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Towards a New Era in Healthcare: Realizing the Promise of Precision Medicine

Precision medicine promises to deliver the most significant changes on the healthcare horizon: improving diagnosis, treatment and patient prognosis. The arrival of precision medicine is imminent: what was once considered a “blip on the horizon” is now just around the corner, and we have an obligation to ensure its successful and appropriate adoption.

Precision medicine will be the driver for deep structural change in healthcare delivery. As well as improved outcomes for patients, it also has the potential to bring significant efficiency savings, transforming health and saving lives and money. For these reasons, it is the chosen focus of the Global Agenda Council on Healthcare. This paper outlines the background of the Council’s work and how it has arrived at precision medicine as a new direction. It also details the Council’s recommendations for action: six priority areas that need support from all key stakeholders if precision medicine is going to succeed and flourish.

Priority Setting: The Council’s First Year

When the Council was first convened in the summer of 2011, its mandate was undecided. The Council previously had worked on issues of health system sustainability, which resulted in the publication of “The Five Bad Habits of Healthcare”1 and a Forum project entitled Scenarios for Sustainability of Health Systems.2 The Council debated its direction for the 2011-2012 term, as the health system work was continuing in a more formalized process. The Council discussed whether “personalized medicine” might be the next relevant topic for its work, as the area has held great, yet still nascent, promise for years. Following careful consideration, it was decided that the Council’s efforts would best be served exploring precision medicine.

From “Personalized Medicine” to “Precision Medicine” Case Study

Ms. H is a 35-year-old woman from Japan who has had a cough for three weeks. Her physician sends her for imaging studies that reveal an advanced tumour, which a biopsy confirms to be a non-small-cell lung cancer. She has never smoked. Can anything be done for her?

Had Ms. H.’s cancer been diagnosed before 2004, her oncologist might have offered her a treatment to which about 10% of patients have a response, with the remainder gaining a negligible survival benefit and experiencing clinically significant side effects. But her diagnosis was made in 2011, when her biopsy tissue could be analysed for a panel of genetic variants that can reliably predict whether the disease will respond to treatment. Her tumour was shown to be responsive to a specific targeted therapeutic, whose administration led to a remission lasting almost a year; her only side effect was a rash.3

As the above scenario demonstrates, precision medicine uses molecular analysis to map a person’s genomic profile that can then lead to a tailored treatment designed to offer the best therapeutic outcome with minimum side effects.4 Recent biotechnological advances have led to an explosion of disease-relevant molecular information that has brought us to within touching distance of achieving the goal of personalized medicine. This approach is expected to deliver significant healthcare benefits at each step of the patient care continuum.

The Council expects that the shift towards personalized medicine will bring with it a new set of challenges. Ensuring a smooth transition will depend on establishing frameworks for regulating, compiling and interpreting the influx of information that can keep pace with rapid scientific developments. Collaboration on a scale not previously seen will be necessary between all major healthcare stakeholders (regulators, clinicians, government, industry, and academic institutions) to address these challenges. In addition, personalized medicine will have profound effects on the dynamic of the patient-health provider axis, which is going through unprecedented change. The successful implementation of personalized medicine will therefore hinge to a great extent on whether and how the clinicians and wider public will adapt to the changes we foresee.

This approach has traditionally been known as “personalized medicine” – making sure the right patient receives the right treatment at the right time. However, the Council concluded that this principle in itself lacks sharpness, and does little to distinguish personalized medicine approaches from well-practiced medicine more generally. The Council explored the definition of the term “personalized medicine”. The Council discussed the assumption that personalized approaches would facilitate more precise healthcare delivery based on individual-specific molecular data – hence, the Council agreed on the preferred term “precision medicine”.

Figure 1 Expected benefits of personalized medicine approaches at different stages of the clinical care pathway

Strategies for Addressing Collaboration Gaps

The Council has identified six fundamental areas for development, which collaborative efforts must urgently address at the national and international level to ensure the success of precision medicine.

  1. More incentives for innovation. The challenges, costs and risks associated with modern medicinal product development are well established. Together with lengthy approval processes, they represent significant impediments to innovation. Conventional industry business models are designed to yield maximum return on investment, and biopharmaceutical partners may be reluctant to invest in new, truly innovative products and diagnostics without having a clear view of how the market is going to reward them, particularly in times of financial crisis and widespread cost-cutting. The Council recommends developing appropriate financial incentives and reimbursement schemes to stimulate innovation. Tax credits and R&D grant schemes as well as a clear commitment to reimburse an innovation premium if precision medicines provide additional value will help foster exciting scientific and technological advances and will also encourage small/non-profit organizations, which would otherwise struggle under the weight of financial costs.
  2. New disease classification systems that incorporate emerging molecular data. A deeper understanding of the molecular mechanisms that underpin health and disease calls for new models of disease classification that include this emerging knowledge. Until now, attempts to incorporate molecular data into disease taxonomy have tended to be subspecialty centred and biologically disconnected, with little integration of data from different biological domains (genome/proteome/transcriptome/metabolome). The Council recommends that a new disease classification system must be developed as a key component of Precision Medicine. This should incorporate all accurate and disease-relevant molecular information as and when it is confirmed and validated. To be truly effective this should be presented in an open access manner, and be introduced into the learning curriculum of future generations of health practitioners.
  3. More streamlined clinical trial regulation frameworks. Recently published figures show that the time taken to gain approval for clinical trials and the financial costs incurred are increasing. Importantly, this increase in regulatory stringency has not been shown to lead to any improvements in patient safety. In the wake of these reports, there have been growing calls for more efficient approval processes. The Council recommends that making these procedures more streamlined will be critical to the success of precision medicine. Approval procedures could be made more efficient by taking into account previous experience with the agent being investigated and/or the study population.
  4. Effective data interpretation and clinical decision support (CDS). Precision medicine’s success will require handling of multi-parametric data and basic proficiency with “–omics” data interpretation and bioinformatics platforms. Current generations of health practitioners will find this additional data burden challenging, and will benefit from computer-based CDS tools, which will provide options regarding test ordering and will aid clinical workflow by providing decision-making algorithms to guide decisions on diagnosis, prognosis and required treatment based on test results. The Council anticipates that the development of appropriate health IT systems that incorporate CDS tools presents a significant logistic and financial challenge and warrants urgent attention.
  5. Stimulation of consumer interest and active participation. There will be significant public uncertainty on legal, ethical and privacy issues concerning open-access data, and these will require careful regulation as well as attention to the security architecture of future health IT systems. Aside from these concerns, it is important that we demonstrate to the wider public that precision medicine can live up to the promise. The Council recommends that safeguarding public interests will require careful regulation and that successful public engagement will require a cultural shift, with patients viewed as participating members of the precision medicine community rather than study subjects.
  6. A rational approach to Health Authority regulatory oversight of precision medicine. The global regulatory oversight of precision medicine, which includes both the therapeutic and associated companion diagnostic, must be one that provides the appropriate mechanism to ensure safety and efficacy (effectiveness) as well as timely premarket review and approval. Given the advancements in understanding the complexity and pathways associated with certain diseases, specifically cancer, the regulatory review process must be receptive to facilitating adoption of target therapeutics that employ innovative and complex diagnostic technologies in the assessment of disease and the clinical state of the patient. This includes such technology as high throughput next generation sequencing. In addition, a less burdensome regulatory path must be provided for rare disease, where there is a significant unmet need and rarity of samples for analysis.

Taking into account the challenges, the Chair of the Council, Professor Lord Ara Darzi, presented a series of recommendations at the Global Agenda Council on Healthcare meeting in Abu Dhabi in October 2011. At the meeting, the Council introduced and subsequently developed key principles, which are outlined in the strategies above, to facilitate the future adoption of precision medicine.

Council’s Next Steps

The Council is committed to highlighting and addressing the challenges and opportunities that are detailed in this report. A successful transition to precision healthcare will depend on the collaboration of all key stakeholders.5

The World Economic Forum remains the main stage for such cross-sector dialogues.

Precision medicine will continue to dominate the Council’s agenda during the coming year. A more detailed, proposed strategy for addressing the challenges outlined above will form the focus of a major collaborative publication from the Global Agenda Council on Healthcare on realizing the promise of precision medicine. This collaborative work is under development and expected to be published prior to the next meeting of the Global Agenda Council on Personalized and Precision Medicine in Abu Dhabi in November 2012.


The opinions expressed here are those of the individual Members of the Council and not of the World Economic Forum or any institutions to which they are affiliated.