Annexes to SWD(2013)436 - Use of '-omics' technologies in the development of personalised medicine - Main contents
Please note
This page contains a limited version of this dossier in the EU Monitor.
| dossier | SWD(2013)436 - Use of '-omics' technologies in the development of personalised medicine. |
|---|---|
| document | SWD(2013)436 |
| date | October 25, 2013 |
The development of pharmaceuticals is a time-consuming process with a high R&D expenditure. Patent protection is essential in order to recoup the R&D investment. However, before pharmaceuticals can be marketed, they must undergo a mandatory approval procedure in order to demonstrate their safety and efficacy. By the time the product is marketed, parts of the patent term have often expired. In order to compensate the patent owner for this loss of effective patent protection, a system of supplementary protection certificates (SPCs) has been established for medicinal products. SPCs prolong the protection of pharmaceutical patents by up to five years.
Moreover, when an application is made for a new indication for a well-established substance, a non-cumulative period of data exclusivity is granted, provided that significant pre-clinical or clinical studies were carried out for the new indication (Article 10(5)). Furthermore, when a product is switched from a prescription medicine to a non-prescription product and this change of classification has been authorised on the basis of significant pre-clinical tests or clinical trials, it enjoys another year of data exclusivity for these data (Article 74a).
A patent is a legal title that can be granted for any invention having a technical character provided that it is new, involves an inventive step and is susceptible of industrial application. A patent gives the owner the right to prevent others from making, using or selling the invention without permission.The unitary effect of a European patent, which is registered in Register for Unitary Patent Protection, is limited to those Member States where the Unified Patent Court has exclusive jurisdiction at the date of registration.
Regulation (EU) No 1257/2012 of the European Parliament and of the Council of 17 December 2012 implementing enhanced cooperation in the area of the creation of unitary patent protection, OJL 361, 31,12,2012.
Council Regulation (EU) No 1260/2012 of 17 December 2012 implementing enhanced cooperation in the area of the creation of unitary patent protection with regard to the applicable translation arrangements, OJ L 361, 31,12,2012. Agreement preceded the accession of Croatia.
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4.2.3.3. Incentives offered by 'orphan' designation
Regulation (EC) No 141/2000 on orphan medicinal products, which provides incentives for the research and development of medicines mainly for life-threatening and rare diseases, has triggered considerable innovation in this area as well. While the benefits offered by this Regulation depend on the fulfilment of its specific designation criteria, an increasing number of products based on a 'personalised approach' have been designated as orphan medicinal products (e.g. based on the use of autologous cells) and are currently under development for marketing authorisation34. An increasing number of 'stratified medicines' involving -omics technologies have been authorised for use in selected populations for orphan conditions such as cystic fibrosis or genetic inherited enzyme deficiency. Companies with an orphan designation for a medicinal product benefit from incentives such as scientific assistance, fee waivers and ten years of market exclusivity after authorisation.
In 2012, the European Union provided EUR 7,49 million for companies of orphan medicines to receive reductions in the regulatory fees payable to the EMA.
Table 1 : Overview of fee reduction types processed in 2012 (in EUR)
| Protocol Assistance (including follow-up) | 4 502 050 |
| Initial Marketing Authorisation | 2 204 370 |
| Inspections | 494 100 |
| Variations | 106 100 |
| Annual fees | 184 100 |
| Total | 7 490 720 |
Orphan medicines have other similarities with personalised medicine, such as high costs for R&D, small target populations, or extensive education required for physicians. Consequently, this may raise affordability issues from the perspective of payers (public health systems and/or private insurers).
4.2.3.4. Incentives for paediatric medicinal products
For paediatric medicinal products, the validity of the supplementary protection certificate can be extended by six months under specific conditions. In addition, medicines developed specifically for paediatric use and with an age-appropriate formulation can also obtain a paediatric use marketing authorisation, offering ten years of data and market exclusivity. For orphan medicines for children, the legislation provides an additional two years of market exclusivity on top of the existing ten years under the EU's Orphan Regulation.
4.2.3.5. Other incentives for the development of medicinal products
The scientific committees of the EMA give scientific advice to companies for the development of medicinal products. Such advice is given case-by-case for a given product under development. Specific provisions in EU legislation35 enable micro, small and medium-sized enterprises developing medicinal products to receive financial and administrative assistance from the EMA. In addition, the possibility of multi-stakeholder scientific advice, with the participation of regulators, patients' organisations and health technology assessment
By April 2011, this number was 26. At the time of orphan designation, most (81 %) of the applications contained data from clinical studies. The majority of active substances designated were either autologous cells or gene therapy products. Other active substances can be described as monoclonal antibodies, recombinant proteins and others (Source: EMA). Commission Regulation (EC) No 2049/2005.
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experts, has recently been introduced for a number of highly innovative medicinal products in the development phase. Scientific advice has notably be given on products in development with specific questions on genomic biomarkers.
In order to facilitate the development of personalised medicine products, the EMA's Pharmacogenomics Working Party has produced a number of reflection papers and concept papers concerning pharmacogenomics in the development and evaluation of medicinal products36. The group provides both valuable input and expertise in developing consistent information and guidelines for '-omics' personalised treatments (including medicines and their companion diagnostics). Another EMA key activity to support personalised medicine is the qualification of biomarkers beyond the procedure of processing individual market authorisation applications or giving scientific advice for the development of individual products37. These scientific opinions, finalised after open consultation with the scientific community, are made available to the public and to sponsors (from academia and industry) in order to support and facilitate further innovative medicine development38.
At international level, EMA and the US Food and Drug Administration (FDA) have concluded the first joint qualification process for biomarkers by qualifying the use of a number of biomarkers39.
Moreover, to support medicine innovation in the EU, the EMA Task Force on Innovation (ITF) provides a scientific platform bringing together expertise in the areas of quality, safety, efficacy, pharmacovigilance, scientific advice, orphan medicinal products and good-practice compliance to advise companies in particular on emerging therapies and technologies^.
4.2.4. Personalised medicines authorised so far
The European Commission consulted the Member States and EMA on the experience with personalised medicines authorised in the European Union. The experience of the EMA includes:
• Assessment of products approved under the centralised procedure41, for which
predictive genomic biomarkers are to be measured before exposure to the treatment — Since 1999, at least 23 personalised medicines with safety or patient selection benefits based on genomic biomarkers have been authorised via the centralised procedure. It should be noted that around 10% of all products authorised via the centralised procedure have genomics biomarkers identified not only for therapeutic indications, but also for contraindications or posology instructions, thus having a major impact on the 'personalisation' of treatment beyond stratification. Submissions for the marketing authorisation of genomically targeted personalised medicines are gradually increasing.
http://www.ema.europa.eu/ema/index.isp?curl=pages/regulation/general/
general content 00041 l.isp&murl=menus/regulations/regulations.isp&mid=WC0b01ac058002958e http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/ 2009/10/WC500004201 .pdf.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/
document_listing_000319.jsp&mid=WC0b01ac0580022bb0#section2.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/
general_content_000349.jsp&mid=WC0b01ac05800baedb.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/
general_content_000339.jsp&mid=WC0b01ac05800baed8.
Authorisation granted by the European Commission after a scientific assessment conducted by the European Medicines Agency.
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• Orphan medicinal products approved by the centralised procedure which follow the personalised medicine approach.
Likewise, some Member States (such as Ireland and Sweden) have authorised medicines at national level or through mutual recognition procedures, where:
• screening is recommended before exposure of patient to the treatment;
• screening is not recommended but information is given in the summary of the product characteristics.
The objective is to ensure the efficacy and safety of the medicines for patients. Challenges and opportunities:
- The revision of the medical device legislation will explicitely include in the definition of IVD companion diagnostics used for personalised medicine and will strengthen the classification system for diagnostics and consequently the procedures for assessing them.
- A better consultation process for companion diagnostics intended to assess patient eligibility for treatment with a specific medicinal product will improve coordination of the regulation relating to pharmaceuticals and diagnostics.
- The revision of the Clinical Trials Directive will simplify the conduct of clinical trials across the EU.
- The current patent reform will provide cost advantages and reduce administrative burden.
- The reinforced pharmacovigilance system on medicinal products will be a new source of information on adverse reactions.
- The current marketing authorisation procedure and the existing incentives can accommodate and accelerate the placing on the market of medicines based on the personalised medicine approach.
5. Factors affecting the uptake of personalised medicine in health care
In a context of public budget deficits and an ageing population, public health budgets in the European Union42 are under considerable strain. Ever-increasing resources are required to treat diseases such as cancers, chronic or degenerative diseases and diabetes43. In contrast with the great expectations for personalised medicine in offering savings for public health budgets through efficiency gains, fears have been expressed that targeted treatment options may put strains on public health care budgets44.
The French Cancer Institute has shown that investing in molecular testing for the use of stratified and targeted medicines can in fact bring significant savings to the public health
Under the Treaty on the Functioning of the EU, the competence for the definition of health policies and for the organisation and delivery of health services and medical care lies with the Member States. In this context, Member States are also competent for decisions on pricing and reimbursement of medicinal products.
EFPIA Disease burden in Europe (2009): each year over 2 million deaths in the EU are caused by cardiovascular disease and 1.4 million by cancer. 80 million people in Europe have some form of allergic disease and the number is increasing. Some 23 million people in Europe are diagnosed as having depression at any one time. Diabetes affects 246 million people worldwide and the number is expected to grow. Alzheimer's disease affects about 26 million people worldwide and the number is also expected to grow. Commission Staff Working Document-Mid-term evaluation of the Health Programme 2008-2013 (COM(2012)83) Communication COM(2008) 666 final.
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sector, as the cost of testing is offset by the reduction in non-effective or inappropriate prescribing. However, there are not enough examples that personalised medicinal products are not only effective but also cost-effective. The experience from orphan medicinal products, i.e. products for small patient populations, shows that these products are often expensive. In the case of personalised medicine, the cost of diagnostic tests adds to the cost of the medicinal product.
A specific characteristic of personalised medicinal products is that, in addition to the need for prescribers of such medicines to have pharmacogenomic knowledge (requiring education and training), they also need to have adequate IT tools and systems at their disposal. Doctors will need to be trained in a number of disciplines in order to understand and to be able to use all the sophisticated tools that will be at their disposal for personalised medicine. And once trained, they should have access to diagnostic and treatment facilities to administer this care in line with the EU principle of health equality and universal access to medicine. This is a further challenge for national health systems.
The effective uptake of personalised medicine approaches in a Member State will depend on acceptance of the medicinal products and the diagnostic tests by the payers, the public health care system and private health insurance. Both medicinal products and diagnostic tests, even if already authorised to be placed on the market, may thus be subject to rigorous evaluations of their cost and clinical effectiveness in comparison with other therapies available to treat the same disease.
The development of personalised medicine will have some impact, not yet assessed, on the pharmaceutical industry (R&D pipeline prioritisation, pricing policies), healthcare professionals (training needs, skills profiles, etc.), patients (affordability and equity issues), and health systems (affordability issues, infrastructure needs, etc.). Availability of prognostic testing may further increase the challenges in designing the appropriate funding models for health systems.
5.1. Health Technology Assessment
Evaluations of impact are already to some extent carried out under a health technology assessment (HTA). The use of HTA has increased in European countries over the last decades, as it has proved to be a useful tool for providing a transparent, non-biased basis for decisions on the uptake of new medicines, medical devices, surgical procedures and other health interventions.
The work of HTA agencies can be different in volume and scope depending on their mandate in the individual Member State. The following table summarises the "Applied criteria for HTA in selected European countries" (Sorenson et al, 2008).
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| Criteria | AT | BE | CH | DE | Fl | FR | NL | NO | SE | UK |
| Therapeutic benefit | X | X | X | X | X | X | X | X | X | X |
| Patient benefit | X | X | X | X | X | X | X | X | X | X |
| Cost-effectiveness | X | X | X | X | X | X | X | |||
| Budget impact | X | X | X | X | X | X | ||||
| Innovative characteristics | X | X | X | X | ||||||
| Availability of therapeutic alternatives | X | X | X | X | X | |||||
| Equity considerations | X | X | X | X | X | |||||
| Public health impact | X | |||||||||
| R&D | X |
As illustrated in the table, all agencies consider first the clinical domains such as the therapeutic benefit and the patient benefit (including quality of life).
HTA assesses aspects such as cost-effectiveness and budget impact as well as patient outcome, safety, organisational, legal, ethical and societal aspects. It can include an evaluation of the therapeutic benefits and economic impacts of the product for patients and society as a whole. As regards personalised medicine, the cost of patient screening/diagnosis is weighed against the savings made by avoiding unnecessary and inadequate use of 'one-size-fits-all' medicinal products and the additional time and expense treating adverse reactions45.
A successful HTA evaluation of personalised medicine requires the HTA methods to take into account the specificities of the technologies involved and to adopt a long-term and societal view of the benefits of such medicine. A recent study of a model for evaluating the economic impact of personalised medicine for breast cancer patients, based on real data from hospitals in Belgium and the UK, indicates potential savings. This study showed a 37% reduction in total patient costs for health care without affecting the average QALY (quality adjusted life years)46. However, this saving is only achievable with upfront investment in diagnostic techniques and electronic health records, which was not included in the cost analysis.
Evidence from European HTA agencies illustrate that so far very few medicinal products based on this approach have emerged. The issue has been raised particularly with regard to so-called companion diagnostics, which can be - and have been - included in technology appraisals for new medicinal products.
For example, the National Institute for Health and Clinical Excellence (NICE) in the UK has published 134 appraisals of health technologies since 2006. Of these, 47 concern diagnostic tests, but only five involve companion diagnostics. The majority of diagnostic tests appraised concern disease severity assessment, followed by imaging tests, protein expression and a few genetic tests. The five companion diagnostics all relate to the treatment of cancer.
5.2. Methodological issues in HTA
Although the examples so far are few, the HTA agencies already see some important methodological challenges in assessing companion diagnostics type health care product.
See, for example, Impact of pharmacogenomics on health care and health economics, International Journal of Pharmaceutical Medicine 15, 95-100 (2001).
http://www.healthpolicyandtechnology.org/article/S221 l-8837(12)00044-5/abstract.
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For example, there is so far no established 'HTA gold standard' for assessing diagnostic tools. Without this, both industry and HTA agencies struggle to sufficiently demonstrate the accuracy of the tests and thereby their effectiveness.
The use of post hoc subgroups as a basis for identifying the relevant marker for the diagnostic test is also a challenge.
In HTA, the relative effectiveness of a given treatment (compared to other treatments) is an important element. However, relevant comparator data may not be generated in the same clinical trial, and will thus usually not be available for the specific target population in question. Moreover,the availability of the test may be an issue.
Another issue is whether the test in question is the most accurate to identify the marker. This has major consequences for assessing the effectiveness of the medicinal product associated with the diagnostic tool. A sub-optimal test may lead to incorrect estimation of the effectiveness of the medicine, which in turn may give rise to a wrong recommendation on use. Therefore, every time a better diagnostic tool is developed, the effectiveness of the associated medicinal product should in principle be re-assessed.
Because personalised medicines represent often 'break-through' treatment, competent authority requires further input from clinical experts or better/more information in industry dossiers in order to ensure better knowledge.
Finally, the number of people responding to the marker has direct implications for the cost-effectiveness of treatment. If a small number of patients respond positively to the marker for example 5%, it means that the cost of using the test on 100 persons should be divided between the five patients who receive treatment. The costs of the diagnostic are important. If more people respond to the marker for example 50%, the total screening costs could be split between ten times more patients, making the use of the diagnostic tool much more cost-effective.In the later case, there are more people to treat and the costs of the diagnostics will be neglictible.
5.3. EU cooperation in HTA
So far, the role and use of health technology assessments have varied considerably between the EU Member States. Through joint actions, the Commission supports cooperation between national bodies responsible for HTA (from EU Member States, EEA and accession countries). The Joint Action EUnetHTA47, which also includes regional organisations and stakeholders' representatives, aims to facilitate the exchange of information and to develop and test common methodological approaches to HTA.
To build on the results of EUnetHTA and strengthen further the cooperation between national HTA bodies, the Commission has established a permanent, voluntary HTA network at European level, in accordance with Article 15 of Directive 2011/24/EU on the application of patient rights in cross-border health care48. EUnetHTA is a network of government-appointed organisations (from EU Member States, EEA and accession countries) and a large number of relevant regional agencies and not-for-profit organisations. The network is expected to produce joint assessments of health technologies which can be reused at national level and can ultimately create synergies and avoid duplication of efforts among bodies responsible for HTA throughout Europe. It should be noted that currently EU cooperation in HTA focuses on clinical issues, as economic, organisational, legal and ethical considerations are more addressed at national/regional level.
EUnetHTA joint action: www.Eunethta.eu.
Directive 2011/24/EU of the European Parliament and of the Council on the application of patient rights in cross-border health care, OJ L 88, 4.4.2011.
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5.4. Pricing and reimbursement
The pricing and reimbursement systems in the Member States also play a role in the effective uptake of personalised medicine. These systems vary from Member State to Member State but across the board a trend is observed towards an increased uptake of so-called "managed entry" agreements whereby continued reimbursement is made conditional upon proven real-life effectiveness. Certain of schemes entail treatment cessation in treatment non-responder patients. Enhanced data collection in the frame of such schemes may further advance the evidence base underpinning personalised medicine. In addition, many Member States apply different procedures to determine, on the one hand, the price and reimbursement status of medicinal products, and, on the other hand, the inclusion of medical devices and in-vitro diagnostic tests in the health insurance system. Economic evaluation helps to assess the "value for money" of an intervention (i.e. the acceptability). In certain cases, budget impact analyses can also be requested to assess the affordability (predicated financial impact of introducing an intervention compared to the current situation).
The Belgian Health care Knowledge Centre published guidelines for economic evaluations and budget impact analysis to introduce methods to process reimbursement application for pharmaceuticals or devices49.
| Reference case methods for economic evaluations | |
| Literature review | Systematic review of up-to-date clinical and economic literature following methodological standards: reproducible search strategy, transparent selection criteria, critical appraisal. |
| Perspective of the evaluation | Costs: Health care payers (federal government + communities + patients). Outcomes: Society. For health-related quality of life, health states should be described by patients on a generic instrument. Health state valuations for these states should come from the general public. |
| Target population | Consistent with the clinical file. Relevant subgroups need to be defined. Post-hoc subgroup analyses only in case of statistical proof of difference in costs or baseline risk between the post-hoc subgroups. |
| Comparator | Economic relevant comparisons are performed on the efficiency frontier. |
| Analytic technique | Cost-effectiveness analysis (CEA) or cost-utility analysis (CUA), choice should be justified. |
| Study design | Economic evaluation based as much as possible on data from head-to-head comparisons between the study product and the comparator. |
| Calculation of costs | Health care costs paid out of the health care budget, by the federal government, the communities and the patients. |
| Valuation of outcomes | Final endpoints. Cost-effectiveness analyses: life years gained for interventions with an impact on mortality. Cost-utility analyses: QALYs, with quality-of-life weights based on empirical data obtained with a generic quality-of-life instrument such as the EQ-5D for which public preference values exist. |
| Time horizon | The appropriate time horizon for the economic evaluation depends on the duration of the impact of the study intervention on relevant outcomes as compared to the comparator intervention. |
| Modelling | Based as much as possible on data from clinical studies comparing the study medication and the comparator, data from validated databases and/or data from literature. Model inputs and outputs consistent with existing data. Face validity checked. Clear presentation of structural hypotheses, assumptions and sources of information. |
| Handling uncertainty | Probabilistic sensitivity analyses for parameter uncertainty. Scenario analyses for analyses of methodological and structural uncertainty. |
https://kce.fgov.be/sites/default/files/page documents/KCE 183C _economic_evaluations_second_edition_0.pdf
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| Presentation of uncertainty around the incremental costs (IC), incremental effects (IE) and ICERs by means of confidence or credibility intervals. Results shown on the cost-effectiveness plane and cost-effectiveness acceptability curve. | |
| Discount rate | 3% on costs and 1.5% on outcomes. |
As regards the costs, most authorities expect to receive information on direct health costs e.g. health services, medications, hospitalisation, etc. However, it is often recommended in the literature to use the societal viewpoint for the economic analysis, i.e. costs and outcomes for society as a whole should be valued. This would include costs borne outside the health care sector, such as productivity losses and travel expenses and stricto sensu also outcomes for patients' family.
Moreover, as regards genetic testing, reimbursement is not necessarily based on added value. Public laboratories might receive a standard budget for each sample examined, independent of the type of sample, the test or the work required to investigate it. According to a report from the OECD this practice may discourage low-volume, technically complex and expensive testing procedures and may drive the centralisation of testing services50. Some stakeholders request increased coordination by national authorities responsible for pricing and reimbursement of medicinal products and medical devices or even the streamlining of national pricing and reimbursement procedures to enable personalised medicine technologies to reap their full potential51.
Challenges and opportunities:
- Challenges include adapting Health Technology Assessment methods to the needs of personalised medicine and developing and maintaining cross border sharing of expertise among HTA bodies.
- The benefits of personalised medicines are expected to offset their costs by efficiency gains. Rigorous evaluation offers the possibility to demonstrate the effectiveness of medical products and medical devices in comparison with other therapies. Successful uptake requires a robust Health Technology Assessment.
- The forthcoming establishment of a permanent, HTA network at European level will allow work models to be established for joint assessments of new health technologies. The information generated can be reused at national level, thereby reducing duplication of work between Member States.
- Challenges also include the fact that there are variable policies in EU as regards pricing and reimbursement52.
6. Conclusion
The development of personalised medicine through the use of -omics technologies offers new opportunities for the treatment of patients in the European Union. Through this approach, health care providers may be able to offer better targeted treatment, avoid medical errors and reduce adverse reactions to medicinal products.
Pharmacogenetics, Opportunities and challenges for health innovation, OECD 2009, p. 109. Europabio.
Priority Medicines for Europe and the World 2013 Update
http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/prioritymedicines_report_en.pdf
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The current regulatory framework for pharmaceuticals offers a number of tools and procedures to ensure that medicines placed on the market have a high quality, safety and efficacy. These tools and procedures have been shown to work well for innovative products and orphan medicines, including therapies relevant to personalised medicine. The pharmaceutical legislation is flexible enough to address current needs and to authorise personalised medicines in a timely manner. The overall regulatory framework allows supporting the field with appropriate scientific guidelines and expert dialogue.
The new framework programme for research, Horizon 2020, and the ongoing revisions of important pieces of legislation address certain challenges identified in the development of these therapies, from basic research up to their placing on the market. The revision of the medical devices legislation will strengthen the oversight of in vitro diagnostics and introduce a better consultation process for companion diagnostics to assess patient eligibility for treatment with a specific medicinal product. The revision of the Clinical Trials Directive is expected to simplify the conduct of clinical trials and consequently facilitate the authorisation of research in therapies using personalised medicine. Moreover, a Health Technology Assessment taking into account the new technologies would provide a methodology for addressing the uptake of personalised medicine.
Personalised medicine is not a revolution but an evolution. Advances in science are expected in such a fast moving field. The European Commission will continue to monitor the developments of personalised medicine in the coming years and maintain a fruitful dialogue with stakeholders.
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Appendix
Research challenges in the development of personalised medicine approaches (identified in the conference "european perspectives in Personalised Medicine" in May 2011 presented by conference session)
The full report is available on: http://ec.europa.eu/research/health/policy-issues-personalised-medicine en.html
Session 1: R&D - the basics
1. Make greater use of genome-wide-association studies and other 'omics' technologies to improve the understanding of molecular disease mechanisms, and by consequence the search for new drug targets.
2. Develop new animal models to test potential treatments for single-gene diseases.
3. Develop new treatments for single-gene diseases.
4. Explore drug targets identified through epigenomics.
5. Maintain Europe's lead in the field of proteomics.
6. Develop new methods for validating biomarkers.
7. Support the commercialisation of new imaging technologies. Develop new imaging standards.
8. Make optimal use of mathematics, computer modelling and simulation to translate information from '-omics' research into clinically relevant products and technologies.
9. Nurture multidisciplinary research bringing together clinicians and -omics specialists.
10. Support and develop standardised data collection and biobanking for disease cohorts. Session 2: Biomarkers in personalised medicine
1. There is a need for high throughput screening platforms to identify biomarkers more quickly and cost-effectively; there is also a need for new validation techniques for candidate biomarkers.
2. Tools and methods need to be developed for the functional analysis of cells.
3. There is a need to standardise how specimens are collected within clinical trials, or for routine purposes and how data-sets are analysed.
4. European guidelines are needed for biomarker qualification and clinical validation.
5. Take advantage of new imaging technologies to understand biological mechanisms, including toxicity, at the molecular, whole organ, and whole body level.
6. Standard Operating Procedures (SOPs) are needed for tissue collection and analytical procedures.
7. A Europe-wide biobanking network is needed.
8. The issue of medicine-diagnostic co-development should be looked at.
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9. Ethically compliant electronic patient records should be developed to inform biomarker research.
Session 3: The tests in humans - clinical aspects and clinical research
1. Develop Europe-wide biobanking.
2. Develop new trial methodologies including adaptive clinical trial design.
3. Promote the development and use of electronic patient records; use these electronic records to inform biomarker research.
4. Provide biomarker studies of generic medicines.
5. Consider the use of all types of biomarkers, not just molecular ones (also functional, imaging, etc.).
6. Many approaches (multi-modality) and not just medicines are needed in achieving personalised healthcare.
7. Standardise insurance requirements for clinical trials in Europe. Session 4: Towards the market and patients - approval process
1. European clinical testing laboratories (both industry and hospital based) require clear
regulatory standards and a stable reimbursement environment. Guidance is needed on which clinical endpoints are considered to deliver patient and societal value; guidance is also needed on innovative clinical trial designs and for the use of retrospectively generated data sets.
2. More investment is required in translational medicine, especially in applied molecular profiling and imaging technologies.
3. A uniform quality framework and delivery infrastructure that maximises patient access is needed for companion diagnostic testing in Europe.
4. More unified and coordinated accreditation procedures should be established for European clinical testing laboratories.
5. There should be a more focused and coordinated effort to use patient registries, patient biological samples, and patient outcome data in a targeted personalised medicine development programme with results published in a timely manner.
Session 5: Uptake in healthcare - post-approval process
1. Research should be undertaken to better measure patient outcomes in the context of personalised medicines.
2. Discussions between medicine regulators, health technology bodies and industry about measuring efficacy/effectiveness should continue.
3. The regulation of the components of personalised medicine (e.g., medicines and diagnostics) should be coordinated. A central role for the EMA was mentioned as a possible solution.
4. Research into viable business models to help support the introduction of personalised medicine and related technologies should be initiated.
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Session 6: In the clinic - practitioner and patient perspectives
1. Explore what the barriers are to achieve clinical added value by the introduction of personalised medicine approaches.
2. Provide more education and training to clinicians in the use of the new diagnostics, and make sure the required facilities are easily available.
3. Make better use of the biomarkers that we already have before introducing new ones.
4. Get all stakeholders to collaborate in translating research into clinical practice, with an emphasis on patient participation.
5. Explore the social consequences, e.g. insurance and employment, for patients who overcome a serious or debilitating disease. Are there mechanisms for reintegrating them into society?
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