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Clinical Trails


Alphabet Soup:


Electronic data capture (EDC) companies are offering more integrated systems as they seek to provide enterprise-wide solutions including such features as clinical trial man-agement system and interactive voice response systems

Will moving EDC toward electronic health records be the ultimate integration play for this niche industry? Vendors also have begun to consider the possibility of merging EDC with EHR, which has the poten-tial of reducing errors by importing clinical trial data directly from electronic patient records. EHR is getting a major push from the federal govern-ment and large companies.

Issues of Early Access

Some fear that the status of randomized clinical trials may be jeopardized because of patients wanting direct access to an investi-gational drug at increasingly earlier stages.

“Often in organized compassionate-use programs, the trialled drug is made avail-able a short time before marketing authori-zation is granted. Named-patient compas-sionate use is often much earlier than this,” Abadie said. “This often hampers develop-ment and the full demonstration of the risk/benefit of the drug. When a new drug in compassionate use is assumed to be much more effective than the active con-trols on the market, the randomized clinical trial may be deemed unethical by many ethics committees. It may end up difficult to prove on solid grounds (i.e., through ran-domized control trials) that the new drug deserves marketing authorization.”

The amount of formal monitoring in compassionate-use programs depends on the drug’s development stage. For cohort programs, control and follow-up is more organized, not being left entirely to the indi-vidual physician with safety data such as serious adverse events nearly always collect-ed. Monitoring, however, remains limited.

“If compassionate use occurs in a non-approved indication, in a marketed product as is frequent with oncology medicines, there is less concern that there’s an under-reporting of serious adverse events within the approved label, as opposed to for a product with no marketing approval at all. Every use of an unapproved drug would provide additional information, and any serious or frequent adverse event that is not reported could potentially provide impor-tant information,

Another contentious issue is that there is no legislation on pricing for compassionate use, the fear being that any price charged for a program product would stick post author-ization.

One argument put forward is that prod-ucts should be provided free of charge to discourage companies from cashing in, and to push for proper clinical trials. However if a trial is already complete and compassion-ate use is for the rollover period before mar-keting authorization, some would surely question the validity of this view.

“Maybe there should be a fair market price arranged pre-approval, related to expected marketed price, or related to price in places where the product is available already. The product should be neither free nor overpriced.” Simon said.

Another suggestion was that products be reimbursed at development cost price to avoid any potential abuse of pre-approval profit making.

Regardless of whether the company pro-vides the drug free or charges, it has to decide at what point it’s safe and reasonable to conduct a program.

“It is important to provide therapeutic modalities to patients in need but as pro-grams are labor intensive, they don’t hap-pen for nothing; they cost the company in manpower. In terms of safety, they have to ask themselves if they can sanction a pro-gram with the data available to-date,” Simon said. “If the drug offers unique prop-erties benefiting patients, then ethically we are obliged to do it,” he added.

Any perceived lack of political will may stem not only from doubts about economic