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European Pharmaceutical Contractor

The Personal Touch

David S Lester of ITHW looks at the impact that personalised medicine has had on the healthcare industry

From a patient’s perspective, management of patient pathology is carried out using the following process: diagnosis, intervention, adherence, compliance and control. Diagnosis is comprised of generally more than one diagnostic test, using technologies such as ‘wet’ assays, genetic tests, imaging and, most importantly, personal reporting (scoring paradigms). Intervention can be therapeutic, but can also come from a device or lifestyle changes. Adherence may be the biggest challenge when considering therapeutics. The patient needs to follow the instructions from the provider and pharmaceutical manufacturer relating to appropriate administration of the drug. Control refers to how effective the drug is, or if there are adverse events. Changes in the treatment of some pathophysiology can be adjusted in each of these four stages, but the patient care cycle relies on all players (provider, patient, pharma and payer) partnering in order to ensure optimal treatment and management of the specific patient’s illness. The present practice of medicine is moving towards this paradigm, largely due to the rapid introduction of new technologies. Personalised medicine (PM) is one of the approaches that is helping to move the practice of medicine in this direction.


The US Congress defines PM as: “the application of genomic and molecular data to better target the delivery of health care, facilitate the discovery and clinical testing of new products, and help determine a person’s predisposition to a particular disease or condition” (1). 

Over the last 50 years, the practice of medicine has been based on epidemiological evidence. This evidence-based medicine approach has focused on population analyses. However, with the increasing use of targeted drugs, it has been found that the use of population analysis has its limitations. In parallel, the last 20 years have seen the explosion of genetics-related technologies, which have provided new insights into the variability of response of individuals (or better-defined populations, such as subpopulations) to specific therapeutic interventions.

The potential for implementation of PM is due to technological advances in areas which include instrumentation and assay development for genotyping and gene expression, HealthIT data management, application of systems biology and pathway analysis, enhanced understanding of receptor-related disease pathologies due to biomedical research in academic institutions and pharmaceutical organisations. As is the established norm for healthcare technologies, the transition from discovery, to commercialisation to adoption is slow when compared to other industries where technologies are the drivers, such as the software industry. In healthcare, it is generally accepted that this transition will take years, compared to months in the software/IT world. This long transition is usually attributed to regulatory issues; however, other factors have even more influence – these include adoption by payers, providers and patients.

As PM programmes progress, it is important to recognise that the patient is becoming more knowledgeable due to the increased availability of knowledge from medical websites (such as and, medicallyrelated social networking (such as and and personal health records (such as, and This means that the patient will take an increasing role in the adoption and adherence of new and existing therapeutic choices. Other influences that should not be neglected in the US healthcare space are private organisations such as the Pharmacy Benefit Manager, MedCo with their ‘Genetics for Generics’ (2) and McKesson with their innovation programmes in clinical trials and molecular diagnostics. It is clear that companies such as these are now positioning themselves to not only embrace these new medical practices, but also influence their direction and implementation.


There are few examples of PM programmes. The best known example is in cancer treatment and relates to Herceptin and breast cancer. Another established case is in infectious diseases: Maraviroc and HIV infection. In all cases to date, the companion test has generally been developed after the approval and release of the drug. There has been discussion that some drugs, such as Iressa, could be ‘rescued’ by the addition of an appropriate companion diagnostic test. The reality is that a new development approach needs to be pioneered for the growth of PM products.

It is the desired strategy that the development of a programme for PM would involve a partnership between a diagnostic company and a pharmaceutical company. This is proposed as the development and regulatory process are significantly different for the two companion products. The diagnostic test needs to be developed and approved in parallel with development and approval of the therapeutic. It is reasonable to consider that the therapeutic clinical trials programmes could serve as the source for the necessary clinical sample and data generation for achieving regulatory approval of the diagnostic test. Technical development of the diagnostic test has to go through various stages. It would be expected that the biomarker(s) for the test would possibly be based on academic research, along with preclinical and/or early clinical development data collected by the pharma or biotech company. For multianalyte tests, a number of associated markers could be identified which would be refined and validated during the clinical trial validation stage. The instrument platform should be technically validated using relevant clinical samples before use in the Phase II study.

The performance of this platform in terms of intra- and inter-patient variability, instrument variability, and so on can all be done before use in Phase II or Phase III clinical trials so as to develop the working prototype from a technical perspective of the instrument and assay that would be used for the diagnostic test for regulatory approval and commercialisation. Phase II, particularly IIb, would be an excellent time for development of the diagnostic as this is when efficacy outcomes can be correlated with the therapeutic results. The data from the diagnostic test can be used as the basis for such factors as patient stratification or efficacy in the Phase III programme that must undergo regulatory approval. In terms of FDA regulatory approval, there still is no process, but there are efforts underway to establish a guidance and process that would bring together the efforts of the Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH). While the US regulatory environment is still unclear, the increased efforts to produce such companion products will help to drive a clear path forward.

On the other hand, this may be an opportunity to change strategies for product launches. For example, DxS (now part of Qiagen) obtained EMEA approval of their K-ras test before submission to the US regulators. Perhaps the non-US market will take the lead in terms of the regulations for approval of PM companion products. However, one thing is clear: the approval of such companion products does not have a clear regulatory pathway in any of the regulatory agencies.


Beyond regulatory approval of a diagnostic or a therapeutic lies the next challenge: that of adoption. The challenges and approaches that are used to achieve adoption of these two distinct classes of products are very different. For the diagnostic, the challenge is to change the standard of practice of medicine. Genomic Health has done a superb job with the acceptance of their breast cancer molecular diagnostic test, Oncotype Dx. The unparalleled success of this test is due to their understanding the standards of practice of breast cancer management followed by subsequent development of a product that is not disruptive to the practice of managing this disease, but rather integrates with this and demonstrates immediate value. In addition, the test relies on a sample being sent to their own Clinical Laboratory Improvement Amendments (CLIA) approved laboratory, ensuring that all sample results will be comparable ( However, as yet, the test is not FDA approved. It would be expected that any companion diagnostic for a therapeutic will have to be approved by some regulatory agency. In terms of the therapeutic, adoption is accompanied by continued Phase IV clinical trials, demonstrating the value of the pharmaceutical and its superiority to other compounds on the market targeting the same stage of the disease.

The PM combination product will most likely demonstrate superiority over compounds in the same class of the disease being treated. However, the PM product may well be focusing on a specific subpopulation, so the overall fear of the commercial team is that PM will cause a reduction in the target market leading to loss of potential revenue. This cannot be overcome by adjusting pricing of the compound as there is a strong push to prevent increased healthcare costs. On the other hand, the present drive in the US for Comparative Effectiveness may provide new opportunities for PM. In this case, the diagnostic may be the factor that demonstrates the superiority of the therapeutic as it could define the responder population. This raises an interesting question in terms of value of the diagnostic. If the diagnostic is a one-time only product that costs, for example, $4,000, and it results in the therapeutic achieving ‘blockbuster’ (over $1 billion) status, how do we determine the remuneration for the diagnostic?

It is also proposed that pharmaceutical companies should be responsible for commercialisation of PM products – pharma has a very extensive sales force that, along with the regional experts and representatives, would simply need to be trained in the use of the diagnostic. This may provide the sales representatives with an additional opportunity for physician contact when promoting their products to the providers. In terms of the payers, there is no doubt that, with the high cost of new therapeutics, particularly biologics, because of the challenges of complex clinical programmes and high cost of manufacturing, having a diagnostic will provide greater confidence to pay for these costly new biological and chemical entities as better efficacy for well-defined populations is ensured.

These arguments are based on what one would consider to be common sense; although this does not mean that they will necessarily be implemented. The healthcare industry acts in a very conservative manner; it is widely said that new technologies take around a decade to become part of clinical practice. In addition, it is an unusual industry as the customer (payer) and the consumer (patient) are different. But the roles of stakeholders are changing, and the patient is becoming more informed and more challenging to their provider. In addition, we are seeing previously ‘quiet’ companies such as MedCo beginning to flex their muscles and exert their influence directly through the patients, hospitals, clinics and providers.

While PM may not be the panacea that was originally proposed when it was first introduced, there is no doubt that it is taking healthcare in the right direction.


  1. S.976 Title: Genomics and Personalized Medicine Act of 2007, A bill to secure the promise of personalized medicine for all Americans by expanding and accelerating genomics research and initiatives to improve the accuracy of disease diagnosis, increase the safety of drugs, and identify novel treatments, 110th Congress 2007-2008, US
  2. The Pink Sheet, October 27, 2009

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David S Lester has held numerous positions in the US Federal Government (NIH and FDA), academia, pharma (Pfizer and Pharmacia) and diagnostic (Gene Express and Theranos). He is presently providing consulting services based on this experience. David's focus in the last few years has been on personalised medicine; a topic on which he regularly speaks and contributes to publications as a thought-leader in this arena.
David S Lester
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