Q: You are the CEO of MolecularMD. Can you tell us what need MolecularMD is addressing with their specific offering in precision medicine?

A: 75% of targeted and immuno oncology investigational therapies in development depend upon diagnostics to support their clinical end points and demonstrate response. The intent of deploying a diagnostic in an oncology drug development is to reduce risk in development, and if not planned and implemented properly, a companion diagnostic can instead add significant risk to regulatory approval of novel oncology agents. MolecularMD is an organization that is entirely focused on supporting drug approvals with advanced diagnostics. Significant risks and gaps in the drug – diagnostic codevelopment process can occur among the commonly adopted partnership models. Risks include misalignment of clinical strategy, business incentives, and development timelines. MolecularMD’s organizational structure, quality systems, and experience is designed to help mitigate the risk profile of drug – diagnostic co development and ensure that all clinical, technical, regulatory, and commercial requirements for the drug development program are met in the face of uncertainty.

Q: The current drug development and commercialization process is highly volatile and competitive. What are some of the biggest issues of the current process?

A: Oncology Drug development programs are challenged with filling their patient cohorts with rare, low prevalence genomic alterations under highly compressed timelines. When this process is adopted by the traditional pharma organizational structures and regulatory processes, this places significant risk on timelines and cloud key decision points impacting eventual approval of therapy.

Q: What are some of the challenges when it comes to incorporating a strategy for companion diagnostics? How can we overcome these challenges?

A: Timing – Drug sponsors struggle with how and when to integrate their codevelopment activities which could potentially lead to a companion diagnostic. Each organization has their own set of experiences and assumptions that go into their decision making and strategic planning for companion diagnostics. The typical result is that programs defer decision making because of the uncertainty and lack of proper funding in translational phases. The key to over coming this challenge is taking a staged diagnostic strategy and setting up partnership that can adapt and execute around key decision point in clinical development and have a partner that can drive such an approach. The Drug sponsor need to actively manage the strategic and tactical objectives of the diagnostic, which can change drastically. The diagnostic can often become disconnected with major regulatory and clinical decision points and this can significantly increase risk downstream. A fit for purpose companion diagnostic strategy that is not solely dependent on one diagnostic path and can adjust quickly with change in therapeutic clinical and regulatory strategy is key to success.

Q: What challenges are there when it comes to defining the commercialization strategy for when marrying a drug with diagnostics?

A: Key questions when defining a commercialization strategy for a drug associated diagnostic:

  • What is the role of the diagnostic with respect to supporting the indication and administration of the therapy? The objective is to commercialize the drug, not the diagnostic, so how must the diagnostic be implemented in order to meet the objectives of the drug commercial strategy and market access?
  • What are the analytical performance and lab reporting requirements necessary to support HCP decision making?
  • What is the state of existing testing (LDT’s) with respect to availability, quality and cost?
  • Given small markets and challenging reimbursements often tied to molecular diagnostics, what is the viability of the business model around the diagnostic and how does the drug commercial plan help to ensure that the test economics support drug commercialization objectives.
  • The commercialization strategy can change all the way up to final indication and regulatory approval of the therapy. The indication for the drug may eventually not require a companion diagnostic at all. What is best approach to manage the Dx commercial plan and agreement given the nature of this uncertainty?
Q: How can we change the field of drug discovery and development to improve the communication and collaboration between diagnostics and pharma companies?

A: The collaboration between diagnostic and pharma companies is highly dependent upon the differences in economic incentives and psychology of the two organizations. The diagnostic leaders must understand how drug development teams are structured and how those teams are managed and incentivized. One of biggest challenges which is often faced is the lack of visibility that diagnostic companies have into the drug development planning and decision making processes. This is the consequence of the fragmented nature of biopharma organizational structures which can create issues with continuity. The establishment and management of a Joint Development Team is a key factor for success to ensuring a good, robust collaboration. All major functions including regulatory, development, IT, quality, commercialization, and clinical must be included in the JPT. Given the focus of the JPT on the development of the diagnostic, the diagnostic partner must take the lead and full accountability on the functioning and organizational culture of this joint team.

Interview with Gabriel Bien-Willner of Palmetto GBA

Q: What does your role entail as the director of the MolDX program at Palmetto GBA?

A: The job directing MolDX is multifaceted; first and foremost the MolDX program is responsible for assessing molecular diagnostic tests on the market and makes coverage and pricing determinations for such tests and technology. This is usually done through local coverage determination policies or technical assessments.

Read More

Interview with Peter Marks of FDA

Q: The CBER’s Regenerative Medicine Advanced Therapy Designation program has been very successful, with about 100 requests for designation in the two years of its existence. Can you please tell us about the program and how it was put together?

A: The Regenerative Medicine Advanced Therapy (RMAT) Designation program came into being as part of the 21st Century Cures Act that was signed into law on December 13, 2016.

Read More

Interview with Calum MacRae of Harvard Medical School

Q: What patient data do we need to better understand the underlying cause of disease and how to prevent it?

A: Medicine at present is highly underdetermined and data poor. To be precise, one must be comprehensive, so medicine (with our consent) will use not only what we currently conceive of as biomedical information, but also data from across our lives.

Read More

Headlines from PMWC 2019 Silicon Valley

A big ‘Thank You’ to all of our presenters and attendees for celebrating 10 years of precision medicine progress with us! PMWC 2019 Silicon Valley was attended by 2000 participants from 35 countries, which included over 400 speakers in 5 parallel tracks!

Read More

Interview with Ken Bloom of Ambry Genetics

Q: Tell us more about your organization/company. What patient population are you serving and which services are you specializing in?

A: Ambry Genetics is a recognized leader in high quality complex genetic testing. We seek to find the genomic cause or contributors to rare diseases, abnormal phenotypes and hereditary disorders.

Read More

Interview with Lee Pierce of Sirius Computer Solutions

Q: What is the state of big data and analytics in healthcare, and how to best use the reams of data available?

A: More than ever, Healthcare organizations are achieving measurable value through use of their data and analytics assets. There is more raw material available than ever to create value. This raw material is the data flowing from internal systems and applications and also from devices and systems external to healthcare organizations.

Read More

Interview with Anita Nelsen of PAREXEL

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: Today’s emerging technologies are making the promise of individualized treatment a reality.

Read More

Interview with Ilan Kirsch of Adaptive Biotechnologies

Q: The Nobel Prize in Medicine was awarded recently to James Allison and Tasuku Honjo 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: For decades cancer was viewed as solely a cell-autonomous condition.

Read More

BMS buys Celgene | Lilly buys Loxo Oncology – Does this Signal a Return to Strong Deal-Making Activities in 2019?

Bristol-Myers Squibb’s blockbuster $74B deal to buy Celgene creates an oncology powerhouse amid industrywide excitement about the rapidly evolving science and explosive growth of the sector. The agreement could signal a return to deal-making for the pharmaceutical industry in the $133B global oncology therapeutics market.

Read More

Interview with Gini Deshpande of NuMedii

Q: What need is NuMedii addressing?

A: NuMedii, has been pioneering the use of Big Data, artificial intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. Artificial Intelligence approaches are a natural fit to harness Big Data as they provide a framework to ‘train’ computers to recognize patterns and sift through vast amounts of new and existing genomic

Read More

Interview with Minnie Sarwal of UCSF

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: Completion of complete sequence data from the human genome project, and the advances in proteomic, microRNA and epigenetic assays added a layer of pathway biology to the understanding of human diseases.

Read More

Interview with Shidong Jia of Predicine

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: We will see accelerated human genome profiling for clinical Dx in 2019 and the coming years as more biomarker-based cancer drugs are gaining approval.

Read More

Interview with Iya Khalil of GNS Healthcare

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 think that there’s a lot of speculation and uncertainty around AI, but I don’t foresee a time when we won’t need physicians.

Read More

Interview with Ilya Michael Rachman of Immix Biopharma Inc.

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.” Besides CAR T-cell therapy what do you think next generation immunotherapies will look like to successfully combat cancer?

A: The next generation of immunotherapies will build on the insights discovered by immunologists like James Allison and Tasuku Honjo and extend them to modify the body’s response to tumors.

Read More

Join me to Kick off PMWC Silicon Valley in the Santa Clara Convention Center, Focusing on Every Element of Precision Medicine

My team worked in collaboration with Bill Dalton, Kim Blackwell, Atul Butte / India Hook Barnard, Nancy Davidson and Sharon Terry to create a program that touches every component of precision medicine while bringing together all of its key stakeholders. Leading participating institutions including Stanford Health Care, UCSF, Duke Health, Duke University, John Hopkins University, University of Michigan and more will share their learnings and experiences and their successes and challenges, as they make precision medicine the new standard of care for all.

Read More
Johns Hopkins
University Of Michigan

The Precision Medicine World Conference (PMWC), in its 17th installment, will take place in the Santa Clara Convention Center (Silicon Valley) on January 21-24, 2020. 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.

See 2019 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

See 2019 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
Get Updates
Sign up for occasional updates on upcoming conferences, news, and other information.
We respect your privacy and will never share your email with anyone.
Something went wrong, please verify your input.
Thank you for signing up!

Get Exclusive Access to the Top PMWC Talks

To receive the most comprehensive news and updates from the field of precision medicine, subscribe to the newsletter here.

Bonus for suscribing, you will get the access code for the top 3 talk videos from January's Precision Medicine World Conference.

You have successfully subscribed, you can access the talks here.