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Addressing the myriad ethical dilemmas with vaccine trials, policies, and access

May 28, 2021/in Hot Topics in Medical Affairs /by Amanda Schmitt

A sophisticated global audience, including IFAPP Academy students, alumni, Global Fellows in Medicines Development, and members of the IFAPP National Member Associations, recently challenged the faculty during a fascinating discourse (Conversations with the Academy, March 30, 2021) on several ethical, policy, and access facets that have emerged as various COVID-19 vaccines are being made available in different nations.

Sandor Kerpel-Fronius, Professor, Semmelweis Medical University, Budapest, one of our eminent panel members, opines….

US, UK, EU, AUS, Japan blocked an IP waiver for “generic” COVID vaccines to be mass-produced… this is an enormous ethical dilemma; how did Medical Affairs in Pharma play into this landmark decision?The technique producing mRNA vaccines might be very difficult and probably not easily manageable. Personally, I think the best approach would be to build production sites by the companies in less-developed regions.
What about covid mRNA vaccines in pregnant women?Yes, they can be used; antibodies were even detected in newborn babies.
What do you think about the recent prolongation of interval between first and second doses? Do you expect, with time, this time period can become even longer? Or, do you think this can lead to the situation that only one dose of vaccine will be sufficient for application?The prolongation is feasible since already after the first dose, marked antibody titers can be measured. Although this will provide some decrease in the protection against infection but will decrease the severity of the infection. However, the booster injection will be needed after the extended interval. Ethically, the advantage of more extensive vaccination of the population over slightly diminished individual protection can be justified.
Given the lethal nature of COVID-19 and the importance of vaccines, what do you think about a degree of flexibility on the part of the regulators for approval of the vaccines, in the first instance, and then follow-up with real-world data?The follow-up of accelerated approval of orphan drugs is increasingly based on RWD. This approach might be possibly used also for vaccines to decrease the time until EUA is approved. However, the successful collection of RWD must be very carefully planned together with the communities receiving these vaccines.
What type of social contract would the panel propose for worldwide distribution of the COVID-19 vaccines?An approach similar to COVAX but it should deal together with WHO to introduce an internationally acceptable unified approach for EUA procedure before donating or marketing vaccines internationally.
Is it ethical that some parts of the world have not as yet received any vaccine due to their geographical location and economic situation and yet 60% of the adults have already been vaccinated in the UK? However, the enormous political pressure of the local citizens for first satisfying local needs will overrule internationally more correct ethical decisions. It might be argued in favor of such approach that rich nations can support international vaccination programs if their economic basis remain functional to support altruistic programs.
Access, I believe, is the most hot-potato ethical issue. Do you offer any solution for equitable access?It would be necessary to improve local vaccine production also in underdeveloped regions making these regions capable for rapidly producing locally vaccines. The technical assistance of advanced producers will be needed.
What is your view regarding informed consent for Human Challenge Trials of vaccines?The fact that the natural history of the disease is not well understood, together with frequent fatal outcomes and lack of treatment, makes challenge studies ethically highly questionable. One should consider that Ethics Committees have to defend physically the subject and psychologically both the subject as well as the performer of such studies. In addition, also the society must be protected from the disastrous consequences of possible tragedies.
I can see the practicality of asking for vaccine passports, but is it ethical? And is it in line with social liberty?Very difficult question. The introduction of such passports would be entirely ethical only after all people requesting vaccination would receive the treatment all over the world.
What happens to those who are generally against vaccines because of religion and personal reasons?Personally, I think that it is not ethical if religion(s) interferes with medical treatments. Those people who refuse vaccination should ethically and administratively accept the possible negative consequences of their decision from the side of the community.

Sandor Kerpel-Fronius, MD, DSc, GFMD

How can learnings from the pandemic facilitate future access to innovative medicines?

May 28, 2021/in Hot Topics in Medical Affairs /by Amanda Schmitt

Conversations with the Academy series webinar, April 30, 2021

The COVID-19 pandemic has taken a grievous and burdensome toll on life, healthcare systems, and the global economy.

On the other hand, the favorable outcome with the accelerated development of vaccines, and the pragmatic utilization of the Emergency Use Authorization (EUA) instrument, has triggered an increasingly empowered patient and advocacy voice for pre-approval access to investigational treatments and biopharmaceutical innovation. This has also drawn attention to revisiting traditional tenets with future approvals of new drugs for very sick patients and the likely longer-term implications for medicines development paradigms and clinical trial design considerations.

As the healthcare and regulatory landscape evolves to tackle the emerging challenges and leverage learnings, fundamental issues are being highlighted around medical, regulatory, and ethical decision-making, increasingly sophisticated systems and data-sharing, and global collaboration and transparency.

What is the purview of the regulatory authorities today? How could the regulatory sciences systems and processes be refurbished to meet future needs? What are the demands on the biopharmaceuticals industry and regulators to remain relevant, offering substantial solutions for the clinical development and technical review of innovative medicines?

Expounding on the imperative for regulatory oversight models to evolve in response to changing healthcare and medicines development needs, David Jefferys, Senior Vice President, Eisai, Lawrence Liberti, Adjunct Assistant Professor, Temple University School of Pharmacy, and Leonard Sacks, Associate Director, US Food and Drug Administration, deliberated on key practices that have arisen, adapted, or accelerated by the pandemic. Which ought to continue in future endeavors, and what policy and scientific initiatives need to be instituted for a meaningful and sustainable change in direction?

The panel emphasized that the COVID-19 pandemic has displayed how researchers, manufacturers, and regulators have been able to expeditiously apply the components of the development toolbox in novel ways to deliver safe and effective vaccines, therapeutics, and devices, in record time.

While several stakeholders may view these events as portending a seismic transition with traditional medicines development and access, the emphasis is now on how and which elements of these experiences might be more broadly extrapolated to the overall cycle of evaluation, manufacturing, regulating, and monitoring innovative medicines.

The challenges notwithstanding, the pandemic has also created alternate avenues for developing and evaluating new medicines, as in the arenas of clinical trials design and conduct, application of digital technologies, remote monitoring and oversight, patient engagement, collaborative working, and knowledge and data sharing. The panel explored learnings from the past year and proposed opportunities to apply these to refining the development and access pathways for next-generation pharmaceuticals.

The discussants elaborated on identifying processes that could contribute to improving discovery, manufacturing, regulatory reviews, and post-authorization assessments. They expanded on the earlier deployment of Real-World Evidence (RWE) and its integration throughout the evaluation lifespan, considering aspects of new trial designs and approaches, including pragmatic trial concepts and the use of RWE and post-approval data.

Appreciating the role of heightened awareness and of collaboration – enhanced partnering between several sponsors and streamlined coordination among agencies – it is evident that promoting competency-based education would be essential to leverage the lessons learned and to bring better medicines to our societies.

The global community has witnessed unprecedented stakeholder engagement and dialogue to identify and enable potential flexibilities and efficiencies within acceptable social and legal frameworks. These have encompassed diverse domains such as supply chain processes, conduct, and reporting of clinical trials, manufacturing, rolling authorization reviews, and integrating post-authorization real-world leanings.

It will be critical to translate these learnings to future clinical assessment and regulatory sciences pathways with non-COVID trials and settings. Importantly, resource prioritization and optimizing development and regulatory models could continue to foster innovation and drive healthcare systems to address unmet medical and social needs.

Even as humanity anticipates the next health challenge….

A recording of this engaging session is available on IFAPP Academy’s YouTube page.

As always, we express our sincere appreciation for the keen insights from our expert faculty and hosts. And, we are motivated by your active participation at these events.

Pravin Chopra, MD, GFMD

Conversations with the Academy Series continues to garner promising participation by the global learning community

January 29, 2021/in Hot Topics in Medical Affairs /by Amanda Schmitt

Conversations with the Academy Series continues to garner promising participation by the global learning community

The December event in the series featured an eminent panel exploring evolving Health Economics Outcomes Research (HEOR) concepts, driven by the changing healthcare landscape and increasing expectations to demonstrate value. Dr. Laurent Pacheco, Managing Director, Syneos Health Consulting; Dr. Seema Haider, International HEOR Advisor; and Dr. Jennifer Stephens, Partner, and Clinical Director, Pharmerit, addressed the challenges and trends in the healthcare market across the globe, with the HEOR audience expanding beyond payers and Health Technology Assessment (HTA) bodies, to a variety of stakeholders, including patients and regulators.

Physicians need HEOR data to inform their treatment decisions to meet the healthcare needs of patients best. Importantly, HEOR brings a real-world perspective. As countries adopt universal healthcare systems and develop HTA frameworks, they need transparent, understandable, and effectively communicated HEOR data to help inform healthcare decision making.

The panel highlighted perspectives to better deliver and communicate HEOR information and real-world data. They emphasize cross-functional collaboration in biopharmaceuticals, patient centricity, and leveraging digital transformation. If you missed this, a recorded session is offered at https://youtu.be/kYmr7IrDtk8

The most recent discussion, Real World Evidence and Real-World Data: from Disease Management to Evaluating Drug Safety and Effectiveness, on January 28^th, seeks to analyze the rapidly increasing interest and relevance of real-world evidence to answer clinical, regulatory, and policy-pertinent questions, which cannot be wholly or directly addressed by traditional randomized controlled trials. The recording will be made available soon on IFAPP Academy’s YouTube page.

As always, we express our sincere appreciation for the keen insights from our expert faculty and hosts. Furthermore, we are motivated by your active participation at these events.

Pravin Chopra, MD, GFMD