The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study

Murphy, Aisling A.; Bere, Nathalie; Vamvakas, Spiros; Mavris, Maria

doi:10.3389/fmed.2021.811855

Cited by 20 publications

(15 citation statements)

References 4 publications

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“…Systematic data collection on the natural history of a disease, for which a (first) drug target is discovered, may be key to making subsequent or parallel drug development and regulatory decision-making more efficient. Early interaction between drug developers, patient organizations, and registry holders is crucial for defining patient-relevant outcomes for clinical trials with a new active substance (Murphy et al, 2021). In addition, parallel consultation between regulators and HTA focusing on RWD collection may accelerate the transition from drug development to clinical practice (European Medicines Agency, 2019).…”

Section: Discussionmentioning

confidence: 99%

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

et al. 2022

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Background: Reference to so-called real-world data is more often made in marketing authorization applications for medicines intended to diagnose, prevent or treat rare diseases compared to more common diseases. We provide granularity on the type and aim of any external data on efficacy aspects from both real-world data sources and external trial data as discussed in regulatory submissions of orphan designated medicinal products in the EU. By quantifying the contribution of external data according to various regulatory characteristics, we aimed at identifying specific opportunities for external data in the field of orphan conditions.Methods: Information on external data in regulatory documents covering 72 orphan designations was extracted. Our sample comprised public assessment reports for approved, refused, or withdrawn applications concluded from 2019–2021 at the European Medicines Agency. Products with an active orphan designation at the time of submission were scrutinized regarding the role of external data on efficacy aspects in the context of marketing authorization applications, or on the criterion of “significant benefit” for the confirmation of the orphan designation at the time of licensing. The reports allowed a broad distinction between clinical development, regulatory decision making, and intended post-approval data collection. We defined three categories of external data, administrative data, structured clinical data, and external trial data (from clinical trials not sponsored by the applicant), and noted whether external data concerned the therapeutic context of the disease or the product under review.Results: While reference to external data with respect to efficacy aspects was included in 63% of the approved medicinal products in the field of rare diseases, 37% of marketing authorization applications were exclusively based on the dedicated clinical development plan for the product under review. Purely administrative data did not play any role in our sample of reports, but clinical data collected in a structured manner (from routine care or clinical research) were often used to inform on the trial design. Two additional recurrent themes for the use of external data were the contextualization of results, especially to confirm the orphan designation at the time of licensing, and reassurance of a large difference in treatment effect size or consistency of effects observed in clinical trials and practice. External data on the product under review were restricted to either active substances already belonging to the standard of care even before authorization or to compassionate use schemes. Furthermore, external data were considered pivotal for marketing authorization only exceptionally and only for active substances already in use within the specific therapeutic indication. Applications for the rarest conditions and those without authorized treatment alternatives were especially prominent with respect to the use of external data from real-world data sources both in the pre- and post-approval setting.Conclusion: Specific opportunities for external data in the setting of marketing authorizations in the field of rare diseases were identified. Ongoing initiatives of fostering systematic data collection are promising steps for a more efficient medicinal product development in the field of rare diseases.

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Section: Discussionmentioning

confidence: 99%

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

et al. 2022

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“…This means collecting and sharing concrete evidence of the value of patient experience in improving outcomes. Ongoing research is investigating how this can be achieved most effectively [25] . Sharing best practice will encourage others to implement approaches that have been shown to work.…”

Section: Patient Empowermentmentioning

confidence: 99%

Patient agency: key questions and challenges – A report from the 1st workshop of the EHC Think Tank Workstream on Patient Agency

Bok¹,

Noone²,

Skouw-Rasmussen³

2022

The Journal of Haemophilia Practice

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Introduction Patient agency refers to the abilities and capabilities of patients to act, contribute, influence and make decisions about their healthcare. It depends on both the willingness of patients to participate and the constraints imposed by healthcare providers, services and systems. To determine the factors affecting patient agency, especially for patients with chronic, rare diseases such as haemophilia requiring lifelong care, it is important to consider the patterns, structures, and mental models that define the ecosystem that patients are a part of, irrespective of their level of engagement. Identifying key challenges At the first workshop of the EHC Think Tank Workstream on Patient Agency in December 2021, participants identified five key themes for in-depth discussion relevant to patient agency: the concept of shared decision-making (SDM), patient empowerment, the spectrum of engagement, cultural change and health literacy. The Iceberg Model was used to unpack challenges by identifying composite factors on four levels: events, patterns, structures and mental models. Summary Across the five themes, four common perceived challenges stand out: uneven relationships between patients and healthcare professionals, services and systems; paternalism and hierarchical cultures; failure to recognise problems; conservatism and resistance to change. Despite some progress towards patient empowerment, a ‘glass ceiling’ prevents patients from driving transformation and taking leadership roles in strategy, policymaking and governance. Patient engagement is fluid and those who could benefit most are least likely to engage. Health literacy is perceived as the problem of the patient, not the system, and patients rather than healthcare providers are typically expected to adapt. Preliminary suggestions for addressing these challenges include behavioural communication training for patients and healthcare professionals, a learning system for empowered patient and family care, and a level playing field for stakeholders to interact equally, leading to mutual acceptance and respect.

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“…Gerade in Therapiegebieten, für die es keine etablierten Endpunkte für die Messung des Therapieansprechens gibt, empfiehlt es sich, geeignete Endpunkte im Rahmen einer wissenschaftlichen Beratung im Vorfeld abzustimmen. Idealerweise werden hierfür auch Patienten(‑Organisationen) einbezogen, damit möglichst alltagsrelevante Endpunkte ausgewählt werden [ 9 ]. Bilden Endpunkte verschiedene, voneinander unabhängige Aspekte des Krankheitsverlaufs ab, ist es zudem möglich, einen Gesamteindruck von der Wirksamkeit zu gewinnen, was gerade bei der Interpretation von Studien mit sehr kleinen Fallzahlen hilfreich sein kann.…”

Section: Position Des Bundesinstituts Für Arzneimittel Und Medizinpro...unclassified

Evidenzbasierte Arzneimittelversorgung bei Seltenen Erkrankungen: die Rolle der Digitalisierung

Naumann‐Winter

Kaiser

Behring

2022

Bundesgesundheitsbl

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ZusammenfassungBei der Wissensgenerierung im Bereich der Arzneimittelentwicklung für Menschen mit Seltenen Erkrankungen (SE) sind besondere Schwierigkeiten zu überwinden. Welche Verbesserungen durch eine zunehmende Digitalisierung erwartet werden, wird in diesem Beitrag aus der Perspektive von 3 Institutionen im Gesundheitswesen aufgezeigt: dem Bundesinstitut für Arzneimittel und Medizinprodukte, dem Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen und dem Gemeinsamen Bundesausschuss.Zunächst wird das Potenzial der Digitalisierung vorgestellt, auch durch eine frühere Zusammenarbeit aller Beteiligten die Effizienz der klinischen Entwicklung und der regulatorischen Entscheidungsprozesse zu erhöhen. Im Anschluss wird argumentiert, dass mit Hilfe der Digitalisierung Hürden bei der Durchführung versorgungsnaher, auch registerbasierter randomisiert kontrollierter Studien abgebaut werden sollten. Hochwertige Registerstudien sollten nicht erst nach der Zulassung, sondern bereits während des Zulassungsprozesses begonnen werden, damit die für Therapieentscheidungen notwendige Evidenz zeitnah nach Zulassung vorliegt. Abschließend wird festgestellt, dass die Verbesserung der Datenlage durch qualitative Verbesserung der Datenquellen und deren Vernetzung unmittelbar den Patient*innen zugutekommt. Verwertbare Evidenz, die über einen längeren Zeitraum – auch über die Zulassung hinaus – generiert werden kann und geeignet ist, in Entscheidungen für das Gesundheitssystem einzufließen, stellt eine effektive Arzneimittelversorgung sicher.Die Institutionen sind sich einig, dass qualitativ hochwertige Indikationsregister als produktunabhängige, stehende Infrastrukturen entwickelt werden sollten, damit bereits früh in der Entwicklung von Arzneimitteln für SE auf hochwertige Daten zurückgegriffen werden kann.

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The Added Value of Patient Engagement in Early Dialogue at EMA: Scientific Advice as a Case Study

Cited by 20 publications

References 4 publications

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

Patient agency: key questions and challenges – A report from the 1st workshop of the EHC Think Tank Workstream on Patient Agency

Evidenzbasierte Arzneimittelversorgung bei Seltenen Erkrankungen: die Rolle der Digitalisierung

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