Pharmaceuticals & Biotech

Going all in on precision medicine

By Pavankumar Anne, Khushboo Garg, Anupurvi Jain, and Nid Ramesh

April 3, 2023 | White Paper | 8-minute read

Going all in on precision medicine

Precision medicine:

Solutions to deliver n=1 healthcare

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Precision medicine-based approaches for drug development and patient care have surged in the past decade, bolstered by evidence of superior patient outcomes, particularly within oncology. Recent studies have shown how use of precision medicine resulted in improvements in patient lifespan. In one study, targeted therapies extended the life expectancy of pancreatic cancer patients by an average of one year.


Traditional pharmaceutical manufacturers continue to invest in more targeted, biomarker-driven indications. Diagnostic and healthcare technology companies are investing resources to transform a surplus of patient data into more personalized, meaningful care that improves patients’ lives in a financially sustainable manner. As a significant proportion of the oncology pipeline continues to target specific biomarkers and investments continue to pour in, treating each cancer patient as “n=1,” or as a sample size of one, is within our reach and will eventually become the norm.


As noted in our new white paper, the oncology market is viewed as the greatest area of opportunity for precision medicine, given its size, disease heterogeneity and the number of approved biomarkers. However, precision medicine has the potential to greatly impact other disease areas, including immunology, neurodegenerative diseases, chronic illnesses and more.


Unfortunately, innovation is slowing due to persistent challenges in discovery and development. Pharmaceutical companies face rising costs in drug discovery and commercialization with a diminishing ability to increase revenue, reinforcing the attractiveness of investing in “all-comer” indications and treatments for diseases with higher prevalence.


More importantly, the current application of precision medicine isn’t always equitable. Certain populations, such as historically marginalized groups and people from lower socioeconomic backgrounds, are often left out of the latest research and therapeutic advancements. As a result, new precision medicine therapies and approaches may not adequately account for characteristics specific to these groups.


Despite these challenges, we think achieving widespread adoption of precision medicine is still within our reach. But first, it’s important to understand what is and isn’t working in this space, and how pharma can successfully deliver on the promises of precision medicine in collaboration with healthcare stakeholders.

Forming a complete picture of the patient with an n=1 mindset

The goal of precision medicine should be to integrate various data points captured from the patient to form one holistic and individualized picture. This picture can be used to develop and deliver proactive care, rather than relying on several disparate pieces of the puzzle to provide reactive care. Patient management should be contextualized through the patient’s entire clinical timeline, including physicians they’ve seen and treatments they’ve received. Every medically significant event and medical intervention should be factored into determining the most appropriate care. 


By doing so, the application of precision medicine can shift from developing treatments that are simply more effective in a subset of patients, based on certain characteristics, to a more personalized and dynamic approach to patient care. 


Achieving n=1 in precision medicine has the potential to maximize quality of life, increase life expectancy, improve disease prognosis and decrease mortality. Precision medicine can also help lower costs by eliminating inefficiencies in the healthcare system. Preventing suboptimal applications of medicine—such as patients receiving suboptimal treatments or medical interventions—can save patients from losing valuable time and improve overall cost-effectiveness within the healthcare system. Shifting the healthcare market towards this future is imperative and requires the close collaboration and coordination of major stakeholders, including pharmaceutical and diagnostic manufacturers, regulatory bodies and policymakers, payers and healthcare systems.

Defining the grand promise of precision medicine

When we think of our ideal world in which precision medicine is delivered optimally to patients, four defining characteristics come to mind.

  1. All biomarkers are known and identifiable
    Using the appropriate tools and techniques, all informative and actionable biomarkers and omics data that have the potential to impact clinical management are known and identifiable in patients.
  2. All biomarkers are actionable
    Healthcare professionals (HCPs) can prevent, diagnose, treat and predict outcomes related to clinical management based on every biomarker or piece of genetic information available in the patient data. These biomarkers may have varying utility but fit together to form one holistic picture of the patient.
  3. All patients are cared for at the right time
    The right patients get the right treatment or behavioral intervention with the appropriate specifications at the right time, with a shift from reactive care to more proactive patient management. 
  4. All stakeholders are incentivized appropriately 
    Misalignment of financial incentives translates to slower progress in patient care. In an ideal world, precision medicine applications are performed sustainably with a patient-centric approach, leading to continued advancement in discovery and delivery of precision medicine-based care. 

Industry actions required to achieve widespread precision medicine adoption

All major stakeholders have an important role to play in bringing us closer to the grand promise of precision medicine. Pharma cannot and should not do it alone. Stakeholder collaboration is necessary to not only address systemic challenges, but also to solve for scale. However, given the financial strength and interconnectedness with other stakeholders, pharma is well positioned to take charge and lead others in the right direction.


To achieve a more sustainable and efficient healthcare system that strives to deliver n=1 care, there are five strategies pharma should consider. And for more insight, make sure to read our new white paper

The future of medicine is n=1

The time to pursue the grand promise of precision medicine is now, and these strategies are just the tip of the iceberg. We believe that the n=1 mindset of care delivery will become a dominant part of most pharma portfolios in the next decade. Strategic planning and foresight are required to successfully execute the solutions we described but doing so will produce a more sustainable and profitable portfolio differentiator in the years to come. 

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