Join our email list to stay up to date on the latest Amyloidosis news.

ARC has published a Health Technology Assessment Toolkit to equip patients and patient groups within Europe, as well as globally, with the tools to advocate on behalf of themselves and other patients for access to new treatments.

The Amyloidosis Research Consortium (ARC) is committed to ensuring that patients and family members affected by any type of amyloidosis receive the best standards of treatment and care from diagnosis right through to end-of-life and everything in between.

Part of this is about equipping patients and patient groups in the US, Europe, and throughout the world, with the knowledge and tools to be able to advocate on behalf of themselves and other patients; to put forward their viewpoint about living with this complex and rare disease and to press for the very highest standards of treatment and care. The ‘patient voice’ and participation in the process of approving treatments is essential and encouraged in many countries and can be extremely impactful in ensuring access to the very best, new treatments.

In response to this, ARC has produced a guide, or Toolkit, about the Health Technology Assessment process in Europe. It is intended to give information about how treatments are evaluated and approved for use in the various member state healthcare systems and to guide patients, and patient groups on how they can participate in the process to ensure that those treatments that are developed for their amyloidosis can be made available in their countries.

ARC recently released a European Health Technology Assessments (HTA) Toolkit, which can be viewed here.

Interview with Kate Morgan, Head of Policy and Access at MPE

As part of this launch ARC interviewed Kate Morgan, Head of Policy and Access at Myeloma Patients Europe (MPE) to hear her perspective about the value of patient engagement with HTA bodies.

Why are European Health Technology Appraisal and payer bodies relevant to patient organizations?

“Once a medicine has been granted national or European marketing authorization, it means it is safe and effective to prescribe for patients. However, patient access to medicines in European countries usually depends on national approval from healthcare systems.

Given each new medicine has a cost to a healthcare system, depending on the price and volume of the medicine required, most European countries have processes in place to provide advice to their health systems on how best to allocate their healthcare resources. Healthcare resources and funding is often limited, so healthcare systems want to ensure that they use these resources in the most effective way. This is particularly relevant in healthcare systems that rely predominantly on funding from taxation or national insurance contributions.

The methods that health technology assessment bodies and payers use to assess the value of a new medicine vary from country to country. The basic principle underpinning decision-making is a health economic assessment of whether the costs of a new medicine outweigh the benefits to the healthcare system. They may also consider wider factors such as unmet need, disease rarity and patient preferences.

As the decisions HTA and payer bodies make impact on patient access to medicines, it is important that patient groups work closely with these bodies, ensuring they are aware of and engage in their decision-making processes where possible.

Some HTA bodies and payers have defined ways of involving patient organizations in their decision-making processes. Ensuring decision-makers are aware of the perspective of the patient and carer community and the impact a new medicine will have on a condition can help them consider the wider benefits of a medicine. Providing this perspective is a very important role for patient organizations.”

Describe the EU HTA and payer body landscape, thinking in terms of commonalities, differences and gaps?

“Most European countries now have systems in place to assess the potential benefits of new treatments in relation to their costs. Whilst the principles underpinning the methodological approach for assessing new medicines are broadly similar in many countries, the processes and the ‘willingness to pay’ threshold can vary significantly from country to country. Patient involvement in decisions and the flexibility that HTA and payer bodies have in decision-making also varies.

Societal and political values, willingness to pay, and the ability to pay, of healthcare systems can lead to differences in decision-making and the ability of patients to access medicines across different European countries. In some Central and Eastern European countries, for example, there are often difficulties accessing high-cost medicines due to systemic issues and restricted health budgets.

Whilst there are ongoing efforts at a European Union (EU) level to better harmonize HTA, there will always be some variation as healthcare spending decisions are powers reserved for member states.”

Briefly summarize the ways in which patient organizations and patients can contribute to the work of HTA and payer bodies?

“The extent to which patients, patient organizations and carers can contribute varies between European countries. Some HTA bodies, such as those in the United Kingdom, France and Germany, have formal processes through which they engage patients and carers in their decision-making. The models for doing this can vary from having permanent patient representatives on decision-making panels through to providing ad hoc opportunities to provide both written and oral evidence to decision-making panels. Patient organizations may also be asked to comment on draft guidance on a decision or to participate in an appeals process.

In most cases, where patient and carer representatives are involved, their contributions are required to focus around the patient and carer experience of the condition and the existing standard of care. They are also asked to provide evidence on lived experience of the new medicine (where possible) and the impact it is likely to have on patients. It is important to build a strong picture for the HTA and payer bodies on this perspective, to help frame their decision.

HTA and payer bodies are interested to understand topics such as the quality of life impact, the impact on patients being able to continue undertaking normal daily activities and other issues such as impact on working and reliance on informal and formal carers. This type of information can be gathered by patient organizations through qualitative and quantitative surveys and through the regular interactions they have with patients, carers and their families.

Having said this, in many countries, the contribution that patients can make can be quite limited. There is still therefore a lot of work to do both on the side of HTA and payer bodies, but also on patients and patient organizations to make the case to be involved.”

What should patient organizations do if there or no obvious ways to work with or engage with their national HTA or payer body?

“My advice would be to collaborate with other patient and carer organizations in your country, develop a strong evidence-based case for support and to lobby the relevant health ministry, department or government body in your country. It is important to point out the benefits of patient involvement for them, as well as to patients and carers.

Pan-European and international networks such as Health Technology Assessment International (HTAi), EUnetHTA and EUPATI have developed best practice models and publications on optimum ways of involving patients and carers in HTA and payer decision-making. These are tools that are very useful in building a case for how patients and cares should be involved in decisions, as they are built on a strong evidence base, cross-stakeholder expertise and consensus.

If you need any advice or help, international patient groups such as ARC and Myeloma Patients Europe are able to help develop a strategy to engage on this important topic, provide examples of how patient advocates from other countries have tackled this problem and also to facilitate contact with expertise if required.

Finally, even where not routinely involved in decision-making, patient and carer organizations should ensure the continuous development of a strong evidence base on the impact that a disease and its treatment have on patient and carers lives. This type of information can help you in advocacy work more widely and in demonstrating to HTA bodies and payers the types of evidence available to help support their decision-making.”

 Is it just about the HTA or payer body, or should patient organizations be influencing evidence generation, medicines optimization and/or health policy at the same time?

“No, it is not just about influencing the HTA or payer body. A common reason why medicines have difficulties being approved in national healthcare systems is due to the evidence not being fit-for-purpose. Pharmaceutical companies need to demonstrate why their medicine is valuable to both healthcare systems and patients, which is often a challenge.

Patient and carer organizations therefore have a much wider role to play. We need to be involved in the shaping of research and clinical trials (as early in the process as possible) to ensure that what is being assessed and measured is relevant to patients, as well as HTA and payer bodies.

Patient organizations should also invest in collecting patient generated health data to provide robust evidence about the needs and perspectives of patients and their families and carers. Only by doing this can the true value and potential benefit of a new medicine be properly assessed.”

 What will the HTA and payer body landscape look like in the next 5-10 years, and what should patient organizations be doing to prepare?

“It will be the norm rather than the exception to have a form of HTA carried out in most countries. Increasingly the way in which new drugs are assessed will become more sophisticated and the role of patients in these assessments will grow. I hope to see earlier, more systematic and flexible approaches to incorporating patient evidence into development and decision-making on new medicines.

The pharmaceutical industry will need to continue to adapt how they generate evidence and the standard and quality of the evidence they submit and be sure to include data that is meaningful to patients.”

Kate Morgan is Head of Policy and Access at MPE. She specializes in health policy, with a specific focus on policies affecting drug development and access in European countries. Kate previously worked for eight years in the Policy Team at Myeloma UK, where she was involved in influencing a range of policy and reimbursement decisions affecting myeloma patient access to good quality care and effective new drugs. She been involved in a wide-range of patient organization networks internationally,  most recently chairing the Blood Cancers Alliance, an information-sharing network of hematological cancer charities in the UK.






Kate Morgan