Are We Entering an Age of Expensive Personalised Medicines?

Since the approval of Pfizer's lung cancer drug Xalkori by the United States Food and Drug Administration (FDA), headlines have been calling into question the future of personalised medicine – and the cost it will bring with it.

Pfizer's own figures suggest the drug will only be of benefit to between 6,500 and 11,000 patients in the United States who are suffering from AKL positive metastatic non-small cell lung cancer, and the small size of this population leads to higher costs.

In clinical trials, the average duration of treatment needed was 22 weeks, at a cost of $9,600 (£6,100) a month – figures which have got some concerned despite the benefits the therapy will bring.

Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizer's Oncology Business Unit, said the approval of the drug represents "a new chapter in personalised therapy for lung cancer".

Of course, Pfizer is by no means the only company targeting personalised medicine as an area for future growth as the pharmaceutical entry leaves its blockbuster phase into a new period of growth spurred by biopharmaceuticals and more targeted treatments.

Roche gained approval this summer for the drug Zelboraf and a companion diagnostic for BRAF mutation-positive metastatic melanoma and drug applications have been submitted to the European Union, Switzerland, Australia, New Zealand, Brazil, India, Mexico and Canada.

Paul Brown, head of Roche Molecular Systems, said: "The cobas BRAF Mutation Test has improved sensitivity, accuracy and speed compared to other commonly used, unapproved detection methods.

"With a personalised medicine now available, all people diagnosed with inoperable or metastatic melanoma should be tested to help determine the best options for treatment."

Signs also suggest an even greater number of companies will be making similar announcements in the future. Research conducted by Tufts Centre for the Study of Drug Development late last year found 94 percent of companies are investing in the area and 100 percent are using approaches like biomarkers to analyse compounds.

Wes Metheny, Pharmaceutical Research and Manufacturers of America (PhRMA) senior vice president, said: "Unsurprisingly, cancer leads the field of personalised medicine research, both in terms of already-marketed technologies and the pipeline.
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He added: "Personalised medicine research is now expanding into metabolic and respiratory therapies as well as virology. What's more, much of this work is used solely to improve a company's understanding of their research, rather than for regulatory approval."

Even if much of the research is not for regulatory approval, authorities are preparing for their future responsibilities in areas of cell therapy, genomics and personalised medicine. The FDA recently launched its Strategic Plan for Regulatory Science, calling for a modernisation in the science used by regulatory agencies.

Commenting on the launch, chief scientist Jesse Goodman said: "This plan will help turn advances in science into products that benefit people, and help FDA assure that happens more quickly and safely. It is win-win-win, because it will help the public, the agency, and developers of new products."

In the UK, funds have been granted by the Technology Strategy Board for research and development in the area of tumour profiling and data capture, with the aim of making the country a leader in the development of personalised medicine and find new, innovative cancer therapies. 

But on both sides of the Atlantic, there are still barriers in place hindering the development of personalised medicines.

Further research from Tufts found that, despite the enthusiasm that exists around the therapies, players are still sceptical about the clinical usefulness of diagnostic tests, which is slowing development in the area.

Joshua Cohen, PhD, senior research fellow at Tufts CSDD and author of the study, explained: "Scientifically, the process of biomarker discovery and validation in general, and parallel development of drugs and companion diagnostics in particular, has been slow.

"Additionally, regulatory and reimbursement issues have limited uptake in clinical practice, particularly with respect to companion diagnostics, but also for drugs lacking effective diagnostics."

With the investment being made in the area, and the success of companies like Pfizer and Roche being so well publicised, chances are that these barriers will not put the brakes on personalised medicine in the long term.

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