Investments in pharma R&D has substantially increased over the last decades. Yet there appears to be no clear correlation to the number of newly approved drugs. This fact is accompanied by ever-increasing healthcare costs, fueled by an aging population and the parallel rise in the chronic disease burden. Late stage drug development is the most costly area of drug R&D and it also has the highest attrition rate with huge financial implications for sponsors. Increasing the probability of success and the number of new chemical entities in pharmaceutical pipelines requires optimization of drug discovery and clinical translational efforts, along with a decrease of late-stage drug failures.

“We are cognizant that the realization of the promise of genomics and ‘big data’ will require generating data and knowledge at an unprecedented scale and no single organization has either the scientific or financial resource to tackle this alone. We have become more mature as a community in finding ways to work together in an ‘open innovation’ space where all parties are comfortable that there will either be no IP emerging from the research or that IP can be managed within the PPP.”

Sally John, PhD, Vice President of Translational Biology, Biogen Inc.

To reverse and overcome these ongoing negative trends, pharmaceutical companies are employing novel strategies that represent a paradigm shift from existing approaches. They are building on innovation, collaboration, and the strengths and input of stakeholders in the health system who share their goal of delivering effective and sustainable healthcare solutions for the population.

One of these new approaches is establishing precompetitive public–private partnerships (PPPs) to solve challenges that are too large for single organizations to effectively address on their own. These partnerships enable researchers to gain valuable insights through pooled knowledge and resources to support advances in the development of new medicines for a wide range of serious and life-threatening diseases.

Public-private partnerships (PPPs):

  • Support open innovation and pre-competitive research
  • Rely on open innovation networks
  • Link the know-how and resources of the pharmaceutical industry with external pools of knowledge, especially in universities and biotechnology companies
  • Have been pioneered in the area of neglected diseases
  • Have been encouraged and fostered by the US Food and Drug Administration and the NIH

Benefits of PPPs:

  • Shared risk and costs that aim to tackle emerging areas of complex biology
  • Bring together expertise and ideas from public and private sectors that lead to greater diversity of thought.
  • Can help sculpt a research landscape conducive to acceleration of the drug discovery process
  • Early delivery of promising new treatments to patients suffering from debilitating diseases.
  • Researchers on both sides gain a new network, learn new practices and share a sense of community and goals

PPP Challenges

  • Establishing the scientific goals, contract terms, and conditions on which all parties agree
  • Maintaining open and transparent communication within a large PPP, critical to building and maintaining trust
  • Different perspectives can sometimes serve as obstacles to effective collaborations, yet when managed and challenged correctly they can also promote innovative thinking

Examples of successful PPP examples:

  • Open Targets: Innovative, large-scale, multi-year, public-private partnership that uses human genetics and genomics data for systematic drug target identification and prioritization
  • FinnGen: Research project that combines genome information with digital health care/electronic health data from Finnish registries
  • Structural Genomics Consortium: Supports the discovery of new medicines through open access research
  • Regeneron Genetics Center – UK Biobank: Generates genetic sequencing data from 500,000 volunteer participants and pairs this genetic data with anonymized clinical data to conduct analyses to learn more about human genetics and biology. UK Biobank is funded by numerous biopharma industry partners who see the value of genetic research in drug discovery and development.

“Genetic research is a game of numbers – you need an enormous sample size to begin to connect the dots between genetic variations and potential links to diseases. In order to obtain this amount of “big data,” it makes sense for public and private organizations to work together to ethically share data for the greater good. By combining public and private resources (both in terms of data and funding), we have more scientific minds analyzing greater amounts of data, in turn accelerating the drug development process and hopefully bringing new therapeutics to people in need faster.”

Aris Baras, MD, Vice President, Head, Regeneron Genetics Center

We have an impressive panel scheduled at PMWC 2018 Duke – September 24-25, Washington Duke Inn, Duke University Campus, focused on “Public-Private Partnerships” and chaired by Dr. Robert Califf (Duke University School of Medicine, former FDA Commissioner). Panel participants include Aris Baras, MD (Regeneron), Sally Jones, PhD (Biogen), Dr. Stacey Gabriel (Broad Institute), Dr. Howard Jacob (Abbvie) which promises to be an engaging discussion.

It is not too late to register for conference attendance and be part of these critical discussions that have the potential to shape solutions to some of our most pressing healthcare challenges.

Best regards,
Tal Behar
Co-founder & President
PMWC

Interview with Andrew Carroll of Google AI

Q: Artificial intelligence (AI) techniques have sent vast waves across healthcare, even fueling an active discussion of whether AI doctors will eventually replace human physicians in the future. Do you believe that human physicians will be replaced by machines in the foreseeable future? What are your thoughts?

A: I believe that applying AI technologies in healthcare will make physicians more valuable, and will make their careers more enjoyable and sustainable.

Read More

Interview with L. Staton Noel III of Panaceutics

Q: What need is Panaceutics addressing?

A: One of the key barriers to making precision/personalized wellness and healthcare products is the contradictory goal of manufacturing individualized products on a large scale. Meeting demand for personalized products can be expensive and logistically challenging.

Read More

Interview with Joe Zhang of Burning Rock Dx

Q: Genomic medicine is entering more hospitals and bringing with it non-invasive technology that can be used to better target and treat diseases. What are some key milestones that contributed to this trend?

A: The milestones in my mind include the first FDA clearance of Next Gen Sequencing instrument, MiSeqDx in 2013, this opened a new door for clinical utilization of parallel gene alteration detection in clinical setting.

Read More

Why are Scientists So Upset About The First CRISPR Babies?

The use of new technologies can provide breakthrough benefits for both patients and providers. However, with increased sharing comes increased risks to the security and privacy of patient data. Currently data is being accumulated across many organizations and initiatives but is often either siloed or simply not accessible. Researchers suggest that patient education tactics can help quell security concerns during patient data sharing.

Read More

Interview with Kara Davis of Stanford

Q: The Nobel Price in Medicine was awarded recently to James Allison and Tasuku for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” What is your first-hand experience the impact that those new drugs had on patients?

A: These immune checkpoint inhibitors have been an incredible demonstration of the ability of the immune system to control and in some cases.

Read More

Interview with Shannon J. McCall of Duke University

Q: Genomic medicine is entering more hospitals and bringing with it non-invasive technology that can be used to better target and treat diseases. What are some key milestones that contributed to this trend?

A: After several years of the promise of precision medicine and abundant clinical trial work, the recent FDA approval of solid-tumor-agnostic therapies dependent on molecular biomarkers has catapulted genomic/precision medicine into the standard-of-care for late stage cancer.

Read More

Interview with Tao Chen of Paragon Genomics, Inc.

Q: Once sequencing has been validated as a clinical solution via trusted workflows, and coinciding with the technological developments driving costs lower, we can expect accelerated human genome profiling for clinical Dx. How soon, do you think, will we see accelerated growth and what can we expect?

A: For whole genome sequencing to be a reliable clinical tool, it will largely depend on the cost of sequencing the genome and our ability to interpret the data.

Read More

Call for Action: The Time is Now for Patient Data Interoperability

The use of new technologies can provide breakthrough benefits for both patients and providers. However, with increased sharing comes increased risks to the security and privacy of patient data. Currently data is being accumulated across many organizations and initiatives but is often either siloed or simply not accessible. Researchers suggest that patient education tactics can help quell security concerns during patient data sharing.

Read More

Interview with Andrew Magis of Arivale

Q: Once sequencing has been validated as a clinical solution via trusted workflows, and coinciding with the technological developments driving costs lower, we can expect accelerated human genome profiling. How soon, do you think, will we see what kind of accelerated growth?

A: I think the acceleration has already begun. Large sequencing projects such as NHLBI Trans-omics for Precision Medicine (TOPMed) and NIH All of Us are sequencing 150,000 and 1 million individuals, respectively.

Read More

Interview with Emily Leproust of Twist Bioscience

Q: NGS is enhancing patient care through improved diagnostic sensitivity and more precise therapeutic targeting. Prominent examples include cystic fibrosis and cancer. What other clinical areas NGS will most likely to change the standard-of-care in the near future?

A: Preventative medicine – using genetic data to identify traits that have the potential to cause harm in the future.

Read More

Interview with Michael Phelps of UCLA

Q: You invented the PET scanner that changed the lives of millions of patients with cancer, brain and heart diseases. What are the potential benefits to patients of combining PET with radio-ablation technologies?

A: PET provides imaging assays of the biology of disease in many diseases today.

Read More

Interview with Daniela Ushizima of Lawrence Berkeley National Lab

Q: Artificial intelligence (AI) techniques have sent vast waves across healthcare, even fueling an active discussion of whether AI doctors will eventually replace human physicians in the future. Do you believe that human physicians will be replaced by machines in the foreseeable future? What are your thoughts?

A: I really hope that human physicians will not be replaced by machines in the foreseeable future.

Read More

Interview with Amy Compton-Phillips of Providence St. Joseph Health

Q: Genomic medicine is entering more hospitals and bringing with it non-invasive technology that can be used to better target and treat diseases. What are some key milestones that contributed to this trend? What technological advancements are driving this change?

A: Genomic medicine is poised to move quickly from the research realm into integration with healthcare delivery, but there is always a time lapse between technology advances and what we do with those advances.

Read More

Interview with James Taylor of Precision NanoSystems

Q: There are various new, emerging technologies that bring us closer towards a cure for life-threatening disorders such as cancer, HIV, or Huntington’s disease. Prominent examples include the popular gene editing tool CRISPR or new and improved cell and gene therapies. By when can we expect these new technologies being part of routine clinical care?

A: Patients are already receiving treatment using novel gene and cell therapies.

Read More

Interview with Julie Eggington of Center for Genomic Interpretation

Q: Together with Robert Burton you founded the Center for Genomic Interpretation (CGI), a non-profit organization. Can you tell us more about CGI and the mission behind it?

A: CGI’s mission is to drive quality in clinical genetics and genomics. CGI works primarily with laboratories, health insurance payers, clinicians, and patients/consumers.

Read More
Johns Hopkins
University Of Michigan

The Precision Medicine World Conference (PMWC), in its 16th installment, will take place in the Santa Clara Convention Center (Silicon Valley) on January 20-23, 2019. The program will traverse innovative technologies, thriving initiatives, and clinical case studies that enable the translation of precision medicine into direct improvements in health care. Conference attendees will have an opportunity to learn first-hand about the latest developments and advancements in precision medicine and cutting-edge new strategies and solutions that are changing how patients are treated.

Agenda highlights:

  • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
    • AI & Data Science Showcase
    • Clinical & Research Tools Showcase
    • Clinical Dx Showcase
    • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
    • Digital Health/Health and Wellness
    • Digital Phenotyping
    • Diversity in Precision Medicine
    • Drug Development (PPPs)
    • Early Days of Life Sequencing
    • Emerging Technologies in PM
    • Emerging Therapeutic Showcase
    • FDA Efforts to Accelerate PM
    • Gene Editing
    • Genomic Profiling Showcase
    • Immunotherapy Sessions & Showcase
    • Implementation into Health Care Delivery
    • Large Scale Bio-data Resources to Support Drug Development (PPPs)
    • Microbial Profiling Showcase
    • Microbiome
    • Neoantigens
    • Next-Gen. Workforce of PM
    • Non-Clinical Services Showcase
    • Pharmacogenomics
    • Point-of Care Dx Platform
    • Precision Public Health
    • Rare Disease Diagnosis
    • Resilience
    • Robust Clinical Decision Support Tools
    • Wellness and Aging Showcase

Agenda highlights:

    • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
      • AI & Data Science Showcase
      • Clinical & Research Tools Showcase
      • Clinical Dx Showcase
      • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
      • Digital Health/Health and Wellness
      • Digital Phenotyping
      • Diversity in Precision Medicine
      • Drug Development (PPPs)
      • Early Days of Life Sequencing
      • Emerging Technologies in PM
      • Emerging Therapeutic Showcase
      • FDA Efforts to Accelerate PM
      • Gene Editing / CRISPR
      • Genomic Profiling Showcase
      • Immunotherapy Sessions & Showcase
      • Implementation into Health Care Delivery
      • Large Scale Bio-data Resources to Support Drug Development (PPPs)
      • Microbial Profiling Showcase
      • Microbiome
      • Neoantigens
      • Next-Gen. Workforce of PM
      • Non-Clinical Services Showcase
      • Pharmacogenomics
      • Point-of Care Dx Platform
      • Precision Public Health
      • Rare Disease Diagnosis
      • Resilience
      • Robust Clinical Decision Support Tools
      • Wellness and Aging Showcase
  • Luminary and Pioneer Awards, honoring individuals who contributed, and continue to contribute, to the field of Precision Medicine
  • 2000+ multidisciplinary attendees, from across the entire spectrum of healthcare, representing different types of companies, technologies, and medical centers with leadership roles in precision medicine
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