Figure 1: Maturity model of patient and Patient Organisation (PO) engagement in Europe (list of countries not exhaustive)
The degree to which patients and patient organisations (POs) can be involved in the HTA review process varies across Europe, and there are as many situations as there are countries (see figure 1 for a high-level overview). Patients and POs contribute to discussions by highlighting, for example, their experience of the disease, their needs and what risks they would consider acceptable in view of the expected or potential clinical benefits.
Some countries like France or Sweden have a formalised process where patients and POs can vote within HTA committees. In contrast, other European countries like Austria, Italy or Portugal do not usually share HTA information nor consult patient representatives. Following the appraisal of a medicine, in some instances patients and POs have the right to appeal or provide input at re-evaluations. For a summary of the key takeaways on patient and PO involvement in local HTAs see figure 2.
Figure 2: Key takeaways on patient and PO involvement in HTAs
The European Medicines Agency (EMA) offers consultations in parallel with the European Network for Health Technology Assessment (EUnetHTA) to harness synergies between regulatory evaluation and HTA. This collaboration currently focuses on evidence-generation plans and patients and POs are regularly invited to share their real-life perspectives and experiences in relation to a particular medicine in their disease area.
In 2019, simultaneous advice from EMA and HTA bodies was provided upon request during the development of 27 medicines. Patients were involved in two thirds of these scientific advice procedures and provided added value in almost every case; sometimes leading to a modification of the development plan to reflect patient inputs.
Underlying our vision is the concept of augmenting patient empowerment through the lens of ‘No decision about me, without me’. We want to see a future where patients and POs are at the centre of medicine development and reimbursement decisions, as they represent real-world patient perspectives and needs. This will help close the gap between “hypothesis” and “reality” when it comes to improving disease outcomes and enhancing health and lives.
Adopting this has many implications. For example, payers, regulators and policymakers will face the challenge of designing decision-making processes that are fair and equitable across all patient representatives and organisations. Negotiations with industry will become more complex and potential budget impacts will need to be balanced carefully.
The life sciences industry will be presented with opportunities for faster access and unique reimbursement models. They will need to adapt their culture and clinical development and commercialisation models to engage with patients and POs much earlier and seek clarity on where decision-making authority sits across patients and payers.
Meanwhile, healthcare providers will need to engage with well-informed patients and POs who may be lobbying for specific treatments. Providers can leverage this increased awareness to enable more patient-to-patient communication, which in turn could lead to improved compliance.
Figure 3: Proposed steps for improving patient empowerment
There are many steps that can be taken to improve patient empowerment. To achieve this, stakeholders will need to rethink every aspect of medicine development and HTA.
All stakeholders should look to establish and adopt a centralised European HTA operating model, building on the cross-border HTA collaboration led by EUnetHTA. This will help drive consistent processes, effective assessments and enable early access to high-value medicines. It will also be important to create pre-competitive collaborative consortiums involving stakeholders across the full value chain, so that they can share lessons learnt and establish best practices.
Meanwhile, payers, regulators and policymakers need to advocate for changes in culture that ensure patient engagement is integrated at every step of the HTA process – as well as assessing alternative funding and reimbursement models to continue to improve patient access to licensed treatments.
Finally, the life sciences industry needs to be ensuring systematic patient involvement upfront in market and disease assessments, clinical trial design and throughout the medicines development process. To facilitate this, readability of lay summaries needs improvement to maximise the value of information exchange and educate POs. This will empower them to provide greater input and influence over HTA decisions.
Most critically, patients and POs should also be looking to engage with these stakeholders as often as they can to advocate and lobby for these changes.
There is a long road ahead for implementing such wide-reaching changes, but the result will be well worth the journey – with better access to treatment, improved ways of working and, ultimately, better patient outcomes.
Vynamic believes there are three critical areas that life sciences organisations should consider as they evolve their patient focus. These are: Define Strategy, Leverage Digital and Measure Impact. Please contact us to learn more about how we might be able to support you in placing the patient at the heart of your business.
Vynamic is a 185+ person healthcare industry management consulting firm headquartered in Philadelphia (US) with offices in London (UK), Boston (US), Raleigh- Durham (US). Vynamic clients include some of the largest companies across Life Sciences, Healthcare Technology, Providers, Public Health and Health Plans. Vynamic helps clients achieve Actionable Strategy, Operational Intelligence, and Healthy Culture. Founded in 2002, Vynamic set out from the beginning to focus first on people to create a healthy culture.
To learn more about Vynamic, visit www.vynamic.com.