What will 2020 bring for medicine and science? – STAT – STAT

By | January 1, 2020

Last year, when we asked science and health care soothsayers to peek ahead to 2019, they told us that methamphetamine use would rise (it did), tumor organoids would near clinical use for personalizing cancer treatment and better targeting clinical trials (that’s happening), and price transparency wouldn’t bring lower health spending (that’s true, too). But nobody predicted the outbreak of lung injuries tied to vaping, the failure and attempted resurrection of Biogen’s Alzheimer’s drug aducanumab, or the restoration of cellular functions in pig brains after death.

We’re back with a new set of predictions for 2020. Let’s see how our experts do this time.

Results for a broad array of approaches to treating Alzheimer’s

Alzheimer’s research and discovery is moving quickly, from possible early detection to treatments — which could be a combination approach: mixes of medicines, or drug and lifestyle combos, that attack the disease from multiple angles. Thanks to increased funding and exploration of new ideas, scientists now know more about dementia than ever before, and are investigating a diverse array of options.


Looking to 2020, the scientific community eagerly awaits the FDA’s review of Biogen’s (BIIB) amyloid-attacking drug, aducanumab. We’re also anticipating results from studies of anti-tau compounds, strategies to reduce brain inflammation, research into oral microbes and gut bacteria, dementia prevention trials, and developments in early detection such as biomarkers. Finally, research looking at lifestyle-related contributions to dementia risk continues to advance, including clinical trials such as U.S. POINTER.

Maria C. Carrillo, chief science officer, Alzheimer’s Association

Tech will change how we get care, but only if companies earn our trust

Technology is ready to enter your doctor’s office in a big way. But there’s an “if.”

With the technology available today and emerging on the horizon, care will hopefully be easier to obtain, understand and afford. In particular, we’re beginning to see advances in artificial intelligence that could help improve the accuracy of your diagnosis. Other tools could help organize medical records and make them more useful so that your caregivers (doctors, nurses, and others) spend less time at computer terminals and more time connecting with you in a human way.

The big “if” is “trust.” These tools, and more importantly, the companies that seek to build and deploy them, must remember that to be successful in health care the patient-doctor relationship, and the privacy of health data, cannot be compromised.

— Dr. David Feinberg, head of Google (GOOGL) Health

Advances for diseases afflicting the world’s poorest citizens

I’m optimistic that 2020 will be a banner year for global health R&D, showing it’s possible to bring the latest in scientific innovation to bear on diseases that affect the world’s poorest citizens. First-generation microbiome-directed foods will show promise in improving newborn gut function. Machine learning algorithms will revolutionize drug discovery and yield promising new compounds for malaria and TB. A new class of remarkably long-acting antiretrovirals for HIV prevention will enter late-phase development. We’ll learn the essential components of human breast milk and how they vary across geographies. Next-generation point-of-care diagnostics will be tested in primary care settings in developing countries. And antenatal corticosteroids will be rolled out around the world to treat lung disorders that are a major cause of newborn mortality. We’ll need these tools and many more like them to meet or beat the best-case scenarios of the 2030 Sustainable Development Goals.

— Trevor Mundel, president of global health, Bill and Melinda Gates Foundation

Digital health culling will begin in earnest

I think 2020 will be the year that digital health consolidation and culling begins in earnest — we are already starting to see it. I doubt there will be much IPO activity outside of what are actually large services business that call themselves digital health but are primarily health services businesses. I think investment dollars may stay the same because a lot of money has been raised, but we will continue to see concentration on fewer companies, particularly those that have actually proven their financial value proposition to clients — the burden of proof is becoming much more onerous, and that is entirely appropriate for a market that ran up a lot on hype or hope, depending on how you look at it. But hope is not a strategy, as the saying goes, so the companies that will garner investment will be the ones committed to doing the hard work of proving clinical and financial ROI to their clients and, finally, a rational growth and profitability plan to their investors.

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— Lisa Suennen, managing director and leader of Digital & Technology Group, Manatt Health

Clinical trial of HIV vaccine will produce vital data

We’re going to see early results in 2020 from a clinical trial testing a novel HIV vaccine candidate designed to stimulate the immune system along the path to producing antibodies that can neutralize a broad range of HIV variants. Not only will these results provide critical information for the HIV vaccine field about whether and how we should further pursue this approach, they may also set a precedent for how structure-based immunogen design can be applied to developing new vaccines against other disease targets that have so far eluded successful vaccine development efforts. Also, we should learn the outcome from a large clinical trial of an infusion of broadly neutralizing antibodies (bnAbs) to prevent HIV infection. Encouraging results will motivate researchers to refine and improve the concept, especially now that the field has discovered more bnAbs and that we have the scientific capabilities to engineer next-generation HIV antibodies to be more potent and more long-lasting.

And while we’re hopeful that these vaccine candidates and antibodies will be efficacious, we need to plan now for making them affordable and accessible to the people who need them most. In 2020, we in the field must collaborate even at these early stages of development to ensure that these new products will be widely and promptly available once approved.

— Dr. Mark Feinberg, president and CEO, International AIDS Vaccine Initiative

FDA will refine how it oversees health tech innovation

2020 is going to bring us better definitions of how does FDA collaborate and communicate with the huge number of data and technology innovators that are really relevant to promoting and protecting health in ways we haven’t learned how to do yet.

That’s going to help us figure out both how to regulate thoughtfully and efficiently, but also it’s going to help point the way for how these data and tech innovators — even the ones we don’t regulate — can do their work in ways that are of the highest quality and most greatly contribute to health.

Dr. Amy Abernethy, principal deputy commissioner, FDA

Increased scrutiny of drug prices in a presidential campaign year

In 2020, we’ll see intensified scrutiny about drug pricing, as the presidential campaign heats up and state and federal policymakers look to pass legislation to support their own reelection. This will slow investment in the industry, as we started to see in the second half of 2019, but not in an overly dramatic way, as advances in science will continue to draw the most savvy investors. More cell and gene therapies will be approved, many as one-time cures, to address the most devastating diseases, but questions about patient access and how to pay for these breakthroughs will continue to dominate the conversation. We will undoubtedly see more efforts to address obstacles to innovative financing methods like value-based arrangements, but serious hurdles remain with no clear course of action. Digital technologies will continue to draw attention from biopharma companies as the industry seeks to better demonstrate the long-term value of therapies by measuring patient outcomes and costs avoided to the health care system.

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— Bob Coughlin, president and CEO, MassBio

Combatting harmful health content online

2020 will see deeper collaboration between patients, health care providers, public health experts, and the technology sector. People have always relied on their community — including friends, family, caregivers, and health care providers — for support around health. We are seeing more of this happen online, including connecting with other people with similar experiences for support and lessons learned; patient-partnered research to accelerate disease understanding; more collaborative communication channels between clinicians and patients. In 2020, emerging partnerships will go deeper to help provide health resources to people where they are, as well as help combat harmful health content that can arise online.

— KX (Kang-Xing) Jin, head of health at Facebook (FB)

Shift away from narrow disease-focused global health programs

I predict we will continue to see a shift away from vertical disease-focused programs towards more comprehensive and health-system focused investments. While meaningful gains have been made through targeted foci on HIV, maternal health or malaria for example, massive disparities still exist around the globe. The opportunity to tackle multiple problems through more systems-based investments – workforce and infrastructure – is significant. There are shifting health burdens including existing communicable and preventable disease and now the epidemic of non-communicable disease, a leading killer globally. Funders and NGOs will recognize that our previous approaches will not address the growing challenges from climate change, increased recognition and diagnosis of mental health, nor the need to address social determinants of health. Vertical based programs will not be enough. The recent signing of the Political Declaration for Universal Health Care in September 2019 will help galvanize this focus. In turn, I think we will see a rise of innovative funding and financing solutions from the private sector and multilaterals to mobilize new resources and to unlock country-wide potential.

— Dr. Vanessa Kerry, CEO, Seed Global Health

Early-stage data for a universal flu vaccine

In 2020, we will see the results of the first component of a multistep approach to developing a universal influenza vaccine designed to induce a response to the stem of the influenza hemagglutinin (HA). Safety and immunogenicity data will become available from a Phase 1 vaccine trial aimed at inducing a protective response against all group 1 influenza A viruses, using a nanoparticle vaccine platform exhibiting the HA stem.

In 2020, we also likely will see substantial progress in our efforts to optimize methods of HIV treatment and prevention. Among our many efforts to achieve the durable control of HIV without daily antiretroviral therapy (ART), a once-monthly, injectable combination HIV treatment (cabotegravir + rilpivirine) likely will reach the clinic for general use. Studies of next-generation ART regimens (e.g. capsid inhibitors) and broadly neutralizing antibodies (bNAbs) requiring even less-frequent dosing for persons with HIV will continue to show promising results. In addition, we will see important result on optimizing HIV prevention, including new tools for pre-exposure prophylaxis (PrEP). These include long-acting antiretroviral drugs (dosed orally, by injection, or as a small implant); infusions or injections of long-acting bNAbs; and topically applied products for rectal and vaginal use.

— Dr. Anthony S. Fauci, director, National Institute of Allergy and Infectious Diseases

‘It’s not just about opioids’ anymore. It’s about stimulants

We are watching very closely the patterns of drug use in the country, among teenagers and adults, which are changing quite rapidly. We’re becoming aware that there is a very significant rise in deaths associated with cocaine and methamphetamine. They’ve more than doubled in the past five years. Now it’s not just about opioids, it’s about psychostimulants.

— Dr. Nora Volkow, director of the National Institute on Drug Abuse

A ‘pivotal year’ for CRISPR-based therapies

2020 will be a pivotal year for engineered cell therapies driven by CRISPR in rare diseases and in oncology, and CRISPR will solidify its place in medicine. While 2019 showed us a glimpse of promising data in transfusion-dependent thalassemia and sickle cell disease, we will see additional data across a range of devastating diseases with significant patient need — including various heme malignancies. The other wave we will see is the use of CRISPR in regenerative medicine to develop implantable cells in a range of organs — whether pancreas, liver or other cells. Finally, we will see additional progress with in vivo approaches using CRISPR, due to the advances we are making in viral and non-viral delivery technologies. CRISPR’s promise continues to become a reality.

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Sam Kulkarni, CEO of CRISPR Therapeutics

Aiming to reduce worldwide deaths of under-5 children

Today, we are closer than ever before to bringing global under-5 child mortality below 5 million. But to get there, we need to provide a complete package to health workers to treat fever, which can kill children in just a few hours. A child under 5 in sub-Saharan Africa gets an average of six bouts of fever a year. A substantial percentage of these are likely to be due to malaria, but also bacterial and viral infections that show up as pneumonia and diarrhea. Undiagnosed and treated, pneumonia, diarrhea, and malaria can weaken and kill children in as quickly as 24 hours. And yet treatment and prevention for these three conditions are extremely cheap.

With the right programs, the right partners and the right levels of funding, we can make sure that a child who has fever gets adequate treatment, whether antimalarials, antibiotics, oral rehydration solution, or simply antipyretics. We have an extraordinary opportunity to drive this change and make 2020 a game-changing year for children – a year when we change the curve of child mortality and accelerate progress toward the Sustainable Development Goals. Millions of children and their families are counting on us.

— Henrietta Fore, executive director, UNICEF

More diagnostic algorithms for consumer devices

My expectations for 2020 include more prospective trials [of health AI], a new patient (consumer) diagnostic algorithm beyond smartwatch atrial fibrillation [capability], and multi-modal neural networks that incorporate deep learning and hybrid models to tackle more complex inputs (e.g. electronic health records plus images plus genomics).

— Dr. Eric Topol, executive vice president, Scripps Research

Reshaping an industry built for scale to one serving smaller groups of patients

Regenerative medicines are among the most promising innovations we have today because of the potential for significant and lasting health outcomes, sometimes in a one-time treatment. In 2020, we will see substantial progress in the race to bring transformative regenerative medicines to patients. This is more than innovation in science and medicine, it is also an industry race to reshape itself to adapt to disruptive innovation. The frameworks, infrastructures, and capabilities that are artifacts of our past were built for scale, serving large patient populations and for chronic, supportive treatments, not necessarily for smaller groups of patients, fragile biomaterials, and curative approaches. In 2020, biopharma will meaningfully advance new regenerative therapies and the pathways that will get them to patients who are in desperate need.

— Rachelle Jacques, CEO, Enzyvant

Explosion in drugs targeted to patients using biomarkers

The Personalized Medicine Coalition is tracking an increase in the number of available drugs that are associated with biomarker-based targeting strategies. The coalition expects this number to exceed 230 in 2020, up from five in 2008 and 132 in 2016. In addition to this increased use of strategies that evaluate patients to determine which therapies are the best match for them, we are likely to see continued movement toward the development of drugs customized for individuals, like CAR T-cell therapies.

— Edward Abrahams, president, Personalized Medicine Coalition

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