Dan Renick comments on the importance of value demonstration for respiratory disease treatments in The Pink Sheet.

The Pink Sheet

May 30, 2016

GSK Leads Way With Real World Data, But Others Follow

By Emily Hayes

Executive Summary

The firm’s hands-off pragmatic trial in the UK paves the way for real world studies in other disease areas where there are many products and arguably little differentiation to sway payers on market access.

Increasingly, health care providers and payers want to see studies reflective of real world situations and proof that health care is being delivered in the most cost-effective way, which was part of the aim of GlaxoSmithKline PLC‘s Salford Lung Study.

The 2,802-patient study compared GSK’s Breo Ellipta, which combines the inhaled corticosteroid fluticasone furoate with the long-acting beta agonist vilanterol, against physician’s choice, to reflect an everyday clinical practice setting (see related story, (“Real World Superiority Data May Not Be Enough To Boost GSK’s Breo In COPD” — “The Pink Sheet,” May 30, 2016). The “real world” study is part of a growing trend to provide better evidence of how a drug performs in clinical practice.

“Respiratory diseases affect broad populations and have long been targets of management for payers, so showing value over the many available competing drugs at the time of launch is becoming increasingly important, otherwise access will be limited until more supporting evidence is available”, Dan Renick, president of Precision for Value, commented in an interview.

Sidney Braman, Icahn School of Medicine at Mount Sinai in New York, noted that FDA approves drugs without assessments of measures that are really important to the medical community or patients, including hospitalization rates and how patients feel – their emotional changes, activity levels and quality of life.

In a sign of FDA’s support for patient-reported outcomes data, the agency recently revised its draft guidance on developing drugs for chronic obstructive pulmonary disease to include a component related to patient reported outcomes.

The new document is identical to draft guidance issued in 2007 with the exception of a two-page appendix describing the use of the St. George’s Respiratory Questionnaire, a patient-reported outcome assessment. The questionnaire has been used in COPD clinical trials as a co-primary and secondary endpoint and is included in labeling for some recently approved products.

Other Outcomes Ahead

Manufacturers are preparing to have their products judged by new standards based on health outcomes.

The American Thoracic Society meeting, held from May 13-17 in San Francisco, featured a number of posters on patient outcomes, including Sunovion Pharmaceuticals Inc.‘s post hoc analysis of a 52-week study for its nebulized long-acting COPD treatment Brovana (arformoteral tartrate) that analyzed the connection between hospitalization rates and patient response in moderate-to-severe COPD, based on the St. George’s Respiratory Questionnaire. The study showed a lower relative risk of hospitalization in responders.

The company also funded a survey about nebulizer use that suggested more education is needed in primary care, where many COPD patients are treated. Nebulizers may be useful for treatment at home for acute exacerbations, potentially saving an emergency room visit.

Genentech Inc. presented three posters for its Esbriet (pirfenidone) in idiopathic pulmonary fibrosis at the ATS meeting, with retrospective analysis showing Esbriet does not increase the risk of cardiovascular or bleeding events over one year compared to placebo and that there was a statistically significant reduction in non-elective respiratory-related hospitalizations over one year – 7% vs. 12% for placebo, with a numerically lower rate of death.

Other manufacturers say that real world data is high on their list of priorities. Tausif “Tosh” Butt, vice president of AstraZeneca PLC‘srespiratory business unit, noted the company’s involvement in the COPD Foundation’s COPD360 program. Launched in March 2015, the program aims to use electronic medical records to collect data for 125,000 patients, including patient-reported outcomes, clinical data and observational research. AstraZeneca was a founding sponsor of the initiative.

“It’s a big deal for us because we want to make sure we can better understand the natural course of disease and impact of treatment and understand whether biomarkers can help us either in diagnostics and in developing targeted treatments,” Butt said in an interview at the ATS meeting.

The company notes that in 2015 it published data on health care utilization patterns, including ER visits, hospitalizations and medication use, for patients taking its Symbicort (budesonide/formoterol) in the International Journal of COPD and has “invested a lot of effort in providing valuable comparative effectiveness research that may help provide additional data to assist payers and clinicians with decisions that will impact their patients.”

The company also recently started the real-world NOVELTY study of COPD and asthma patients, which “aims to identify novel phenotypes and endotypes in COPD and asthma patients to examine patient characteristics and treatment patterns. It will be one of the largest, global prospective longitudinal clinical, observational studies in obstructive lung disease,” the company said.

Novartis notes that it has conducted several real world retrospective studies in respiratory diseases investigating a variety of issues, including treatment patterns and clinical and economic outcomes.

The company has planned the prospective non-interventional LUMIERE study about the real-world effectiveness of COPD treatments, which will track outcomes, including the rate of hospitalizations for exacerbations.

“We have the analysis plan to address potential biases that may rise from a non-randomized, observational study. We are confident that this study will provide the real-world evidence for COPD combination drugs. In this study, we will track multiple outcome measures including hospitalizations due to exacerbations,” the company said.