How Patients and Patient Advocacy Groups Get New Treatments Approved

Did you know that it takes an average of $800 Million and 8 years for a drug manufacturer to complete research and development, prove safety and efficacy in multiple clinical trials? (read more at More so, in the arena of drugs for small population — “orphan” — illnesses, drug companies have little incentive to go down this road when they can only sell any resulting drug to a few hundred people. Compare that to 113 million prescriptions for high blood pressure drugs in 2005 alone!

The FDA, recognizing this disincentive to develop better treatments for orphan diseases, has developed strategies to allow, encourage, and make possible the development of drugseven for small populations.

Because so much of the cost of drug development happens long before a drug ever (might) be solda hurdle to even getting startedthe FDA has modeled some new concessions that could allow drugs to be released earlier to needy populations. These are tied to ongoing followup through a new program called REMS (Risk Evaluation and Mitigation Strategy). Now, with well documented and consistent survey data of a drug’s impact, a manufacturer may be allowed to release a drug that has not completed

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traditional drug-to-market clinical trials, when circumstances like a too small population exists, which can allow people with no good treatment solutions to have access to medicines in ways they never could, while still providing the FDA with all data necessary to ensure safety. Recording good quality data on the effects of drugslike rituximabbeing tried for P/P, helps those drugs gain FDA approval if there is evidence they are effective. This opens the door to better access, more insurance coverage, and better options.

In addition to sharing data about medications and disease history on an aggregate, de-identified basis, patients support the development of new drugs by participating in clinical trials. It would be a challenge for a manufacturer to even be able to run a clinical trial in many rare diseases as the patients are far apart, not well connected and, by definition, few. The more patients connect to patient advocacy groups representing rare disorders, the better chance that a drug study could be offered to more people to allow for meaningful proof of effectiveness. This is true also of tissue-bank collections. With an extensive spectrum of blood/tissue samples available for analysis, opportunities for seeing patterns that may provide answers, increase. Most drug manufacturers of orphan drugs begin their clinical trials process by reaching out to patient organizations to help them get connected to their relevant community. Because medical information is protected, no patient/member names may be disclosed, however, information about a trial may be sent out and patients can investigate further if they are interested.

How does this relate to you?

The IPPF is launching a new science advocacy campaign in January. Titled “1010 in 2010, we are determined to get a minimum of 1000 (plus a few to spare) “data-sets”. That’s you all! We need 1000 disease history/medication experiences recorded so that the numbers are (statistically) significant to define treatment opportunities.

This data will be used when our Medical Advisory Board (MAB) and P/P experts from around the world gather at the NIH in the Fall of 2010 to define consensus and advise on protocols.

Now, all this data collection will be gathered in an online surveyno phone calls, no appointments (unless you want), just log in, enter your meds, improvements and flaresat midnight, in your pajamas, on your cell phone, whatever works for you. Watch the IPPF website as the numbers rise and find out interesting facts from the data! We appreciate what you contribute to helping everyone have access to better treatments!

Опубликовано в Issue 60 - Spring 2010
The P/P Registry has been approved by the Western Institutional Review Board (WIRB) and is actively enrolling participants.