Health technology assessment (HTA) in England, France and Germany: What do matched drug pairs tell us about recommendations by national HTA agencies?

Aims: To explore health technology assessment (HTA) outcomes of matched drug pairs by national agencies in Germany (Gemeinsamer Bundesausschuss, GBA), France (Haute Autorité de Santé, HAS) and England and Wales (NICE). Methods: We considered published GBA decisions, HAS reports and NICE guidance from January 2011 to June 2018. HTAs of matched pairs were compared overall, and for non-cancer and cancer drugs separately. We further analyzed the role of additional attributes related to cancer therapies. Results: Matched pairs show higher concordance for GBA/HAS than for GBA/NICE and HAS/NICE. Overall, NICE evaluated technologies more favorably than GBA and HAS. GBA appraisals of cancer drugs, however, tended to be more positive than cancer-related recommendations by NICE and HAS. Conclusion: The findings indicate substantial variations in HTAs, although cancer-related outcomes seem to diverge less than non-cancer results.

Governance-Transformation durch Richterrecht? Juristische Diskurse zur Selbstverwaltung im Gesundheitswesen

Wesentliche Teile des Sozialversicherungsrechts wurden in Deutschland durch juristische Fachdiskurse und Gerichtsurteile bestimmt. Deren Folgen und Fernwirkungen blieben in der Regel unberücksichtigt. Dies betrifft neuerdings verstärkt die Selbstverwaltung im Gesundheitswesen. Von deren Spitzengremium „Gemeinsamer Bundesausschuss“ festgelegte medizinische Behandlungsnormen werten Juristen als Eingriff in das Grundrecht auf Leben und körperliche Unversehrtheit (Art. 2 II 1 GG), der dem von Verbandsvertretern bestellten Gremium nicht zustehe. Die der „Legitimationskettentheorie“ des Bundesverfassungsgerichtes folgende Wertung offenbart ein monistisches, von überkommenen Souveränitätslehren geprägtes Staatsverständnis, von dem sich die meisten westlichen Demokratien gelöst haben. Die daran anknüpfende Rechtsprechung tangiert das ordnungspolitische Gefüge von Staat, Mark und Organisationsgesellschaft. Der Beitrag erörtert einschlägige Gerichtsurteile und diskutiert kritische Einwände sowie demokratietheoretische Alternativkonzepte.

Aktuelle Betrachtung der Tonsillektomie und Tonsillotomie

What is right? Tonsillotomy or tonsillectomy? Moreover, when and what has to be preferred? Is the discussion never ending? No, and this is good. Tonsil surgery is and will remain a frequent surgical procedure for the otolaryngologist. Especially, because many children are operated, and because – also rare – significant complications can occur, a quality-controlled benefit-risk-analysis is important. Part of this is systematic analysis of the evidence of the benefits and risks of tonsillotomy and tonsillectomy, as it was performed recently by the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG) by order of the Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA). One result was the implementation of the tonsillotomy as a regular service paid by the insurance companies. Nevertheless, the discussion is not yet concluded. Actually, a randomized controlled trial is in preparation sponsored by the G-BA to proof the non-inferiority of tonsillotomy against tonsillectomy for the treatment of patients with recurrent acute tonsillitis. The present article on ENT specialist knowledge should put the reader, especially ENT residents, in a position to advice patients and their relatives on the pros and cons of tonsil surgery.

PP142 Health Technology Assessment – A Major Bottleneck In Patient Access?

IntroductionConditional marketing authorization (CMA) and accelerated assessment (AA) have been introduced to expedite the development of and access to therapies in Europe. However, to reach patients medicines must also be publicly reimbursed. This research evaluated the reimbursement of therapies which have received European CMA or underwent AA.MethodsMedicines that received CMA or underwent AA between January 2012 and December 2017 were identified. Appraisals of these medicines conducted by major European payer bodies were obtained from relevant websites and key data were extracted.ResultsOut of the 38 medicines that received a CMA, 83 percent (19/23) were assessed by the National Institute for Health and Care Excellence (NICE) and received positive decisions, compared with 57 percent (16/26) by the Scottish Medicines Consortium (SMC) (defined as recommended/restricted), 74 percent (14/19) by Gemeinsamer Bundesausschuss (G-BA) (defined as any level of additional benefit), and 29 percent by Haute Autorité de Santé (HAS) (amélioration du service médical rendu I-III). The median delay between CMA approval and positive health technology assessment (HTA) outcome was 13 months for NICE, 11 months for SMC, 7 months for G-BA, and 5 months for HAS. Thirty-two medicines underwent AA. Of these, 68 percent (17/25) were appraised by G-BA and received positive outcomes, compared with 29 percent (7/24) by HAS, 90 percent (19/21) by SMC, and 86 percent (18/21) by NICE. The median delay between AA approval and positive HTA outcome was 7.4 months for G-BA, 7.9 months for HAS, 11.7 months for SMC, and 11.8 months for NICE.ConclusionsCMA has expedited regulatory approval for products that address severe unmet needs. However, many of these products fail to gain public reimbursement, and even when they do there is a significant delay. AA provides market authorizations two months earlier than standard centralized assessment. Although high rates of positive payer outcomes have been achieved, the products typically experience substantial additional delays in securing public reimbursement. A parallel, cooperative approach among regulatory and HTA bodies across Europe is required to truly expedite patient access.

OP19 Does The HST Represent A Best Practice Model For Ultra-Orphan HTA?

IntroductionUltra-orphan therapies (prevalence: <1:50,000) can have trouble meeting Health Technology Assessment (HTA) clinical- and cost-effectiveness criteria, set by HTA bodies to inform reimbursement decision-making, due to low patient numbers limiting the supporting clinical evidence generated and high per-patient prices. Since 2013, National Institute of Health and Care Excellence (NICE) appraise Highly Specialised Technologies (HST) (“for use in the provision of services for rare and very rare conditions”) using a distinct appraisal framework. This research compares NICE HST appraisal outcomes with corresponding guidance by other HTA bodies.MethodsAll NICE HST technology guidance was screened (1 January 2013–6 November 2018) alongside corresponding guidance by Gemeinsamer Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), Scottish Medicines Consortium (SMC), and National Centre for Pharmacoeconomics (NCPE).ResultsNICE have published eight HST guidances all with positive recommendations after a median of 21 months (range: 7–38) after European Marketing Authorization (MA). An additional eight HST have guidance in-development despite having European MA for a median of 12 months (range: 2–46) with 5/8 having draft guidance issued, all being “not recommended”. Of the 18 HSTs with NICE guidance published/in-development, 29 percent (2/7), and 33 percent (2/6) have been assessed with positive outcomes (definition: “recommended”/”accepted”/“conditional”/”restricted”) by SMC, and NCPE, respectively vs. 100 percent (9/9) by G-BA (definition: any additional benefit), and 50 percent (5/10) by HAS (definition: ASMR I-III). Median delays between European MA and positive appraisal outcomes were seven (G-BA), nine (HAS), 12 (NCPE), and 19.5 months (SMC).ConclusionsAlthough all NICE HST final guidances to date have been positive, few technologies have completed this process after substantial delays from MA. Other cost/QALY HTA bodies (i.e. excluding the G-BA and HAS clinical-assessment HTAs) have shown low appraisal and recommendation rates for these technologies; therefore, ultra-orphan technologies may require a distinct appraisal process/framework but the HST may not (yet) represent best-practice.

Die externe Qualitätssicherung im Krankenhausrecht

Quality assurance has recently come to the forefront of German health legislation. The author discusses objectives and methods of quality management in the hospital sector and investigates their implementation in social law and hospital law while also taking into account constitutional law. The focus of this treatise are the quality measures by the G-BA (Gemeinsamer Bundesausschuss) [Federal Joint Committee] and implementations of quality standards in German hospital planning. The author presents a comprehensive overview with a distinctive consideration of the differing legislative powers of the Federal Government and the federal states, consequences of subjective rights and current problems of the execution of single measures like minimum quantity regulation. Result: the consolidation of the democratic legitimation of the G-BA is required. A clear delimitation of the competences of the Federal Government and the federal states is necessary. The author has been working as a lawyer focusing on German hospital law for several years. The publication is aimed at lawyers and legal practitioners in health law.

ESCAlate – Ein adaptiver Behandlungsansatz für Jugendliche und Erwachsene mit ADHS

Zusammenfassung. In der Behandlung der adulten ADHS haben sich sowohl pharmakologische Interventionen als auch psychosoziale Behandlungen als wirksam erwiesen. In der täglichen klinischen Routine wird die Behandlung jedoch weniger von den Ergebnissen klinischer Studien als von Behandlungsrichtlinien und Anforderungen nationaler Institutionen (Gemeinsamer Bundesausschuss) beeinflusst. Der Hauptaspekt dieser Vorschriften ist die Anforderung, dass ein stufenweiser Behandlungsansatz, beginnend mit niederschwelligen Interventionen, am sinnvollsten für die Behandlung bei adulten ADHS-Patienten ist. Leider gibt es bis heute fast ausschließlich klinische Studien, welche die Auswirkungen einzelner Therapiestrategien erforschen. Das gestufte Versorgungsmodell scheint noch nicht ausreichend validiert. Genau hier möchte die im Folgenden beschriebene ESCAlate-Studie ansetzen. Bei ESCAlate handelt es sich um eine randomisierte, kontrollierte Studie. Es sollen 279 Patienten im Alter zwischen 16.00 und 45.11 Jahren in das Behandlungsprogramm, welches sich in mehrere Abschnitte gliedert, aufgenommen werden. In einem ersten Behandlungsabschnitt werden die Patienten nach dem Zufallsverfahren in drei Gruppen eingeteilt: Psychoedukation im Einzelsetting (PE), Telefonassistierte Selbsthilfe (TASH) oder Wartekontrollgruppe. Alle Patienten der Wartegruppe erhalten nach dreimonatiger Wartezeit eine Behandlung mit TASH. Im zweiten Behandlungsabschnitt werden die Patienten entsprechend der Schwere ihrer persistierenden Symptome in die drei Gruppen Full-Responder, Partial-Responder und Non-Responder eingeteilt. Patienten, welche als Full-Responder eingestuft wurden erhalten ein verhaltenstherapeutisch orientiertes Coaching. Partial-Responder erhalten ebenfalls dieses Coaching, wobei in dieser Gruppe der Zufall entscheidet, ob die Patienten zusätzlich ein Neurofeedbacktraining (NF) erhalten. Non-Responder erhalten eine pharmakologische Behandlung mit Methylphenidat. Hier entscheidet ebenfalls der Zufall, ob die Patienten zusätzlich ein Neurofeedbacktraining erhalten. ESCAlate zeichnet sich durch eine relativ naturalistische Stichprobenzusammensetzung aus, da auf hochspezifische Ein- und Ausschlusskriterien verzichtet wurde, um eine Patientenstichprobe zu erhalten, die die Patienten der alltäglichen klinischen Routine in den Praxen widerspiegelt. Die Wirksamkeit einer evidenzbasierten Intervention mit gestufter Behandlung wird durch primäre (Verringerung des Schweregrads der ADHS-Symptome) und sekundäre Ergebnisse (funktionelle Ergebnisse; z.B. Lebensqualität, Ärger-Management, Steigerung des psychosozialen Wohlbefindens) untersucht. Prädiktoren für therapeutisches Ansprechen bzw. Nicht-Ansprechen werden bei jedem Schritt der Intervention evaluiert. Darüber hinaus können eventuelle geschlechtsspezifische Unterschiede untersucht werden.