Workshop on market access for personalised medicine


Workshop on market access for personalised medicine

In the healthcare industry, this shift from a “one-size-fits-all” to a tailored approach is already impacting the development of new products. Today between 12 and 50% of current clinical pipelines involve personalised medicine, and between 2006 and 2010, the pharmaceutical industry investment in personalised medicine research increased by a mean of 75 per cent, with an additional increase of 53 per cent predicted by 2015 [1]. For patients, it means that, by developing better diagnostic tools which allow better targeting of individual characteristics, treatments could become more effective and safer.


From a public health management perspective, a better understanding of disease causality and the benefit-risk of a medical intervention allows the targeting of healthcare to patients who are most likely to derive benefits and /or least likely to suffer adverse events. This will not only improve patients’ outcomes and quality of life [2], but also increase healthcare efficiency through targeted value for money. With concrete case studies, the Third EuropaBio Workshop on Personalised Medicine brought together industry expects in Health Technology Assessment, economic strategy and pricing and reimbursement (P&R) to look into the market access challenges and opportunities for personalised medicine.


Four presentations gave an overview of the market access issues in personalised medicine:


1.    Recap from the first workshop
By Ludovic Lacaine, EuropaBio

2.    Market access challenges and opportunities for personalised medicine
By Andrea Rappagliosi, GSK

3.    Toward the Appropriate Valuation of Diagnostics: NICE Pilot Advice Project Case Study
By Vince Thomas, Roche Diagnostics

4.    Better access to molecular tests: the INCa/Pfizer partnership
By Dr. Allen Crook, Pfizer

5.   Implementation and availability of biomarker tests: KRAS Example
By Vanessa Stevens, Merck Serono


In addition, the workshop backgrounder can be downloaded here.


Following the presentations a discussion ensued concluding in particular that there is today no consensus among the industry on the best approaches to market access for personalised medicine. However it is clear that a flexible framework of legislation, regulation and guidelines is needed to overcome the hurdles identified – appropriate valuing of diagnostics, limited co-ordination of P&R methodologies and assessments by national authorities and narrow health technology assessment processes.


The increasing number of authorised products already approved with personalised components such as pharmacogenomics induces a variety of economic impacts on patients, payers and the industry. For payers in particular, personalised medicine offers the potential to target patient populations who will most benefit from a therapy while reducing unnecessary health interventions associated with side effects, thus allowing for an improved allocation of resources across the whole healthcare system. However, several grand challenges remain, which must be addressed trough a multi-stakeholder dialogue:

·       How do you increase investments in diagnostic test R&D and ensure rapid access, uptake and wide spread use of those tests?

·       How do you ensure diagnostic test quality by regulation and at the same time encourage innovation?

·       How to reward the value of the drug / diagnostic combination?

·       How to encourage cooperation and maintain competition between companies?


To realise this potential, a number of hurdles will also need to be overcome:


·       The use of diagnostic testing has steadily expanded, moving from simple diagnosis into screening, predisposition, prognosis, prevention, and therapy monitoring. However there is a vicious circle in the P&R of diagnostics which sees poor reimbursement leading to less funding for public or private research, little incentive to do properly designed clinical studies, lower level of evidence and few recommendations for clinical use, and therefore poor valuing of diagnostic. The UK’s National Institute for Clinical Excellence (NICE) has had some recent improvements to its evaluation pathway. In 2009, a programme was started which is expected to speed up the uptake of innovative diagnostics by improving the understanding about the nature of diagnostics and what is necessary to encourage this field.

The experience with NICE revealed that there is a need to improve the P&R schemes for diagnostics in general but also to allocate more funding to studies and clinical trials demonstrating the clinical utility of diagnostics. The value of diagnostics has not, so far, been appreciated. However this is beginning to change, in part due to the fact that pharmaceutical, biotech and diagnostic companies are increasingly collaborating in the development of companion diagnostics for medicinal products, thus breaking silos between disciplines.


·       Molecular testing is a key part of personalised medicine’s promise of better and more targeted healthcare improving patient outcomes. However, problems in obtaining samples for tests, regulatory issues and education of the value of these tests still hamper this goal. With national P&R regimes diverging from one EU Member States to another, uptake rates of molecular testing also vary greatly across and within countries, leading to uneven access to these technologies for patients.

As highlighted by the example of the public-private partnerships between the Institut National du Cancer (INCa) and Pfizer solutions exist allowing molecular testing for three specific oncology indications to become widely available across France. This partnerships – in response to the French Cancer Plan which states that there should be equal access to innovative and existing treatments, and that access to molecular testing should be expanded – was recognised as an innovative approach to increase access to diagnostic tools and it should act as a blue-print going forward.


·       Targeting the right patient population with the right drug using stratification tools such as diagnostic testing can provide significant clinical benefits, as illustrated in by the example of the biomarker and KRAS testing. However it is only as a result of a concentrated awareness campaign aimed at demonstrating the clinical utility of the KRAS testing that its adoption dramatically increased over the years. There is still a lot of room for improvement and particularly as regard to the awareness of physicians and P&R authorities on the value of testing. An holistic approach to healthcare funding and incentivising for diagnostic testing is needed. This needs to be kept in mind considering the current emphasis on cutting costs and budget constraints across a lot of EU Member States


The workshop concluded that it is crucial to raise awareness that personalised medicine should be considered in a holistic approach looking at policy and regulatory issues, market access problems and development/implementation of adequate incentives for innovators. Educating all stakeholders involved in P&R decision about this concept and particularly the value of diagnostics in personalised medicine will be essential to ensure an appropriate access to these technologies for patients.


[1] Tufts Center for the Study of Drug Development, “Personalized Medicine Is Playing a Growing Role in Development Pipelines,” Impact Report, 12 (November/December 2010): 6.
[2] D. L. Veenstra & M. K. Higashi; Assessing the Cost-Effectiveness of Pharmacogenomics. AAPS Pharmsci 2000; 2 (3) article 29

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