To explore the potential challenges and opportunities the JCA presents and understand to what extent the patient perspective will be incorporated, Cencora brought together a panel of experts for an in-depth webinar discussion. The session was moderated by Herbert Altmann, Head Commercialization and Access Solutions Europe, at PharmaLex, a Cencora company. Panelists included Antonella Cardone, CEO, Cancer Patients Europe (CPE), Matias Olsen, Senior Manager, Public Affairs and Policy, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), and Casper Paardekooper, Partner, Vintura, a Cencora company.

In the lead up to the webinar, we published an article on the patient perspective. The webinar takes that discussion further and into additional areas impacting patient involvement. 

Will including the patient voice improve access to innovative medicines?

The implementation of the JCA will begin with oncology drugs and advanced therapy medicinal products (ATMPs) in January 2025, so it is a central topic for the European cancer community, Cardone noted. Although CPE welcomes the spirit of the Health Technology Assessment Regulation (HTAR), Cardone said it is not clear when and where patients will be involved in the process, under which criteria and how patients’ input will be taken into consideration.

While all indications are that the framework will incorporate the patient voice, “the devil will be in the details of the final implementing acts”, Paardekooper noted . “We need to ensure patients are heard and truly involved in the process and it’s not just a tick-the-box exercise.”

In disease areas such as cancer, patients are often at the forefront of informing both other patients and clinicians about innovative treatments in the pipeline, Olsen said. “It’s a natural fit to involve patients in the HTA, not only because it concerns them, but also because patients have unique experiences that should be leveraged,” he said. “This is why some national authorities are involving patients in the HTA in a systematic manner. Patients can provide important information about, for example, what types of outcomes to look at, which will help HTA bodies to determine that outcomes that have been studied are ones that matter to patients.” 

“Where the patient voice will be of particular importance is with rare diseases, where there is often less natural history of the disease itself, and regarding the standard of care, which can vary between member states,” Olsen added. 

Overcoming the challenges with embedding the patient voice

Cardone listed a number of challenges the JCA process must confront. One is finding a way to standardize when and how in the JCA process the patient input is expected, and then integrating that input in the decision-making process. 

“We would like to see a more pragmatic approach to setting the scope,” she said. “Ideally, a common comparator and common endpoints should be identified to avoid duplication and achieve the spirit of accelerating access to new treatments. We also must consider primary endpoints and patient reported endpoints. Additionally, patient preference surveys, run by patient organizations, real-world data on the technology or the history of the disease, and real-world outcomes should be considered by the assessors.”

Cardone pointed to existing best practice for facilitating and standardizing patient input in the JCA, such as the PREFER project for patient preference , and the Summary of Information for Patients (SIP) template, which is currently being piloted by the U.K,’s National Institute for Health and Care Excellence (NICE)³. All this will be key to avoiding the risk of inconclusive recommendations.

The wording of the draft framework refers to the inclusion of patient experts, which Paardekooper noted sounds good in theory but there is a risk that an individual patient won’t have the broader expertise needed. “It’s important that we have patient representatives that talk on behalf of broader groups of patients and that are also a little bit more literate in the process,” he said. “It also should be remembered that while it’s a central European process, it is being done in collaboration with all the different European member states and HTA bodies. So, patient advocacy groups in these countries need to have a voice.”

He added that there is also a need to educate all stakeholders, especially patient advocates, on the details of the legislation so they can participate. 

Cardone agreed that involving individual patients poses a challenge, including potential conflict of interest. 

“Patient organizations not only bring the views of the community, but it would be much easier to control and check the conflict of interest of an organization rather than an individual,” she said. “We share this concern with other stakeholders, such as health care providers. We are discussing this issue also within a multi stakeholder platform called the European Access Academy , as we would like to identify the role of each stakeholder in the HTA process.” 

 

Ensuring involvement of all relevant stakeholders to increase the quality of assessments

While the JCA seeks to do away with burdensome processes, it’s important not to lose elements of the current process that work, Olsen told the panel.

“Currently, companies and HTA bodies engage and learn from one another to inform the assessment process while being careful to ensure assessment decisions aren’t unduly influenced,” he said. “This can even begin before the start of HTA, with early pipeline meetings where companies can inform HTA bodies about upcoming technologies, so assessors can prepare for methodological challenges that might arise.” 

Olsen added that early scientific advice, such as Joint Scientific Consultations can help companies prepare for an eventual HTA submission, and that scoping meetings can help HTA bodies consider the relevant standard of care and how that varies across member states. What are the current products in use? What is the right comparison for a new product? This is where companies can provide valuable information based on their own insights, Olsen said, adding that it is important to maintain the scoping meeting in the new procedure. “If that is lost, there is a risk of discontinued or delayed assessments, which would delay patient access to innovative treatments.”

 

Preparing patient organizations for the new JCA process

While patient organizations at the European level are better prepared for the JCA, associations at a national level are lagging behind, Cardone said.  There are training modules available to help patient organizations and advocates participate in the HTA process^5,6. 

“We are working with our members and associates to prepare them to be involved in the HTA system,” Cardone said. “The challenge, however, is that there might not be enough patients, especially at a country level, willing to be involved in the system. So, while we need to prepare patients, the system also needs to be simplified to facilitate more patients to be involved.”

Industry can also play a role in helping to embed the patient voice in the JCA, panelists noted. 

“The purpose of any medical innovation is to improve patient outcomes, so it’s important that companies engage with patients to better understand which outcomes really matter to them,” Paardekooper said. “By teaming up with patient advocacy groups, companies can advocate for transparent, harmonized and pragmatic processes that really matter to patients in clinical practice.”

Paardekooper also highlighted the role companies could play in supporting and educating advocacy groups on the new JCA process. Additionally, companies should be working with their respective industry associations, such as EUCOPE and the European Federation of Pharmaceutical Industries and Associations (EFPIA) at the European level as well as local associations. 

Cardone said that such engagement should be conducted early on, ideally by Phase 1 clinical trials. “When involving patients in designing a clinical trial, it is important to report back to the patients on how their inputs have been taken into consideration,” she said. By the time the HTA process begins, she added that it is important that patients and industry remain separate. 

 

Keeping streamlined processes and the care of patients top of mind

As Cardone said during the discussion, a priority for all stakeholders should be to reduce waste of resources and avoid duplication, because ultimately this will help to accelerate patient access to innovative medicines while saving money. 

Paardekooper summarized by urging the HTA coordination group that is taking over the JCA preparation to include patients as a key and equal stakeholder in the process. 

 

Listen to the webinar recording to hear the views of the panel of experts and to learn more about the role of patients in the JCA.

 

The contents of this article may contain opinions of the webinar panel and do not represent the opinions of Vintura, PharmaLex or their parent Cencora. Vintura, PharmaLex and Cencora strongly encourage readers to review the references provided with this article and all available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto.

¹Regulation on Health Technology Assessment, European Commission. https://health.ec.europa.eu/health-technology-assessment/regulation-health-technology-assessment_en

²PREFER Patient Preferences, Innovative Medicines Initiative. https://www.imi-prefer.eu/

³Summary of Information for Patients (SIP), Health Technology Assessment International. https://www.nice.org.uk/guidance/GID-TA10656/documents/committee-papers-2

⁴European Access Academy, https://www.euaac.org/ 

⁵EUCAPA - European Capacity Building for Patients, https://www.eucapa.eu/

⁶HTA4Patients: Bringing Health Technology Assessment closer to patients, EUPATI. https://eupati.eu/hta4patients/