at stake … and ensure informed consent is rigorous – that they really do understand all the risks,” she said.

Ohid Yaqub, a science policy expert at Britain’s University of Sussex, pointed to WHO guidelines on the issue, which among other things say human challenge trials “might be considered when the disease an organism causes has an acute onset, can be readily and objectively detected, and existing efficacious treatments … can be administered … to prevent significant morbidity, and eliminate mortality.”

Yaqub warned of a risk to public trust in science and medicine if these criteria

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U.K. Plans ‘Challenge Trials,’ Which Will Intentionally Give People COVID-19 to Test Vaccines

Syringe on gauze pad with vaccine bottles
Syringe on gauze pad with vaccine bottles

Credit – Getty Images—Jonnie Miles

On Oct. 20, researchers at the Imperial College of London announced plans for the first human challenge study of COVID-19, which involves deliberately infecting volunteers with the virus that causes the disease, in order to test the effectiveness of vaccines.

The strategy is controversial, as researchers have to weigh the risks of infection against the benefits of learning how well the various vaccine candidates can fight that infection. The strongest argument in favor of the studies has to do with time. If cases of COVID-19 are waning, then the likelihood that people who are vaccinated would get exposed to and potentially infected with the virus naturally declines as well, and it takes researchers longer to accumulate enough data to tell if a vaccine is effective or not. By intentionally exposing people to the virus after they have been vaccinated, researchers can shrink this timeline significantly.

Scientists have used the model to test vaccines against a number of different diseases, including the very first one against smallpox—Edward Jenner infected his son with cowpox, and then exposed his son to smallpox as a way to test his theory that exposure to the former would protect his son from infection by the latter. Scientists tested an H1N1 influenza vaccine by exposing people to the flu, and did the same with a cholera vaccine and the bacterium that causes it. But the strategy requires a solid base of information about both the disease and the vaccine in order to justify the risks. More recently, for example, scientists considered intentionally infecting volunteers with the Zika virus to test vaccines against that disease, but ultimately decided they didn’t have enough data to justify the risk.

Adair Richards, honorary associate professor at the University of Warwick who last May published guidelines on how to ethically conduct human challenge studies, notes that during a pandemic, the risk of delays in developing treatments should be considered alongside the risks to volunteers who are intentionally exposed to disease. “There is a moral weight to inaction as well as action,” he says. “There is an unseen risk if we don’t do [these studies]. We send a lot of doctors, nurses and care workers to work every day, and some will get really sick and die of COVID-19 in the next few weeks. [Those] few weeks count—that’s the unseen risk.”

More than 38,000 people in the U.S. agree, and have registered their intention to volunteer for challenge studies on 1DaySooner.org, an online recruiting group—despite the fact that no such studies have been planned in the country yet.

The London-based scientists still need to submit a detailed proposal to regulatory agencies on how they could conduct their study. If the proposal is approved, the team won’t start exposing any volunteers until January. Before that, they will first need to determine what dose of SARS-CoV-2, the virus that causes COVID-19, is safe to give to people but can still produce

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