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Premier Research's New Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges - Premier Research

Premier Research's New Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges - Premier Research | Rare diseases | Scoop.it
A new survey of clinical trial decision makers commissioned by Premier Research reports that nearly two-thirds (64%) of respondents said that among the most difficult factors in recruiting for a rare disease or orphan drug clinical trial was not...
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Premier Research's New Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges - Premier Research

Premier Research's New Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges - Premier Research | Rare diseases | Scoop.it
A new survey of clinical trial decision makers commissioned by Premier Research reports that nearly two-thirds (64%) of respondents said that among the most difficult factors in recruiting for a rare disease or orphan drug clinical trial was not...
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CentoMD: World's largest genetic mutation database for rare diseases

CentoMD: World's largest genetic mutation database for rare diseases | Rare diseases | Scoop.it
CENTOGENE AG announces the launch of the world's largest genetic mutation database for rare diseases, CentoMD.
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The first test for the self-diagnosis of rare diseases

The first test for the self-diagnosis of rare diseases | Rare diseases | Scoop.it

Available in the USA the first direct-to-consumer genetic carrier test for genetic diseases. Developed by the firm 23andMe, it has now been approved for commercialization by the Food and Drug Administration.

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Rare diseases: How patients and parents cope with with little-known illnesses - Scottish Daily Record

Rare diseases: How patients and parents cope with with little-known illnesses - Scottish Daily Record | Rare diseases | Scoop.it
ONE in 17 people in the world suffer from a rare disease and 30,000 of those people live in Scotland.
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Scotland approves rare cancer drug

Scotland approves rare cancer drug | Rare diseases | Scoop.it
Scotland becomes the first part of the UK to make a new drug available to patients with a rare form of cancer.
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Social media and the rare disease community

Social media and the rare disease community | Rare diseases | Scoop.it
Social media has opened up an infinite amount of possibilities for patients and patient advocates, creating a large online community.  This powerful platform has enabled rare disease stakeholders to raise awareness of diseases, fight for…
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How to access the Brazilian Orphan-Drug Market

How to access the Brazilian Orphan-Drug Market | Rare diseases | Scoop.it
Providing access to Orphan Drugs has many different opportunities and challenges, which vary market-to-market. Devaney Baccarin, Presidente & CEO of BaccarinD joined us at the 4th Annual World Orphan Drug Congress USA in 2014 to discuss…
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Karyopharm Announces Orphan Designation Granted by FDA for Selinexor (KPT-330) in Multiple Myeloma

Karyopharm Announces Orphan Designation Granted by FDA for Selinexor (KPT-330) in Multiple Myeloma | Rare diseases | Scoop.it
Karyopharm Announces Orphan Designation Granted by FDA for Selinexor (KPT-330) in Multiple Myeloma Newton, Mass. – January 8, 2015 – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today announced that its lead drug candidate…
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Call for guest bloggers

Call for guest bloggers | Rare diseases | Scoop.it
  This opportunity is open to all would-be commentators from all corners of the rare disease industry – from students, blogger, journalists, researchers to academics. We would want you to join us at the World…
Freya Smale's insight:

This opportunity is open to all would-be commentators from all corners of the rare disease industry – from students, blogger, journalists, researchers to academics.

 

We would want you to join us at the World Orphan Drug Congress USA 2015 for 2 days in Washington D.C. and join our in-house marketing team in an effort to cover the amazing content that will be presented at the event.

 

We want to hear from a wide range of voices reflecting all levels of seniority and experience in the industry and we hope it will become a significant forum for debate on the rare disease landscape.

 

This is your chance to get involved! If you’re interested in joining us, please contact me today at freya.smale@terrapinn.com

 

What do guest bloggers get?


As a guest blogger you will benefit in a few ways…

 

You get the chance to attend the 2 day conference for FREEYou get access to the pre-conference day workshops for FREEYou get a big audience seeing your post on our Total Orphan Drugs blog, LinkedIN and TwitterYou get to share your views on the current issues in the industry and have fun for 2 days at the eventYou get to network with the industry
 

To be considered for the role, please email me on freya.smale@terrapinn.com and I will get back to you very soon.

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England's National Health Service announces DNA project to fight cancer, rare diseases

England's National Health Service announces DNA project to fight cancer, rare diseases | Rare diseases | Scoop.it
England's NHS announced 11 new Genomics Medicine Centres Monday, as part of a new DNA scheme that aims to tackle cancer and rare diseases.
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Pharma Marketing Blog: 43% of New Drugs Approved by FDA in 2014 were for Treatment of Rare Diseases

Pharma Marketing Blog: 43% of New Drugs Approved by FDA in 2014 were for Treatment of Rare Diseases | Rare diseases | Scoop.it

Preliminary data announced recently by the FDA shows that 2014 is shaping up to be a banner year for orphan drugs, which are drugs that treat "rare" diseases/disorder affecting fewer than 200,000 people in the U.S.


Orphan drug approvals represent 43% of all new drug approvals in 2014. Today, orphan drugs have the potential to turn into blockbusters with annual sales over $1 Billion.


Read more here.



Via Pharma Guy
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Pharma Guy's curator insight, December 16, 2014 12:39 PM


When a dumb reporter asked "Willie" Sutton, the famous bank robber, why he robbed banks, he is reported to have said "Because that's where the money is." Duh!

Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an "orphan drug" -- ie, a drug for a disorder affecting fewer than 200,000 people in the U.S. -- you would likely have gotten a response such as "because there is an unmet medical need" or something similar.

Today, however, orphan drugs also have the potential to turn into blockbusters; ie, be where pharma's money is at.


Read, for example, "New Big Pharma Economies of Scale: Less Patients Needed to Reach Blockbuster Sales")

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The Rise of Superbugs - Consumer Reports

The Rise of Superbugs - Consumer Reports | Rare diseases | Scoop.it
Consumer Reports looks at the rise of superbugs, noting that these dangerous infections that are resistant to antibiotics are spreading and growing stronger, with dire consequences.
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FDA grants orphan drug designation to Reolysin for malignant gliomas - Healio

FDA grants orphan drug designation to Reolysin for malignant gliomas - Healio | Rare diseases | Scoop.it
The FDA today granted orphan drug designation to pelareorep for the treatment of patients with malignant gliomas, according to a press release from the drug’s manufacturer.The FDA based its decision in part on results of three brain cancer studies...
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Horizon to acquire orphan drugs in $1.1bn Hyperion takeover - Royal Society of Chemistry

Horizon to acquire orphan drugs in $1.1bn Hyperion takeover - Royal Society of Chemistry | Rare diseases | Scoop.it
Horizon will buy Hyperion and add new drugs to its rare disease portfolio
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Orphan drugs to account for 19% of sales by 2020 - Managed Care magazine (blog)

Orphan drugs to account for 19% of sales by 2020 - Managed Care magazine (blog) | Rare diseases | Scoop.it
They are relatively cheap to develop, since clinical trials are small and “the lack of alternative treatments give orphan agents an advantage when up for regulatory review,” according to a report by Evaluate, a life science market intelligence...
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There are More Orphan Drugs and FDA Approvals Than Ever Before

There are More Orphan Drugs and FDA Approvals Than Ever Before | Rare diseases | Scoop.it
A new analysis indicates that the number of orphan drug submissions, designations and approvals are rising at a record pace.

Via Pharma Guy
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Shattered chromosome cures woman of immune disease - Science Now

Shattered chromosome cures woman of immune disease - Science Now | Rare diseases | Scoop.it
Newly scrambled DNA erases genetic defect that caused frequent childhood infections
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Precision Medicine: Beginning of the End of Orphan Drugs

Precision Medicine: Beginning of the End of Orphan Drugs | Rare diseases | Scoop.it
If every disease is a rare disease, none of them are. It's the unspoken elephant in the room as the U.S. and the world makes headway into the long-sought advance of precision medicine. The Orphan…
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Strategic U.S. Regulatory Considerations for Drug Development for Serious & Life Threatening Medical Conditions

Strategic U.S. Regulatory Considerations for Drug Development for Serious & Life Threatening Medical Conditions | Rare diseases | Scoop.it
In last week’s State of the Union (SOTU) address, President Barack Obama backed the Orphan Drug industry, and bought to the forefront of the nation something this industry has known for a while: medicine is…
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Obama’s Precision Medicine Initiative: What It Means for Orphan Drugs

Obama’s Precision Medicine Initiative: What It Means for Orphan Drugs | Rare diseases | Scoop.it
In last night's State of the Union (SOTU) address, President Barack Obama brought to the forefront of the nation something that we in the Orphan Drug industry have known for a while: medicine is becoming…
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Was 2014 the EMA’s Breakthrough Year?

Was 2014 the EMA’s Breakthrough Year? | Rare diseases | Scoop.it
A record number of EMA orphan drug approvals breaks out of previous stagnation in the EU The USA has had it's best ever year for orphan drug approvals, with a total of 41 being given…
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Shire to buy US rival in $5.2bn deal

Shire to buy US rival in $5.2bn deal | Rare diseases | Scoop.it
UK pharmaceutical company Shire has clinched a deal to buy US rival NPS Pharmaceuticals for $5.2bn (£3.4bn).
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FDA defiant on orphan exclusivity rules even after court judgement - In-PharmaTechnologist.com

FDA defiant on orphan exclusivity rules even after court judgement - In-PharmaTechnologist.com | Rare diseases | Scoop.it
The US FDA says it will stick to its policy on orphan drug exclusivity despite a conflicting court ruling.
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