Karen Hickman, a Global Health Division Senior Program Officer at the Bill & Melinda Gates Foundation mused that it might be because of the culture of taking healthcare systems for granted. However, she also spoke on the lack of at-home diagnostics capabilities for a number of infectious diseases. 

Why diagnostics matter

William Hait, a Global Head at Johnson & Johnson shared two comments on Heichman’s point about diagnostics, First, he made the economic argument: “We know the earlier you diagnose cancer, the better the outcome, and the differences in outcome are a huge cost saving; the same with infectious diseases.” Second, Johnson & Johnson created a diagnostics leg that they simply view as a cost of manufacturing, a business model other companies might seriously consider.

Of course, most pharmaceutical companies don’t have those types of resources, but that’s why small biotechs are so important. They have to have a “compelling case” and find those niche areas that will attract investors. 

But what are tools without policy and community structural change

As Jeremy Levin, BIO Chairman and CEO of Ovid Therapeutics Inc. pointed out, much of this conversation about diagnostics is moot without strong policy. “It really doesn't matter how good the diagnostics are, it really doesn’t matter how wonderful the digital is. At the end of the day, it will require that we have intent and a purpose and a set of mechanisms and policies that put this in place in the medical system that we have, otherwise we are going to revisit this again.”

Vicki Seyfert-Margolis, CEO & Founder of My Own Med talked a little bit about why lower-income and poor communities are hit harder by the pandemic and why the next phase of pandemic can’t happen without addressing it. “There's a number of different issues. One of them of course is access to healthcare, another is trust and cultural context. I think in the use of our digital technologies we can't overlook building trust and having the right people that are part of the interface and part of the communication strategy for the deployment of these tools. What we're seeing is that if you aren't working with people who are trusted in the community, who understand the culture of the community...You can't be successful. Even with the latest, greatest technologies.”

After providing the foundation of the discussion on patterns of use, coverage, and access to cancer, prenatal, and rare & undiagnosed disease genomic testing across the U.S., the panel took a deep dive into the issues. 

It’s important to have shared beliefs among collaborators. In an ideal world, the utilization of innovative genomic technologies should result in 1) improved outcomes, 2) reduced costs and 3) improved patient experience. 

Success that brings benefits for patients, innovators, and the healthcare system has incredible promise, but there must be evidence that it works. 

The discussion circled around the fundamental challenge for improving access issues for genomic technologies being the lack of evidence for the clinical utility of these technologies. Without that, it will continue to be an uphill battle for these incredibly exciting and innovative tools at our disposal. 

Overall Takeaway

Focusing on patient access and coverage of genomic testing utilization can reveal important implications for pharmaceutical, diagnostic, and data platform development, as well as market penetration.

- Jeremy Isenberg 

What programs are available for coronavirus-related assistance?

The government has created a lot of programs to help American business through the pandemic:

1. Paycheck Protection Program for companies with fewer than 500 employees. This program allows for loans for up to 2.5x monthly payroll. The first eight weeks are forgivable, essentially turning the loan into a grant. After eight weeks, companies can apply for forgiveness.
2. Mainstreet Lending Program provides a liquidity alternative for companies not eligible for the PPP loan. Companies with fewer than 15,000 employees or less than $5 billion in annual revenues. All loans are required to be repaid along with other restrictions.
3. Employee retention credit for companies that did not receive a PPP loan. This refundable employment tax credit is for employers whose operations were disrupted due to orders from the government related to the pandemic or had a significant decline in gross receipts.
4. Payroll tax deferral for companies that did not receive a PPP loan. This referral allows employers to defer the deposit and payment of the employer’s share of social security tax, with 50% of what is deferred due December 31, 2021, and the remainder due December 31, 2022.
5. Unemployment insurance for furloughed workers. Furloughed employees who are eligible for unemployment may be eligible for an additional amount of unemployment for 2021 to pay for medical costs, too.
6. Emergency leave employer credit. Eligible employees may take up to two weeks of emergency paid sick leave for qualified conditions related to coronavirus, up to a certain salary cap. It allows an employee to take up to 12 weeks for paid family medical leave due to conditions related to coronavirus, up to a certain salary cap.

Have small biotechs benefited?

Many companies have met requirements for financial assistance. Some simply cannot prove the loans are “necessary.” Smaller companies are now competing with larger companies for assistance. And many pre-revenue companies do not fit into loan categories, creating obstacles for small biotechs.

Justin Field, SVP of Government Affairs @nvca explains that the pandemic’s assistance programs were created through a traditional view of the economy and many companies have been “fitting square pegs into round holes” to be able to qualify. #BIODigital

— I Am Biotech (@IAmBiotech) June 11, 2020

Many companies are finding it hard to plan a way to pay back Mainstreet Lending Program funds. Others burned through cash thinking they were guaranteed the PPP loan, but did not qualify. The panelists agreed the government must keep assistance simple.

However, some businesses have benefitted greatly from unemployment insurance for furloughed workers.

Pending legislation

Proposed legislation would change the PPP forgiveness provision to allow businesses with a 60% payroll cost and 40% operating cost eligible for loan forgiveness.

What else can be done?

• Monetize R&D credits
• Include the Orphan Drug Credit

And particularly for companies working on COVID-related activities:

• Double R&D credits for U.S. venture capital startups in the biotech industry
• Workplace indemnification – make companies not liable for COVID cases if they have shown a process of compliance with social distancing and sanitation requirements

– Cassie McKay

The importance of external partnerships

“While our internal R&D engine is producing high-quality programs, the external biopharma enterprise is substantial and there’s plenty of innovative science being pursued,” he said.

“We’re always looking for the best science. We’re looking for high quality in programs. We’re looking for the most promising opportunities to find programs that have the potential to impact substantial patient populations, and to complement, again, our internal efforts,” with different approaches and new technologies.

Workplace and culture in the era of COVID-19

It’s not feasible to stay on top of all the science, so Dr. Settleman relies heavily on the leadership team to ensure they’re pursuing the best possible science—“while also recognizing that we can’t do everything.” Sometimes, you have to make difficult decisions when you prioritize resources.

R&D continues, even though COVID-19 is far from over, he added.

“These are interesting and challenging times for us, and it is especially important that we stay connected with each other.” In-person, daily interactions with colleagues are important, but it’s possible to maintain engagement and connectivity, with things like virtual meetings and town halls, video updates on the science, and twice-weekly virtual coffee chats with small groups of colleagues. Pfizer also has an internal website to post comments, photos, and videos to share experiences and tips, like working remotely or online educational tools for employees’ kids.

“We’re actually now connecting with each other in ways that are more personal,” which is somewhat of a silver lining.

The importance of mentorship

Mentorship is extremely important, especially in the biopharma industry, he said.

“We frequently fail, even with lots of really smart scientists working very hard on these problems because biology is extremely complex and still relatively poorly understood. In mentoring my colleagues, I emphasize the importance of scientific rigor and good decision making to improve our chances of success.”

“If we don’t keep the bar for rigor as high as possible our task becomes even more difficult.” And part of this includes knowing when to terminate projects, he said.

How do small biotechs know if they’re ready for Pfizer?

Good scientific thinking, strong execution, and substantial quality “is what it’s all about,” he said.

“We certainly recognize it when we see it.”

– Samantha Sault and Andrew Segerman

Valerie Paradiz of Autism Speaks, speaks from the perspective of being, herself, on the autism spectrum and explained why autism patients might be much more willing to participate if they were not required to go to a physical trial site. “I know that as a person with autism working as a professional in the field, that we need more options” she explained.

Valerie Paradiz of @autismspeaks: “We need more options for people with #autisim in terms of medications and advancements” #BIODigital pic.twitter.com/MoqnXzRKiK

— I Am Biotech (@IAmBiotech) June 11, 2020

Decentralized clinical trials could add more patients to the trial because they are not required to go to travel to one site. Technology is used to communicate and have remote interaction with the participants. It is exciting and promising but is not without its pitfalls. Digital application requires both training and compliance on the part of the patient. 

As the FDA’s Isaac Rodriguez-Chavez noted in response to Kalamegham’s disclosure that she was often careless when it came to keeping up with her food intake app, “The reason why your electronic recording of your food intake failed is because you were not trained and you were not proficient in that technology and in decentralized clinical trials, it is vital that after participants are screened and selected, that the inclusion criteria also stipulates proficiency in the use of the technology, otherwise it is a futile exercise.”

The FDA is in the process of developing a new guidance on decentralized clinical trials. The agency recognized that these new ways of collecting clinical data and adding convenience to participants will ultimately advance drug development and the approval of new medicines. At the same time, FDA wants to ensure that the same safety standards and rigor of data quality and integrity are maintained. 

Resistance to change the way that clinical trials are conducted is both practical and stubborn. Wade Ackerman of Covington noted, “there is a tried and true pathway for doing clinical trials and does any one sponsor want to take the risk of having the FDA believe their data is not reliable?”

It seems inevitable that decentralized clinical trials may one day be a part of drug development. Integrity is key and when that is assured, they can become a reality.

- Theresa Brady 

“Ethics has moved front and center”

From enforced quarantines to wearing masks to scarce medical resources to research surrounding vaccines, we must consider ethics, said Caplan.

While we hope for a vaccine at warp speed, challenges and ethical hurdles like public trust, mandates, and clinical trials can arise, he explained.

"I have a moral obligation to try something"

Bateman-House looks at two areas: access to investigational medicines and clinical trials, and how to treat patients, looking at the moral obligation of how to treat patients when we don’t have an approved therapeutic or standard of care. 

She’s seen situations in which doctors knew they didn’t have validated evidence for a specific treatment, but believed they had a moral obligation to do something--even if their own institutions had policies against it. 

Daily during the COVID crisis, professionals are facing ethical discussions around trials, data, analysis, patients, protocols, and efficacy.

Follow the science, be transparent, and collaborate globally

Follow the science, said Aliu. It’s important to understand the science when discussing “compassionate use” requests outside the approved label. For example, science has guided Novartis on decisions about which studies to participate in.

Dr. Waldstreicher added the need to be transparent. Johnson & Johnson created a bioethical framework specific to COVID-19 in the face of an unproven benefit – “a bioethical framework on how to allocate our supply in the face of no proven efficacy and safety, but only in the face of either biologic hypotheses or in vitro data.”

Dr. Kim emphasized the need to collaborate globally and prioritize vaccine research. Inovio is prioritizing its candidate COVID vaccine research while also prioritizing patient safety versus speed and urgency.

Learn more about Inovio’s vaccine:

Continue the conversation

• As COVID develops and bioethics heighten, it’s important to continue conversations surrounding:
• The need to bolster the peer review system.
• Transparency around potential conflicts of interest.
• Vaccine distribution.
• The need to develop communicators who can present information to the public.
• The role of public health to help make decisions about vaccine distribution for different population groups.
• The need to let science lead.
• Exploring opportunities for partnerships and collaboration.
• Not cutting corners in vaccine development – “If you cut corners you hit walls”.
• The crisis of public trust.
• Equity in access and who is involved in research.
• How to justify work on COVID-19 to other patient groups. 

During the Q&A, Bateman-House shared this resource on how to prepare potential COVID-19 study participants.

They started by talking about the "restructuring" at FDA; read more about that in STAT News if you need to catch up.

Status of COVID-19 diagnostics

There has been a "heavy burden of work coming in" on diagnostic tests, said Dr. Marks. But we are starting to have antibody and diagnostic tests that people are confident in.

Status of COVID-19 therapeutics and vaccines 

CDER is working towards a “cohesive approach” on trials, said Dr. Stein, working with NIH, academics, and industry to make “tremendous progress in terms of developing strategies.”

“In a short period of time, a number of trials are moving towards development and even the execution phase,” he continued.

CDER is focusing on “drugs that are most readily available now, which are repurposed drugs” as well as rapidly developing trial capacity for monoclonal antibodies, then identifying the next drugs that are most promising.

“What we’ve been trying to do is make sure we have an organized approach in how we are managing the huge increase in submissions.” They have created a pathway to “triage” and distribute work across divisions within the Office of New Drugs.

The centers are trying to stay aligned so there’s consistency, added Dr. Marks.
“We’re trying to be very fair about how we prioritize things, not favoring one sponsor over another,” but of course, certain drugs “rise to the top.”

How does FDA prioritize which products to review first?

"This is really about being a good portfolio manager," said Dr. Marks. "We need to make sure what’s coming up front and what's very urgent gets taken care of, while keeping an eye on the fact that there are some things that may take longer to get there that may turn out to be very important products."

"You just can't dismiss things" based on where they are in development right now, because they may be very important later. 

"We intend to be as broad was we can," added Dr. Stein, to find compounds that might have potential and move quickly if they are effective.

The importance of combinations

For therapeutics, combinations will need to be considered thoughtfully, said Dr. Stein.

The pandemic has led to greater efficiencies

FDA's virtual work environment "has worked out surprisingly well," said Dr. Stein. The agency has had large weekly staff meetings and figured out best practices across the organization.

Industry meetings have gone smoothly as well, he said.

Dr. Marks said they've been able to "shave months off" guidance practices.

"Paradoxically, some of the guidance that we’ve gotten out during this crisis is better guidance. It’s concise, it’s clear, it’s to the point, it contains the scientific information that it needs to. It’s better than guidance that we spent months to years getting out," added Dr. Marks.

"We shouldn’t be as scared of that, because industry likes guidance," he continued. "Maybe isn’t so bad if we streamline how we do it.”

However, the speed may not be sustainable, he said, unless they add staff.

"The per-unit output of staff" has increased 20-30% as they work from home. 

"We can't continue this for the long haul."

We have to learn from this pandemic

“At the end of the day we better be putting down things in our pandemic playbook," said Dr. Marks. "Some of these are things we should have known, but some of them are things that are just unanticipated because of the enormity of this."

“Right now, at least as a regulator, what keeps me up at night…the journey has only really begun.”

“I’m very aware of the fact that we have a long way to go here, and at FDA, part of what we have to do now is we can’t get complacent.”

“Believe it or not, we have to be ready for the fact that a second wave could get worse,” he concluded. “If we want to do our jobs right, we have to think through all the possibilities here.”

– Samantha Sault

It's the last day of BIO Digital, and we're starting bright and early today! Here's what we'll cover here on the blog:

9 AM ET/6 AM PT – FDA Town Hall

10 AM ET/7 AM PT – Bioethics Through the Lens of a Pandemic 

1 PM ET/10 AM PT – Patient-Centric 2.0: Are We Ready for De-Centralized Trials? (Sponsored by Genentech)

2 PM ET/11 AM PT – Digital Health: Innovation Meets Regulation 

3 PM ET/12 PM PT – Leading Through the Crisis: Speaking Up and Out on Equity, Diversity and Inclusion Issues 

4 PM ET/1 PM PT – Protecting Our Long-Term Commitment to Care in a Post-Pandemic World (Sponsored by Sanofi)

6:30 PM ET/3:30 PM PT – Fireside Chat with Dr. Jeff Settleman, Chief Scientific Officer for Oncology R&D, Pfizer Inc. (Sponsored by Pfizer Inc.)

6:30 PM ET/3:30 PM PT – Federal Economic Recovery Efforts for Small Biotech Companies 

7:30 PM ET/4:30 PM PT – Access to Personalized Medicine Drives Utilization of Genomic Tests and Targeted Therapies (Sponsored by Genentech)

7:30 PM ET/4:30 PM PT – Nature Biotechnology and Scientific American WORLDVIEW: Whiplash! How COVID-19 Will Radically Change Biomedical R&D Across the Globe