the National Cures Agenda
TCTF supports an “alternative initial approval” process that would give drug manufacturers the legal ability to introduce new drugs into the market as well as give payers the ability to reimburse patients for new, qualifying drugs.
Pharmaceutical companies support getting new medicines into the market as soon and as safely possible. Real-use information is invaluable for improving products, defining safety parameters, and identifying sub-patient populations that achieves significantly better treatment results. Even FDA Phase III trials, despite long durations of testing and expense, cannot predict with certainty all the effects from new drugs which can later surface when in real-use among a heterogeneous population.
Allowing early access to promising new drugs shortens the amount of time it takes for a drug to go from the laboratory to patients. This would have an enormously positive effect on patients. It would literally save lives. Accelerated access and a concomitant information flow would greatly improve pharmaceutical research and development decisions and spur innovation.
TCTF supports the establishment of a centralized database of patient information and outcomes (observational data) when a new drug is used. Big data computer systems have the ability to gather, segment, and disseminate medical information based on an array of variables to identify patient sub-populations in order to get the right medicines to the right future patient. Major software companies already do this.
Providing the legal framework allows companies, research institutions, doctors and patients to exchange information more rapidly and comprehensively so a diversity of participants can contribute to medical discoveries. But patients alone would have the ability to permit public access to their personal medical information.
The competitiveness of the pharmaceutical industry is currently dependent on scientific skill, the control of information, competitive intelligence, and patents owned. Unlike academic and some government institutions, which have to publish findings to gain more funding, corporations are motivated to keep information internal. Even the FDA withholds information from the public in an iterative process designed to assess a drug. This approach does not allow new information and discoveries made in trials to be shared. Instead, companies have to redesign new trials to meet new end-points required by the FDA, adding time and expense to a drug’s approval. The ability to finds cures resides in sharing information widely and exchanging data freely, but neither government approval processes nor private corporations currently have the incentive to do so.
TCTF supports decriminalization of a patent holder (drug corporation) from providing information about drugs used "off-label, to allow information exchanges to be accessible so patients can choose to share data voluntarily, and provide an FDA post-market approval designation without having to resubmit drugs to FDA trials.
While in trials, the FDA guards information about a drug’s performance. Medicines prescribed off-label have no formal system to gather information about their use. And current law forbids companies (patent holders) from marketing or discussing a medicine’s use for something other than for what it was originally tested and approved. The only source of medical information is when it is submitted voluntarily by a patient because patient-outcome exchanges are unregulated.
Another major impediment to utilizing medical information in the search for cures is the confusing array of intellectual property rights that govern the use and dissemination of information, including the extent to which companies, government, doctors and patients may share those rights.
Fortunately, the FDA does not have the authority to intervene directly between doctor and patient, and so cannot restrict the use of a prescribed drug. However, because of this situation, both the FDA and the drug companies have no ability to gather patient-outcomes for assessment. But, it does restrict the medical system from the widespread application of new knowledge which has been shared informally among doctors who know each other, or choose to publish in medical journals about a particular drug’s effectiveness.
TCTF supports a restructuring of "compassionate use" so patients would be allowed, under the guidance of their doctors, but without having to get permission from the government, to have access to investigational new drugs which have passed FDA Phase I safety trials and successfully completed one or more Phase II trials.
Patient-consumers in consultation with their doctors should be the ultimate decision makers when it comes time to pursuing treatment options and using medication, including medicine that has yet to be approved by the FDA. The problem is that today's information systems are fragmented and/or vertical and need to be horizontal. Commercial models such as MyTomorrows are available in Europe, which links patients seeking alternatives to doctors and medicines globally. These avenues of access are not available in the United States because current US law restricts access and information so tightly we don't even have a path, much less an avenue of access. It is a deeply personal choice, rooted in the individual's fundamental rights, to try to preserve life even by using medicines not yet fully approved. In life-threatening situations, it is inhumane to restrict patient access.