Mar 6 2014, 12:01am CST | by Forbes
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.
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Source: Forbes Business
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