PHAR announced today the final results of a study, published in Current Medical Research and Opinion, which found that pre-launch predictions of the budget impact of newly developed drugs tended to be considerable overestimates of their actual sales. The study examined the accuracy of 25 publicly released predictions for the cost of drugs launched in a variety of indications since 2012. Overall, the predicted budget impact was 5.5 times the actual budget impact of the drug. Ten estimates were off by more than $1 billion: 8 overestimated drug sales by more than a billion; and 2 underestimated it by at least that amount. Overestimating budget impact may lead to early access restrictions, higher copays, and other changes that ultimately impact patients. More information about the study is available here and the detailed results can be viewed here.

Research focused on idiopathic pulmonary fibrosis (IPF) conducted by PHAR and supported by Genentech, Inc. was well-represented at the CHEST Annual Meeting 2016. One study was selected as an oral presentation and two were presented as posters. The oral presentation reported on the prevalence of comorbidities in Medicare IPF patients versus disease free controls, and whether it increased over time. The posters presented analyses of mortality, and cardiovascular burden and medication use among IPF patients. The posters can be accessed on the PHAR website here.

On August 16, the Journal of Oncology Practice (JOP) published a joint guidance statement from ASCO and the American Academy of Hospice and Palliative Medicine (AAHPM) regarding high-quality palliative care as delivered as part of routine cancer care. PHAR supported the development of this important guidance by moderating a meeting of experts using the RAND/UCLA modified Delphi panel process. With this method, over 900 potential palliative care services were rated by more than 20 participants on multiple dimensions. Those services which were both highly ranked and which had a high level of agreement were included in the final guidance. Read the full study here.

A new PHAR study compared predictions of health care costs made prior to the introduction of new drugs to the actual sales of the drugs. Fourteen drugs were examined: two cholesterol lowering drugs; three drugs to treat various forms of cancer; three for hepatitis C; two for obesity management; and one each for cystic fibrosis, heart failure, psoriasis, and diabetes. In many cases, sales predictions were dramatically overestimated, sometimes by billions of dollars. In one example, the Institute for Clinical and Economic Review (ICER) predicted the one-year cost of the two PCSK9 inhibitors would be $7.2 billion. Based on the initial quarters of reported sales, PHAR found that the actual number will be closer to $83 million, an overestimate of $7.1 billion. Asked to comment on the findings, Dr. Broder said, “Overestimating drug costs by so much cannot lead to good decision making… it is likely that patients feel the negative effects of such predictions in the form of early access restrictions and higher copayments.” More details about the study are available here.

In association with the researchers at Genentech, Inc., studies conducted by PHAR were a strong presence at the American Thoracic Society Conference (ATS) 2016. One was selected as an oral presentation and three were presented as posters. The study chosen for oral presentation reported on the overall in-hospital mortality, length of stay (LOS), and hospitalization cost in patients with IPF admitted to hospitals in the US, and the posters presented a more in-depth set of data on the mortality and resource utilization of IPF. A study on whether allergic status is associated with increased number of asthma exacerbations was also presented. The posters can be accessed on the PHAR website here.

In response to the rise of effective but expensive new cancer treatments, several organizations have put forth “value frameworks” for oncology. These frameworks, produced by organizations including the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), and the Institute for Clinical and Economic Review (ICER), are tools designed to help doctors, patients, and insurance companies determine how much value a treatment provides relative to its cost. Researchers at PHAR, Tufts, UCLA, and Memorial Sloan Kettering Cancer Center, collaborating on the first published evaluation of the reliability of these frameworks, found only fair to good consistency between different individuals using the same framework to evaluate the same drug. They also found that using each of the three different frameworks could lead to significantly different conclusions about the relative value of cancer treatments. These findings suggest it may be premature to use these value frameworks in treatment decision-making without further evidence of their reliability and validity. The study results were presented at the 2016 ASCO meeting.

This retrospective study used insurance claims data to examine the extent of twice-daily use of dornase alfa among cystic fibrosis patients. The study was done in partnership with researchers at Genentech, Inc. and presented at Academy of Managed Care Pharmacy Managed Care & Specialty Pharmacy Annual Meeting in San Francisco. See the full poster here.

Five studies conducted by teams at PHAR were presented at the Academy of Managed Care Pharmacy Managed Care & Specialty Pharmacy Annual Meeting in San Francisco. These studies cover a wide range of diseases and methods including cost effectiveness study of treatments for peripheral t-cell lymphoma, an analysis of insurance claims among cystic fibrosis patients, and an examination of patients with idiopathic pulmonary fibrosis using the National Inpatient Sample. The five posters can be found here.

A study conducted by the researchers at PHAR was selected for oral presentation at Endocrine Society’s 2016 Annual Meeting in Boston in April. The study used data from medical records at 8 US endocrine centers to describe treatment outcomes for Cushing’s Disease patients . The findings of the study can be viewed here. Including this one, there were 5 poster presentations of studies by PHAR. All posters presented at ENDO can be accessed here.

An abstract submitted by researchers at PHAR and Jazz Pharmaceuticals was highlighted by the American Society of Hematology (ASH) at their 57th Annual Meeting. The study estimated the total 100-day and one-year costs associated with inpatient hematopoietic stem cell transplantation, stratified by type of conditioning regimen and other potential contributors to cost. Researchers found that total per patient healthcare costs ranged from $182,000 to $409,000 in the one-year period post transplant. The poster can be accessed here.