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This is a shareable personal research tool to keep track of Wikipedia categories, articles, reliable source (references) etc related to the history of Pharmacoeconomics, orphan drugs.

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Pharmacy and Pharmacology


Categories

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Category:Health sciences > Pharmaceutical sciences (10 C, 9 P) > Category:Orphan drug companies > Catalyst Pharmaceuticals

B Bausch Health Beacon Pharmaceuticals Benzer Pharmacy Bevacizumab Biopharmaceutical Busulfan C Capecitabine CVS Health D Delcath Systems Diplomat Pharmacy E Express Scripts G Gilead Sciences H Humana I Interferon beta-1a M Melphalan O Omnicare P Palbociclib R Rituximab S Sodium thiosulfate Sofosbuvir Specialty pharmacyStromagen T Trastuzumab W Walgreens Boots Alliance Walgreens Health Services Z Zalcitabine

Category:Pharmaceutical industry [Category:Pharmaceuticals policy Pharmaceuticals policy]


Category:Food and Drug Administration > Category:Breakthrough therapy

Category:Orphan drug companies This category is for articles about companies that produce orphan drugs. Therapies that have received orphan status in the United States and Europe are available from the U.S. Food and Drug Administration and the European Commission, respectively: FDA List of Orphan Designations and Approvals, European Commission Register of designated Orphan Medicinal Products


Clade

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This is an experimental use of clades:

Pharmacy benefit management
Specialty pharmacy<br />)
label1<br />)Cite error: There are <ref> tags on this page without content in them (see the help page).
label 1<br />)
<br />)

orphan drug

)

Alglucerase
)

3
(est. 1852, acq. 1954)

4(acq. 1959)

5
(est. 1866, acq. 1986)[1][2]

Manufacturers Hanover<br />(merged 1961)

Manufacturers
Trust Company
(est. 1905)[3]

2
(est. 1873)

Breakthrough drug therapy<br />
2<br />(formerly Morgan Guaranty Trust)<br />(merged 1959)

Guaranty Trust Company
of New York
(est. 1866)

2("The House of Morgan")

Food and Drug Administration<br />)
Online pharmacy<br />1)[5]

3

orphan drug<br />
4

Alglucerase
Imiglucerase

 

2

 

3

 

4

 

5

6

6
(acq. 2005)

Timeline

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See Timeline orphan drugs a sandbox compilation of significant events related to legislation, research, marketing etc of orphan drugs.[6]

October 10, 1962 The Kefauver-Harris Amendment became law partly as a result of the thalidomide tragedy. Kefauver's bill enhanced drug regulation by forcing pharmaceutical companies to prove to the FDA that their drugs were both safe and effective before they were introduced into the US market. The FDA received authority to regulate advertising of prescription drugs and to establish good manufacturing practices. The law required that all drugs introduced between 1938 and 1962 had to be effective. An FDA - National Academy of Sciences collaborative study showed that nearly 40 percent of these products were not effective. A similarly comprehensive study of over-the-counter products began in the 1970s.[7]

1970s A similarly comprehensive study of over-the-counter products began in the 1970s.[7]

1976 Henry G. Grabowski's article "Drug Regulation and Innovation: Empirical Evidence and Policy Options" was published by the American Enterprise Institute for Public Policy Research. Grabowski [8] Grabowski is Professor Emeritus and Director of Program in Pharmaceutical Health Economics, Duke University and a member of the National Academy of Sciences, the Institute of Medicine, the Federal Trade Commission, the General Accounting Office and the Office of Technology Assessment. *Henry G. Grabowski, Professor Emeritus and Director of Program in Pharmaceutical Health Economics, Duke University. National Academy of Sciences, the Institute of Medicine, the Federal Trade Commission, the General Accounting Office and the Office of Technology Assessment contributes regularly to the American Enterprise Institute for Public Policy Research . Grabowski [8]

1981 Genzyme was founded by Sheridan Snyder, George M. Whitesides[9] and scientist Henry Blair in 1981 and is primarily devoted to finding drugs that would cure enzyme deficiency conditions that were essential to human survival and which usually afflict a very small percentage of the world’s population. Drugs used to treat such conditions are considered to be “orphan drugs.” In 1986, the company went public, raising $27 million. In February 16, 2011, Sanofi acquired Genzyme for $20.1 billion.[10]

1983 The Orphan Drug Act (ODA) of January 1983, passed in the United States, with lobbying from the National Organization for Rare Disorders and many other organizations,[11] is meant to encourage pharmaceutical companies to develop drugs for diseases that have a small market.[12] Under the ODA orphan drug sponsors qualify for seven-year FDA-administered market Orphan Drug Exclusivity (ODE), "tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance[13]: 660  and and may get clinical trial tax incentives.[12]


1989 conservative Heritage Foundation proposed an individual mandate as an alternative to single-payer health care.[14]

1990 The Brattle Group formed in 1990 to provide "consulting services and expert testimony in economics, finance, and regulation to corporations, law firms, and public agencies. It hires internationally recognized experts, and has had strong partnerships with leading academics and highly credentialed industry specialists around the world."[15]

1991 Alglucerase (trade name Ceredase) was approved by the FDA in 1991[16]: 123  and was the first drug approved as an enzyme replacement therapy.[17] "Acquisition cost of alglucerase is $US3.70 per unit (1994 US dollars); thus, a dosage regimen of 60 IU/kg bodyweight administered every 2 weeks for a patient weighing 70kg costs $US404,040 per year."[18] It was withdrawn from the market[19][20][21] due to the approval of similar drugs.[22] By 2008 this treatment costs about US$200,000 annually for a single person and should be continued for life. The rarity of the disease means dose-finding studies have been difficult to conduct, so controversy remains over the optimal dose and dosing frequency.[23] Due to the low incidence, this has become an orphan drug in many countries, meaning a government recognizes and accommodates the financial constraints that limit research into drugs that address a small population.

1997 The European Organisation for Rare Diseases (EURORDIS), was founded in 1997 as part of patient self-advocacy[24] and it now represents "30 million people affected by rare diseases throughout Europe."

1997 A Wall Street Journal investigative journalist reported that one doctor alone made dozens of Rhone-Poulenc Rorer (RPR)-funded presentations openly promoting RPR's Lovenox, a blood clot medication for Off-label uses that were not approved by the FDA. RPR is "68% owned by Paris-based Rhone-Poulenc SA."[25]

2010By 2010 Idursulfase (brand name Elaprase), manufactured by Shire and used for the treatment of Hunter syndrome, was one of the most expensive drugs ever produced, costing US$375,000 per patient per year.[26][27]

2011 Montreal-based Enobia Pharma Inc. initially developed asfotase alfa (brand name Strensiq).[28] In 2011 Alexion Pharmaceuticals acquired Enobia Pharma and therefore asfotase alfa and continued research into its usage as treatment for an enzyme replacement therapy for [[Hypophosphatasia (HPP), "an inherited and life-threatening ultra-rare metabolic disorder that leads to progressive damage to multiple vital organs, including destruction and deformity of bones."[28] "A significant portion of the research and development" of asfotase alfa was undertaken in Canada, and "Canadian clinicians now have global expertise in the treatment of HPP."[28] "Many analysts expect the drug to pass the "blockbuster" mark of $1 billion a year at some point and maybe reach $2 billion."[29]

March 23, 2010 The US Patient Protection and Affordable Care Act, (PPACA), (ACA) or Obamacare, signed 23 March 2010 by President Barack Obama a significant regulatory overhaul of the U.S. healthcare system partly to decrease Medicare spending.[30][31][32][33][34] The PPACA consisted of a combination of measures to control healthcare costs, and an expansion of coverage through public and private insurance: broader Medicaid eligibility and Medicare coverage, and subsidized, regulated private insurance. Senate Republicans threatened to block appointments to relevant agencies, such as the Independent Payment Advisory Board[35] and Centers for Medicare and Medicaid Services.[36][37] Conservative groups such as Heritage Action provided lobbying support for the linkage between the ACA and the government shutdown.[38][39][40]

2013 "These 25 biotechnology firms are ranked by their market capitalization for the latest quarter that they furnished figures on their numbers of outstanding shares."[41][41]

2014 Orphan drugs were more lucrative than non-orphan drugs to companies in the pharmaceutical industry that focus on orphan drugs. They "offer opportunities for greater returns on investment compared to other companies, owing to factors such as the higher pricing, financial incentives, accelerated regulatory filings and smaller sales force requirements for orphan drugs." This theory was questioned in Morel et al (2014).[42]

2014 KuicK Research released their "Global Orphan Drug Market Outlook 2018" research report "offering comprehensive insight on recent trends, regulatory development related to US orphan drug market, Europe orphan drug market & Asia orphan drug market. The Report also gives insight on more than 600 orphan drugs in clinical pipeline and 231 marketed orphan drugs."[43] "The global pharmaceutical industry has been experiencing a slow growth in recent years owing to many factors such as expiration of patents, competition from generic drugs segment, exhausting pipelines, and an increasingly stringent regulatory framework. It is most likely that many blockbuster drugs would lose their exclusivity in the next 5 year horizon. Thus, due to significant competition from generics and the current economic situation, the focus area of the pharmaceutical companies is undergoing a paradigm shift from manufacturing traditional essential medicines to investing in the new business model, which is also called orphan drugs. The returns on investment from orphan drugs are expected to help the pharma companies to overcome the impact of revenue loss due to expiry of patents of blockbuster drugs."[43]

2014 EvaluatePharma published their Orphan Drug Report, which said that, "Over 7,000 different types of rare diseases and disorders exist, with more being discovered each day, and large pharmaceutical and biotech companies are structuring to participate and leverage the growing orphan market. “Orphan drug sales will make up 19% of the total share of prescription drug sales by 2020, totaling $176 billion. And they’ll grow at an annual rate of nearly 11% per year through the end of the decade, compared with about 4% for drugs treating larger populations." pharmaceutical industry According to a 2014 report by Andreas Hadjivasiliou, published by Evaluate Pharma, in the last few years, the orphan drug market has become increasingly lucrative because the cost of clinical trials for orphan drugs is substantially lower than for other diseases—trial sizes are naturally much smaller than for more common diseases with larger numbers of patients— and there is a lack of alternative therapies for rare diseases. Small clinical trials and little competition place these "orphan agents" at an advantage when they come up for regulatory review. There is a further reduction to the cost of development because of the tax incentives in the Orphan Drug Act 1983. On average the cost per patient for orphan drugs is "six times that of non-orphan drugs, a clear indication of their pricing power."[44] Partly as a result of the 1983 US Orphan Drug Act, Japan adopted it in 1993 as did the European Union in 2000.[44] Although there are much smaller orphan disease populations are the smallest, the cost of per-patient outlays are the largest. Hadjivasiliou argues that there will be more pressure on pharmaceuticals that "represent the biggest budgetary drain" particularly as more people with rare diseases—in the United States for example—will be eligible for public subsidies through the Affordable Care Act.[44]


2014 The London School of Economics published a 2014 refereed article comparing the outcomes and consequences of Health Technology Assessments (HTA) processes that determine expected value for money for Orphan Drugs (ODs) among European Union (EU) member states (MS). The outcome of assessments differs significantly across the EU[45] with consequences for access to medicines across member states (MS) [2]. Orphan drugs (ODs). For example "90% of a sample of 60 ODs" were available in "France, was found to the Netherlands and Denmark but "only one third were available in Spain, Greece and Romania [6]. Financial considerations often limit access to pharmaceuticals in many EU member states. A survey undertaken in 2014 "reported that nearly a quarter of the 22 European countries surveyed (Estonia, Latvia, Lithuania, Poland and Romania) restricted access to ODs due to budgetary constraints. Based on the same survey, only 5 countries always granted access to ODs, while several authorised access but with possible restrictions, such as prior authorisation."[7][46]: 84 

2015 The Orphan Drugs & Rare Diseases Global Congress 2015 took place in Europe.

2015 In 2015 a White Paper on biotech was published which said that, From 1975 to 2015 many countries adopted measures to promote the commercial development of Drugs for rare diseases (DRDs) s and facilitate patient access to these treatments.[28]

2015 Health Canada had only approved half of FDA-approved DRDs for sale in Canada. In some cases it takes six years for FDA-approved drugs to enter the Canadian market.[28]

2015 Various governments use different standards for Health technology assessments (HTA) regarding orphan drugs and other treatments for diseases that are not rare. Canada's Canadian Agency for Drugs and Technologies in Health (CADTH) uses the same standards for both.[28]


2018 [47]

2012

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The FDA approved NPS Pharmaceuticals’ teduglutide (trade name Gattex, for the treatment of short bowel syndrome in December 2012. In an interview with Forbes journalist at the 2012 Forbes Healthcare Summit, NPS CEO, Francois Nader, described how Dan Drucker, a University of Toronto endocrinologist discovered teduglutide.[48]
"In 2012 corporate America accounted for more than three-quarters of the $3.3 billion spent on lobbying in Washington, DC. General Electric was the market leader, spending $21.4m, and Google came second, with $18.2m."[49]
According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investing in orphan drug Research and Development partly due to the U. S. Orphan Drug Act (ODA) 1983 and similar Acts in other regions of the world and also driven by "high-profile philanthropic funding."[50][13]
By 2012,

"the revenue-generating potential of orphan drugs [was] as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success."

— Gaze and Breen 2012

2011

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The period between 2001 to 2011 was the "most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals."[13]: 660  For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an "impressive 25.8 percent, compared to only 20.1 percent for a matched control group of non-orphan drugs."[50]: 6  By 2012 the market for orphan drugs was worth USD$637 million compared to the USD$638 million matched control group of non-orphan drugs, Thomson Reuters.[50]


2009

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Genzyme, a Sanofi Company undertook clinical trials starting in July 2009 comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 patients.[51]
According to the Canadian Organization of Rare Diseases (CORD) 80% of rare diseases affect children and "more than 50% of rare disorders are inherited."[52]: 1  Prader–Willi syndrome and Hunter syndrome were used as examples of rare diseases with no cure.[52] By 2007 Idursulfase (brand name Elaprase), manufactured by Shire, was approved for the treatment of Hunter syndrome.[53] By 2010 Idursulfase (brand name Elaprase), manufactured by Shireit was one of the most expensive drugs ever produced, costing US$375,000 per patient per year.[26][27]

2008

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By 2008 the "most common criticism of orphan-product legislation has been the very high cost of treatment with some of the drugs."[54][55]
By 2008 most of the orphan drugs appraised had cost-effectiveness thresholds "well in excess of the ‘accepted’ level and would not be reimbursed according to conventional criteria."[56]
In 2008 Health Canada provisionally licensed the Swiss company Santhera Pharmaceuticals's Idebenone (trade name Catena, Raxone, Sovrima) for treatment of Friedreich's ataxia.[57] Durhane Wong-Rieger, President of Canadian Organization for Rare Disorders (CORD), spoke on behalf of Sanhera's Catena, arguing that it was "essential that governments, insurers and treatment centers combine their efforts and assist Friedreich's Ataxia patients in accessing this new therapeutic advancement." Idebenone was provided free of charge in Quebec, where there is a particularly high occurrence of Friedreich's Ataxia.[57] In all the other Canadian provinces it is only provided by private insurers.[58]: 4  Idebenone was removed from the Canadian market in 2013 due to lack of effectiveness.[59] Idebenone was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer's disease and other cognitive defects[60] and met with limited success. The .In 2012 the The Cochrane Database of Systematic Reviews on antioxidants and other pharmacological treatment in Friedreich Ataxia[58] In July 2008 idebenone was licensed provisionally in Canada for treatment of Friedreich ataxia. Since then it has been provided free of charge in one of the ten provinces, Quebec, but in the other provinces it is only provided by private insurers. In November 2008, the European Medicines Agency refused marketing authorisation for idebenone in Europe (EMA 2009). The US Food and Drugs Administration has not authorised idebenone for use in Friedreich ataxia. According to the European Federation of Hereditary Ataxias (euro-ATAXIA) by 2012 those the cost of idebenone was high if patients had bo purchase in private pharmaceutical sales.[58][61]

2007

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In an effort to reduce the burden on manufacturers applying for orphan drug status, the FDA and EMA agreed in late 2007 to utilize a common application process for both agencies. However, the two agencies will continue to maintain separate approval processes.[62]
"According to a report by the Center for Public Integrity, congressmen are outnumbered two to one by lobbyists for an industry that spends roughly $100 million a year in campaign contributions and lobbying expenses to protect its profits."[63]
By 2007 the use of economic evaluation methods regarding public-funding of orphan drugs, using estimates of the incremental cost-effectiveness, for example, became more established internationally.[64] The QALY has often been used in cost-utility analysis to calculate the ratio of cost to QALYs saved for a particular health care intervention.[65][66] By 2008 the National Institute for Health and Care Excellence (NICE) in England and Wales, for example, operated with a threshold range of £20,000–£30,000 per Quality-adjusted life year (QALY).[56] By 2005 doubts were raised about the use of economic evaluations in orphan drugs.[64] As early as 2005 McCabe et al argued[67][68] that rarity should not have a premium and orphan drugs should be treated like other pharmaceuticals in general.[67][68] Drummond et al[68] argued that the social value of health technologies should also be included in the assessment along than the estimation of the incremental cost-effectiveness ratio.
  • 2006 Oldani who worked for Pfizer, Inc. as a pharmaceutical salesperson from 1989 to 1998, drew heavily on his auto-ethnographic experiences while working in the industry" for his PhD dissertation.[69]: 23 

2005

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From 1998 to 2005, the "pharmaceutical and health products industry spent more than $800 million in federal lobbying and campaign donations at the federal and state levels in the past seven years."[70] This was more than any other industry.[70]
2005 Maskus and Reichman co-edited International Public Goods and Transfer Technology under a Globalized Intellectual Property Regime. Grawbowski's article "Increasing R&D Incentives for Neglected Diseases: Lessons from the Orphan Drug Act.[71]

2004

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Genzyme reportedly received more than $800 million in revenue in 2004 from imiglucerase alone.[54]: 2041 [72] Gaucher's disease affects fewer than 20,000 patients in the USA.[54]: 2041 [73]

2003

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"Drugs such as imiglucerase, an enzyme replacement therapy developed by Genzyme to treat Gaucher's disease; and other orphan blockbuster drugs have led to calls for modification of the legislation. Treatment with imiglucerase might cost as much as US$400,000 per year per adult patient.[54]: 2041 [74]
Medicare Modernisation Act of 2003,[75] a federal law of the United States, enacted in 2003,[76] was the largest overhaul of Medicare in the public health program's 38-year history. It was signed by President George W. Bush.[77] The MMA's most touted feature is the introduction of an entitlement benefit for prescription drugs, through tax breaks and subsidies. The benefit is funded in a complex way, reflecting diverse priorities of lobbyists and constituencies. As of February 2009, the projected net cost of the program over the 2006 to 2015 period was $549.2 billion.[78][79] Walter Jones, R-N.C., called the vote "the ugliest night I have ever seen in 22 years." Jones claimed, "The pharmaceutical lobbyists wrote the bill," says Jones. "The bill was over 1,000 pages. And it got to the members of the House that morning, and we voted for it at about 3 a.m. in the morning."[63] Billy Tauzin, R-La, steered the bill through the house.[63] "In 2003 alone, the [pharmaceutical] industry spent nearly $116 million lobbying the government."[70]
In a 2015 article in The Economist, the role of lobbyists in this act was criticized,

"A classic case of selfish lobbying wrapped in a cloak of selflessness was the Medicare Modernisation Act of 2003. Thanks to the pharmaceutical industry’s lobbyists, this brought new prescription-drug benefits to millions of older Americans, but without any attempt to control costs through means-testing or bulk-buying. John Friedman, an economist at Brown University, estimated that as a result the drugmakers would gain benefits of $242 billion over a ten-year period—a healthy return on the $130m the industry spent on lobbying in the year the law passed."

— The Economist 2015

2002

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"From 1996 to 2001 the pharmaceutical sales force in America doubled, to a total of 90,000 reps."[80]

See also

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Notes

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References

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  1. ^ "Specialty Pharmacy Drug List" (PDF). Magellan RX Management. 2015. Retrieved 5 October 2015.
  2. ^ Cite error: The named reference NYT_Magellan_20150716 was invoked but never defined (see the help page).
  3. ^ Gleason, Alexander G. C.; Starner, C. I.; Ritter, S. T.; Van Houten, H. K.; Gunderson, B. W.; Shah, N. D. (September 2013). "Health plan utilization and costs of specialty drugs within 4 chronic conditions". Academy of Managed Care Pharmacy. 19 (7): 542–8. PMID 23964615. Retrieved 11 October 2015.
  4. ^ ref
  5. ^ ref
  6. ^ "The Orphan Drug Act (as amended)". U.S. Food and Drug Administration. Retrieved 2007-09-24.
  7. ^ a b "Brochure: The History of Drug Regulation in the United States".
  8. ^ a b Henry G. Grabowski (1976), Drug Regulation and Innovation: Empirical Evidence and Policy Options, Washington, D.C: American Enterprise Institute for Public Policy Research
  9. ^ Michael Rosenwald (August 2010). "Can Nanotechnology Save Lives?". Smithsonian Magazine. Retrieved August 16, 2012.
  10. ^ Regan, James (February 3, 2011). "Sanofi could announce Genzyme deal next week – report". Reuters.
  11. ^ Henkel, John (1999). "Orphan Drug Law Matures into Medical Mainstay". FDA Consumer. U.S. Food and Drug Administration. Retrieved 14 February 2009.
  12. ^ a b Pollack, Andrew (30 April 1990). "Orphan Drug Law Spurs Debate". The New York Times. Retrieved 15 February 2009.
  13. ^ a b c Orphan drug development: an economically viable strategy for biopharma R&D, 15 August 2012, doi:10.1016/j.drudis.2012.02.005, retrieved 29 June 2015 {{citation}}: Unknown parameter |authors= ignored (help)
  14. ^ Roy, Avik (February 7, 2012). "The Tortuous History of Conservatives and the Individual Mandate". Forbes Magazine.
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  16. ^ "WHO Drug Information" (PDF), World Health Organization., Regulatory Matters, vol. 5, no. 3, 1991
  17. ^ Deegan PB, Cox TM. Imiglucerase in the treatment of Gaucher disease: a history and perspective. Drug Des Devel Ther. 2012;6:81-106. doi: 10.2147/DDDT.S14395. Epub 2012 Apr 18. PMID 22563238 (free full text)
  18. ^ Whittington. "Alglucerase. A pharmacoeconomic appraisal of its use in the treatment of Gaucher's disease". Pharmacoeconomics date=January 1995. 7 (1). GOA: :63–90. {{cite journal}}: Missing pipe in: |journal= (help)
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  23. ^ Cite error: The named reference Grabowski2008 was invoked but never defined (see the help page).
  24. ^ Houyez F (2004). "Active involvement of patients in drug research, evaluation, and commercialization: European perspective". J Ambul Care Manage. 27 (2): 139–45. PMID 15069992.
  25. ^ Tanouye, Elyse, Off the Label: Staffers of Drug Maker Say it Pushed Product For Unapproved Uses, Wall Street Journal date=15 September 1997, pp. A1, A7 {{citation}}: Missing pipe in: |publisher= (help)
  26. ^ a b "Drug approved to treat rare but potentially deadly disease". Retrieved 2011-04-29.
  27. ^ a b Herper, Matthew (22 February 2010), Health Care: The World's Most Expensive Drugs, Forbes, retrieved 5 July 2015
  28. ^ a b c d e f Biotech White Paper (Canada) (PDF), March 2015, retrieved 6 July 2015, To be confirmed: From 1975 to 2015 many countries adopted measures to promote the commercial development of Drugs for rare diseases (DRDs) s and facilitate patient access to these treatments. Health Canada had only approved half of FDA-approved DRDs for sale in Canada. In some cases it takes six years for FDA-approved drugs to enter the Canadian market. Various governments use different standards for Health technology assessments (HTA) regarding orphan drugs and other treatments for diseases that are not rare. Canada's Canadian Agency for Drugs and Technologies in Health (CADTH) uses the same standards for both. Montreal-based Enobia Pharma Inc. initially developed asfotase alfa (brand name Strensiq). In 2011 Alexion Pharmaceuticals acquired Enobia Pharma and therefore asfotase alfa and continued research into its usage as treatment for an enzyme replacement therapy for Hypophosphatasia (HPP), "an inherited and life-threatening ultra-rare metabolic disorder that leads to progressive damage to multiple vital organs, including destruction and deformity of bones." "A significant portion of the research and development" of asfotase alfa was undertaken in Canada, and "Canadian clinicians now have global expertise in the treatment of HPP."
  29. ^ Alexion Pharma Looks To Life Beyond Its Blockbuster, Nasdaq via Investor's Business Daily, 8 July 2014, retrieved 6 July 2015
  30. ^ Pear, Robert (July 7, 2012). "Health Law Critics Prepare to Battle Over Insurance Exchange Subsidies". New York Times. Retrieved July 7, 2012.
  31. ^ Krugman, Paul (January 31, 2010). "Krugman calls Senate health care bill similar to law in Massachusetts". PolitiFact.com. Tampa Bay Times. Retrieved August 29, 2012.
  32. ^ "ObamaCare Survives the Supreme Court: 5 Takeaways". The Week. June 28, 2012. Retrieved June 30, 2012.
  33. ^ Elmendorf, Douglas W. (March 30, 2011). "CBO's Analysis of the Major Health Care Legislation Enacted in March 2010" (PDF). Congressional Budget Office. Retrieved July 15, 2012.
  34. ^ Elmendorf, Douglas W. (June 21, 2011). "CBO's 2011 Long-Term Budget Outlook" (PDF). Congressional Budget Office. p. 44. Through those changes and numerous others, the 2010 legislation significantly decreased Medicare outlays relative to what they would have been under prior law.
  35. ^ Goddard, Teagan (May 17, 2013). "Blocking the Medicare Reform Board Won't Stop Reform". WonkWire.RollCall.com.
  36. ^ Cohn, Jonathan (May 24, 2010). "Save Donald". The New Republic.
    Cohn, Jonathan (July 6, 2010). "Meet The Don". The New Republic.
  37. ^ Cohn, Jonathan (July 19, 2011). "The New Nullification: GOP v. Obama Nominees". The New Republic.
  38. ^ Moody, Chris (October 9, 2013). "Meet one of the conservative advocacy groups behind the GOP's government shutdown strategy". Yahoo! News.
  39. ^ Joseph, Cameron (October 9, 2013). "Heritage Action leader: Paul Ryan's shutdown offer off-target". The Hill.
  40. ^ Miller, Zeke J. (September 30, 2013). "Hidden Hand: How Heritage Action Drove DC To Shut Down". Time.
  41. ^ a b Alex Philippidis (22 July 2013), Top 25 Biotech Companies of 2013; Which firms made this list this time?, retrieved 6 July 2015
  42. ^ Morel T, Popa C, Simoens S (January 2014), "Market watch: Are orphan drug companies the pick of the pharmaceutical industry?", Nat Rev Drug Discov., 13 (1), National Center for Biotechnology Information (NCBI), U.S. National Library of Medicine (NLM), doi:10.1038/nrd4205{{citation}}: CS1 maint: multiple names: authors list (link)
  43. ^ a b Global orphan drug market to reach US$ 120 billion by 2018, New Delhi: Kuick Research, 7 Feb 2014, retrieved 20 March 2014
  44. ^ a b c Hadjivasiliou, Andreas (October 2014), "Orphan Drug Report 2014" (PDF), EvaluatePharma, retrieved 28 June 2015
  45. ^ Kanavos P, Nicod E, van den Aardweg S, Pomedli S. (2010), "The impact of health technology assessments: an international comparison", Euro observer, 12 (4): 1–7{{citation}}: CS1 maint: multiple names: authors list (link)
  46. ^ David Tordrup, Victoria Tzouma, Panos Kanavos (August 14), "Orphan drug considerations in Health Technology Assessment in eight European countries" (PDF), Rare Diseases and Orphan Drugs: An International Journal of Public Health, 1 (3), London School of Economics: 86–97, retrieved 6 July 2015 {{citation}}: Check date values in: |date= (help)CS1 maint: multiple names: authors list (link)
  47. ^ "The Global Orphan Drug Market 2018: Forecast to Reach $285.8 Billion by 2023 -". Research And Markets. 2018-09-11. Retrieved 2019-02-07.
  48. ^ Roy, Avik (2 May 2013), Are Drugs for Ultra-Rare Diseases the Future of Biotech? Francois Nader of NPS Weighs In, Forbes, retrieved 6 July 2015
  49. ^ "The Washington wishing-well: The unstoppable rise in lobbying by American business is bad for business itself", The Economist, 13 June 2015, retrieved 2 July 2015
  50. ^ a b c "The Economic Power of Orphan Drugs" (PDF), Thomson Reuters, 2012, retrieved 29 June 2015 {{citation}}: Unknown parameter |authors= ignored (help)
  51. ^ "Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy:24-Month Results". Blood. 124 (21): 1406–14. 6 December 2014. {{cite journal}}: Unknown parameter |authors= ignored (help)
  52. ^ a b Wong-Rieger, Durhane (1 April 2009), Effect of Rare Diseases on Patients and Families (PDF), retrieved 5 July 2015
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