Dear Colleague:

In the last month humanity's historic struggle with COVID-19 has reshaped our lives and fostered a new appreciation of science, technology, and the principles of personalized medicine.

This edition of Personalized Medicine Today offers a snapshot of how the landscape of personalized medicine has changed.

After being driven to their homes by a series of broad-based social distancing orders that have stifled the global economy, many leaders in health care have concluded that the widespread use of diagnostic testing for COVID-19 could help identify low-risk populations who could then safely return to work as well as high-risk populations that should remain sheltered in place. Earlier testing along these lines, they contend, may have saved lives and spared us from a devastating economic recession.

To make sense of the widely variable effects of the new virus in patient populations with similar characteristics, researchers have begun to explore how the extent of viral exposure and genetic factors influence the course of the disease in different patients. These efforts may guide more effective approaches to isolating and treating the right patients at the right time.

The Personalized Medicine Coalition is proud to be in the thick of these conversations as its members have stepped up to help combat the virulent new enemy (see Resources on COVID-19: A List of Links Published by Members of the Personalized Medicine Coalition).

PMC, which is well-positioned to maintain the full scope of its productivity in this new environment, has taken steps to ensure that Congress recognizes the importance of funding for the National Institutes of Health and the Food and Drug Administration at this important moment in the history of health care and personalized medicine.

The Coalition also continues to educate policymakers and the public about the importance of personalized medicine and to commission studies about its clinical and economic utility in order to build a base of support for the field.

We welcome your thoughts as we pursue a brighter future for patients and health systems in the midst of this global crisis.

Sincerely yours,

Edward Abrahams
President
Personalized Medicine Coalition

• As Congressional Budgeting Negotiations Continue Amid COVID-19 Crisis, Proponents for Personalized Medicine Seek Increases for NIH, FDA

Proponents for personalized medicine advocated for increases to the budgets of NIH and FDA last month as House Appropriations Committee Chairwoman Nita Lowey (D-NY), pictured here, continued to work with her colleagues in the House and Senate to develop successful Congressional budget negotiations for 2021.

With the Congressional appropriations process beginning amid the COVID-19 crisis, proponents for personalized medicine advocated last month for increases to the budgets of the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), two agencies that help provide a foundation for personalized medicine by investing in the biomedical research underpinning the field and ensuring the efficient advancement of the tests and treatments underpinning many personalized health care strategies. Advocacy efforts were designed in part to ensure that NIH and FDA are well-positioned to continue advancing personalized medicine when the crisis abates.

In written testimonies submitted to the members of the relevant House and Senate subcommittees, the Personalized Medicine Coalition has advocated for increases of at least $3 billion and $70 million for NIH and FDA, respectively, over fiscal year 2020 appropriated funding levels. With reference to the challenge of combatting COVID-19 without losing momentum in other areas, the testimonies note that additional investments in the work of these agencies will "bring us one step closer to a future where every patient benefits from an individualized approach to health care."

• PMC Outside Witness Testimony: "Fiscal Year 2021 House Appropriations Committee Subcommittee on Labor, Health and Human Services, Education and Related Agencies Appropriations Testimony"
• PMC Outside Witness Testimony: "Fiscal Year 2021 Agriculture, Rural Development, Food and Drug Administration, & Related Agencies Appropriations Testimony"
• PMC Outside Witness Testimony: "Fiscal Year 2021 Senate Appropriations Committee Outside Witness Testimony"

• Economic Consequences of Nationwide Social Distancing to Combat COVID-19 Add Significance to Conversations About Need for More Targeted Approaches to Prevention, Treatment
  
• Thought Leaders Consider Extent of Viral Exposure, Genetics as Potential Measures to Underpin Personalized Health Care Strategies to Predict, Combat Severe COVID-19 Infections
  
• Educational Piece Published as Part of USA Today Insert, Economist Article on Personalized Medicine and Drug Development Boost Global Awareness of Field

In an opinion piece in The Timmerman Report titled “Knowledge is Power: Don’t Give Up on Diagnostic Tests for COVID-19,” PMC Board Member Michael Pellini, M.D., Managing Partner, Section 32, a venture capital firm, calls for a national testing program that would help the United States combat COVID-19 by targeting the most intensive medical interventions and deploying resources to those who need them most — something that personalized medicine has long promised to do.

With the global economy stagnating as countries around the world adopt sweeping social distancing measures to combat the COVID-19 pandemic, leaders in health care are increasingly calling for national testing and surveillance systems that can stratify the regions of any given country into low-risk areas where people can safely return to work and high-risk areas where people should remain sheltered in place. The crisis has added another layer of significance to conversations about the future of personalized medicine, which has long promised to target the most intensive medical interventions and deploy resources to those who need them most.

The new imperative to adopt diagnostic testing that can underpin targeted prevention and treatment strategies is summarized in a recent opinion piece published in The Timmerman Report on March 28 by Personalized Medicine Coalition Board Member Michael Pellini, M.D., Managing Partner, Section 32, a venture capital firm. Pellini laments the fact that following "a decade of breathtaking progress" in science and technology, citizens in every corner of the United States have been forced into virtual exile to avoid this new virus. Praising the efforts of companies including Abbott, Roche, and Thermo Fisher Scientific to meet the need for diagnostic testing for COVID-19, Pellini calls for a national program to "track the spread of this particularly virulent enemy, to understand its characteristics, and to reveal how different people respond to it."

Pellini's contentions have been echoed elsewhere.

For example, in an op-ed for The Wall Street Journal titled "The Road Back to Normal: More, Better Testing," former Food and Drug Administration commissioner Scott Gottlieb, M.D., and Lauren Silvis, who served as Gottlieb's chief of staff at FDA prior to becoming a Senior Vice President at Tempus, a “data-driven precision medicine” company, call on Congressional leaders to fund a “sentinel surveillance system” that “allows cases to be identified and tracked in real time without overburdening providers with data entry and case reports.” Such an intelligence system, they contend, would facilitate smarter return-to-work policies.

• The Timmerman Report: "Knowledge is Power: Don't Give Up on Diagnostic Tests for COVID-19"
• The Wall Street Journal (subscription content): "The Road Back to Normal: More, Better Testing"

In an article that demonstrates a shift in thinking toward personalized health care strategies to prevent and treat COVID-19, Siddhartha Mukherjee, M.D., D.Phil., contends in a recent article for The New Yorker that in addition to measuring the spread of the SARS-CoV-2 virus "across people," we need to begin measuring its prevalence "within people."

In a development reminding us that health care's shift toward a paradigm of personalized medicine is rooted in scientific principles of human heterogeneity that are applicable across disease states, scientists have begun to observe that humankind would be in a better position to combat the devastating effects of the new SARS-CoV-2 virus if we understood the molecular reasons why certain patients are more vulnerable to infections or life-threatening symptoms than others. Their observations have inspired interest in studying the immune reactions of patients with certain genetic characteristics after exposure to the virus in varying proportions.

In an article published in The New Yorker for April 6, Siddhartha Mukherjee, M.D., D.Phil., of Columbia University reviews the medical evidence suggesting that the amount of a virus a person is exposed to as well as the amount of the virus the person sheds after infection may be strong predictors of disease severity and contagiousness in an individual patient. And the propensity of the SARS-CoV-2 virus to cause unpredictably severe illness in some patients with no obvious risk factors like age, diabetes, or a weakened immune system has prompted researchers around the world to begin searching for genetic characteristics that may influence how easily the virus can penetrate a patient's respiratory cells.

If studies along these lines generate new insights about the viral and genetic characteristics of the patients who are most likely to fall seriously ill and spread the SARS-CoV-2 virus to others, the use of diagnostic tools to measure these factors may save lives across the globe by identifying high-risk patients who should take greater precautions to protect themselves from infection. The insights may also be used to target early and more aggressive interventions to infected patients who are most likely to develop serious complications.

• The New Yorker (subscription content): "How Does the Coronavirus Behave Inside a Patient?"
  
• Science magazine: "How Sick Will the Coronavirus Make You? The Answer May Be in Your Genes"
• CNN: "The Mystery of Why the Coronavirus Kills Some Young People"

In an invited article submitted as an unpaid editorial contribution to an insert recently published in the USA Today by a company called Mediaplanet, PMC President Edward Abrahams explains the origins and importance of personalized medicine in cancer care.

Informed by the emergence of powerful personalized treatments for patients with cancers, rare diseases, and other conditions, two articles recently published in The Economist and as part of an educational insert in The USA Today explain the evolution and implications of personalized medicine. In addition to increasing the public's awareness of personalized medicine, the articles demonstrate that the field's proponents are making progress in informing key decision-makers about the personalized medicine's significance to the future of health care.

In an invited article submitted as an unpaid editorial contribution to an insert published in The USA Today by Mediaplanet, a content marketing company, Edward Abrahams, President, Personalized Medicine Coalition, explains the origins and importance of personalized medicine in cancer care, with reference to landmark therapies approved in the area since the emergence of Herceptin (trastuzumab) in 1998.

In the article from The Economist, the magazine cites statistics from PMC's Personalized Medicine at FDA: A Progress & Outlook Report to explain how personalized medicine has reshaped drug development.

"Examining illness molecule by molecule allows pharmaceutical researchers to understand the pathways through which cells act according to the dictates of genes and environment, thus seeing deep into the mechanisms by which diseases cause harm, and finding new workings to target," the article in The Economist reads.

• Mediaplanet: "How Personalized Medicine Is Revolutionizing Cancer Treatment"
• The Economist (subscription content): "New Drugs are Costly and Unmet Need is Growing"

To support its members' efforts to combat COVID-19, the Personalized Medicine Coalition has published Resources on COVID-19: A List of Links Published by Members of the Personalized Medicine Coalition. The list is available for download in every edition of this newsletter and on PMC's home page.

Please contact PMC Membership & Development Director Kayla Smith if you have member links you would like to share.

• Resources on COVID-19: A List of Links Published by Members of the Personalized Medicine Coalition

In recognition of its successful efforts to apply personalized medicine to reduce health care costs, Coriell Life Sciences recently received an Excellence Award in Cost Containment from the Pharmacy Benefit Management Institute (PBMI). The award recognizes an institution that has made exceptional contributions to the practice of pharmacy management.

"We are thrilled to present Coriell Life Sciences with this award for their Enterprise PGx program," said PBMI Senior Vice President for Research and Data Innovation Sharon Glave Frazee, Ph.D. "This turn-key solution combines pharmacogenomic testing and medication review to help identify and ensure patients are on the right medication for them."

• Coriell Life Sciences press release
  

In two moves the company hopes will help facilitate more widespread clinical adoption of pharmacogenetic (PGx) testing, Invitae has acquired YouScript, a clinical decision support and analytics platform, and Genelex, a privately held PGx testing company. By offering a PGx program that combines testing and interpretation services at the point of care, Invitae hopes to deliver a solution that makes it easy for physicians to act on PGx tests, which provide genetic information that can be used to guide drug selection and dosing decisions by indicating the pace at which a patient's body will likely metabolize certain drugs.

"Simply detecting pharmacogenetic variation is not nearly enough to make the information clinically useful," said Robert Nussbaum, M.D., Chief Medical Officer, Invitae. "Combining Genelex testing with clinical decision support in the EMR using YouScript software enables clinicians to easily navigate this information when making prescription choices at the point of care."

• Invitae press release
  

Blueprint Medicines
Blueprint Medicines is developing a new generation of highly selective and potent kinase therapies to dramatically improve the lives of patients with genomically defined diseases. The company's approach is rooted in a deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases. Blueprint's ability to identify novel drivers of disease, coupled with its proprietary library of novel and diverse chemical compounds, gives the company a unique ability to craft kinase therapies against new and difficult-to-drug targets.

The Next Generation Dx Summit brings together more than 800 professionals from the diagnostics industry. The summit offers insight from the comprehensive programming and networking with key opinion leaders.

Event website

The 16th Annual Personalized Medicine Conference at Harvard Medical School will explore the science, business, and policy issues shaping the landscape and outlook for personalized medicine.

Event website

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