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Risky Business: The FDA And Drug Development For The Fatally Ill
Photo Credit: Forbes Business

Risky Business: The FDA And Drug Development For The Fatally Ill

Mar 6 2014, 12:01am CST | by

If you’ve ever waited for treatment in the ER—for yourself or perhaps for a child suffering a broken bone or an asthma attack—then you’re familiar with how agonizing it can be, knowing that the...

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20 weeks ago

Risky Business: The FDA And Drug Development For The Fatally Ill

Mar 6 2014, 12:01am CST | by

If you’ve ever waited for treatment in the ER—for yourself or perhaps for a child suffering a broken bone or an asthma attack—then you’re familiar with how agonizing it can be, knowing that the answer might be just on the other side of the swinging doors. For parents of seriously ill children, this kind of waiting can last for years. And when treatment options are still stuck in the development, testing, or approval processes, the wait can cost patients their lives.

The FDA has set up these processes for good reason; they protect the public from experiencing severe, unexpected side effects due to unsafe drugs, which could actually make people even sicker than they already were. But for seriously ill patients, sometimes the risk is worth taking if there’s any chance of increasing their survival rates. In these cases, the burdensome approval process to get drugs on the market—which can take up to 18 years—is detrimental both to pharmaceutical companies and to patients. The recent controversy over the FDA’s refusal to approve eteplirsen, a drug intended to treat Duchenne muscular dystrophy, is a prime example of this. Eteplirsen has shown some promising results in clinical trials, but the data still isn’t satisfactory enough for FDA approval, since it has a hard time assessing benefit and risk across a spectrum of perspectives and experiences. Patients and their families beg for access to the drug, to no avail.

Unlike other industries, pharmaceutical companies are also legally restricted from directly marketing to consumers or including them in the prototyping phase of the design process. This can prevent consumers from providing vital feedback that could shape drug effectiveness, as well as from learning about experimental options when they are available. With most conditions, the risks of experimentation would be too high. But if you had a fatal disease and were told you only had one year to live, wouldn’t you prefer to be allowed to make your own choice?

Sharon Terry, an Ashoka Fellow, and Genetic Alliance have developed a platform that facilitates the responsible engagement of the FDA with patients such as these, and they’re getting patients directly involved in voicing their concerns about the current system. The FDA’s initiative is called Patient-Focused Drug Development (PFDD), and it is designed to assess risks and benefits for specific patient communities.  Genetic Alliance is crowdsourcing input for the FDA to hear from patients.  Terry hopes to increase the available pool of information—information about patients’ genetics and other personal data—to help accelerate discovery. She’d like to see a systemic solution across all diseases, rather than solutions that work on a case by case basis.  To do that, she and her partners are putting in place a structure to empower disease groups and patients to vocalize their needs to the FDA and determine how and with whom their data is shared. Currently Genetic Alliance works with eight disease groups on PFDD and has enabled patients from each community to serve as guides with the goal of getting input from patient communities. It hopes to expand its work to more diseases in the next few years, and eventually to every disease.

Terry’s initiative has recently been awarded a $1 million contract from the Patient Centered Outcomes Research Institute to be a part of the National Patient Centered Clinical Research Network . This news could mean big things ahead for patients with genetic diseases.

If their work is successful, the FDA will begin to distinguish between the process for approving standard use drugs and the process for approving drugs that are designed to treat or cure severe disease. With increased collaboration between pharmaceutical companies and patients with a high risk tolerance, the FDA can gain data to stand on, which could help speed up drug patent approval. Patients would also be able to participate in drug design, thus improving the development process.

By challenging some of the obstacles that prevent access to potentially life-saving medicine, it may be possible to empower families to stop waiting and start acting. Risky? Maybe. But thousands of families risk even more every day.

This post is part of a series of essays on the power and potential of feedback loops to dramatically increase social impact (see the first one here ). This work is being catalyzed by Feedback Labs with support from the Rita Allen Foundation .

Come join us here .

Source: Forbes Business

 
Update
10

6 weeks ago

RM47mil KWSG contributions still unclaimed

Jun 9 2014 7:54am CDT | Source: Business Times Malaysia

Cambodia's Famous Battambang Circus
KUALA LUMPUR: About RM47 million of contributions in the Teachers Provident Fund (KWSG) still remain unclaimed, the Dewan Rakyat was tol ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
9

6 weeks ago

Gold shop lost almost RM1mil

Jun 9 2014 3:50am CDT | Source: Business Times Malaysia

Sydney FC v West Ham United - Football United New Zealand Tour 2014
KANGAR: A gold shop owner lost almost RM1 million after after the safe on in his shop was b ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
8

6 weeks ago

Motion to debate MAS losses in Dewan Rakyat rejected

Jun 9 2014 3:39am CDT | Source: Business Times Malaysia

Federal Aviation Administration Bans All US Flights To Israel
KUALA LUMPUR: AN emergency motion to debate the losses incurred by Malaysia Airlines last year, amounting to RM1.2 billion was rejected by the Dewan Raky ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
7

6 weeks ago

MH370 Tragedy: Hisham: RM27.6 mil spent on 1st phase of SAR

Jun 9 2014 2:11am CDT | Source: Business Times Malaysia

KUALA LUMPUR: Malaysia spent some RM27.6 million in its first phase of the search operations for missing Malaysia Airline flight MH370, said Acting Transport Minister, Datuk ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
6

6 weeks ago

9.1m litres of diesel seized in a month

Jun 8 2014 1:11am CDT | Source: Business Times Malaysia

INDIA-CHINA-DIPLOMACY-TRADE
PUTRAJAYA: The Domestic Trade, Cooperatives, and Consumerism ministry has seized some 9.1 million litres of diesel and property worth RM58 million since mounting ‘Operasi Diesel Selatan’ in the southe ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
5

7 weeks ago

Girl, 9, awarded RM2.78m compensation for medical negligence

Jun 6 2014 4:56am CDT | Source: Business Times Malaysia

Government Weekly Cabinet Meeting
KUALA LUMPUR: A nine-year-old girl who suffered brain damage during her birth at a government hospital was awarded over RM2.78 million in compen ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
4

7 weeks ago

Malaysia's total trade in April up 12pc

Jun 5 2014 11:52pm CDT | Source: Business Times Malaysia

KUALA LUMPUR: Malaysia's total trade in April 2014 rose by 12 per cent from a year ago to RM123.86 billion due to growing trading activities, International Trade and Industry Minister Datuk Seri Must ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
3

7 weeks ago

Works Ministry to spend RM20m for upgrading works at 50 accident black spots

Jun 4 2014 11:35pm CDT | Source: Business Times Malaysia

Iskandar Johor Open - Previews
JOHOR BARU: The Works Ministry will implement upgrading works at 50 accident prone locations in the country that have been identified this year involving an al ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
2

7 weeks ago

Najib launches loan scheme for Ramadan traders

Jun 4 2014 10:24pm CDT | Source: Business Times Malaysia

EurAsia Cup presented by DRB-HICOM - Day One
PUTRAJAYA: Prime Minister Datuk Seri Najib Razak today launches RM45 million Ramadan ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 
Update
1

7 weeks ago

Residents bring up objection against Kidex to Suhakam

Jun 4 2014 4:49am CDT | Source: Business Times Malaysia

PETALING JAYA: A group of 20 Petaling Jaya residents held a meeting with the Human Rights Commission of Malaysia (Suhakam) over their objection against the proposed RM2.2 billion Kinrara Damansara S ...
Source: Business Times Malaysia   Full article at: Business Times Malaysia
 

 

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