Meeting of the MEP Interest Group on Brain Health and Neurological Conditions
November 14, 2023 | European Parliament, Brussels
On November 14th, 2023, the MEP Interest Group on Brain Health and Neurological Conditions hosted an in-person event in the European Parliament, Brussels, entitled ‘Patient Involvement in HTA processes: Disparities in Access across Europe from the Neurology Perspective’. The event’s main objective was to discuss the challenges of Health Technology Assessment processes at European and national level and learn how patient involvement could be applied to yield an improvement in access disparities across Europe. The event was attended by EU policymakers, healthcare professionals and representatives of patient organisations.
Orla Galvin, Executive Director of EFNA opened the meeting commenting that it is critical that the brain health and neurological patient community are more involved in HTA processes.
MEP Alex Saliba (S&D, Malta); Co-Chair of the MEP IG on BHNC led the opening remarks reminding the audience that this interest group is a patient led initiative to ensure health policy solutions align with neurological patient needs. MEP Saliba highlighted challenges in access.
Not all EU countries represent the same opportunity for industry. The average EU rate of availability of medicinal products is 45%. Larger central EU countries such as Germany and Italy have over 80% availability, with many Eastern EU countries having less than 20 % availability such as Latvia and Estonia, and others such as Malta and Turkey having 10% or availability or less.
Time is of the essence:
The EU average time to availability (that is the time from marketing authorization to the date of availability to patients) is 517 days. However again we see countries such as Germany (128 days), Denmark, Netherlands, and Sweden under 330 days, yet for Spain it is 629 days, Greece 674, Portugal 702, Poland 827, and Romania 918 days. This translates to significant delays in access for people living with neurological conditions, many of whom live with chronic and progressive brain and neurological conditions and adds to inequity across Europe. With significant differences in rates of availability and time to availability between countries, novel approaches to HTA protocols are needed. Indeed, the Commission has begun this progress with revisions to the directive and regulations laying down Union procedures for new joint clinical assessments and joint scientific consultations.
Key principles of the new joint HTA regulations are:
- that it is only on the clinical component-no economic assessment or recommendations on pricing or reimbursement.
- Driven by EU HTA bodies who remain responsible for drawing conclusions on added value for their health systems
- High quality, timeliness and transparency
- Use of joint work in national HTA processes
- Input from independent experts
- Stakeholder engagement and inclusiveness
- Progressive implementation
The timeline for new regulations is:
- Jan 2025: New oncology and advance medicine therapy
- Jan 2028: Orphan medicinal products
- Jan 2030: All new medicines will come under the scope of the new regulation
This means all stakeholders must engage to prepare. We all have a role to play.
HTA interest is growing. Patients have knowledge, perspectives and experiences that are unique and contribute to essential evidence for HTA. Learnings can come from others in this space such as the 2022 European Medicines Agency Patient Engagement Framework.
Astri Arnesen, EFNA President, reminded the audience that EFNA has been around for over 21 years advocating for patients, caregivers and families affected by neurological conditions and this work was recognised with WHO-Europe accrediting EFNA with Non-State Actor status this year. She addressed the unfair disparities in the years of delays in access between countries in Europe, and praised the politicians and policy makers for working with us emphasising the need for innovative and sustainable solutions. Indeed, reflecting this is timely as neurology has the second highest therapeutic area of EMA approvals.
In setting the scene Mr David Schwicker, ORPHA Strategy, outlined HTA as a service to society, a process of providing evidence-based information for healthcare decision making.
Healthcare budgets and reimbursement remain national Member State competencies, accordingly, HTA is a national process. However different HTA and Pricing Reimbursement legislation, processes, guidelines, resources, priorities, values, and decision-making bodies can lead to a variation of outcomes in HTA recommendations and pricing negotiations across Europe, not to mention duplication of work for developers with multiple HTA submissions. This has a major impact on the population of Europe with ONE in THREE people being affected with a neurological condition throughout their life-course. Only ONE in FIVE people living with a neurological condition have access to affordable therapeutics that satisfactorily manage their condition- this means 80% of people do not (data from an EFNA study, 2022). He states however, that value is complex concept. Value is in the eye of the beholder- patients/families, yet caregiver cost is not included in value proposition. HCPs want to know real-world performance- such as tolerability and drug-drug interactions. There are national, regional and hospital budgets with many stakeholders and many levels.
David emphasised the additional challenges for neurology for HTA- namely the uncertainties with neurological conditions. The invisible and sometimes immeasurable symptoms, uncertainty of data, condition progression and registry data. Moreover the situation is worse for rare neurology, the rarer the condition the greater the challenge. He indicated that is timely for greater patient involvement to improve research and innovation and HTA processes to ultimately improve neurology patient access in the EU.
Dr. Anke Peggy Holtorf, HTAi shared a pre-recorded video- ‘Patient involvement in HTA in Europe: Recommendations from a 360-perspective view of current patient involvement practices’.
Dr Holtorf reflected on the fact that patient involvement has been around since the 1970’s and how in the present day there is unfortunately still high variability on how patient involvement is carried out. She recommends recent study reports on the major values and standards in patient involvement in HTA emphasising relevance, fairness, equity, legitimacy and capacity building. She described how patient involvement impacts the HTA recommendations, staff and processes, and the patients involved regarding awareness of HTA.
Recent work from the HTAi reports the barriers for patient involvement including: patients not knowing about the opportunities as to how to get involved, the need for improved knowledge, capability and resources which patients do not have. HTAi work investigated what would motivate patients to become involved and the major responses were to have it government mandated, access to training, and explicit published processes. HTAi also learned that for those involved in HTA in many countries, they did not have enough information and did not know how their input would be used.
Dr Holtorf indicated patient involvement can be in each step, from horizon scanning to appraisal or evaluation. She highlighted recent study recommendations such more collaborations between HTA organisations and patient organisations, more guidance, an early alert system to give more time, and reporting back on how info was used. She stated that patients serve as advisors on each step, essentially serving as quality assurance. The 360 Review helped to identify best practices such as good preparation, early alerts, stakeholder collaboration and evalution and feedback, all of which lead to improved quality and effectiveness.
Jana Hlavacova, Section for Legislature & Law, Ministry of Health, Czech Republic, informed the audience that she is also a patient advocate living with MS and has worked with the EFNA member EMSP. Jana provided what the audience agreed was an example of best practice in Patient Involvement in HTA processes. Jana described how training for patient involvement began 3 years before patient involvement in HTA processes was embedded into law in 2022 for Orphan Medicines and Products. Jana indicated that the HTAi template, as mentioned by Dr Anke Holtorf, was used to establish this process and highly recommends it.
In the Czech Republic they are trying to make sure patient involvement is mainstream throughout healthcare policies. From Jana’s perspective, without it we are missing the equity and quality patient involvement brings to decision making but to really make a difference we need political will and dedicated staff. Critical to the process is finding a patient organisation partner because of its legitimacy and its being a democratic way of representing patients. If you have a chair at the table for patients you need to make sure they can sit on it- meaning we have to create favourable conditions- ensuring appropriate levels of information, education and financing.
In the Czech Republic there are 25 people on the patient council which directly as a unit provide advice to the minister. Patient involvement in HTA has led to timely access for patients, progressing from each individual applying for reimbursement to having a systematic way of reimbursement.
Patient Involvement in HTA in the Czech Republic occurs in two ways- first in the assessment phase, directly from patient with the condition, and then the appraisal phase, where those involved in making the binding decision include patients from patient organisations who do not have the condition.
Stella O’Brien, Lay Representative, NICE UK, provided a virtual presentation on ‘Patient involvement part of the solution for health and social care systems’.
Stella began her presentation addressing the role of NICE- to get the best care to patients fast, while insuring value for the taxpayer. NICE must recover the cost of the appraisal from the company marketing the product. Members of NICE committees go through an independent and open application process, the committee is made of statisticians, health economists, clinicians from many sectors and lay persons, for each appraisal they are joined by clinical experts and expert by experience, that is patients or caregivers.
Lay persons and experts by experience have the support of NICE’s PIP (Public Involvement Program) team which is world leading and gold standard for patient involvement. PIP provides training and support through the appraisal process so they can make fully informed decisions. Lay members can act as a bridge to the patient experts and the societal perspective. Just because a particular outcome is measurable doesn’t mean that it is an outcome that is meaningful to patients, and just because something cannot be measured doesn’t undercut it’s value.
She explained how NICE asks companies to provide SIPs (Summary of Information for Patients) in plain language, as part of the evidence package for an appraisal, which cannot be marketing material, and this is checked by NICE. It turns out that SIPs which were intended for patients are actually useful to everyone on the appraisal committee and dismantle any barriers the technical language may erect.
Stella was shocked how few countries have patient involvement in HTA processes. Particularly when PPI in decision-making is gaining momentum everywhere. HTAi, the Institute for Clinical and Economical review in the US and NICE, all encourage equal patient and public involvement in these processes. According to Stella, we are not an unnecessary overhead, we are part of the solution for sustainable healthcare systems. We are the reality of nothing about us without us.
Dr Orla Galvin asked the audience to reflect on why we hosted this meeting, and that was to help tackle the delays in access faced by people living with neurological conditions. She reminded the audience that two thirds of people with neurological conditions have to visit three clinical sites before accessing a diagnosis and that 40% of women wait over one year for a diagnosis while this figure is 29% for men. HTA is not the only delay in access but it is something that can be improved upon.
Ms July Spoony, (European Patients’ Forum (EPF)), commented on their ongoing work on the new HTA regulation and its implementation, stating that we have patient involvement embedded in the regulation but we do not know the practical implementation of this. EPF is part of the HTA stakeholder network, which informs the work of the Commission on the regulation. The Commission need to provide us with the information around what constitutes conflict of interest of patient involvement, and from the patient perspective this needs to be reasonable and realistic. EPF is also working with EURORDIS on training for patient involvement at in HTA at the EU level.
Mr David Schwicker encouraged the audience to take note of the key inflection points on the revisions to the HTA regulations and open consultation periods (which are short).
Albert Counet, President, Belgian Huntington’s Disease Association, described how 30 years ago we would not be having this multi-stakeholder conversation around reimbursement and that indeed we have come a long way. As a representative of the rare disease community he is very grateful for that. However, he still has concerns about the discrepancies between countries, between prevalence of conditions and the difficulties within a country to reach agreement, at hospital level and with insurance. He took the opportunity to highlight the need for patient registries so that an appropriate budget could be applied.
Dr Orla Galvin closed the session reminding the audience of EFNA’s forthcoming free e-learning modules and materials on HTA and on the EU Health Data Space. She thanked MEP Alex Saliba and the MEP Interest Group on Brain Health and Neurological Conditions for hosting the meeting, as well as the sponsors for affording us the potential for this much needed discussion – Pfizer, Roche, Novartis and Alexion.