The Budget Impact of Orphan Drugs in the US: A 2007-2013 MIDAS Sales Data Analysis December 8, 2014 Oral Presentation at the 56th ASH Annual Meeting and Exposition, Session: 902. Health Services and Outcomes Research Malignant Diseases: New/Refined Prognostic Schemes/Pharmacoeconomics Victoria Divino 1 *, Mitch Dekoven, MHSA 1 *, Weiying Wang, MPH 2 *, Michael Kleinrock, MA 1 *, R. Donald Harvey, PharmD 3, Rolin L. Wade, RPh,MS 1 * and Satyin Kaura, MSci, MBA 4 * 1 IMS Health, Fairfax, VA; 2 MKTXS, Raritan, NJ; 3 Winship Cancer Institute, Emory University, Atlanta, GA; 4 Celgene, Summit, NJ
Background Study Background The United States (US) Orphan Drug Act (ODA) of 1983 established incentives for the development of drugs that treat rare or orphan diseases. Approximately 25-30M Americans, or 8-10% of the US population, have one of the diseases defined as rare, and thus may benefit from the ODA. 1 The ODA has been successful in stimulating orphan drug development to treat a variety of previously underserved conditions. Despite the clinical value of orphan drugs, payer sensitivity to orphan drugs is increasing due to the perceived potential impact on payers drug budgets. Despite these payer concerns, little evidence/data has been generated as to the actual burden of orphan drug expenditures in the US. Figure 1. Orphan Drug Designations/Approvals 1983-2012 Rare Diseases and FDA Perspectives from the Office of Orphan Products Development (OOPD) Presented by Katherine Needleman, MS, PhD, RAC, Director, Orphan Products Grants Program FDA/OOPD at the IRDiRC Conference, April 16 & 17, 2013 2 1. Handfield R, Feldstein J. Insurance companies' perspectives on the orphan drug pipeline. Am Health Drug Benefits. 2013 Nov;6(9):589-98.
Objectives Study Objectives To measure the total annual expenditures on branded orphan drugs between 2007-2013 in the US. The specific objectives were: To estimate total orphan drug expenditures in the US from 2007-2013, and to evaluate total orphan drug expenditures as a proportion of total pharmaceutical expenditures. To evaluate future orphan drug expenditures (2014-2018) and as a proportion of total future pharmaceutical expenditures. 3
Overview Methods Overview This analysis utilized the US FDA Orphan Drug Product designation website to identify the comprehensive list of drugs which have been approved by the FDA and given orphan status in the US since the establishment of the ODA (1983) through the end of 2013 (N=365 unique products). Only branded products (N=356) were considered in this analysis. The IMS Health MIDAS database was used to assess orphan drug and total drug expenditures in the US between 2007-2013. o IMS MIDAS is the gold standard for audited biopharmaceutical sales, a unique analysis tool and on-line system, and the benchmark measure in the industry. IMS MIDAS integrates and extends the IMS Health building blocks that are the IMS National Audits which measures sales volume at the product pack level. The primary analysis focused on expenditures for 291 out of the total 356 (82%) total orphan drugs that were orphan only, approved for a single orphan designation only or for multiple orphan only designations. In a secondary analysis, expenditures for 65 out of the total 356 (18%) orphan drugs, identified as partial orphans, approved for both orphan and non-orphan indications, were adjusted. Utilizing several US sources, in-depth research was conducted in order to determine a disease factor to apply to those sales so only orphan indication sales were considered in the analysis. o The Cowen and Company Equity Research Health Care Therapeutic Categories Report, manufacturers audited financial reports, an analysis of US physician office claims data, the diagnosis value in MIDAS, epidemiology in the US. 4
Analyses Analyses Total expenditures for the branded orphan drugs, measured in the MIDAS dataset, were calculated annually from 2007-2013. Orphan only drug expenditures were reported for the primarily analysis. Additionally, adjusted partial orphan drug expenditures, and adjusted total orphan drug expenditures (orphan only + partial orphan drugs) were reported. Orphan drug expenditures were further evaluated as a proportion of total annual 2007-2013 US pharmaceutical drug expenditures sourced from MIDAS. Expenditures were recorded at the time of the sale. All expenditures were adjusted to 2014 USD using the Bureau of Economic Analysis GDP deflator. Trends in total orphan drug expenditures were assessed over time (e.g. major therapeutic category of orphan indications). A future trend analysis was evaluated (via 2 methods) to estimate orphan drug sales from 2014-2018. Future estimated orphan drug sales were evaluated out of projected future total drug sales based on IMS Health Market Prognosis forecasts. 5
2007-2013 Orphan Drug Expenditures Results Table 1. 2007-2013 Orphan Drugs Captured 2007 2008 2009 2010 2011 2012 2013 # Orphan only drugs captured N=252 # Partial Orphan drugs captured N=64 # Total Orphan drugs captured N=316 Orphan Only Drug Expenditures ($) Total Drug Expenditures ($) % Orphan Only / Total Drug Measure 2007 2008 2009 2010 2011 2012 2013 Partial Orphan Drug Expenditures ($) 1,724,112,665 2,000,120,316 2,269,512,397 2,981,964,166 3,312,563,575 3,637,321,362 4,204,897,912 Total Orphan Drug Expenditures ($) 15,029,971,619 17,067,138,132 19,394,965,957 23,094,548,844 26,124,999,476 28,030,524,508 30,048,066,281 % Orphan Only / Total Orphan 88.5% 88.3% 88.3% 87.1% 87.3% 87.0% 86.0% % Partial Orphan / Total Orphan 11.5% 11.7% 11.7% 12.9% 12.7% 13.0% 14.0% % Partial Orphan / Total Drug 0.5% 0.6% 0.7% 0.9% 0.9% 1.1% 1.2% % Total Orphan/ Total Drug 4.8% 5.5% 6.0% 6.8% 7.5% 8.5% 8.9% Orphan Only drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations. Partial Orphan drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures. Total Orphan drugs = ( Orphan Only drugs + adjusted Partial Orphan drugs) 6 169 172 181 190 203 208 227 41 44 45 50 49 55 59 210 216 226 240 252 263 286 Table 2. 2007-2013 Orphan Only Drug Expenditures (2014 USD) 2007 2008 2009 2010 2011 2012 2013 13,305,858,954 15,067,017,816 17,125,453,561 20,112,584,678 22,812,435,902 24,393,203,146 25,843,168,369 311,384,270,346 310,104,657,654 324,774,764,623 340,551,781,912 346,473,406,816 330,083,171,834 337,442,018,948 4.3% 4.9% 5.3% 5.9% 6.6% 7.4% 7.7% Table 3. 2007-2013 Partial Only and Total Orphan Drug Expenditures
Results Figure 2. 2007-2013 Orphan Drug Expenditures out of Total US Drug Expenditures 4E+11 $311.4B $310.1B $324.8B $340.6B $346.5B $330.1B $337.2B Total Pharmaceutical Expenditure 3.5E+11 3E+11 2.5E+11 0.5% 0.6% 4.3% 4.9% 0.7% 5.3% 0.9% 0.9% 5.9% 6.6% 1.1% 1.2% 7.4% 7.7% Partial Orphan (Adjusted) 2E+11 1.5E+11 1E+11 5E+10 0 95.2% 94.5% 94.0% 93.2% 92.5% 91.5% 91.1% 2007 2008 2009 2010 2011 2012 2013 Orphan Only Non-Orphan (Adjusted) Orphan Only drugs = orphan drugs approved for a single orphan designation only or for multiple orphan only designations. Partial Orphan drugs = orphan drugs approved for both orphan and non-orphan indications, adjusted to include only orphan indication expenditures. Total Orphan drugs = ( Orphan Only drugs + adjusted Partial Orphan drugs) 7
Results Oncology was the most common therapeutic class, with 101 (32.0%) out of the total 316 orphan drugs identified with an orphan oncology indication $35.0B 35,000,000,000 Figure 3. 2007-2013 Orphan Drug Expenditures by Therapeutic Class $30.0B 30,000,000,000 $25.0B 25,000,000,000 $20.0B 20,000,000,000 $15.0B 15,000,000,000 $10.0B 10,000,000,000 $5.0B 5,000,000,000 $0 - $30.0B $26.1B $28.0B 62.0% 59.3% $23.1B 64.7% $19.4B 66.5% $15.0B $17.1B 68.7% 67.7% 70.7% 40.7% 38.0% 35.3% 33.5% 32.3% 29.3% 31.3% 2007 2008 2009 2010 2011 2012 2013 All Other Oncology- Related 8
Future trends (2014-2018) suggest a slowing down in orphan drug growth 9 Results Methodology #1: A linear trend line was created using 2007-2013 sales Orphan drug expenditures of $33.5- $44.2B, were estimated for 2014-2018, representing 8.8-9.5% of total U.S. drug expenditures. Methodology #2: Linear trend lines were created to estimate number of orphan drug approvals and average cost in 2014-2018 based on 2007-2013 data. Future costs were estimated as the number of new approvals x the average cost per drug, added to prior total year orphan drug sales, for each year Orphan drug expenditures of $34.9- $54.9B were estimated for 2014-2018, representing 9.2-11.8% of total US drug costs. IMS Health Market Prognosis recently forecasted 2014-2018 US total drug sales of $380.8-$465.0B.
Discussion Discussion While a select few orphan drugs may have high costs, the population using these orphan drugs is small, and total orphan drug expenditures, when considered out of total drug expenditures, is relatively minimal. For the primary analysis of 252 orphan only drugs, expenditures represented 4.3-7.7% of 2007-2013 total US drug sales. Adjusted drug expenditures for 64 partial orphan drugs represented 0.5-1.2% of total US drug sales between 2007-2013. o Our disease factoring analysis suggests that the majority of unadjusted partial orphan drug sales are for non-orphan indications. Following adjustment, orphan indication costs were minimal and represented only 7.2-10.5% of total unadjusted partial orphan drug expenditures in 2007-2013, respectively. Total orphan drug expenditures represented 4.8-8.9% of total US drug expenditures, in 2007-2013. Future trend analysis suggests that while orphan drug expenditures in 2014-2018 will increase, there will be a slowing down in growth, and growth will remain fairly stable as a proportion of total drug expenditures. 10
Limitations There are a number of limitations to note with this analysis. Discussion MIDAS database coverage is limited with respect to certain products and/or manufacturers. o The forecasted total drug expenditures from IMS Market Prognosis provide more comprehensive estimates. As our MIDAS estimates may be underestimated due to coverage limitations, our future trend analysis of orphan drug expenditures out of total forecasted drug sales may be further underestimated. Finally, there is the potential for off-label use of orphan drugs. Because the sales data do not have indications associated with sales, it is unknown if, or to what extent, these drugs are being used for off-label indications. 11
Conclusions Discussion The ODA has led to an increase in the number of approved orphan drugs, and while associated orphan drug expenditures have increased, these drugs benefit many people with previously underserved rare conditions. The annual expenditures on orphan drugs are small relative to total pharmaceutical drug expenditures. Orphan drug expenditures have remained fairly stable as a proportion of total pharmaceutical expenditures and future trend analysis suggests growth is slowing down. Concerns that growth in orphan drug expenditures may lead to unsustainable drug expenditures do not appear to be justified. 12