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HTA Workshop Series (Virtual)

1 January 2027 @ 08:00 - 31 July 2027 @ 17:00 GMT

Aligning Evidence, Value and Sustainable Access in Rare Neuromuscular Diseases

Virtual Series | January – July 2027

Bringing together HTA bodies, payers, regulators, patient organisations, clinicians, researchers and industry to address the challenges associated with the assessment and adoption of innovative therapies for rare and ultra-rare neuromuscular diseases.

Why This Series?

The rare disease landscape is changing rapidly. Gene therapies, precision medicines, devices and disease-modifying treatments are creating unprecedented opportunities for patients with devastating and previously untreatable conditions.

This workshop series will create a practical forum for collaboration, knowledge exchange and solution-focused discussion, helping stakeholders navigate the evolving HTA and access environment.

Pre-register now to reserve your place

To receive programme updates, speaker announcements and registration information.
Contact: education@treat-nmd.com

 

What We’ll Explore

  • Evidence generation and assessment in rare and ultra-rare diseases.
  • Managing uncertainty through real-world evidence, patient registries and long-term data collection.
  • Patient-centred value assessment and incorporation of patient perspectives.
  • Collaborative approaches to improving HTA processes and timely patient access to innovative therapies.

The Workshop Series

Workshop 1: Evidence & HTA Foundations
Defining the Evidence Standard in Rare Neuromuscular Diseases

Workshop 2: Value, Pricing & Sustainability
What Is Value and How Do We Pay for It?

Workshop 3: Managing Uncertainty & Policy Alignment
Building Confidence in Long-Term Decision Making

Workshop 4: From Challenges to Solutions
Developing Practical Recommendations for Better Access

Who Should Attend?

  • Pharmaceutical, Biotechnology and MedTech Companies
  • Market Access and HEOR Professionals
  • Patient Organisations
  • Clinicians and Researchers
  • Payer and Reimbursement Bodies
  • Regulatory Authorities

Session Dates and Times

  • Tuesday 19 January 2027 – Webinar 1
  • Tuesday 16 March 2027 – Webinar 2
  • Tuesday 27 April 2027 – Webinar 3
  • Tuesday 22 June 2027 – Webinar 4

Workstreams

Participants will contribute to discussions within one of the following workstreams:

  • Gene Therapy
  • Small Molecules
  • Ultra-Rare Disease
  • Medical Devices & Technology

Registration & Pricing

The full four-session workshop series is £2,995.

A 50% discounted rate (£1,497.50) is available for:

  • Academics studying ultra-rare diseases
  • Patient organisations

Places are limited and will be offered on a first-come, first-served basis. Pre-registration is required, and completion of the form does not guarantee a place.

Programme Outcomes

The series aims to generate practical outputs that support future policy development, evidence generation and access planning, including:

  • Workshop reports
  • Consensus recommendations
  • White papers and future guidance documents

Apply Now

Join an international community committed to improving access to innovative therapies for people living with rare neuromuscular diseases.

 

For Sponsors & Partners

Sponsorship and partnership opportunities provide a platform to:

  • Demonstrate commitment to patient access
  • Engage with key decision-makers and stakeholders
  • Contribute to discussions shaping future access frameworks
  • Support the development of practical recommendations and outputs for the rare disease community

Details