Terminal or Seriously Ill Patients· Terminal or Seriously Ill Patients· Ability to Access Experimental Ability to Access Experimental Therapies Therapies By I&C President Ryan Witt By I&C President Ryan Witt [email protected][email protected]
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Seriously Ill Patients' Access to Experimental Therapies
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8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
The ability of a person The ability of a person, who has a, who has a disease with no approveddisease with no approvedtherapy availabletherapy available, to access a therapy while it is in its, to access a therapy while it is in itsexperimental stages of study either in that study experimental stages of study either in that study or or outside of outside of that study.that study.
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
Studies in humans, often to determine a therapy orStudies in humans, often to determine a therapy ordevice·s safety and efficacy (the ability to produce andevice·s safety and efficacy (the ability to produce aneffect ).effect ).
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
There are There are 3 phases3 phases in Clinical Trials prior to FD A approvalin Clinical Trials prior to FD A approval
PreclinicalPreclinical ² ² cell culture and in animalscell culture and in animals
Phase 0Phase 0 ² ² 1010--15 patients to study if drug works the way it·s15 patients to study if drug works the way it·sexpected, based on preclinical studies; no data onexpected, based on preclinical studies; no data on
safety/efficacy safety/efficacy
1.1. Phase IPhase I ² ² maximum tolerable dose, safety, and metabolismmaximum tolerable dose, safety, and metabolism
2.2. Phase IIPhase II ² ² safety and effectiveness (200safety and effectiveness (200--500 ppl )500 ppl )
3.3. Phase IIIPhase III ² ² safety and effectiveness compared to SoC ( 500safety and effectiveness compared to SoC ( 500--
2000 ppl )2000 ppl )FD A Marketing Approval (exclusivity )FD A Marketing Approval (exclusivity )
Phase IV Phase IV ² ² postpost--approval safety surveillanceapproval safety surveillance
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
Why is enrollment so low? Why is enrollment so low?
1.1. Patients do not know about, or understandPatients do not know about, or understand
what is offered in, clinical trials. what is offered in, clinical trials. Physician·s do not inform them (time, paper work; Pt may Physician·s do not inform them (time, paper work; Pt may
not be the ¶ideal· candidate, physician may not feel patientnot be the ¶ideal· candidate, physician may not feel patient
would be able to afford it, and more ) would be able to afford it, and more )
Janine Kresin Janine Kresin
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
Physicians are bad people ² they simply don·t have the time orthe tools to deal with all of their patients· searches for clinicaltrials (imagine 10 patients coming to a physician with 20 trialseach ), as well patients may not be ideal for clinical trial settingsand may not be able to afford a therapy.
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
3.3. Insurers will not pay for tests to show you are eligibleInsurers will not pay for tests to show you are eligible4.4. Can·t pay for therapy or to travel to clinical siteCan·t pay for therapy or to travel to clinical site5.5. Getting Getting the therapy isthe therapy is randomrandom
6.6. Therapy may NOT be effective or safe Therapy may NOT be effective or safe
anyway (~7% of CTs in phase I are approved by anyway (~7% of CTs in phase I are approved by the FD A as safe and efficacious )the FD A as safe and efficacious )
Seriously Compromising IllnessesSeriously Compromising Illnesses7.7. Too weak to travel Too weak to travel8.8. Want to be close to work, family, and friends Want to be close to work, family, and friends
*Given aforementioned reasons, many patients do not enroll.*Given aforementioned reasons, many patients do not enroll.
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ProgramsPrograms instituted by pharmaceuticals / drug sponsorsinstituted by pharmaceuticals / drug sponsors to offerto offertheir experimental therapy to patientstheir experimental therapy to patients n ot n ot enrolled inenrolled incorresponding clinical trials.corresponding clinical trials.
Protocol:Protocol: Pharmaceutical asks the FD A,Pharmaceutical asks the FD A,
FD A approves/denies,FD A approves/denies,
Subsequent Expanded Access program is instituted or not.Subsequent Expanded Access program is instituted or not.
How to Apply?How to Apply?
Talk to your physician, contact the pharmaceutical / therapy Talk to your physician, contact the pharmaceutical / therapy sponsor, approval / denialsponsor, approval / denial --> receive therapy in mail> receive therapy in mail
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Compassionate Use (CU)/ Single PtCompassionate Use (CU)/ Single Pt
Investigational New Drug (IND)Investigational New Drug (IND) For anFor an individual patient individual patient applying to access an experimental therapy, outsideapplying to access an experimental therapy, outside
of the corresponding clinical trial / experimental study.of the corresponding clinical trial / experimental study. Protocol:Protocol:
Patient requests access to the desired therapy through a licensed physician,Patient requests access to the desired therapy through a licensed physician, Physician asks pharmaceutical,Physician asks pharmaceutical,
Pharma approves / denies; and, pending approval by pharmaPharma approves / denies; and, pending approval by pharma ---- FD A request is submitted and final FD A approval is denied or granted. ( And,FD A request is submitted and final FD A approval is denied or granted. ( And,
YES, sometimes the FD A is the one to deny you access. ) YES, sometimes the FD A is the one to deny you access. )
How to apply?How to apply? Talk with your physician, contact therapy sponsor, then FD A Talk with your physician, contact therapy sponsor, then FD A
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
1.1. Many don·t know this opportunity exists; physicians don·t tellMany don·t know this opportunity exists; physicians don·t tellthem; researchers don·t know about it and pharmas don·tthem; researchers don·t know about it and pharmas don·tbroadcast it.broadcast it.
2.2. Pharmas are worried aboutPharmas are worried about
Getting suedGetting sued
Bad press.Bad press.
As well As well
3.3. Phase II Limitations: ThePhase II Limitations: The more experimentalmore experimental the study the study , the, the
less likely it is the FD A will approve this accessless likely it is the FD A will approve this access due to moredue to moresafety concerns and less proof of efficacy safety concerns and less proof of efficacy ..
4.4. Incentives for therapy sponsors are uneconomical and do notIncentives for therapy sponsors are uneconomical and do notgive patients the full scope of opportunity.give patients the full scope of opportunity.
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
Title 21, Sec 312.34 of FD A rules and regulations implies the Title 21, Sec 312.34 of FD A rules and regulations implies theFD A is open to approving this ´experimental accessµ on anFD A is open to approving this ´experimental accessµ on anExpanded Access or Compassionate Use basis, however theExpanded Access or Compassionate Use basis, however theFD A neglects to uphold this for therapies in phase II trial andFD A neglects to uphold this for therapies in phase II trial and
prior.prior. Link toLink to FD A·s exact protocol for Treatment INDsFD A·s exact protocol for Treatment INDs
ollection Results to seriously ill patient care:Results to seriously ill patient care:
a )a ) Need cliniciansNeed clinicians to deliver therapy rather than simply to deliver therapy rather than simply
physicians, as these programs are effectively a ´studyµ andphysicians, as these programs are effectively a ´studyµ and
clinical trial protocols must be followed.clinical trial protocols must be followed.b )b ) Patient can rarely combine investigational therapiesPatient can rarely combine investigational therapies with with
currently approved therapies.currently approved therapies.
Ex:Ex: JOSEPH DOUGHERTY JOSEPH DOUGHERTY
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
3.3. Compassionate Use / single patient INDsCompassionate Use / single patient INDs
4.4. Emergency Use (not covered )Emergency Use (not covered )
While only 6% of therapies in phase I clinical trial reach final While only 6% of therapies in phase I clinical trial reach finalFD A approval (are proven to be safe and effective ), this isFD A approval (are proven to be safe and effective ), this isenough hope for some.enough hope for some.
Joshua Thompson Joshua Thompson
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies
Pharmas, regulators, and physicians are people tooPharmas, regulators, and physicians are people too ² ²
compassionate, yet don·t like to be cornered; are faced with theircompassionate, yet don·t like to be cornered; are faced with their
own constraints and demands on them, are sometimesown constraints and demands on them, are sometimes
desensitized to seriously ill patients, and have a limited time.desensitized to seriously ill patients, and have a limited time.
They are human, too. This isn·t a movie They are human, too. This isn·t a movie ² ² this is your and your lovedthis is your and your lovedone·s lives. Put yourself in others shoes before you act.one·s lives. Put yourself in others shoes before you act.
8/9/2019 Seriously Ill Patients' Access to Experimental Therapies