Terminal or Seriously Ill Patients’ Ability to Access Experimental Therapies By I&C President Ryan Witt [email_address]
Remember <ul><li>Ask  Q s </li></ul><ul><li>Always  ask “ Why/how, What, and When” </li></ul><ul><li>Try to  u nderstand E...
Access to what? <ul><li>The ability of a person , who has a  disease with no approved therapy available , to access a ther...
Four Methods of Access to Experimental Therapies <ul><ul><ul><li>Clinical Trials </li></ul></ul></ul><ul><ul><ul><li>Expan...
Clinical Trials (CTs) <ul><li>Studies in humans, often to determine a therapy or device’s safety and efficacy (the ability...
Phases of Testing <ul><li>There are  3 phases  in Clinical Trials prior to FDA approval </li></ul><ul><ul><li>Preclinical ...
Why do we care? <ul><li>Seriously ill patients – no effective or safe therapy </li></ul><ul><li>CTs represent the most inn...
Enrollment in Clinical Trials <ul><li>However, despite this benefit…  FEW  with serious illnesses take advantage of this o...
Why is enrollment so low? <ul><li>Patients do not know about, or understand what is offered in, clinical trials. </li></ul...
This is not because… Physicians are bad people – they simply don’t have the time or the tools to deal with all of their pa...
Other Reasons <ul><li>Patients do not meet the eligibility requirements </li></ul>
More Reasons <ul><li>Insurers will not pay for tests to show you are eligible </li></ul><ul><li>Can’t pay for therapy or t...
Access to Experimental Therapies  outside  of a Clinical Trial
Expanded Access (EA) Programs <ul><li>Programs  instituted by pharmaceuticals / drug sponsors  to offer their experimental...
Compassionate Use (CU)/ Single Pt Investigational New Drug (IND) <ul><li>For an  individual patient  applying to access an...
Devil’s in the Details <ul><li>Four key   problems  with Expanded Access / Compassionate use protocols </li></ul>
This is the story of <ul><li>Joshua Thompson, ALS </li></ul><ul><li>Abigail Burroughs, cancer </li></ul><ul><li>My grandfa...
Problems are <ul><li>Many don’t know this opportunity exists; physicians don’t tell them; researchers don’t know about it ...
1.  People Don’t Know <ul><li>Some people don’t know the opportunity to access experimental therapies outside of correspon...
2.  Pharma Worries <ul><li>Getting sued </li></ul><ul><li>Bad press </li></ul>
3. Phase II Limitations <ul><li>Title 21, Sec 312.34 of FDA rules and regulations implies the FDA is open to approving thi...
4. Incentives <ul><li>Direct cost recovery </li></ul><ul><li>External-of-trial data collection </li></ul>
Incentive: Direct Cost Recovery <ul><li>Through an Economist’s Eye </li></ul><ul><li>Effective price ceiling at a firm’s a...
Incentive: External-of-trial Data Collection <ul><li>Results to seriously ill patient care: </li></ul><ul><li>Need clinici...
Terminal Patient Access <ul><li>All of these reasons help explain why only  8 of over 8,000 open clinical trials for cance...
 
Review <ul><li>Four ways  to access experimental therapies: </li></ul><ul><ul><li>Clinical Trials </li></ul></ul><ul><ul><...
What to do? <ul><li>Consult your physician about  clinical trials ,  compassionate use , and  expanded access  -- if you h...
What Are We Doing? <ul><li>Bringing innovation to you at the  fastest rate possible  is our goal. </li></ul><ul><li>Commun...
Remember <ul><li>Pharmas, regulators, and physicians are people too – compassionate, yet don’t like to be cornered; are fa...
 
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Seriously Ill Patients Access To Experimental Therapies

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  • 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&amp;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.
  • Seriously Ill Patients Access To Experimental Therapies

    1. 1. Terminal or Seriously Ill Patients’ Ability to Access Experimental Therapies By I&C President Ryan Witt [email_address]
    2. 2. Remember <ul><li>Ask Q s </li></ul><ul><li>Always ask “ Why/how, What, and When” </li></ul><ul><li>Try to u nderstand EVERYTHING! </li></ul>
    3. 3. Access to what? <ul><li>The ability of a person , who has a disease with no approved therapy available , to access a therapy while it is in its experimental stages of study either in that study or outside of that study. </li></ul>
    4. 4. Four Methods of Access to Experimental Therapies <ul><ul><ul><li>Clinical Trials </li></ul></ul></ul><ul><ul><ul><li>Expanded Access (EA) programs </li></ul></ul></ul><ul><ul><ul><li>Compassionate Use (CU) protocol </li></ul></ul></ul><ul><ul><ul><li>Emergency Use </li></ul></ul></ul>
    5. 5. Clinical Trials (CTs) <ul><li>Studies in humans, often to determine a therapy or device’s safety and efficacy (the ability to produce an effect). </li></ul>
    6. 6. Phases of Testing <ul><li>There are 3 phases in Clinical Trials prior to FDA approval </li></ul><ul><ul><li>Preclinical – cell culture and in animals </li></ul></ul><ul><ul><li>Phase 0 – 10-15 patients to study if drug works the way it’s expected, based on preclinical studies; no data on safety/efficacy </li></ul></ul><ul><ul><li>Phase I – maximum tolerable dose, safety, and metabolism </li></ul></ul><ul><ul><li>Phase II – safety and effectiveness (200-500 ppl) </li></ul></ul><ul><ul><li>Phase III – safety and effectiveness compared to SoC (500-2000 ppl) </li></ul></ul><ul><ul><li>FDA Marketing Approval (exclusivity) </li></ul></ul><ul><ul><li>Phase IV – post-approval safety surveillance </li></ul></ul>
    7. 7. Why do we care? <ul><li>Seriously ill patients – no effective or safe therapy </li></ul><ul><li>CTs represent the most innovative therapies available. </li></ul><ul><li>In the case of current technology, this is -- </li></ul><ul><ul><ul><li>Targeted therapies </li></ul></ul></ul><ul><ul><ul><ul><li>Bivalent antibodies </li></ul></ul></ul></ul><ul><ul><ul><ul><li>Novel inhibitors </li></ul></ul></ul></ul><ul><ul><ul><ul><li>Gene Therapy with innovative delivery techniques and payloads </li></ul></ul></ul></ul>
    8. 8. Enrollment in Clinical Trials <ul><li>However, despite this benefit… FEW with serious illnesses take advantage of this opportunity. </li></ul><ul><ul><li>For cancer: an estimated 3 to 5 percent of all adult patients ever enroll </li></ul></ul>
    9. 9. Why is enrollment so low? <ul><li>Patients do not know about, or understand what is offered in, clinical trials. </li></ul><ul><ul><li>Physician’s do not inform them (time, paper work; Pt may not be the ‘ideal’ candidate, physician may not feel patient would be able to afford it, and more) </li></ul></ul><ul><ul><li>Janine Kresin </li></ul></ul>
    10. 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.
    11. 11. Other Reasons <ul><li>Patients do not meet the eligibility requirements </li></ul>
    12. 12. More Reasons <ul><li>Insurers will not pay for tests to show you are eligible </li></ul><ul><li>Can’t pay for therapy or to travel to clinical site </li></ul><ul><li>Getting the therapy is random </li></ul><ul><li>Therapy may NOT be effective or safe </li></ul><ul><li>anyway (~7% of CTs in phase I are approved by </li></ul><ul><li>the FDA as safe and efficacious) </li></ul><ul><li>Seriously Compromising Illnesses </li></ul><ul><li>Too weak to travel </li></ul><ul><li>Want to be close to work, family, and friends </li></ul><ul><li>*Given aforementioned reasons, many patients do not enroll. </li></ul>
    13. 13. Access to Experimental Therapies outside of a Clinical Trial
    14. 14. Expanded Access (EA) Programs <ul><li>Programs instituted by pharmaceuticals / drug sponsors to offer their experimental therapy to patients not enrolled in corresponding clinical trials. </li></ul><ul><li>Protocol: </li></ul><ul><ul><li>Pharmaceutical asks the FDA, </li></ul></ul><ul><ul><li>FDA approves/denies, </li></ul></ul><ul><ul><li>Subsequent Expanded Access program is instituted or not. </li></ul></ul><ul><li>How to Apply? </li></ul><ul><ul><li>Talk to your physician, contact the pharmaceutical / therapy sponsor, approval / denial -> receive therapy in mail </li></ul></ul>
    15. 15. Compassionate Use (CU)/ Single Pt Investigational New Drug (IND) <ul><li>For an individual patient applying to access an experimental therapy, outside of the corresponding clinical trial / experimental study. </li></ul><ul><li>Protocol: </li></ul><ul><ul><li>Patient requests access to the desired therapy through a licensed physician, </li></ul></ul><ul><ul><li>Physician asks pharmaceutical, </li></ul></ul><ul><ul><li>Pharma approves / denies; and, pending approval by pharma -- </li></ul></ul><ul><ul><li>FDA request is submitted and final FDA approval is denied or granted. (And, YES, sometimes the FDA is the one to deny you access.) </li></ul></ul><ul><li>How to apply? </li></ul><ul><ul><li>Talk with your physician, contact therapy sponsor, then FDA </li></ul></ul>
    16. 16. Devil’s in the Details <ul><li>Four key problems with Expanded Access / Compassionate use protocols </li></ul>
    17. 17. This is the story of <ul><li>Joshua Thompson, ALS </li></ul><ul><li>Abigail Burroughs, cancer </li></ul><ul><li>My grandfather, lung cancer </li></ul><ul><li>Many others – Alzheimer’s, ALS, cancer, and more. </li></ul>
    18. 18. Problems are <ul><li>Many don’t know this opportunity exists; physicians don’t tell them; researchers don’t know about it and pharmas don’t broadcast it. </li></ul><ul><li>Pharmas are worried about </li></ul><ul><ul><li>Getting sued </li></ul></ul><ul><ul><li>Bad press. </li></ul></ul><ul><li>As well </li></ul><ul><li>Phase II Limitations: The more experimental the study , the less likely it is the FDA will approve this access due to more safety concerns and less proof of efficacy . </li></ul><ul><li>Incentives for therapy sponsors are uneconomical and do not give patients the full scope of opportunity. </li></ul>
    19. 19. 1. People Don’t Know <ul><li>Some people don’t know the opportunity to access experimental therapies outside of corresponding experimental studies exists. </li></ul><ul><li>Ex: my grandfather. </li></ul>
    20. 20. 2. Pharma Worries <ul><li>Getting sued </li></ul><ul><li>Bad press </li></ul>
    21. 21. 3. Phase II Limitations <ul><li>Title 21, Sec 312.34 of FDA rules and regulations implies the FDA is open to approving this “experimental access” on an Expanded Access or Compassionate Use basis, however the FDA neglects to uphold this for therapies in phase II trial and prior. </li></ul><ul><ul><li>Link to FDA’s exact protocol for Treatment INDs </li></ul></ul><ul><li>I.e., Abigail Burroughs & Joshua Thompson </li></ul>
    22. 22. 4. Incentives <ul><li>Direct cost recovery </li></ul><ul><li>External-of-trial data collection </li></ul>
    23. 23. Incentive: Direct Cost Recovery <ul><li>Through an Economist’s Eye </li></ul><ul><li>Effective price ceiling at a firm’s average total cost, depleting any incentive for a sponsor to provide this opportunity to patients. </li></ul><ul><li>Example: </li></ul><ul><li>Provenge </li></ul>
    24. 24. Incentive: External-of-trial Data Collection <ul><li>Results to seriously ill patient care: </li></ul><ul><li>Need clinicians to deliver therapy rather than simply physicians, as these programs are effectively a “study” and clinical trial protocols must be followed. </li></ul><ul><li>Patient can rarely combine investigational therapies with currently approved therapies. </li></ul><ul><li>Ex: JOSEPH DOUGHERTY </li></ul>
    25. 25. Terminal Patient Access <ul><li>All of these reasons help explain why only 8 of over 8,000 open clinical trials for cancer offer a corresponding Expanded Access program to terminal patients . </li></ul><ul><ul><li>(PHARMAS don’t care to start programs -- no incentives and too much fear!) </li></ul></ul>
    26. 27. Review <ul><li>Four ways to access experimental therapies: </li></ul><ul><ul><li>Clinical Trials </li></ul></ul><ul><ul><li>Expanded Access programs </li></ul></ul><ul><ul><li>Compassionate Use / single patient INDs </li></ul></ul><ul><ul><li>Emergency Use (not covered) </li></ul></ul><ul><li>While only 6% of therapies in phase I clinical trial reach final FDA approval (are proven to be safe and effective), this is enough hope for some. </li></ul><ul><ul><ul><li>Joshua Thompson </li></ul></ul></ul>
    27. 28. What to do? <ul><li>Consult your physician about clinical trials , compassionate use , and expanded access -- if you have a serious illness. </li></ul><ul><li>ClinicalTrials.gov </li></ul><ul><li>Other CT search engines </li></ul>
    28. 29. What Are We Doing? <ul><li>Bringing innovation to you at the fastest rate possible is our goal. </li></ul><ul><li>Community-based education and resource sharing </li></ul><ul><li>Building an interface to empower you to make a difference. </li></ul><ul><li>Working to integrate these innovations and patient options into physicians’ workflow. </li></ul>
    29. 30. Remember <ul><li>Pharmas, regulators, and physicians are people too – compassionate, yet don’t like to be cornered; are faced with their own constraints and demands on them, are sometimes desensitized to seriously ill patients, and have a limited time. </li></ul><ul><ul><ul><li> They are human, too. This isn’t a movie – this is your and your loved one’s lives. Put yourself in others shoes before you act. </li></ul></ul></ul>

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