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PTC sued by an angry parent in search of PTC124
PTC Therapeutics has been on a roll over the last few months. But the biotech hit a loud bump when the New York Times featured the story of one mother's legal fight to gain access to PTC124 for her son, who suffers from Duchenne muscular dystrophy. PTC just inked a $437 million deal with Genzyme for PTC124, which has demonstrated promising results for treating a segment of the Duchenne MD population. Cheri Gunvalson says that the experimental therapy is her son's last and best hope. But PTC isn't providing the therapy to anyone who isn't in the clinical trial and the boy, who can no longer walk, isn't eligible. Gunvalson is asking for access as part of a compassionate use approach that a number of drug developers have in place, but PTC CEO Stuart Peltz has said that there's been too little experience with the mid-stage therapy to provide it outside of a trial.
The Times notes that for many people an experimental drug is their only hope. But the Abigail Alliance for Better Access to Developmental Drugs took their case for broader access to experimental cancer therapies to court last year--and lost.
- read the article in the New York Times
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Comments
By techdiligence | Posted 2:36pm | July 18, 2008
In the Abigail Alliance case, the court did reach the correct constitutional conclusion; it is now up to Congress to propose legislation allowing access to unapproved drugs for compassionate use. Unfortunately it will almost certainly take a further decision by the Supreme Court, or success in a suite such as this one, to get the attention of Congress.
As a chemist concerned with the development and production of API for clinical trials, I know that any increase in the demand for material outside of the current system for supplying clinical trials will be far, far more expensive than even the most expensive approved, marketed drug that currently exists. But this is strictly a financial point and could possibly be solved by allowing tax-credits or other financial inducements to the pharma/biotech industry. Not politically attractive, but since an unapproved, unmarketed drug is only being made by one company and there is no commercial production facility either off-shore or domestic, any financial arrangement would be completely limited to one drug for one company for non-commercial use; hardly a get-rich scenario.
As a brother of a leukemia patient, I am not insensitive to the desires of those who are running out of options, and their families. I firmly believe that expanded availability of experimental drugs outside of the current system of designed clinical trials would show good utility of drugs in completely unexpected niches that would not be discovered otherwise in standard clinical trial designs. However, anything that damages the collection of data under controlled conditions will destroy the entire system for determining the safety and efficacy of a new drug, so any data from “un-controlled†use must NOT be included in data from a designed clinical trial. Perhaps the creation of a “Phase X†(“X†for “experimentalâ€) could be a solution; those receiving the drug in an otherwise "uncontrolled" clinical setting could not be counted in the data for the designed, approved clinical trials, but their results would be publicly available for analysis by any method, by any party. Such a Phase X trial could also be the vehicle for covering the legal and ethical concerns of in the face of voluntary consent which cannot be "informed", human experimentation when there is no information that the drug might be benefit, indemnification, etc.
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