The personalized medicine marathon

The full version of this personalised medicine feature can be read in the April issue of our digital magazine: http://www.pharmtech.com/ptedigital0410

Personalised medicine — often defined as the right treatment for the right person at the right time — is slowly treading closer to reality and has been hailed as a potential way to avoid unnecessary treatments, reduce the number of adverse drug reactions, increase treatment efficacy and, ultimately, improve health outcomes. In the US, the market for personalised medicine is already estimated at more than $230 billion, according to a report by PricewaterhouseCoopers (PwC);¹ however, this figure is forecast to rise by 11% annually. By 2015, it will have almost doubled in size to reach more than $450 billion.

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"When the Wright Brothers took flight in 1903, few could imagine that in the space of less than 70 years a man would set foot on the moon," says the PwC report. "Similarly, we believe the 21st century will give rise to advances in genomic and proteomic science that cannot yet be envisioned, and will yield results on a similarly grand scale."

The potential promise of personalised healthcare has been fuelled by recent advances in genomics and proteomics, which have improved our understanding of the variances between individuals' genetic profiles. This knowledge offers the opportunity to identify which individuals are susceptible to which health conditions, as well as the ability to match the right drugs to patients based on their genetic make up. According to the US Institute for Systems Biology, personalised medicine has four attributes:

• Personalised — genetic variations drive treatment.

• Predictive — conditions that a person might contract in the future can be identified early on.

• Preventative — a shift in focus from illness to wellness.

• Participatory — patients can make informed choices and take responsibility for their own health.

According to Kalorama Information's report,² Pharmacodiagnostics, the path to personalised medicine lies in pharmacodiagnostics — the pairing of a diagnostic test to a therapeutic. The report claims there are two new applications for this. "The first is to narrow the field of application for drugs in development. That is, not only will genomic and proteomic testing be used to identify targets, but also to identify subpopulations of individuals for selection in clinical trials. This was something that was unthinkable to pharmaceutical companies not long ago."

The second application is to select a therapeutic for a patient that will have the greatest efficacy and a lower probability of harmful side effects. This type of personalised treatment is already a reality for certain conditions, such as cancer. The diagnostic classification of cancer has historically been based on the organ or tissue location where it originated in the body. Now, however, it is possible to classify some cancers by the genes that are expressed, the tumour's cell surface proteins and other molecular attributes. Trastuzumab (Herceptin; Genentech), for instance, is only prescribed for patients whose genetic tests reveal an over-expression of the protein HER2 due to a gene mutation.

Barriers to adoption

Unfortunately, there are also numerous barriers hindering pharmacodiagnostics, including a healthcare financial system that is dependent on visible disease symptoms and gross clinical classifications. "Simultaneously, the ability to classify diseases into distinct molecular subcategories challenges traditional pharmaceutical business models that focus on "one-size-fits-all" drugs," says the Kalorama report.

The need for a change in traditional business models is also echoed by the PwC report, The new science of personalised medicine, which describes personalised medicine as a "disruptive innovation" because it will require new approaches, new relationships and new ways of thinking.

The PwC report estimates the core diagnostic and therapeutic segment of the personalised medicine market (comprising mainly pharmaceutical, medical device and diagnostic companies) to be worth approximately $24 billion in the US and expects it to grow by 10% annually to reach $42 billion by 2015. However, the biggest opportunities in personalised medicine lie outside of the traditional healthcare sector. The personalised medical care portion of the US market (including telemedicine, health information technology and disease management services) is estimated to rise from its current value of $4–12 billion to more than $100 billion by 2015. Meanwhile, the nutrition and wellness market (comprising retail, complementary and alternative medicines offered by consumer products, food and beverage, leisure and retail companies) will rise from $196 billion to more than $290 billion by 2015.

"As the boundaries between traditional healthcare offerings and wellness products and services blur, and as the trend towards consumer-focused healthcare accelerates, companies outside the health industry are finding new opportunities," says the report, which means that traditional pharma and biotech companies will need to be wary of new competitors entering the market. The report adds: "Companies from outside the traditional healthcare arena, from PepsiCo to Procter & Gamble, could be formidable competitors, due to their skills and experience in targeting consumers, who are contributing to the rapid growth of the wellness market."

In particular, the PwC report stresses that collaboration will be key in this new business model. "Unless regulatory and reimbursement models are aligned with the requirements of personalised medicine, the new science is not likely to advance far," says the report. "Pharmaceutical and diagnostic companies, payers and regulators can work together to create an economically viable, sustainable process for commercialising targeted diagnostics and therapeutics."

In light of the future expectations of personalised medicine, Pharmaceutical Technology Europe spoke with numerous experts to find out more about some of the latest developments and trends driving the sector.

PTE Digital extracts

Progress in molecular diagnostics

Molecular diagnostics is one of the fastest growing segments of the in vitro diagnostics market and, within this, personalised medicine is at the forefront. In recent years, we have witnessed a significant demand both from pharmaceutical companies for partnerships and also from laboratories for products, both of which have been driven by the commercial and clinical benefits of adopting a personalised medicine approach. In particular, cancer is leading the field with all new cancer drugs in development now having an associated biomarker programme.

Read in full at www.pharmtech.com/ptedigital0410

Stephen Little

DxS, a QIAGEN company

Personalised cancer therapy

T cell therapy is being assessed for the treatment of cancer, which could be applicable to a whole range of cancers. It is, however, a patient specific treatment, which requires production of a large number of cells to a GMP standard in a short period of time for an individual patient — this poses a number of challenges. Even though the cost is high, the benefits appear large (currently for melanoma patients at least) and the key advantage is that this is a one-off treatment that has long-lasting benefits.

Read in full at www.pharmtech.com/ptedigital0410

Robert Hawkins

Christie Hospital, UK

Glucocorticoid therapy

Numerous drugs have been developed that target the glucocorticoid receptor (GR); however, recent research shows that genetic factors will shape the future of personalised glucocorticoid therapy. Several SNPs corresponding with functional domains of the human GR protein and mRNA have been identified, and follow-up studies have shown these are associated with the GR's sensitivity to cortisol. Therefore, mutations in the human GR gene may cause glucocorticoid resistance.

Read in full at www.pharmtech.com/ptedigital0410

Erno Vreugdenhil

University Leiden, The Netherlands

References

1. The new science of personalized medicine (PricewaterhouseCoopers, October 2009).

2. Pharmacodiagnostics (Kalorama Information, New York, USA, April 2009).