RWE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 16.

In this update, we discuss recent US FDA guidance offering more specific guidelines on appropriate study design and analysis to support causal inference for non-interventional studies and the launch of the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) public electronic catalogues. We also highlight an article recommending assessing data quality and suitability prior to protocol finalization and a Journal of the American Medical Association-endorsed framework for using causal language when publishing real-world evidence studies. Finally, we explore the potential of large language models to automate the development of health economic models.

Access in all areas? a round up of developments in market access and health technology assessment: part 4.

In this latest update, we look at recent developments in market access including the pricing agreement of Libmeldy® by the Beneluxa Initiative, the financial impact of managed entry agreements in Italy and the restructuring of Agenzia Italiana del Farmaco (AIFA). We also highlight the collaboration between FINOSE and the New Expensive Drug (NED) section of the Nordic Pharmaceutical Forum.

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 15.

In this latest update we discuss real-world evidence (RWE) guidance from the leading oncology professional societies, the American Society of Clinical Oncology and the European Society for Medical Oncology, and the PRINCIPLED practical guide on the design and analysis of causal RWE studies.

Access in all areas? A round up of developments in market access and health technology assessment: part 3.

In this latest update, we explore some of the key updates in market access over recent months including the UK's voluntary scheme for branded medicines pricing, access and growth (VPAG), the first drugs funded by the Innovative Medicines Fund in the UK and the Direct Access Scheme in France, and, finally, the new Institute for Clinical and Economic Review (ICER) value assessment framework in the USA.

Is the price right? Paying for value today to get more value tomorrow.

BACKGROUND: Contemporary debates about drug pricing feature several widely held misconceptions, including the relationship between incentives and innovation, the proportion of total healthcare spending on pharmaceuticals, and whether the economic evaluation of a medicine can be influenced by things other than clinical efficacy. MAIN BODY: All citizens should have access to timely, equitable, and cost-effective care covered by public funds, private insurance, or a combination of both. Better managing the collective burden of diseases borne by today's and future generations depends in part on developing better technologies, including better medicines. As in any innovative industry, the expectation of adequate financial returns incentivizes innovators and their investors to develop new medicines. Estimating expected returns requires that they forecast revenues, based on the future price trajectory and volume of use over time. How market participants decide what price to set or accept can be complicated, and some observers and stakeholders want to confirm whether the net prices society pays for novel medicines, whether as a reward for past innovation or an incentive for future innovation, are commensurate with those medicines' incremental value. But we must also ask "value to whom?"; medicines not only bring immediate clinical benefits to patients treated today, but also can provide a broad spectrum of short- and long-term benefits to patients, their families, and society. Spending across all facets of healthcare has grown over the last 25 years, but both inpatient and outpatient spending has outpaced drug spending growth even as our drug armamentarium is constantly improving with safer and more effective medicines. In large part, this is because, unlike hospitals, drugs typically go generic, thus making room in our budgets for new and better ones, even as they often keep patients out of hospitals, driving further savings. CONCLUSION: A thorough evaluation of drug spending and value can help to promote a better allocation of healthcare resources for both the healthy and the sick, both of whom must pay for healthcare. Taking a holistic approach to assessing drug value makes it clear that a branded drug's value to a patient is often only a small fraction of the drug's total value to society. Societal value merits consideration when determining whether and how to make a medicine affordable and accessible to patients: a drug that is worth its price to society should not be rendered inaccessible to ill patients by imposing high out-of-pocket costs or restricting coverage based on narrow health technology assessments (HTAs). Furthermore, recognizing the total societal cost of un- or undertreated conditions is crucial to gaining a thorough understanding of what guides the biomedical innovation ecosystem to create value for society. It would be unwise to discourage the development of new solutions without first appreciating the cost of leaving the problems unsolved.

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 14.

In this latest update we highlight: a publication from the US FDA regarding the definitions of real-world data (RWD) and real-world evidence (RWE); a publication from academic researchers on a demonstration project for target trial emulation; a publication from the National Institute of Health and Care Excellence (NICE) on the 1 year anniversary of their RWE framework; and a publication from NICE and Flatiron Health on the utility of US RWD for initial UK health technology assessment decision making.

Access in all areas? A round up of developments in market access and health technology assessment: part 2.

In this latest update, we explore the Inflation Reduction Act (IRA) enacted by the US Congress in August 2022, with the Centers for Medicare and Medicaid Services (CMS) recently releasing the list of the first ten drugs it will negotiate prices on. We also cover the consequences of price controls and rigid value assessment in Germany which have led to the withdrawal of a number of medicines. It will be important to see how the IRA balances cost-saving with holistic value assessment, incentives for innovation and patient access to treatment.

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 13.

In this latest update we highlight a report from the European Medicines Agency on their use of real-world evidence (RWE) in decision making, RWE reporting guidance from the Canadian Agency for Drugs and Technologies in Health and highlight some new data demonstrating the value medicines for spinal muscular atrophy have brought patients.

Access in all areas? A round up of developments in market access and health technology assessment: part 1.

In this new series reviewing recent developments in market access, we highlight publications investigating health technology assessment (HTA) guidance, review processes and outcomes across the world and discuss how forthcoming changes in the HTA and regulatory environment in the European Union may allow for more consistency in decision making.

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 12.

In this latest update we highlight the final results from the RCT-DUPLICATE initiative, the publication of guidance from Haute Autorité de Santé (HAS), the joint viewpoint from the Institute for Quality and Efficiency in HealthCare (IQWIG) and the Belgian HealthCare Knowledge Center, and a position from the European Organization for Research and Treatment of Cancer (EORTC). Finally, we discuss how the NICE RWE framework has been implemented to allow consideration of RWE external control arms.