Four scenarios for the future of medicines and social policy in 2030.

The future of medicines is likely determined by an array of scientific, socioeconomic, policy, medical need, and geopolitical factors, with many uncertainties ahead. Here, we report from a scenario project, analyzing various trends, crucial and complex developments in the medicines' space. From a range of 'critical uncertainties' we derived two scenario drivers: global convergence, ranging from very high (trust and solidarity), to very low (fragmented ecosystems); and disease orientation, ranging from public health first to interceptive medicine. This resulted in four contrasting portraits of the future of medicines and social policy: deprioritizing the high-end; sustainable flow; transformative healing; and global divide. All those involved in drug discovery and development can use these for strengthening preparedness for the crucial challenges ahead.

Global consumption of prescription opioid analgesics between 2009-2019: a country-level observational study.

BACKGROUND: Opioid analgesics play a key role in pain management but providing access while mitigating risk of misuse and dependence remains a challenge. Tracking global consumption of all opioids over time can help identify emerging patterns and drivers of use. METHODS: Prescription opioid analgesic consumption was estimated for 76 countries between 2009 and 2019 using IQVIA MIDAS data. We reported country-level consumption trends in morphine milligram equivalents (MMEs), assessed differences in consumption between high-income (HICs), upper-middle income (UMICs), and low- and lower-middle income countries (LMICs), and identified country-level socioeconomic drivers of consumption using fixed-effects panel regression models. FINDINGS: Global opioid consumption rate declined from 216·3 to 151·5 morphine milligram equivalents per 1,000 inhabitants per day (MID) between 2009 and 2019, with consumption declines in the US and Germany. Overall, consumption rates increased in HICs by a median 36·6 MID (IQR, -7·5 -124·5) with substantial heterogeneity between countries. Median consumption rates were lower in UMICs (23·6 MID) and LMICs (8·3 MID) compared to HICs (345·1 MID) and increased by median 10·4 and 3·7 MID from 2009-2019, respectively. Consumption rates were associated with income (coefficient 18·84, 95% confidence interval 3·8-33·9) and trade (13·59, 1·3-25·8) in UMICs, and physician density (1·95, 1·2-2·7) in LMICs. Tramadol consumption rate increased in the study period and accounted for a relatively large proportion of total opioid volume consumed across all country-income groups. INTERPRETATION: Substantial heterogeneity in global opioid consumption patterns reflect the challenges involved with providing adequate access to opioid treatment while avoiding potential misuse.

Assessing Participation Burden in Clinical Trials: Introducing the Patient Friction Coefficient.

Protocol design complexity, and associated study volunteer burden, negatively impact patient recruitment and retention as well as overall research and development productivity. Complex protocols reduce the willingness of potential clinical trial participants to enroll and reduce retention rates. There have been few systematic assessments of protocol design characteristics to determine the burden placed on study volunteers, although such an assessment would offer a compelling opportunity to optimize trial designs and improve recruitment and retention performance. To be useful, an assessment would need to be patient-centric, and focused on the factors that influence participation throughout the clinical trial. Such an assessment would also need to accommodate the unique cost-value trade-off compared with current treatment patterns that each participant makes when choosing to participate and remain in a clinical trial. This article proposes a new methodology to quantify patient burden: the clinical trial patient friction coefficient (PFC). A case example is provided to illustrate the utility of the PFC. A number of applications for the PFC are envisioned: standardizing patient burden assessment to evaluate clinical trial design feasibility, shedding light on the impact of patient burden on clinical trial economics and performance, and conducting sensitivity analyses to identify factors that most reduce patient burden and improve the performance and efficiency of clinical trials.

Reconciling the Seemingly Irreconcilable: How Much Are We Spending on Drugs?

BACKGROUND: Estimates of drug spending are often central to the public policy debate on how to manage healthcare spending in the United States. Nevertheless, common estimates of prescription drug spending vary substantially by source, which can inhibit productive policy dialogue. OBJECTIVES: To review publicly reported estimates of drug spending and uncover the underlying methodological inputs that drive the substantial variation in estimates of prescription drug spending. METHODS: We systematically evaluated 5 estimates of drug spending to identify differences in the underlying methodological inputs and approaches. To uniformly assess and compare estimates, we developed a model to identify the inputs of 3 primary components associated with each estimate: numerator (How is drug cost measured?), denominator (How is healthcare cost measured?), and population (What group of individuals is included in the measurement?). We then applied standardized methodological inputs to each estimate to assess whether variation among estimates could be reconciled. We then conducted a sensitivity analysis to address important limitations. RESULTS: We found that the 18.8 percentage point range in the publicly reported estimates is predominately attributed to methodological differences. Reconciling estimates using a standardized methodological approach reduces this range to 4.0 percentage points. CONCLUSIONS: Because variation in estimates of drug spending is primarily driven by methodological differences, stakeholders should seek to establish a mutually agreed upon methodological approach that is appropriate for the policy question at hand to provide a sound basis for health spending policy discussions.

Preparing for the incoming wave of biosimilars in oncology.

With the imminent arrival of oncology biosimilars in the therapeutic paradigm, stakeholders including a clinician, specialist nurse, patient advocate, regulator and economist provide their perspective on optimising the uptake of these new agents in the treatment of cancer. A number of key messages emerge, based on the discussion that took place during a session of the European Society for Medical Oncology's Annual Congress, ESMO Madrid 2017. First, for successful integration of biosimilars into the global healthcare paradigm, informing and educating the full scope of stakeholders, including clinicians, nurses, pharmacists and patients, is primordial. Success is dependent on providing solid evidence and ensuring all voices are heard. Second, for oncology medicines, much can be learnt from the growing experience of approved biosimilars in other disease indications, with success stories for patients, their healthcare providers and healthcare budgets alike. Finally, effective sustainability of the impact on healthcare budgets and the redirection of these savings require education and transparency.

The future of drug development: the paradigm shift towards systems therapeutics.

Progress in cell biology, genetics, molecular, and systems pharmacology is the driving force behind a current paradigm shift in drug research. This paradigm shift shapes new avenues for advanced treatments that are commonly referred to as 'systems therapeutics'. Systems therapeutics differ in many ways from current drugs because they target biological networks rather than single transduction pathways, and affect disease processes rather than physiological processes. Here, we examine how the paradigm shift towards systems therapeutics will change current scientific concepts of the interactions between drugs and diseases, the organization of research and development, as well as the clinical use and therapeutic evaluations of therapeutic interventions.

Has The Era Of Slow Growth For Prescription Drug Spending Ended?

In the period 2005-13 the US prescription drug market grew at an average annual pace of only 1.8 percent in real terms on an invoice price basis (that is, in constant dollars and before manufacturers' rebates and discounts). But the growth rate increased dramatically in 2014, when the market expanded by 11.5 percent-which raised questions about future trends. We determined the impact of manufacturers' rebates and discounts on prices and identified the underlying factors likely to influence prescription spending over the next decade. These include a strengthening of the innovation pipeline; consolidation among buyers such as wholesalers, pharmacy benefit managers, and health insurers; and reduced incidence of patent expirations, which means that fewer less costly generic drug substitutes will enter the market than in the recent past. While various forecasts indicate that pharmaceutical spending growth will moderate from its 2014 level, the business tension between buyers and sellers could play out in many different ways. This suggests that future spending trends remain highly uncertain.

Why life sciences companies need to tap technology to gain a competitive edge-and what that means for the chief information officers (CIO) role.

Fundamental changes in the marketplace for medicines, as well as the rapid and continuing evolution of technology are bringing new challenges and opportunities for life sciences companies and their contribution to healthcare systems. These changes bring pressure on chief information officers (CIOs) within the healthcare industry to play an increasingly strategic role in advancing business success and delivering digital transformation.

Decline in economic returns from new drugs raises questions about sustaining innovations.

The sales and financial returns realized by pharmaceutical companies are a frequent topic of discussion and debate. In this study we analyzed the economic returns for four cohorts of new prescription drugs launched in the United States (in 1991-94, 1995-99, 2000-04, and 2005-09) and compared fluctuations in revenues with changing average research and development (R&D) and other costs to determine patterns in rewards for pharmaceutical innovation. We found that the average present values of lifetime net economic returns were positive and reached a peak with the 1995-99 and 2000-04 new drug cohorts. However, returns have fallen sharply since then, with those for the 2005-09 cohort being very slightly negative and, on average, failing to recoup research and development and other costs. If this level of diminished returns persists, we believe that the rewards for innovation will not be sufficient for pharmaceutical manufacturers to maintain the historical rates of investments needed to sustain biomedical innovation.

Prescription drug spending trends in the United States: looking beyond the turning point.

Annual growth in real prescription drug spending averaged 9.9 percent during 1997-2007 but has slowed since 2003, falling to 1.6 percent in 2007. More patent expirations, increased generic penetration, and reduced new product innovations have contributed to this turning point. We document trends and identify underlying components: declines in the role of blockbuster drugs, increased importance of biologics and vaccines relative to traditional pharmaceuticals, and a changing medication mix away from those prescribed principally by primary care physicians toward those mostly prescribed by specialists. We conclude with policy implications.