Moving the new Personalized Medicine to the Clinic

I am on the Advisory Board for a conference to be held 13-14 May in San Francisco on Personalized Medicine from the perspectives of regulators, biotech and pharma interest, health service funders and patient users of new and emerging technologies in this area.

Trusted doctor-patient relationships form a long recognized key underpinning basis for ensuring as effective as possible disease prevention and treatment. That relationship needs to be supported by a strong evidence base on clinical and cost-effectiveness and safety in use of medicines and supporting diagnostics and devices.

Thanks to economies arising from progress in gene technology (Moore's Law applied to medicine) and advances for exponential increase in active partners in this field (Metcalfe's Law applied to medicine), costs of genetic, genomic and other technologies to stratify diagnosis and treatment choice are becoming increasingly affordable in clinical practice.

The Summit is a one-day conference that will gather biotechnology and pharmaceutical experts and healthcare stakeholders as keynote speakers and panel discussants on legal, regulatory, funding and other key issues that will promote research and development, growth and effectiveness  in the short to medium term horizon for emergence of personalized medicine for clinical care.

The summit is co-hosted by the Personalized Medicine Coalition and Foley & Lardner LLP and is supported by major academic, clinical and industry patrons Life Technologies, Cancer Treatment Centers of America and the California Institute of Regenerative Medicine.

See the Personalized Medicine Summit website for more on the conference and how to register.

Selected papers from the conference will be published in the international journal Health Policy and Technology.

Progress on Personalized Medicine? Updates from Harvard.

@HealthMed The 8th annual Personalized Medicine Conference took place at Harvard this week - a joint venture of Harvard Medical School, Harvard Business School and Partners Healthcare, lead by Professor Raju Kucherlapati, from the HMS Department of Genetics. Worth checking the excellent archive of past programmes, presentations and podcasts.
Meantime, some of the highlights?
- An excellent narrative on the partnership between Plexxikon (Peter Hirth) and Roche Diagnostics (Suzanne Cheng) to create a companion diagnostic/therapeutic pairing for vemurafenib (Zelboraf), the first FDA approved pairing for BRAF V600E positive metastatic melanoma
- Further case studies illustrating successful drug development using genetic approaches
- Personal case studies on the impact, clinical value and ethical and clinical challenges of genomic screening: from Joe Beery, Life Technologies, on detecting unrecognised treatable serious early childhood disorders, to John Lauerman, Bloomberg News, on consequences of sequencing for asymptomatic adults - questions on penetrance and future screening for onset e.g.  of metabolic disease and cancers

Harvard Medical School: New Research Building - Avenue Pasteur.

- Clinical potential, and regulatory and reimbursement challenges to introducing molecular diagnostics into clinical care pathways
- Leadership award to Randy Scott, In Vitae, whose discussion points included the relevance of Moore's Law (technology advancing) and Metcalfe's Law (people factors: exponential increase in interaction as network expands) to developments in personalized medicine
- Business models and their governance for use of genetic information
- A North Virginia (John Vockley, Inova) pioneering series of projects aiming to assess outcomes of neonatal genomic sequencing: from insight into preterm labour to prospective longitudinal follow-up to adulthood, supported by multi-generation family member sequencing combined with clinical histories
- The US Air Force Programme on Patient-Centered Precision Care (Dr Cecili Sessions), in partnership with the Coriell Institute and Johns Hopkins University,  aims of which include understanding the impact on health-related behaviour of providing personal genetic information on remediable medical disorders and on drug responses.
- A business school case study led by Professor Richard Hamermesh, Director of the HBS HealthCare Initiative, on reactive and proactive responses for development of companion diagnostics (1).
- Pros and cons of liberal vs. restrictive approaches to IP for genetic and other molecular diagnostics
- Engaging the policy community and the public in ethical, clinical, reimbursement and adoption issues for new diagnostics and treatments aimed at personalizing medicine, including case studies from the American Medical Association and the American Assocation for Cancer Research.

Personalized Medicine Conference website 
Companion and coupled diagnostics

Companion diagnostics to personalise medicine

@HealthMed What common link is there between confusion, dementia, dyspepsia, heart failure, hypertension, liver failure and stroke (apart of course from excess alcohol in some)? They are all syndromes - clusters of symptoms and physical signs with many possible causes, risk factors and co-morbidities.
Earlier approaches to treating these health-related conditions were largely based on trial on error until the most effective treatment(s) were identified. While pragmatic, in many that may lead to a delay in achieving effective disease control, while exposing some patients to unnecessary risk of adverse drug reactions.
The relatively new term 'companion diagnostics' describes the concept that coupling careful selection of biomarkers of disease and risk factor phenotype, of therapeutic response and early warning of risk of adverse reactions, will provide a more rapid route to safe and effective drug selection and monitoring.
Major current challenges include the relative immaturity of research evidence on cost and clinical effectiveness of combining companion diagnostics with specific therapeutic strategies. A major potential driver to improve systematic assessment of companion diagnostics will come from the extension of the activities of NICE (the UK's National Institute for Health and Clinical Excellence) from pharmaceutical and other treatments into assessment of diagnostic technologies (Diagnostics Assessment Programme), including companion diagnostics. This will be complemented by the role of NICE in commissioning new research where significant gaps are identified. 
A further important challenge is the fragmentation of diagnostic services within clinical health services. There are practical reasons for having multiple local diagnostic capacity for serious acute illnesses for which very rapid access to sophisticated diagnostics is potentially life-saving or critical to minimise preventable complications, from early selection of effective treatment. Where there is the luxury of more than a few hours delay without significant risk to the patient, e.g. while using 'holding' empirical treatment, there needs to be more research on relative effectiveness of local compared with remote lab diagnostic strategies. There are also practical issues to be resolved among biotechnology companion diagnostic developers where joint licensing agreements are needed when multiple diagnostics are indicated from different source providers. And standardisation of testing is needed across national health service and private laboratories to ensure that results of these new diagnostic tests are validated.
And of course when there is a single test, implementation is more straightforward. However where multiple tests are needed within a companion diagnostics portfolio, there will need to be a regular programme of induction and refresher education for prescribers on how best to use and interpret results of this new testing strategy. This will be particularly important for interpreting the currently less familiar genotype based tests.