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Evidence-based indications for the planning of PET or PET/CT capacities are needed

Purpose To identify evidence-based indications for PET/PET–CT scans in support of facilities planning and to describe a pilot project in which this information was applied for an investment decision in an Austrian region. The study updates a Health Technology Assessment (HTA) report (2015) on oncological indications, extending it to neurological indications and inflammatory disorders. Methods A systematic literature search to identify HTA reports, evidence-based guidelines, and systematic reviews/meta-analyses (SR/MA) was performed, supplemented by a manual search for professional society recommendations and explicit “not-to-do’s”. A needs-assessment was conducted in the context of the pilot study on investing in an additional PET–CT scanner in the Austrian region of Carinthia. Results Overall recommendations for indications as well as non-recommendations for the three areas (oncology, neurology, and inflammatory disorders) were compiled from the 2015 PET–HTA report and expanded for a final total of ten HTA, comprising 234 (positive and negative) recommendations from professional societies and databases, and supplemented by findings from 23 SR/MA. For the investment decision pilot study in Carinthia, 1762 PET scans were analyzed; 77.8% were assigned to the category “recommended evidence-based indications” (54.7%), “not recommended” (1.8%) or “contradictory recommendations” (21.3%). The remaining could not be assigned to any of the three categories. Conclusions The piloting of PET capacity planning using evidence-based information is a first of its kind in the published literature. On one hand, the high number of PET scans that could not be ascribed to any of the categories identified limits to the instructive power of the study to use evidence-based indication lists as the basis for a needs-assessment investment planning. On the other hand, this study reveals how there is a need to improve indication coding for enhanced capacity planning of medical services. Overall recommendations identified can serve as needs-based and evidence-based decision support for PET/PET–CT service provision

Utilisation of the ESMO-MCBS in practice of HTA

It is highly appreciated that the European Society of Medical Oncology has developed a system to assess new oncologic compounds according to their value to patients. Consequently, offering decision-support to those who either want to use the new cancer therapies in clinical practice but cannot keep up-to-date with all therapy options or, alternatively, to those who have to decide whether or not to fund new oncology medicines or exclude from reimbursement due to their low value. This is particularly important with ever-rising prices for new oncology medicines which have increased up to ten fold in recent years

Approval of cancer drugs with uncertain therapeutic value: a comparison of regulatory decisions in Europe and the United States

Policy Points Regulatory agencies may have limited evidence on the clinical benefits and harms of new drugs when deciding whether new therapeutic agents are allowed to enter the market and under which conditions, including whether approval is granted under special regulatory pathways and obligations to address knowledge gaps through postmarketing studies are imposed. In a matched comparison of marketing applications for cancer drugs of uncertain therapeutic value reviewed by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), we found frequent discordance between the two agencies on regulatory outcomes and the use of special regulatory pathways. Both agencies often granted regular approval, even when the other agency judged there to be substantial uncertainty about drug benefits and risks that needed to be resolved through additional studies in the postmarketing period. Postmarketing studies imposed by regulators under special approval pathways to address remaining questions of efficacy and safety may not be suited to deliver timely, confirmatory evidence due to shortcomings in study design and delays, raising questions over the suitability of the FDA's Accelerated Approval and the EMA's Conditional Marketing Authorization as tools for allowing early market access for cancer drugs while maintaining rigorous regulatory standards. Context: Regulatory agencies are increasingly required to make market approval decisions for new drugs on the basis of limited clinical evidence, a situation commonly encountered in cancer. We aimed to investigate how regulators manage uncertainty in the benefit-risk profiles of new cancer drugs by comparing decisions for the world's two largest regulatory bodies—the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA)—over a 5-year period. Methods: We systematically identified a set of cancer drug-indication pairs for which data on efficacy and safety was less complete than that required for regular approval at time of market entry from 2009 to 2013, as determined by the FDA's use of Accelerated Approval (AA) or the EMA's use of Conditional Marketing Authorization (CMA) pathways, and matched these across the two agencies. Using publicly available information, we compared regulatory pathways and outcomes, final approved indications, and postmarketing obligations imposed by the agencies. Findings: We identified 21 cancer drug-indication pairs that received FDA AA, EMA CMA, or both. Although most applications relied on identical pivotal trials across the FDA and the EMA, regulatory pathways often differed; 57% of indications received either FDA AA or EMA CMA, and regular approval by the other agency. After approval, the EMA more often accepted single-arm studies to confirm clinical benefit compared to the FDA (75% vs. 29% of indications), and the FDA more commonly requested randomized controlled trials (85% vs. 50%). Forty-one percent of confirmatory trials after FDA AA were conducted in different populations than the approved indication, compared to 13% after EMA CMA. Both agencies relied primarily on surrogate measures of patient benefit for postmarketing obligations. After a median follow-up of 7.25 years, 40% of FDA and 61% of EMA postmarketing obligations after AA and CMA, respectively, were delayed. Conclusions: US and European regulators often deemed early and less complete evidence on benefit-risk profiles of cancer drugs sufficient to grant regular approval, raising questions over regulatory standards for the approval of new medicines. Even when imposing confirmatory studies in the postmarketing period through special approval pathways, meaningful evidence may not materialize due to shortcomings in study design and delays in conducting required studies with due diligence

Policy change and the National Essential Medicines List development process in Brazil between 2000 and 2014 : has the Essential Medicine concept been abandoned?

Brazil has had a National Essential Medicines List (EML) since 1964. From 2000 to 2010, five consecutive evidence-based editions were produced, building on the essential medicines concept. In 2012, the government changed course to establish a new paradigm, introducing adoption of new medicines as the main aim within the recommendation process. The objective of the paper is to report efforts to develop Brazil´s national EML, policy changes from 2000 to 2014, discussing results, challenges, and perspectives. Brazilian EML history and development process were collected from legislation, minutes, reports and legal ordinances, from 2000 to 2014. The Brazilian EML and the WHO Model lists were compared using the Anatomical Therapeutic Chemical system. Overlap between lists was verified and linear trends were produced. Type of membership, inclusion criteria, procedures, flow and listed medicines varied greatly between the selection committees acting before and after 2012. Paradigm-changing legislation aiming at linking list compliance to public financing in 2012 produced (i) greater importance given to political and administrative stakeholders, (ii) increasing trends in number of medicines over the years, (iii) decrease in use of WHO Model List as a reference, (iv) substitution of an essential medicines list review and update process by an adoption decision output. Other issues remained unchanged. Insufficient efforts for list implementation, such as lack of physician education, presented consequences to the health system. Substantial efforts were made to produce and update the list from 2000 to 2014. However, continuous and intense health litigation disproves process outcome effectiveness

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