Seriously Ill Patients Access To Experimental Therapies
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There are four main methods for terminally or seriously ill patients to access experimental therapies: clinical trials, expanded access programs, compassionate use protocols, and emergency use. However, few patients enroll in clinical trials or access experimental therapies through other means due to various barriers. These barriers include lack of awareness about opportunities, concerns from drug companies about liability and bad publicity, limitations on access to early-stage experimental therapies, and lack of incentives for drug companies to provide access. Efforts are needed to help more patients learn about and navigate options to access potentially life-saving experimental treatments.
![Terminal or Seriously Ill Patients’ Ability to Access Experimental Therapies By I&C President Ryan Witt [email_address]](https://image.slidesharecdn.com/seriouslyillpatientsaccesstoexperimentaltherapies-100404201250-phpapp01/85/seriously-ill-patients-access-to-experimental-therapies-1-320.jpg)
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Seriously Ill Patients Access To Experimental Therapies
- 1. Terminal or Seriously Ill Patients’ Ability to Access Experimental Therapies By I&C President Ryan Witt [email_address]
- 10. This is not because… Physicians are bad people – they simply don’t have the time or the tools to deal with all of their patients’ searches for clinical trials (imagine 10 patients coming to a physician with 20 trials each), as well patients may not be ideal for clinical trial settings and may not be able to afford a therapy.
- 13. Access to Experimental Therapies outside of a Clinical Trial
Editor's Notes
- A terminal patient’s ability to access experimental therapies refers to a terminal patient’s, a person whose prognosis is inevitable and imminent death, ability to access therapies in experimentation in humans (clinical trials) or pre-clinical investigations, outside of those experiments. For example, if you had terminal cancer and thought Avastin might benefit you, but it was in phase I clinical trial and you didn’t meet the eligibility requirements to enter into that trial. It is access to this therapy and this patient’s desire that we seek to meet. This demand is currently met by two very similar FDA protocols: Expanded Access programs and Compassionate Use (single patient IND) protocol. Expanded Access refers to when a therapy sponsor (a manufacturer or distributor of the therapy) believes their therapy would benefit these desperately ill patients and would like to provide this service. For these institutions, they would apply to the FDA with a set of eligibility requirements of who they believe would benefit from their therapy, and the FDA would grant or deny this request to provide this service. If they accept, then the sponsor can provide it. Compassionate Use refers to a single patient who believes he or she might benefit from a therapy. If this were the case, he or she would talk to his/her physician, who would contact the therapy-in-question’s sponsor to ask if the sponsor is willing to provide this service. If they are, these three parties take their case to the FDA, who then approves or disapproves of this use.
- Single Patient Investigational New Drug (IND)
- Why – because, by definition, serious illnesses do not have very effective therapies approved for them. Additionally, clinical trials and experimental settings are quite often the location where the most therapeutic innovation is being developed or validated. Moreover, a drug in phase I is the same drug that may eventually get approved; and, if you are battling a terminal illness – with no effective therapy approved – you may want to explore these potentially safe and efficacious innovations.
- Drug sponsors = manufacturers / makers of therapies
- Joshua Thompson - http://www.nytimes.com/2009/05/17/health/policy/17untested.html?_r=3&ref=health Abigail Burroughs - http://www.abigail-alliance.org/ My grandfather - http://innovationandchoice.com/voices-of-the-people/marshallwitt/ Others - http://innovationandchoice.com/voices-of-the-people/
- Some people don’t know this opportunity exists as an option. The FDA currently does not approve this access for therapies in phase II clinical trial and prior. Thirdly, the incentives for providing this access to terminal patients are uneconomical and not good for patients.
- **Fixing this issue might also be applicable to improving efficient enrollment in clinical trials because a patient battling a terminal disease would be more likely to search for applicable clinical trials if they felt they may still be able to access that therapy even without participating in those trials.
- Implication : https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfCFR/CFRSearch.cfm?fr=312.34 VERSE Reality : Talk to Abigail Burroughs and Joshua Thompson - http://www.abigail-alliance.org/ (their contact info is on their website)
- Reason Provenge wasn’t issued on an Expanded Access basis, even after it was recommended for final FDA approval by an advisory panel by unanimous vote, is because the sponsor of the therapy was a small pharmaceutical and could not afford to do this.
- As one can see, there is no motivation for a pharmaceutical or drug sponsor to undertake this transaction and provide this service with this direct cost recovery limit. (The dotted-red line indicates a price ceiling at Average Total Cost, prohibiting anyone from ever having an incentive to undertake an action.) Furthermore, even if the kindness of corporate CEOs’ and investors’ hearts were that generous, society wants these sponsors to offer these programs. The reason for this is because profits allows smaller pharmaceuticals and researchers -- most often the most innovative of the group, and those with the fewest resources -- to have an incentive to provide this service, giving people fighting very serious illnesses all the opportunity the United States can when it comes to fighting for their lives and improving their health. We want these firms to participate to stimulate opportunity for our family members and our loved ones.
- Source – clincialtrials.gov
- Joshua Thompson’s ALS progression reversed, when he was given miracle drug Iplex, while on life support.