Day 2: ACCESS Programmes Congress

Conference Agenda


Expanded Access Programmes - Day 2

Day 2: Wednesday 21st September 2022


  • Deep-dive into the reality of healthcare access around the world
  • Streamlining the process and navigating early access regulations globally
  • What does the future of health access look like? The interconnected ecosystem where all healthcare stakeholders work together
  • The ethical imperative for gene therapy companies to proactively develop expanded accesspolicies
  • Obstacles developers face in providing expanded access to gene therapy products
  • Conditions with limited windows for treatment deserve special consideration in expandedaccess

Lisa Kearns, Division of Medical Ethics, NYU Grossman School of Medicine

  • The main challenges in the execution of an EAP for cell and gene therapies are:
  • Treatment is generally available in a restricted number of specialist centres, so patients needto travel for treatment, and
  • The treatment is generally high-value, particularly in the case of rare indications.
  • The presentation will highlight lessons learnt and innovative solutions for EAPs and cross-border programs for cell and gene therapies         
  • Companies should anticipate the need for EA and should have a written policy
  • The policy should be available to the person entitled to expanded access
  • Companies must be accountable to the patients sending expanded access requests
  • Sharing of data analysis is crucial for improvement


  • Overview of Karyopharm Expanded Access Program (KEAP)
  • Importance of knowing your patient (so that the appropriate dosing and supportive care canbe recommended)
  • Following up on patient’s status post treatment initiation
  • Discuss the steps followed in KEAP and highlighting the benefits seen.

Trinayan Kashyap, Director, Global Expanded Access Lead, Karyopharm Therapeutics Inc

2:00 - 2:30 pm, 2:35 - 3:05 pm - ROUND TABLE DISCUSSIONS
Divided between 2 sessions of 30 mins. (5 mins. to change table). Attendees have 2 round table options to attend

  • Alternative access schemes offer flexibility to policymakers but often lack transparency and clear rules.
  • The uncertainties allow for dynamic responses to weaknesses in the main drug approval and reimbursement systems
  • Challenges facing the reimbursement system
  • Understanding different payer strategies currently in place

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence BlueCross

  • The importance of patient advocacy in providing much-needed information on EAP’s available for patients
  • Collaborating with one another to enable a much richer understanding of patient needs
  • Patient groups’ importance in working with health authorities to advance policies that accelerate patient access to investigational medicines
  • Creating a state of the art payer engagement strategies to address challenges in pricing and reimbursement