Welcome to this month's P4A Let’s Talk Rare podcast episode by Partners4Acess. Today, Georgie and Owen are joined by Leonard Mazur, CEO of Citius Pharmaceuticals, to discuss their innovative therapy, Lymphir, for cutaneous T-cell lymphoma (CTCL). Leonard shares his journey with Citius, from its founding in 2013 to the recent resubmission of Lymphirto the FDA, and discusses the drug's potential to alleviate severe itching for the 21,000 annual CTCL patients in the U.S. The conversation also covers Citius's commercialisation plans and ongoing trials exploring Lymphir’s use for other conditions. Join Georgie, Leonard, and co-host Owen for an insightful discussion on how Citius is committed to improving patient quality of life through groundbreaking treatments!

Leonard explains how Citius acquired Lymphir in 2021, a drug facing regulatory challenges that they’ve addressed for FDA resubmission, with potential approval expected. They discuss Lymphir’s benefits, especially for CTCL patients, including its potential to relieve severe itching and outline the company's commercialisation plans with Eversana. Leonard emphasises the team’s extensive expertise as vital to navigating the regulatory process. They also explore Lymphir’s off-label potential for other cancers, with ongoing trials at major universities. The conversation closes on the rewarding impact of pharmaceutical work on patient lives, underscoring Citius's commitment to addressing unmet medical needs through innovative treatments.

Leonard Mazur Bio:

Leonard Mazur is a seasoned entrepreneur and executive with over five decades of experience in the pharmaceutical industry. He is known for his skill in founding and growing multiple healthcare companies. Currently CEO of Citius Pharmaceuticals, Leonard previously co-founded Leonard-Meron Biosciences and Akrimax Pharmaceuticals. He also led Triax Pharmaceuticals as COO, specialising in dermatology, and successfully sold his dermatological company, Genesis Pharmaceuticals, to Pierre Fabre in 2003. His extensive background spans roles in sales, marketing, and business development at Medicis, ICN, Knoll Pharma, and Cooper Labs. Born in Germany, Mazur holds an MBA from Temple University, where he also earned his undergraduate degree. He is an Ellis Island Medal of Honor recipient.

Episode Resources:

• Leonard Mazur on LinkedIn

• Citius Pharmaceuticals Website

• Owen Bryant on LinkedIn

• Georgina Rack on LinkedIn

• Partners4Access Website 

• P4A Let’s Talk Rare podcast on Apple Podcasts
  

In this episode of P4A Let's Talk Rare, hosts Georgie and Owen Bryant from Partners For Access are joined by Wes Michael, founder and president of Rare Patient Voice LLC. Wes shares the organization’s journey from its beginnings as a community project for hemophilia patients to a global platform that connects rare disease patients and caregivers with healthcare professionals. Rare Patient Voice has awarded over $30 million to participants and completed more than 210,000 projects, with Wes highlighting the company’s organic growth through patient referrals and advocacy partnerships.

Wes discusses the challenges of reaching patients nationwide, the rewarding impact of patient involvement in healthcare, and the essential role of patient-centricity in shaping drug development and health technology assessments. He emphasizes that patients' voices, as the end-users, are critical for creating effective healthcare solutions. Looking ahead, Wes shares his enthusiasm for expanding Rare Patient Voice into clinical trials and increasing its presence in Europe.

The episode concludes with a call to action for patients and caregivers to join the Rare Patient Voice community and share personal stories to further understanding and awareness of rare diseases.

Wes Michael Bio:

A market research industry veteran of over 40 years, Wes earned his MBA from the University of Chicago Booth School of Business and a BA in English from the University of Pennsylvania. In 2013, Wes launched Rare Patient Voice, based on a novel approach to building a market research panel that enables patients and caregivers to share their voices with researchers and companies developing products to improve patients’ lives. He structured the company on the core values of being a good patient resource, a good client partner, a good employer to staff, and a good citizen in our community. Since then, Wes has seen the company grow, expanding from the US to Canada, the United Kingdom, Spain, Italy, France, and Germany, completing thousands of projects, and providing customized service to hundreds of clients. He hopes that RPV’s work can help the lives of patients and caregivers who have participated in our studies and those who come after.

Episode Resources:

• Wes Michael on LinkedIn

• Rare Patient Voice LLC Website

• Owen Bryant on LinkedIn

• Georgina Rack on LinkedIn

• Partners4Access Website 

• P4A Let’s Talk Rare podcast on Apple Podcasts
  

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Aurelija Luko, the CFO, and Darren Callanan, the Global Digital Lead at Partners4Access. Join them as they discuss the recent job cuts in the pharma and biotech industry and the reasons behind them.

Aurelia explains that redundancies are not limited to the pharma industry and are influenced by factors like mergers, expiring patents, and macroeconomic instability. Darren adds that high drug development costs and pricing pressures also contribute to layoffs. However, he suggests that companies can use digital tools to enhance efficiency and innovation to address industry challenges. They also discuss a digital launch platform and a social listening tool created by Partners4Access. 

Aurelia highlights that companies are looking to increase shareholder value and reduce costs. Darren adds that thanks to the high cost of drug development and downward pressure on prices, companies are forced to narrow their focus and compete in the same space, leading to layoffs.

The conversation then shifts to discuss the digital launch platform created by Partners for Access. Darren explains that the platform streamlines the access and utilization of launch materials, ensuring that affiliates have access to the most up-to-date and compliant versions. This helps overcome the challenge of inconsistent distribution and saves time for the teams. 

Darren also mentions another tool called RarePulse, a social listening platform. This tool collects and analyzes data from social media and other sources to provide insights on key opinion leaders, unmet patient needs, and other relevant topics. He emphasizes that social media listening allows for a broader understanding of patient experiences and can help shape primary research and launch strategies.

Aurelija Luko Bio:

Aurelija’s work at P4A includes overseeing all accounting activities, from managing financial risk to setting budgets. Also, to ensure smooth day-to-day finance operations within the business.​ Aurelija is a Chartered Accountant with 10+ years of experience; she holds an MSc in Financial Economics from the University of Exeter and a BSc in the same field. ​

​Before P4A, she worked for small start-ups and big listed companies in healthcare, consulting, and property industries. She is a problem solver who can adapt well to new situations and review and improve the internal controls within the organization.

Darren Callanan Bio:

Darren Callanan is the Global Digital Lead at Partners4Access. He works in a specialist consultancy team to develop digital solutions addressing healthcare delivery barriers. Darren holds a master’s degree in Biotechnology and Business Management from the University of Warwick and an honors degree in Physiology from the National University of Ireland, Galway. His expertise includes strong communication and interpersonal skills, supported by a robust scientific background across various therapeutic areas. Previously, he worked at S3 Connected Health.

Episode Resources:

• Aurelija Luko on LinkedIn

• Darren Callanan on LinkedIn

• Owen Bryant on LinkedIn

• Georgina Rack on LinkedIn

• Partners4Access Website 

• P4A Let’s Talk Rare podcast on Apple Podcasts
  

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Pina Haberl, Senior Director, and Sam Morrison, Executive Director at Partners4Access. Join us as we discuss the crucial role of patient experience data in drug development. 

The conversation explores the need for drug developers to understand patient perspectives and gather data on their experiences, preferences, and needs. Pina and Sam highlight how patient experience data can improve drug development and clinical trial design while differentiating products in the market. They also address challenges in collecting and integrating patient data and the importance of involving patients early in the process. 

Also discussed is how some regulators and health technology assessment (HTA) bodies have started to accept patient experience data in their decision-making processes, but there is still uncertainty and inconsistency in how it is used. 

There is a call for more collaboration among stakeholders, including payers, policymakers, and patient groups, to establish best practices for collecting, analyzing, and utilizing patient experience data. Pina emphasizes the need for patients to have a seat at the table and be heard in decision-making processes. She mentions initiatives, such as partnering with patients and conferences, but acknowledges that there is still a long way to go.

A study published in Cambridge University Press investigated the utilization of patient preference (PP) data in health technology assessment (HTA) bodies worldwide. An exploratory survey by the HTA International Patient and Citizen Involvement Interest Group gathered insights from 40 members across twelve countries. Results showed that 82.5% of respondents formally considered PP data in HTA reviews, yet only 39.4% reported its frequent inclusion in assessments due to time and resource limitations. 

PP data moderately influenced decision-making processes and outcomes, with a higher impact on decision quality. Participants suggested patient advocacy groups should play a primary role in generating and submitting such evidence. The study highlights barriers to broader integration and proposes collaborative workshops for further investigation.

Pina (Josephina) Haberl Bio:

Pina is a goal-driven, solution-oriented, hands-on, and team-spirited professional with over 10 years of proven Market Access in consulting and industry expertise. She thrives on teamwork and excels at delivering actionable and innovative solutions to succeed in meeting business challenges. 

She specializes in developing and executing Market Access strategies and tactics that ensure success for pharmaceuticals, medical devices, and diagnostics in an international, cross-functional environment. Her understanding of the intricacies and specificities of respective healthcare systems and the diplomatic needs of diverse teams is critical in delivering results and ensuring patients have access to innovations.

Sam Morrison Bio:

Sam is an Executive Director at Partners4Access (P4A) with over 15 years of experience in market access consultancy. She has gained considerable insight into health systems across Europe and the USA, as well as other major markets, including Japan, China, and Brazil, as well as many Central and Eastern European markets. Sam has conducted many market access due diligence projects, and as pricing and reimbursement have become more essential to the licensing process, she is used to the time pressures and the balance between commercial, clinical development, and market access these projects require.

Episode Resources:

• Pina (Josephina) Haberl on LinkedIn
• Sam Morrison on LinkedIn
• Owen Bryant on LinkedIn
• Georgina Rack on LinkedIn
• Partners4Access Website 
• P4A Let’s Talk Rare podcast on Apple Podcasts

Welcome to this month's episode of Let’s Talk Rare: The Life Science Podcast brought to you by Partners4Access. Host Georgie records this episode at the World EPA Conference in Amsterdam. She holds a panel discussion with Juliette Sinclair-Spence, Sandrine Ruiz, 

Neil Grubert, and Seema Sondhi, and together we shared our experiences at the EPA conference. 

We also shared our excitement for AI and patient experience topics, as well as the importance of sustainability and finding new pricing approaches in healthcare. Gain insights on inclusivity and involving patients in drug development. Get ready for a meaningful, engaging conversation that will leave you inspired.

Take a deep dive into the benefits of tiered pricing, the challenges and concerns of companies and HTA agencies in joint HTA assessments, the importance of inclusivity, and the importance of involving and educating others.

Juliette Sinclair-Spence: Bio and Quote

LinkedIn

Juliette is the Funder and Chairman of the AK Eye Foundation and is a committed 𝐀𝐜𝐚𝐧𝐭𝐡𝐚𝐦𝐨𝐞𝐛𝐚 𝐊𝐞𝐫𝐚𝐭𝐢𝐭𝐢𝐬 (𝐀𝐊) 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐀𝐝𝐯𝐨𝐜𝐚𝐭𝐞. She has experienced this rare eye disease, and her key learning was that there is much that needs to be done on many fronts, including prevention, education, diagnosis, treatment, research, and support. Her passion for fighting against the disease led her to set up the first worldwide foundation focused on Acanthamoeba Keratitis.

 “I think involving patients in the drug development process is so important. We need to hear their voices and understand their journeys.”

Sandrine Ruiz: Bio and Quote

LinkedIn

Sandrine, or Sandy as she likes to be called, is the Senior Director of MarketAccess, Pricing, and HEOR at Immunocore. She is an experienced Pricing & Market Access professional with 25+ years in the biopharmaceutical industry. She has hands-on experience in giving strategic and science-based input across the pharmaceutical development workflow, from early stage to late stage, into Market/Patient Access, Pricing & Reimbursement, Payer Strategy, Real World Evidence, Clinical Development, Clinical Operations, and Business Development.

“I was really intrigued by the panel discussion on tiered pricing. It's such a hot topic right now, and it was interesting to hear the different perspectives on patient access. I'm also fascinated by digital information and the new trends in our field.”

Neil Grubert: Bio and Quote

LinkedIn

Neil is an Independent Global Market Access Consultant and a multilingual pharmaceutical market access specialist with 30 years of experience tracking the global prescription drug and self-medication markets. He has spearheaded the establishment and growth of Decision Resources’ international market access business. As the author of more than 150 reports covering 20 mature and emerging markets, multiple therapeutic areas, and numerous industry issues, he has earned a reputation for extensive knowledge of market access environments around the world.

“Yes, tiered pricing is definitely a challenging issue. Speaking of challenges, the EU HTA implementation has been causing many uncertainties for companies and HTA agencies. We need to navigate it carefully.”

Seema Sondhi: Bio and Quote

LinkedIn

Seema is the Head of Payer Value and Access for the Vaccine Business Unit at Takeda. She is passionate about patient access to innovative medicines and has worked diligently in her country, regional, and global roles to ensure access to pharmaceuticals, vaccines, medical devices, and orphan drugs. This has been achieved through key activities such as influencing global clinical trial development to ensure the collection of appropriate evidence, all the way through to launching products at prices that allow good levels of patient access and ensuring continued access post-launch via mechanisms such as innovative access schemes.

“Inclusivity is such an important aspect of our work. We can't just think about it; we have to be involved and educate others actively. Patients need to understand the questions asked and the impact of their input.”

Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines.

Together they explore the EU joint HTA legislation coming into force in January 2025, and the importance of patient experience data. Nick explains that the challenges for rare patients remain the same, with diagnosis being the first hurdle. He highlights the progress being made in genetic diagnoses and screening but emphasizes the need for more harmonization in Europe. 

Finally, they touch on the concept of patient experience data, which refers to the inclusion of patient perspectives and outcomes in decision-making processes. Nick emphasizes the importance of this data in understanding rare conditions and leveraging it for better access to treatments. They also discuss the various events and initiatives planned by Genetic Alliance for  the upcoming Rare Disease Day on February 29th, to raise awareness and share stories of rare conditions.

Nick Meade Bio:

Nick Meade is Head of Policy at Genetic Alliance, the largest alliance of organizations supporting people with genetic, rare and undiagnosed conditions in the UK. He has over twenty years of experience in the field and is a member of the NIHR Advanced Therapy Medicinal Product (ATMP) Group. 

Episode Resources:

Nick Meade on LinkedIn

Genetic Alliance website

Partners4Access website  

P4A Let’s Talk Rare podcast on Apple

Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bottom of our hearts, we would not have gotten here without you. 

A special episode calls for special guests, and joining us today are our in-house expert and Managing Partner at P4A, Sophie Schmitz, our host from 2018-2021, Aparna Krishnan, and Prasan Subedi, Access Strategy Team Lead at Pfizer. Together, we walk down memory lane and relive the high points and challenges of the past five years, and share insights on rare diseases and cell and gene therapies.

Aparna Krishnan Bio:

Aparna has over 10 years of experience in the pharmaceutical and healthcare industry. As part of the senior leadership team, her primary focus is on strategic corporate engagement, legal, financial operations, and human resources. She has previously worked as Manager of Life Sciences at IHS Markit and as Consultant at Parexel’s Commercialization unit, among others specializing in market access, pricing, and reimbursement.

Sophie Schmitz Bio:

Sophie is the managing partner at Partners 4 Access, a company that believes in a sustainable rare disease world, which means helping our clients unlock the true opportunity of orphan drugs and ATMPs. Her 20+ year career spans various disease areas and medical devices, successfully supporting the strategy development and operationalization for orphan drugs. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising clients on price and market access strategies, with the ultimate goal of optimizing fair access for all stakeholders in rare diseases.

In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a friendly and engaging tone, this podcast offers valuable insights into healthcare and the potential of digital solutions.

RJ Kedziora Bio:

Mr. Kedziora is the co-founder of Estenda Solutions, a leading company specializing in custom software and data analysis for healthcare and medical companies. With a remarkable journey spanning over 30 years, he possesses a deep understanding of designing, developing, and deploying successful software projects. His extensive experience enables him to provide valuable guidance and innovative insights, resulting in cost-effective solutions that improve patient outcomes.

Mr. Kedziora received his M.B.A. from West Chester University and a Bachelor of Science in Computer Science from Duquesne University where he received the Excellence in Computer Science Award. He has spoken at numerous technical and healthcare conferences on a variety of topics and written or co-authored multiple articles focused on healthcare information technology, several published in peer-reviewed scientific journals.

Social Media Links:

FACEBOOK (BUSINESS): https://www.facebook.com/EstendaSolutions

LINKEDIN: https://www.linkedin.com/in/rjkedziora/

INSTAGRAM: https://www.instagram.com/estendasolutions/

TWITTER: https://twitter.com/Estenda

YOUTUBE: https://www.youtube.com/channel/UCuAfCbIFW0DmeI5PDS1PXcg

Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Georgie and Owen are joined by Iola Forster, Head of Publications and Portfolio at Karger Publishers, to discuss the future of patient engagement and the role of technology in healthcare. 

Together, they cover the future of HCPs (healthcare practitioners) and patient engagement, highlighting the importance of technology and patient empowerment in driving this evolution. They also emphasize the need for equitable access to healthcare information.

The conversation touches on the changing landscape of patient communication over the past fifteen years, with the advent of digital platforms and social media. They discuss how digitization has democratized access to health information and created opportunities for patients to connect with others facing similar conditions.

They further explore the impact of digitization on the orphan drug and cell and gene therapy world, emphasizing the role of patient involvement in content creation and the use of multimedia resources like infographics to cater to diverse learning styles.

Iola Forster Bio:

Iola Forster is the Head of Publications and Portfolio at Karger Publishers. She has over fifteen years of experience in the publishing industry and is skilled at fostering partnerships between the life sciences, healthcare clients, and Karger. Iola brings a wealth of knowledge and expertise to the table.

Episode Resources:

Iola Forster on LinkedIn

Karger Publishers website

Partners4Access website  

P4A Let’s Talk Rare podcast on Apple

Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of proactive preparation for the upcoming changes. 

The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of ATMPs and oncology medicines to not adopt a wait and watch strategy; the risks of ignoring this change and the potential impact on clinical development plans, commercialization strategies, and patient access; the opportunities the EU market presents; and the need to start planning and adapting internal processes to successfully navigate the new regulatory landscape.

Chloe Sheppard Bio:

Chloe serves as a Senior Consultant at Partners4Access, working closely with clients to craft custom solutions for their pricing and market access obstacles. Her motivation lies in transforming the immense potential of advanced medicines into tangible therapeutic benefits for patients.

Akshay Kumar Bio:

Akshay, a Partner at Partners4Access, brings over 13 years of experience assisting pharmaceutical companies in devising market access and pricing strategies that align with their broader commercial and organizational contexts. His expertise spans not only product-level solutions but also process improvements and capability enhancement. In addition to market access, Akshay is well-versed in various other aspects of drug commercialization, including opportunity assessments, product positioning, forecasting, customer insights, and organizational design.

Episode Resources:

Chloe’s LinkedIn

Akshay’s LinkedIn

Partners 4 Access Website

In this episode we will be talking to Alejandro (Alex) Dorenbaum, M.D., CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing drugs for patients with #rare mitochondrial diseases, a high unmet disease with no current treatments available. Their lead candidate #Mavodelpar has recently completed enrolment for their pivotal STRIDE clinical trial. STRIDE is a global, randomized double-blind, 6 months, placebo-controlled trial designed to assess the efficacy and safety of Mavodelpar.

Alex also discusses the importance of engaging early with #patients and patient organisations for #PMM across the world to truly understand the patient journey, their challenges & daily routines which help to shape the study design. This ensures successful enrolment and patient retention throughout the clinical trial, something Reneo have successfully completed throughout the development of Mavodelpar. By engaging early with patients, clinicians & regulators, they have completed enrolment in record time, this is especially difficult for #rarediseases as it is often difficult to find the patients and something Alex is extremely proud of.

Reneo Pharma: https://reneopharma.com/

Reneo is a clinical-stage pharmaceutical company focused on the development of therapies for patients with rare genetic diseases including mitochondrial diseases with significant unmet medical needs.

LinkedIn: Alejandro Dorenbaum

https://www.linkedin.com/in/alejandro-alex-dorenbaum-m-d-b01a6ab/

JustGiving: https://www.justgiving.com/fundraising/partners4access-b-v?utm_source=copyLink&utm_med[…]ampaign=pfp-share&utm_term=0c6d8180e0f3440b8383845adedb6513