Webinar Report: “Implementing Real World Data to Make Adaptive Pathways Work”

On 18 October 2017, the ADAPT SMART consortium presented a webinar hosted by the Estonian Ministry of Social Affairs. The webinar was the final event of eHealth week, an international conference occurring as part of Estonia’s Presidency of the European Council, and discussed how Eastern European countries are implementing real-world data (RWD) tools that can be used to provide the data infrastructure needed to make Adaptive Pathways work.

Medicines Adaptive Pathways to Patients, or MAPPs, promise to provide a tool for placing new, innovative medicines on the market for those patients with the greatest unmet need by harnessing RWD to validate the effectiveness and safety of new therapies. The ADAPT SMART (Sustainable, Multi-stakeholder Approach from Research to Treatment outcomes) consortium enables coordination of activities, supporting investigation into methodologies and dialogue to develop workable concepts for MAPPs.

The webinar began with a presentation by the Estonian Ministry of Social Affairs discussing their best practices in the use of electronic health data and patient records, and how they are facilitating improved treatment pathways. Ain Aaviksoo, Deputy Secretary General on E-services Development and Innovation, said, “our central database accessed by doctors, patients and hospitals, called Digilugu, gives exactly the same feedback to the patients as it gives to the family doctors and the hospitals; it is completely transparent.” Deputy Secretary Aaviksoo also highlighted that the patient has complete control over what personal data is shared with the various members of the healthcare value chain. When asked what percentage of the population actively manages their data, he answered, “Currently the data is actively managed by 30% of the patients, and by ‘actively’, we mean they log in to their data portal at least once a month.”

Stanimir Hasurdjiev, the Secretary General of the Patient Access Partnership, focused his presentation on the current situation regarding the challenges and solutions to harnessing RWD in Central and Eastern Europe (CEE). Amongst the many factors, he pointed out that political instability is a primary cause, impacting the ability to implement health data solutions. “Even if we have governments that are willing to implement the tools needed, very often they change and then everything starts over with the new administration,” said Mr. Hasurdjiev, pointing out that this lack of political certainty is the key hindrance to committing resources to long-term health IT projects.

Presenting the evolution of IMI for projects that impact the use of patient data for improved access and earlier discussions on regulatory requirements with key stakeholders, Thomas Allvin, Executive Director Strategy and Healthcare Systems at EFPIA, emphasized that building upon the success of a series of IMI projects such as EHR4CR, EMIF, and GetReal has lead to the Big Data for Better Outcomes initiatives under IMI2, “where we are really diving into the use of RWD for solutions to specific disease areas,” said Allvin.

Representing the ADAPT SMART consortium, Programme Manager Pieter Stolk addressed several of the concerns being raised about early access harnessing RWD in the context of MAPPS. Pieter pointed out that, “Randomised clinical trials (RCTs) are the methodology with the highest internal validity. But for an efficient increase in the benefits and risks of new treatments, we need to embrace the full evidence spectrum, not only RCTs, pragmatic trials, and different types of observational studies.” He concluded by saying, “RWD complements rather than replaces RCTs: the right study type [should be used] for the right question – where feasible.”

The final portion of the webinar was opened up to questions and answers from the audience. Bror Jonzon, from the Swedish Medical Products Agency, asked, “Is randomised clinical trial data not considered to be ‘real world data’ under the current definition?” Pieter Stolk emphasized that ADAPT SMART had used the definition of RWD from the GetReal project, and the assumption that a clinical trial can generate RWD, depending on the setting of the study.

The webinar concluded with Ain Aaviksoo emphasizing that, “It is vital that we make use of the opportunities that are available to us, not only in a theoretical way, but through running actual projects that we can use. Under the Presidency, we’ve put together a data ‘coalition of the doing’ and we are keen to meet the relevant people − clinicians, researchers and those in industry − to make this initiative real.”

A complete video archive and all of the slides presented in this webinar can be found at here.

The ADAPT SMART Coordination and Support Action (CSA) acts as a neutral collaborative framework to establish the platform that will engage with all relevant stakeholders, including patients, industry, SMEs (small- and medium-sized enterprises), regulators, health technology assessment bodies (HTAs), payers (national and European networks), clinicians, governments/policy makers (national authorities, as well as the European Commission’s DG SANTE and DG GROW, and European networks). In addition to engaging in a dialogue with relevant stakeholders, the ADAPT SMART consortium aims to align understanding on the impact of MAPPs, to share findings between all stakeholders, and to allow the field to actively work towards MAPPs implementation.