Rare Diseases Weekly Roundup

In this episode of Let’s Talk Rare, as we commemorate World Rare Disease Day, Georgie and Owen are joined by Louise Fish and Dan Lewi to discuss all aspects surrounding rare diseases, from key challenges patients face in getting access to life-saving medicines, the clinical trial burden, EU pharmaceutical strategy and more. The topics covered include: Why genetic screening of newborns in the UK lags behind the EU, funding rare disease research, clinical trial burden, patient registries, patient experience data and its relevance, revamping data sharing with families, and how pharma can involve patients earlier. Louise Fish Bio: Louise is a senior leader with over 25 years of executive and non-executive board experience in health and social care in charitable, public and commercial sectors. She is passionate about improving the NHS and social care services by listening to and learning from the experiences of patients and their families. She has a strong understanding of how to drive change. Dan Lewi Bio: Dan is

The P4A team have hand-picked trends to look out for in 2023. This episode we are joined by special guest Neil Grubert who will be discussing the topics in further detail, alongside our P4A experts, Shrishti & Jodie. The topics covered are: EU Pharmaceutical Strategy, OMP Legislation, Joint HTA Assessments - cross-country HTA cooperation, Expansion of CAR-T & Hospital Exemptions across Europe and finally the Patient - Putting patients at the forefront of drug developers. Neil Grubert Bio:  Neil has worked in Industry for over 20 years starting at Decision Resources as a researcher in 1997 and leaving as Vice President of Global Market Access Insights in 2013. Currently he is an independent MA consultant and has been doing this for the last 8 years. LinkedIn: https://www.linkedin.com/in/neil-grubert/ Hosts: Georgie Rack & Owen Bryant P4A Speakers: Srishti Gupta, Senior Consultant & Jodie Lyons, Analyst Produced by: Operations

Did you miss the World Orphan Drug Congress Europe 2022, held in Sitges, Barcelona in November? If you did, don't worry P4A have you covered! This month's special  podcast was part recorded live at the conference and part recorded back in London with members of the team that attended! We will be speaking to a few presenters & sponsors to discuss their presentations, key learnings and their personal highlights from the conference. Also the P4A team will be talking about our favourite sessions and the key takeaways from the conference. Special thanks to our guests for their insights and learnings throughout the podcast. Presenters: Georgie Rack & Owen Bryant Guests: Alexander Natz, Secretary General at EUCOPE - https://www.eucope.org/ Leon van Wouwe, Clinical Innovation Director at VOLV Global https://www.volv.global/ Wing-yun Cheung, General Manager at Terrapinn & organiser of WODC EU - https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm Chloe Sheppard, Consultant at P4

 Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, with innovative orphan drugs and cell and gene therapies, launch strategies be? With the latest decision from Janssen to avoid the German market altogether for x2 Rare Oncology innovative drugs, will this be a trend we are likely to see continue?  Join Stefan Walzer & Fisentzos Stylianou discuss the new bill, in regards to the biggest changes that impact orphan drug (OD) manufacturers. Will OD manufacturers still see Germany as the first go to market within Europe and what does this means for rare disease patients?  Will there be delays to new treatments or will manufacturers decide not to launch in Germany at all to protect the price of their new drug?  We will be discussing this and so much more! If you are a drug manufacturer planning your launch strategy, this podcast is for you!  Presenters: Georgie Rack & Owen Bryant Guests Stefan

P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie Schmitz are back with us to carry on the discussion in part 2 on true patient empowerment and really looking at the WHY’s! Why should drug manufacturers involve & listen to and involve the patients. What are the implications, if any, for drug manufacturers who do not include the patient voice. How can we bring all stakeholders together to work completely new approaches to medicine approval and patient access. Lastly, we will look at initiatives or services our panel are offering to try to bridge the gaps and barriers for sustainable patient access. P4A’s’’Let’s talk rare’’ monthly podcasts are available wherever you listen to your podcasts. If you are a drug manufacturer and you haven’t yet listened, this needs to be at the top of your to do list! If you missed out on part 1 - listen here: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb Hosts: Georgie Rack (G-

This month we have a special 2-part series focused on Patient Empowerment, with special guests Laurence Woollard, On the Pulse, Neil Bertelsen, Independent Consultant & Sophie Schmitz, Managing Partner at P4A. This podcast is a MUST to listen too if you are a company looking to successfully develop and commercialize an orphan drug or ATMP The first part will focus on how do we ensure there is genuine patient involvement at all stages of drug development? What are the challenges and barriers for patients living with a rare disease vs drug developers within the rare/ultra-rare sphere? The second part will solely focus on the WHY! Why should drug manufacturers, payors, regulators and HTA bodies involve and listen to the patients? What do they bring to the table? We will also dive into the implications of not including the patient voice and use examples of companies that have successfully managed to do this and the benefits of doing so. Laurence Woollard, Owner On The Pulse Laurence Woollard is founder and di

This month we have put together a slightly different episode with a twist. Georgie & Owen will take you behind the scenes at P4A and discuss 'What makes P4A an award-winning global market access consultancy?' Why we do what we do every day, and what it means to each and every member of staff. Pride, Passion & Partnership are the core values at P4A, and we take a closer look into each one. Later in the show we have a very special announcement from our Managing Partner, Sophie Schmitz. Host: Georgie Rack, Communication Executive & Owen Bryant, Creative Director Produced By: Operations Awards P4A have won in 2022 (so far!) - https://partners4access.com/about/awards/ 1. BOBI Awards 2022 - Analyst Team of the Year Link: https://www.bhbia.org.uk/bobi-awards/award-winners/bobi-2022 2. Benelux Enterprise Awards 2022- Best Orphan Drug & Gene Therapy Experts Link: https://www.eubusinessnews.com/awards/benelux-business-awards/#ourwinners 3. International Life Science Awards 2022 - Best Cell & Gene Th

In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and how this came about. We then discuss JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies and how the joint HTA process will link to national processes. EUnetHTA21 and how the methodology is evolving with G-BA & IQWIG increasingly involved, will this be a major influence in the development of final methodology? Finally, we look at how Pharma companies should be best preparing for the implementation of the regulation. Host: Chloe Sheppard Guest Speaker: Alexander Natz Produced by: Operations team Helpful links: #EUHTAPriorities – EUCOPE’s Five Priorities for a Successful EU HTA Procedure Sounds of Science Podcast – Episode 1: A Future-Proof HTA system in Europe? https://partners4access.com/whitepapers/pan-european-joint-hta-what-does-the-future-hold-for-innovative-ther

Part 1 featuring Victor Maertens, Government Affairs Manager at EUCOPE In this episode Victor and Aparna will be focusing on Advanced Therapy Medicinal Products (ATMPs). We will start by looking at the main challenges for manufacturers in the ATMP approval process and what is required from an HTA perspective. We then discuss EUCOPE’s position paper on the different payment models, including the motivation for the report and key learnings. We look at sustainable patient access and whether the report addresses the affordability issues surrounding ATMP’s drug pricing and if this is even possible in the current landscape. The EU’s revision of Blood, Tissue & Cells legislation and lastly the future of ATMPs for manufacturers and patients. Host: Aparna Krishnan Guest speaker: Victor Maertens Produced by: Operations team About Victor Maertens: https://www.linkedin.com/in/victormaertens/ Victor Maertens is the Government Affairs Manager for the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Vict

In 2020 China’s pharmaceutical market passed an important milestone, a Deloitte report cited that China was expected to grow its pharmaceutical market volume by 2020 to $220 billion (USD) becoming the second largest market behind the US. The commercial potential is huge, so what do we know about the orphan drug (OD) potential in China? Fisentzos Stylianou discusses China’s healthcare system, key challenges for OD manufacturers, Spinraza’s commercialisation journey in China, and incentives available to OD manufacturers in China. Reference: https://www2.deloitte.com/cn/en/pages/life-sciences-and-healthcare/solutions/life-sciences.html Presenter: Aparna Krishnan, Partner – Global Operations Contributor: Fisentzos Stylianou, Analyst Producer: Operations team

Over the last few years, the Middle East has piqued the interest of orphan drug manufacturers, and some have prioritised launching in this region before European markets. Akshay Kumar and Lavni Varyani discuss what is driving this behaviour, the orphan drug infrastructure within the region, and opportunities, barriers, and key considerations for manufacturers aiming to launch in the Middle East. Presenter: Akshay Kumar, Partner, P4A Contributor: Lavni Varyani, Founder, Pharma Business Partners Producer: Operations team

For this Rare Disease Day episode, Janet and Nick discuss a range of topics associated with the rare disease community and patient advocacy. They address new-born screening, patient registries, the UK rare disease framework, the NICE methods review and much more! Presenter: Georgie Rack, Communication Executive Contributors: Janet Bloor, CEO of Duchenne Nexus Advocacy (DNA) and Nick Meade, Director of Policy and Joint Interim Chief Executive of Genetic Alliance UK Producer: Operations team

Lots of anticipated changes are expected in the orphan drug, cell and gene therapy space and 2022 is going to be an exciting year. Listen to our latest podcast on the top trends in drug development, HTA and pricing from Akshay Kumar and Andrea Bernardini to help you navigate the year ahead. Presenter: Georgie Rack Speakers: Akshay Kumar & Andrea Bernardini Produced by: Ops team

Sophie Schmitz and Joanna Fernandes discuss bluebird bio’s innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and Joanna will be looking at the fall of Zynteglo from two sides: from a company perspective and from the perspective of the EU environment. Presenter: Georgie Rack, Communication Executive Contributors: Sophie Schmitz, Managing Partner and Joanna Fernandes, Senior Consultant Producer: Operations team

This episode featuring James Mackay, President & CEO of Aristea Therapeutics. James provides insights on his experience with setting up a biotech and challenges associated, industry climate on spin off opportunities, Aristea’s pipeline, collaborations and US PRMA reforms. Presenter: Aparna Krishnan Contributors: James Mackay, President & CEO of Aristea Therapeutics Producer: Operations team About Aristea Therapeutics, a San Diego-based clinical-stage immunology-focused drug development company developing novel therapies for serious, rare inflammatory diseases.

In this episode P4A’s Richard Wang and Adama Anozie discuss Hospital Exemption (HE) which is an emerging access pathway for Advanced Therapy Medicinal Products (ATMPs). Main topics of discussion include the benefits and potential downsides associated with this pathway, the EU regulations involving HE and recent events in Italy, Spain and France, as well as the implications of HE on the traditional pharma model, the need for collaboration between big pharma and hospitals/institutions to develop HE therapies and much more! Presenter: Richard Wang Contributor: Adama Anozie Producer: Operations Team

In this episode P4A’s Senior Client Relationship Director, Bruce Chin discusses his experiences from the in person World Orphan Drug Congress USA 2021. Topics of discussion included travel and safety precautions due to COVID, Bruce’s favourite sessions, key learnings from the sessions, and much more! Presenter: Georgie Rack Contributor: Bruce Chin Producer: Operations Team

In this episode Akshay Kumar and Richard Wang discuss how through innovative technology, decentralised clinical trials came to the limelight, facilitating remote access and ensuring continued operation of clinical trials. In addition, we discuss the future of decentralised clinical trials in a post-COVID world, its implications and demands within the industry. Presenter: Akshay Kumar Contributors: Richard Wang Producer: Operations Team

In this episode, the P4A team take a deep dive into the Brazilian pharmaceutical market and discuss its potential for access to orphan drugs, cell and gene therapies. The overall Latin American pharma market is forecast to grow by 9% per year through to year 2028. However, major markets in this region such as Brazil have some major barriers to access. Listen in to learn more.  Presenter and Contributor: Ciaran Cassidy Producer: Aparna Krishnan

Its almost six months since President Joe Biden came to office but the role of the head of the country's key drug regulator, the US Food and Drug Administration (USFDA) is yet to be filled. In this episode, the P4A discusses the absence of a USFDA commissioner and its impact. Also, we understand the significance of recent remarks of the Center for Biologics Evaluation and Research (CBER) director Peter Marks on the need for consistency in the manufacture of cell and gene therapies.  Presenter and Contributor: Max Rex Producer: Aparna Krishnan