Talk:Orphan drug

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Are orphan drug initiatives in effect outside of US/Europe - ie. Australia/Asia (China, Japan, Australia), Russia, Africa etc. --- or are these covered by the previously mentioned facilities?--ZayZayEM (talk) 00:21, 16 September 2008 (UTC)

New "controversies" content added in 2 places[edit]

This is great content in general but the execution is ax-grindy and fails NPOV. Happy to include but this but we'll need to make this neutral.

first chunk of content added in description of the law

Eculizumab (brand name Soliris) is designated as an orphan drug under the United States Orphan Drug Act (ODA).[1] The ODA is meant to encourage pharmaceutical companies to develop drugs for rare disorders such as aHUS and PNH that have a small market because their patient population is too small to attract pharmaceutical research.[2]

"Public resources went into understanding the molecular basis of the disease, public resources went into the technology to make antibodies and finally, Alexion, to their credit, kind of picked up the pieces."

— Sachdev Sidhu 2015
Under the law, companies that develop such a drug (a drug for a disorder affecting fewer than 200,000 people in the United States) may sell it without competition for seven years,[3] and may get clinical trial tax incentives.[3]


second chunk of content added to new "Controversies" section

Eculizumab (brand name Soliris), developed, manufactured and marketed by Alexion Pharmaceuticals, described by Forbes as the most expensive drug in the world,[1] costs approximately USD$409,500 a year in the United States (2010),[1] €430,000 per year for ongoing treatment in the UK[2] [3] and $500,000 a year in Canada (2014) and.[4] The actual cost of manufacturing Soliris' monoclonal antibodies is less than "1 per cent of the price of the drug."[5]

In the case of the rarest diseases that afflict fewer than 10,000 people, biotech companies who own the only approved drugs to treat those diseases "can charge pretty much whatever they want."[4] "Before testing Soliris for PNH, Alexion tested the drug for rheumatoid arthritis, which afflicts 1 million Americans. The trials failed. But if it had worked for arthritis, Alexion would likely have had to charge a much a lower price for this use, as would have to compete against drugs that cost a mere $20,000." Alexion started selling Soliris in 2008 making $295 million in 2007 with its stock price rising to 130% in 2010.[4]

In April and May 2013, a controversy arose in Belgium when the media revealed that the government had refused to pay for a seven-year-old boy's treatment because Soliris was too expensive. The boy's medicine cost 9,000 euros every two weeks.[6] On May 4, 2013, De Standaard reported that a press relations (PR) agency working for Alexion had helped the boy's parents communicate their story to the press.[7] It was also reported that the parents had believed their benefactor was a Dutch organization for patients, and that the PR agency acted with permission from Alexion.[8][9] Several politicians stated that the company was attempting to 'blackmail' the government, charges which Alexion denied.[10] By May 7, 2013 an agreement had been reached to reimburse the medicine.[11][12]

Pharma, the Belgian pharmaceutical industry's association, opened an internal investigation into the affair, for possible breach of the association's ethical standards by Alexion. However, on June 12, Alexion received a court gag order against Pharma, preventing it from communicating its investigation. At the same time, Pharma opened a court case against Alexion Pharma Belgium. The gag order was revoked by the end of September 2013,[13] but the case was still pending in March 2015.[12]

By 2015 industry analysts and academic researchers agree that the sky-high price of so-called orphan drugs, that treat these ultra-rare diseases, is no longer related to research, development and manufacturing costs.[5] The price of orphan drugs is arbitrary and has become more profitable than traditional medicines.[5]

According to Sachdev Sidhu, a University of Toronto scientist who spent years researching monoclonal antibodies, "the underlying elements in Soliris, for a U.S. biotech company,"[5] claimed that 80 or 90 per cent of Soliris research and development was done by publicly-funded university researchers working in academic laboratories.[5]

"Public resources went into understanding the molecular basis of the disease, public resources went into the technology to make antibodies and finally, Alexion, to their credit, kind of picked up the pieces."

— Sachdev Sidhu 2015

In December 2014 the government of Ontario, Canada was in ongoing price negotiations with the Soliris manufacturer, the only drug approved by Health Canada to treat aHUS. Patients who require the apply for it on "compassionate grounds" "on a case-by-case basis for individuals who have been urgently hospitalized due to an immediate life-, limb-, or organ-threatening complication." Soliris is already "funded by the Ontario government for the treatment of another rare illness, paroxysmal nocturnal hemoglobinuria (PNH), through a bulk-buy deal reached by the provincial premiers in 2011."[4]

In February 2015, Canada’s drug-price regulator took the rare step of calling a hearing into Soliris, accusing Alexion of exceeding the permissible price cap under the Highest International Price Comparison (HIPC).[14] In June 2015, the Patented Medicine Prices Review Board (PMPRB) under the Canadian Patent Act, held a preliminary hearing in Ottawa, Ontario to examine allegations. John Haslam, President and General Manager of Vaughan, Ontario-based Alexion Canada, was named as one of the respondents.[15] Alexion charges Canada $700,000 per patient per year—more than anywhere else in the world.[5] Alexion denies the claim. By 2011 some Canadian provinces had already negotiated secret discounted bulk-buy deals regarding Soliris.[4][14]

"This drug [Soliris] is forcing us to have to rethink how we say yes and how we say no when it comes to prescription drugs."

— Michael Law, University of British Columbia


  1. ^ a b Herper, Matthew (February 19, 2010), "The World's Most Expensive Drugs", Forbes, retrieved June 25, 2015 
  2. ^
  3. ^
  4. ^ a b c d e Gallant, Jacques (December 4, 2014), Toronto woman with rare disease fights province for life-saving but costly drug Soliris, which costs $500,000 a year, would treat Toni Vernon's blood disease, but the health ministry is holding back, retrieved June 25, 2015 
  5. ^ a b c d e f Crowe, Kelly (25 June 2015), "How pharmaceutical company Alexion set the price of the world's most expensive drug: Cost of one of world's most expensive drugs shrouded in corporate secrecy", CBC News, retrieved 25 June 2015  line feed character in |title= at position 85 (help)
  6. ^ "Viktor (7) moet elke twee weken infuus krijgen van 9.000 euro". April 30, 2013. 
  7. ^ "Pr-bureau van farmabedrijf adviseerde ook ouders Viktor". May 4, 2013. 
  8. ^ "Farmabedrijf Alexion heeft Viktor "gebruikt"". May 4, 2013. 
  9. ^ "Farmabaas fluit Alexion terug in zaak-Viktor". May 6, 2013. 
  10. ^ "Detiège: "Dit is chantage van het farmabedrijf"". May 2, 2013. 
  11. ^ "Medicijn Viktor vanaf juli terugbetaald". May 7, 2013. 
  12. ^ a b "Contract van Onkelinx met farmareus opent doos van Pandora". 2015-03-23. Retrieved 2015-03-24. 
  13. ^ "Spreekverbod opgeheven". Retrieved 2015-03-24. 
  14. ^ a b Blackwell, Tom (February 3, 2015), "World’s most expensive drug — which costs up to $700,000 per year — too expensive, Canada says", National Post, retrieved June 25, 2015 
  15. ^ "Motions and Exhibits" (PDF), Government of Canada, Ottawa, Ontario, 2015, retrieved June 25, 2015 

Hi Jytdog, I have recently added orphan drugs to my list of topics I follow and contribute to in Wikipedia and will continue to do so. I have no problem with my content being edited removed if the team deems it to be someway inappropriate and there is a solid academic argument for its removal.Oceanflynn (talk) 14:59, 29 June 2015 (UTC)

In regards to the material based on the 2014 Evaluate Pharma publication[1] any removal of the content would require a rebuttal with full citations for each point argued by Hadjivasiliou. If you do a very basic Google scholar search for orphan drugs you will find that the economic challenges mentioned above are listed as issues of concern to policy-makers globally and they will only become intensified. This deserves a space in a well-rounded Wikipedia article.Oceanflynn (talk) 15:42, 29 June 2015 (UTC)


  1. ^ Hadjivasiliou, Andreas (October 2014), "Orphan Drug Report 2014" (PDF), EvaluatePharma, retrieved 28 June 2015 

— Preceding unsigned comment added by Oceanflynn (talkcontribs) 15:07, 29 June 2015 (UTC)

almost all that is inappropriate for an article Talk page. I will reply on your Talk page where you posted the same thing. and I'm am asking you to revise the above to focus on content relevant to this article, not on contributor - not on you, and not on me. Jytdog (talk) 15:14, 29 June 2015 (UTC)
I do not approve of loose labeling and I do not have time to engage.Oceanflynn (talk) 15:42, 29 June 2015 (UTC)
Thanks for editing your remarks in this dif. Will review your new edits later and discuss then. Jytdog (talk) 15:44, 29 June 2015 (UTC)

Proposed changes[edit]

Working on editing this article for a class at the University of Michigan. The talking points are below, the link to our edits is also below


Orphan Drugs classification

    Pends on the country you are in
         In America it is any disease with < 200,000 patients
         In Europe it is any disease with less than 5 in 10,000
         In Japan it is any disease with less than 50,000 patients

Costs sections

    Very small customer base
         This forces companies to increase the price of the drugs exponentially (See graph)
         The government attempts to intervene with tax breaks and other incentives up to 50% off on R&D
              This is extremely important as this is the primary overhead cost that must be allocated to customers
                   In a normal market, with more customers, this R&D cost can be spread very thinly
                        With a smaller group of customers/patients this manufacturing overhead is dumped on the very few that require the drug
              Much of this comes from phase 3 clinical trials
                   orphan drugs cost anywhere from 50% to a quarter of non orphan drugs in this phase
    Grants for phase 1-3 clinical trials
         With much of the R&D coming from the clinical trials the government has tried to further incentives the drug manufacturers approximately 30 million is granted every fiscal year

Abuse of the system (New Section)

         Because of all these incentives given to Orphan drug companies many non-orphan drug companies try to take advantage of them it has become a practice to register one's drugs as orphan drugs, even though they have more uses than that
              an example of this is the drug Crestor, which can be used to treatment of pediatric homozygous familial hypercholesterolemia (age 0 through 16).                  cite:
                   This got them the tax breaks and funding they needed
                        once receiving the funding they marketed that the drug could be used to treat cholesterol in all diabetics.
                   In total: they cut their R&D costs almost in half and were able to generate much higher margins than they originally predicted

Wbigley (talk)

Wbigley (talk) I apologize for the format, I pre wrote this on a word document and the numbers did not transfer to wikipedia. — Preceding unsigned comment added by Wbigley (talkcontribs) 21:36, 16 March 2016 (UTC)

Please do not add badly formatted content here again. You haven't provided any sources so none of us can tell if this is WP:OR or based on reliable sources. Jytdog (talk) 00:31, 17 March 2016 (UTC)

Peer review and responses during the educational assignment in Winter 2016[edit]

Topic Peer Review 1[edit]


The introduction to this article is clear to non-experts on the topic of Orphan Drugs. However, the formatting in your Sandbox is off and does not have the same presentation as that of the original Wikipedia page. I found it confusing to review the Sandbox because I struggled to follow the formatting so I would definitely look into this. Some of the links to existing pages are not in existence so be sure to cross check this before the next submission of the project. In your Sandbox, many of the important terms are not linked so this is also something to look out for. Even those with links in the initial article are not in your Sandbox. For the most part, the contents of each section seem to justify its length. I do however, feel that you could go more in depth regarding some of the subjects and clarify their relation to Orphan Drugs. For example, I think you should add more information regarding Rare Disease Act, Regulatory harmonization, and "other countries" because they are brushed over rather quickly.

As far as "other countries" goes, the edits you have made regarding classification could fit here. The highlighted examples in the article do a good job of exemplifying Orphan Drugs, but once again I feel that you can go more in depth with these rather than just linking users to a different page. It seems as though there are an extensive amount of topics covered on the page and not that much information associated with these topics.

I think you did a good job contributing to the page and adding useful information on the topic. I also think that you could add a little more!


There are no figures included in this article. I think that you could definitely incorporate a molecular model of an example of an orphan drug.


The references are formatted strangely on the Sandbox page but everything seems to be cited on the original Wikipedia page. The original reference list does include non journals, which is totally fine.

Overall Presentation[edit]

As I stated above, there are several things that need to be fixed with the physical appearance of the page. Ultimately, I struggled to even locate some of the changes you made. This may be due to the formatting but also could just be because tracking the edits was difficult. I do think that the changes made are valuable and that you did a good job of further explaining Orphan Drugs. I feel that fixing the formatting to follow that of the original page will be beneficial to the reader. I also think that going further in depth into certain topics would also be very helpful! Overall, it is a very interesting topic and fascinating to read.

Lhochhauser (talk) 23:35, 21 March 2016 (UTC)Lhochhauser

Response to Peer Review[edit]

Thank you for your Review we went in and fixed the wonky formatting that appeared in our sandbox so now it appears like a proper Wikipedia article. We added more links to our added sections and added more links to the preexisting sections of the Wikipedia article to make it a bigger part of the overall Wikipedia community. We took your advice and used a figure of a orphan drug in the article but we decided to make it Crestor an orphan drug that gamed the system and was marketed to a large consumer base. We fixed the references section and added new references of our own so we are now responsible for references 16,22,23,24, and 31. Zachduff (talk) 14:13, 5 April 2016 (UTC)

Topic Peer Review #2 (by Natalie)[edit]


The introductory section is clear and accessible for everyone. Good job making it clear what orphan drugs are and their importance. However, I found the quote included in the bottom of the section to be introduced a little awkwardly, and suggest you add another sentence leading into it besides "by 2012." None of the sections are unreasonably long, but maybe you could add more to the "Regulatory harmonization" and "other countries" sections, or instead of "other countries" being it's own section, add that to the EU part and just do a new "outside of the U.S." section. Most of the important terms have links to pages, but not every link worked for me. For the most part there was no redundancy or duplicative content that I noted, but maybe you don't have to have a whole section for the "rare diseases act", and instead integrate it into another section, since there already exists a whole wikipedia page for that.


There were no figures, so you will have to add those. But even if you don't find any available, maybe you could make a chart with the information you already have relating to the cost of Orphan drugs.


On the original wikipedia page which most people will access, the references look fine to me. It looked a little weird on sandbox but I guess that doesn't matter as long as it works on the original page.

Overall Presentation[edit]

Good job overall. I think you could consolidate some of the smaller sections to make the reading flow more smoothly appearance-wise, or simply elaborate on these topics to make them more lengthy and justify their having their own section. But overall the content is clear and organized in a way that makes sense. Nice job-- didn't notice any bias or major problems. Just adding figures will solve one of the major problems. It was interesting and accessible to read.

Ngreifer (talk) 00:15, 27 March 2016 (UTC)ngreifer

Response to Peer Review[edit]

Thanks for your input we did decide to add more information to the existing sections of the article but we decided instead to focus on the relationship of Orphan Drugs and AIDS in the effectiveness section because we found a very interesting journal article about this relationship and thought it would be a valuable and interesting addition to the article. We were able to find a figure of Crestor to add to the article. We fixed the references in our sandbox and the formatting to accurately reflected the final Wikipedia page. Wbigley (talk) 14:25, 5 April 2016 (UTC)

Suggestions from ChemLibrarian[edit]

It's a little difficult to tell your additions since the original article was quite long to start with. I see that you added a paragraph at the end of the Effectiveness section. One paragraph in Cost. and one section on Abuse of public-funding for orphan drugs. Is that all?

Here are a few suggestions before you post your edits to the main space.

  1. It seems that you messed up some references in the original article like Reference 1 in your Sandbox version. Please be careful not to carry those mistakes back to the main article. Please only copy/paste the paragraphs you added one by one.
  2. The last sentence of your Abuse sections says "With more and more orphan drug's being filed every year it seems that companies are looking to take advantage of these loop holes to minimize research and development cost and make a larger profit margin.". It reads like a statement you make. Is there any reference mentioned this statement? If so, cite it. If not, please be careful with making a statement like this because it would make it your opinion piece instead of an encyclopedia article only reflecting what's been reported.

ChemLibrarian (talk) 19:21, 5 April 2016 (UTC)

External links modified[edit]

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the following is almost all unsourced and is dramatically incomplete; not sure this is encyclopedic at all:

Industry involvement[edit]

Key orphan drug developers are:

These pharmaceutical companies work together with national bodies such as the U.S.'s National Organization for Rare Disorders (NORD), Avella Specialty Pharmacy[1] and the European Organization for Rare Diseases (EURORDIS) to advance this field.


  1. ^ Christopher Geoffrey McPherson (1 July 2014). "Avella Specialty Pharmacy attorney fulfills dream through medicines". AZCentral. Retrieved 5 July 2014. 

-- Jytdog (talk) 14:43, 22 October 2016 (UTC)