For health systems, personalised medicines can lead to decreased use of ineffective treatments, reduced hospitalisations and other costs associated with chronic conditions, and more efficient use of healthcare resources. But there are also a number of environmental and organisational challenges that currently prevent the effective uptake of personalised medicines and potentially hinder their development. A range of stakeholders including leading payers, policymakers and healthcare professionals have called for efforts to better understand these challenges to support target goals in patient access for new therapies while also maximising their positive impact on health systems in the UK and throughout the EU.
To better understand the factors that can affect access to personalised medicines, CRA recently completed an analysis (sponsored by EFPIA/EBE) focusing on new personalised immunotherapies and assessed the extent to which they are made available in the UK, Denmark, France, the Netherlands and Poland. These countries collectively represent a broad range in terms of geography, reimbursement mechanisms, approaches to health technology assessments (HTAs), treatment infrastructure and healthcare policies.
The analysis highlights a clear need for innovative strategies to address challenges and to prioritise personalised medicines within a diverse range of healthcare models. New approaches might require continued focus on better management of care that reflects the advantages personalised treatments can deliver, broader investment in next-generation diagnostics technologies and testing infrastructure, and better alignment of value assessment data to support their use for regulators and HTA bodies.
New funding models for diagnostics
Many stakeholders agree that the lack of clear funding mechanisms for new diagnostic services can significantly restrict access to some personalised medicines. Historically, investment in companion diagnostics was linked to the value of an individual therapy to make development commercially viable. As diagnostic approaches move toward testing that is not disease specific (such as tumour profiling or WGS), this traditional funding strategy may no longer apply. Stakeholders may need to consider new strategies in funding for diagnostics that take into account factors associated with individual markets and disease area.
Some potentially more effective models currently being implemented across the EU include centralised funding, a diagnosis-related group (DRG)-based approach, and a self-pay approach. The centralised funding model may support broader infrastructure investment and higher levels of access, whereas the DRG-based approach used in the UK involves competitive fee-for-service laboratories that could increase competition, leading to quicker innovation and reduced prices that have a corresponding positive impact on access. While the self-pay approach offers some benefits, plans could be limited over the long term. EU markets might also consider a hybrid model that involves some centralised funding and funding associated with testing for certain conditions.
Coordination of care
Efforts to improve coordination of care for patients across EU markets are also important to help expand access to personalised medicines and may require new investments in healthcare infrastructure and expertise. Some markets are taking steps to improve efficiency and quality of patient care in oncology while managing budgetary challenges by either centralising care according to tumour type, developing a network of accredited hospitals and specialised treatment centres, or delivering care through a ‘hub-and-spoke’ delivery model. In the Netherlands, for example, the concentration of care and expertise for melanoma and ovarian cancer in select hospitals has resulted in increased uptake and more effective use of personalised treatments. The UK, on the other hand, uses a hub-and-spoke system that encourages use of integrated information and communication technology networks to support electronic patient records and real-time data sharing. This approach allows clinicians working across different geographies and in various facilities, including hub hospitals and more specialised cancer centres, to collaborate in patient management and treatment more effectively.
While there is not necessarily a single model or solution that can be broadly applied, the alignment of expertise and strategies to support access to innovative medicines and better coordination of care are critical to realising the full range of potential benefits of personalised treatments. Better coordination of care also presents the opportunity for faster diagnosis and treatment and improved outcomes.
Flexibility in value assessments and reimbursement
The HTA systems in many EU countries can be barriers to both access and reimbursement of innovative medicines including personalised therapies. For example, stringent HTA guidelines in the UK have created numerous challenges in meeting cost-effectiveness thresholds to achieve positive recommendations from the National Institute for Health and Care Excellence. Consequently, all recently approved innovative medicines in the UK have required some form of patient or managed access scheme to reduce costs to within accepted thresholds.
Within this framework, recent progress in the clinical development of personalised medicines has prompted the European Medicines Agency (EMA) to become more flexible when reviewing data to expedite approvals and support rapid patient access while also ensuring that new treatments demonstrate sufficient levels of safety and efficacy. Since 2003, several personalised cancer therapies have launched in Europe based on results from Phase 2 single-arm trials or Phase 3 trials with limited data instead of full randomised control trials. Concurrently, many payers continue to see large, comparative randomised clinical studies as the gold standard. They are often reluctant to reimburse for new drugs based on limited clinical data that does not meet previous standards in risk-benefit assessments. This misalignment can create a gap between payers and regulators that can limit access to new therapies.
Several plans have emerged to try to address this gap, including the Medicines Adaptive Pathways to Patients initiative, parallel consultations between the EMA and HTA bodies, and accelerated pathways viewed as a continuum to collect input from all relevant stakeholders. Many drug developers are also collecting significant levels of real-world data to mitigate payer concerns about limitations in data at launch. Several EU markets have established patient registries to collect real-world safety and efficacy data to keep track of patients who are using conditionally approved products and to support ongoing clinical assessments. UK regulators from the Medicines and Healthcare Products Regulatory Agency recently introduced an early access to medicines scheme, which gives patients with some serious and potentially life-threatening conditions access to medicines pre-marketing authorisation with the ability to collect real-world data to support subsequent HTAs.
It is increasingly clear that new and previously untried strategies and healthcare policies will need to be implemented to support the continued development of personalised medicines and target goals in patient access in the years ahead. Efforts to expand access to personalised therapies will likely require collaborations between multidisciplinary stakeholders including payers, policymakers, manufacturers, regulators, HTA bodies and clinicians. Sharing best practices related to advanced diagnostics, funding mechanisms, HTA systems, coordination of care and other important issues will also be essential to ensure equal and rapid access across different EU markets. Without this level of change implemented in the near term, many beneficial and potentially life-saving therapies may not reach patients who can benefit from treatment.
Anthony Barron is an associate principal in the Life Sciences practice of Charles River Associates (CRA) based in Brussels. The views expressed herein are the author’s and not those of CRA or any of the organisations with which the author is affiliated.
