Providing patients access to novel medical products can be challenging to navigate for sponsors and trial participants alike.
To learn more about expanded access, compassionate use, Right to Try laws, I invited Dr. Alison Bateman-House, PhD, MPH, MA, an Assistant Professor in the Division of Medical Ethics at NYU Grossman School of Medicine.
Dr. Bateman-House is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies ethical issues concerning access to investigational medical products and which consists of patient advocates, clinicians, members of industry, current and former FDA staffers, lawyers, and academics, among others.
She advises biopharmaceutical companies, patient advocacy organizations, governmental, and non-governmental entities about clinical trial design and non-trial access programs, and she serves as ethicist for numerous data safety monitoring boards overseeing clinical trials.
She has published and spoken extensively on non-trial access to investigational medical products, equity in clinical trials, individualized therapeutics, the history and ethics of using humans as research subjects, and public health ethics.
Sponsor(s):
This podcast is brought to you by Florence Healthcare. Florence eliminates chaotic workflows in clinical research operations with remote access and digital workflow platforms. More than 12,000 study sites, sponsors, and CROs in 45 countries trust them to accelerate their operations. To learn more, visit https://florencehc.com
This podcast is brought to you by Slope. Slope drives operational excellence for highly complex, sample-intensive, early-phase clinical trials. The platform transforms chaotic clinical trial supply chains into protocol-specific operational workflows for sponsors, CROs, clinical research sites and labs. Learn more at https://www.slopeclinical.com/
SELECTED LINKS FROM THE EPISODE:
Dr. Alison Bateman-House – New York University Profile
New York University Grossman School of Medicine
New York University Janssen Pharmaceutical Compassionate Use Advisory Committee
Working Group for Compassionate Use and Preapproval Access
United States Food and Drug Agency – Right to Try
Right-to-Try Laws: Hope, Hype, and Unintended Consequences (2015)
Medicare – A federal health insurance program in the United States for people age 65 or older and younger people with disabilities
Medicaid – A government program in the United States that provides health insurance for adults and children with limited income and resources
Books:
Social Transformation of American Medicine by Paul Starr (Amazon, Barnes & Noble)
Gospel of Germs by Nancy Tomes (Amazon, Barnes & Noble)
Pure and Modern Milk by Kendra Smith-Howard (Amazon, Barnes & Noble)
Show Notes:
[0:20] Dr. Allison Bateman background
- Assistant Professor in the Division of Medical Ethics at NYU Grossman School of Medicine
- Co-chair of the Working Group on Compassionate Use and Preapproval Access
- Non-Voting Chair of the New York University Janssen Pharmaceutical Compassionate Use Advisory Committee for infectious disease and neurology and psychology
[2:31] Sponsor Message: This podcast is brought to you by Florence Healthcare. Florence eliminates chaotic workflows in clinical research operations with remote access and digital workflow platforms. More than 10,000 study sites, sponsors, and CROs in 45 countries trust them to accelerate their operations. To learn more, visit https://florencehc.com
[4:23] – Purpose of clinical trials
- Get safety and efficacy data about a medical product normally to bring product to market
- Indication expansion
- Most people join to seeking personal benefit from the study product
[6:49] Clinical trial access and trial participants
- Clinical trials are the only way to get access to drugs without market authorization
- Often seen by patients as a “Hail Mary” when in need of treatment
- Expanded access is a legal term term for access to medication that is not in a trial and pre-approval
- Majority of clinical trials still fail
[13:12] Explaining Expanded Access and the Right to Try
- Right to Try is a federal law that passed in 2018
- Similar to expanded access but it removes the FDA requirement
- Expanded access patients still need to do an informed consent that has been IRB approved
- Expanded access must be done with a willing doctor
- Expanded access is broader than Right to Try
- 1000s of patients each year get access to study products through Expanded Access
- Sponsors prefer expanded access because it is more collaborative with the FDA than going around them through Right to Try
[19:31] General misconceptions about clinical trials for subjects
- People assume study drugs are “safe” or a “miracle”
- Subjects don’t understand the differences between Phase 1, Phase 2, and Phase 3 studies
[22:52] General misconceptions sponsors have about Expanded Access?
- Sponsors can be both private companies or public entities
- Sponsor high turnover means there is always a need for training and retraining
- Make sure you’re speaking to all the patients that may want your product even those initially excluded from the study
- Patient advocacy is very important
[25:24] How can trials be designed to improve access to studies?
- Balance between who wants the product and what is the best population to study the product
- If people who want your drug are outside of the cohort that is best to study the drug on then its important to figure out how you’re going to get them access
- Sometimes the approach is a second trial for a different population, sometimes its better to use expanded access
[31:35] The role of payers in the expanded access and trial design space
- Accelerated approval lets drugs get to market faster with less data if there is an unmet need
- If after approval the confirmatory trial does not support the claims then the product is supposed to be stripped of approval by the FDA
- US government payers like Medicaid, Medicare or Veterans Affairs will back out of paying if confirmatory trail does not support
[35:03] Sponsor: Florence Healthcare (https://florencehc.com/) & Slope Clinical (https://www.slopeclinical.com/)
[37:14] Core pillars of an expanded access program
- Think of expanded access like flipping your inclusion and exclusion criteria for the study
- Limiting factor is often how much money sponsors will spend to support it
- Often expanded access is done outside of US which is unfair to those that want it in US but didn’t qualify for the trial
[40:52] Using data from expanded access
- One example of overdose reversal drug getting FDA approval through expanded access as trial would be unethical
- Expanded access data is becoming more acceptable than it used to be historically
- Sometimes the additional data is called pivotal data or supplementary data
- Expanded access data is similar to real world data but necessarily the same
[44:59] Site Selection for rare disease clinical trials
- Challenge is the population is typically very small
- Limited number of specialists for rare disease feeds into an even more limited number of research centers
- Sometimes different regional laws means trials are easier to do in different parts of the world
- Ethical consideration of should you be doing research in a region you don’t intend to bring the drug to
[51:42] Compassionate Use
- Compassionate use encompasses expanded access, Right to Try, and pre-approval access
- Patients need to go through their doctor for pre-approval access
- For expanded access IRB is required but for Right to Try IRB is not required but hospital may request one anyways
[57:43] Dr. Alison’ Bateman-House’s 2015 paper on Right to Try
- 4 key things: inequitable access, terminal illness, healthcare provider qualifications, informed concent
- Was vocally opposed to Right to Try because of fear of patient exploitation
- Example of potential avenue of exploitation was the removal of IRB review of informed consents
- Initial fear that Right to Try could be a means of profit for companies
- Right to Try is largely not being used so there is less to fear
[0:20] Typical Day for Dr. Alison Bateman-House
- Teaching, conferences, or leading a demonstration. Frequently meeting with trial sponsors
- No day is completely the same
- Frequently working with program managers
- Also traditional academic writing grants and looking for funding
- New York University (NYU) Compassionate Use & Preapproval Access (CUPA)
[1:08:29] Dr. Bateman-House’s recommended resources
[1:12:08] Sponsor Message:
This podcast is brought to you by Slope. Slope drives operational excellence for highly complex, sample-intensive, early-phase clinical trials. The platform transforms chaotic clinical trial supply chains into protocol-specific operational workflows for sponsors, CROs, clinical research sites and labs. Slope is trusted by industry leaders in complex early-phase clinical trials from top 50 pharma and CROs to emerging biotechs, and a global site network including NCI cancer centers and AMCs. Learn more at https://www.slopeclinical.com/
