60 episodes

A monthly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit www.partners4access.com/podcasts/

Rare Disease, Cell & Gene Therapy Monthly RoundU‪p‬ Partners4Access

    • Science
    • 4.7 • 6 Ratings

A monthly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit www.partners4access.com/podcasts/

    Monthly RoundUp - March 2021

    Monthly RoundUp - March 2021

    The world of orphan drugs is at the cusp of a transformation in the post COVID era. In this episode, the team discuss the impact of the pandemic on value demonstration of orphan drugs. In order to be successful, manufacturers of precision medicine will need to focus on carving out a niche for their drugs and support health systems to find the right patient for their treatment at the right time. 

    The team also explore the role of digital tools such as Artificial Intelligence (AI) and telemedicine in helping drugmakers optimise manufacturing and treatment delivery. This combined with a multi-stakeholder approach will provide the recipe for success for next generation`niche-busters'.

    Presenter and Contributor : Akshay Kumar

    Other contributors: Richard Wang, Andrea Bernardini and Erfan Akbraian

    Producer: Aparna Krishnan

    • 13 min
    Monthly RoundUp - February 2021

    Monthly RoundUp - February 2021

    In this episode, the P4A team discussed the impact of cost-containment measures in Germany, the European Commission's recent move to revise legislation on orphan medicinal products and potential legislative reforms in the US under the new Biden administration. 

    Presenter: Jens Leutloff

    Contributors: Chloe Sheppard, Max Rex

    Producer: Aparna Krishnan

    • 22 min
    Rare Disease Day special episode: Reclaiming the rare disease patient’s voice

    Rare Disease Day special episode: Reclaiming the rare disease patient’s voice

    In this Rare Disease Day special episode, we focus on the impact of COVID -19 on rare disease patients. Some of the key challenges for patients during this pandemic include disruption in their access to treatments, struggle to continue participation in clinical trials, lack of access to vital equipment such as PPE (Personal Protective Equipment) and also to healthcare personnel. We speak to Genetic Alliance chief executive officer Jayne Spink, patient Nicola Whitehill and Danielle Myers, mother and carer of patient 10 year old Dylan Myers.

    Presenter: Aparna Krishnan

    Contributors: Jayne Spink, Nicola Whitehill and Danielle Myers

    Producer: Aparna Krishnan

    More information on :

    Genetic Alliance can be found at https://geneticalliance.org.uk/

    Nicola Whitehill's blog: https://blog.raynaudsscleroderma.co.uk/2017/04/scleroderma-raynauds-rare-disease.html?m=1 

    Dylan Myers' story: https://m.facebook.com/dylansstory/  and https://www.treeofhope.org.uk/dylansstory/

    • 36 min
    End of year 2020 review

    End of year 2020 review

    In this episode,  the P4A team review all the key events of the year 2020 in the orphan drug, cell and gene therapy world. The emergence of COVID-19 pandemic has caused disruption but also opportunities for the biotech industry. From  change in regulations, new stakeholder collaborations to impact of Brexit and US drug pricing reforms,  a full round up of 2020. So do listen in!

    Presenter and Contributors: Sophie Schmitz  and Akshay Kumar

    Producer: Aparna Krishnan

    • 18 min
    Special episode: Gene Therapy access from a specialty pharmacy perspective

    Special episode: Gene Therapy access from a specialty pharmacy perspective

    This episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective.  The senior team at Orsini  provide insight on their experience of Zolgensma access including key manufacturer criteria, payer landscape along with challenges and lessons learnt. 

    Presenter: Aparna Krishnan

    Contributors: Dave Frobel, Senior Vice President, Trade & Tom Shaughnessy, Senior Vice President, Health Plans    

    Producer: Aparna Krishnan

    About Orsini Healthcare: Founded in 2009, Orsini is a leading independent specialty pharmacy focused on rare conditions and gene therapies. Orsini was one of the first specialty pharmacies to provide Zolgensma, the first gene therapy for Spinal Muscular Atrophy. Today, Orsini has access to 40 limited distribution drugs, has more than 525 direct payor contracts and is recognized as a reimbursement expert in managing both medical and pharmacy benefits.

    • 20 min
    Weekly RoundUp: February 29, 2020

    Weekly RoundUp: February 29, 2020

    This week is a special episode celebrating Rare Disease Day 2020.  Our guest speaker is Annie Kennedy, head of policy and advocacy at Every Life Foundation, a US based non profit organisation that works to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. Here she speaks about the organisation's initiatives on drug access. 

    To know more about the foundation, visit https://everylifefoundation.org/ 

    Presenter and Producer: Aparna Krishnan

    Contributor: Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases

    • 17 min

Customer Reviews

4.7 out of 5
6 Ratings

6 Ratings

ChristinaPos1234 ,

Great!

I was searching for a pharma analysis podcast for a while and finally found one. Well done Partners4Access!

Scarab09 ,

Excellent podcast!

Essential listening for orphan drug industry.

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