Are neurologists ready to embrace gene therapy? - Physician Views


As recently noted by Forbes, the biotech bull market of 2013 has helped to revive an approach to drug development that had become largely abandoned and pursued by only a few devoted believers. Between the beginning of last year and early April 2014, however, 11 different companies specialising in gene therapy raised $618 million from venture capitalists and the public markets.

Momentum for this approach has been driven not only by a more palatable financing environment, but the relatively recent approval of the world's first gene therapy product (uniQure's Glybera for the treatment of lipoprotein lipase deficiency) and the steady accumulation of clinical data that have helped to shape more robust sentiment around the commercial opportunity of these products.

Parkinson's disease – an area bereft of significant clinical breakthroughs in recent years – has emerged as a focal point for a handful of gene therapy developers such as Oxford BioMedica and Voyager Therapeutics. The latter is hoping to replace the use of current therapies, which raise dopamine levels in Parkinson's disease patients, by developing a gene therapy that will trigger the patient's brain to produce more dopamine.

Reasons to Purchase

With this in mind, FirstWord is polling US and EU-5 based neurologists to ascertain how practising physicians currently view and evaluate the gene therapy development space. Specifically we are asking them...

  • How familiar they are with gene therapy
  • What they perceive to be the key benefits of gene therapy?
  • What are their specific concerns with gene therapy?
  • What is the highest priority issue that needs resolving over the next 12 months?
  • Which stakeholder group they expect to play the most important role in driving usage of gene therapy?

1.Executive summary
2.Empowered, aware patients
3.The pharma-patient relationship
3.1.Overlapping agendas in healthcare
3.2.Access to medicines
3.4.Communality of interests between patients and industry
3.5.Clinical-trial data transparency
3.6.Patient organisation fears of co-option
3.7.The effect of corporate reputation on relationships
3.8.Financial influence on patient groups
3.9.Emphasis on transparency
4.Programme and funding challenges
5.Recognise the interests of various stakeholders
5.1.Market conditions count
5.2.The call for free and frank discussion
5.3.Underlying concerns about the relationship
5.4.A mutually beneficial overlap of interests
6.Patient-centric medicines
6.1.Clinical-trial recruitment
6.2.Remodelling the interface between patients and industry
6.3.What do patients want?
6.4.Understanding the patient body
7.The impact of digital
7.1.Reaching beyond patient organisations
7.2.AllTrials and transparency
7.3.The business of pharmaceuticals
7.4.Pharma's social media lag
7.5.The patient thirst for information
7.6.The internet of things
7.7.The role of medical apps
7.8.Game-changing media
7.9.Pharma's reluctance to engage with patients
7.10.A new sphere of influence created by patient advocates online
8.The dynamics of patient empowerment and market change
8.1.does industry fit in?
8.2.Information needs beyond benefit-risk
8.3.Patients want information, not promotion
8.4.A relationship based on trust
8.5.Differences of interpretation can lead to confusion
8.6.Using third parties to provide information
9.How to engage with patient groups
9.1.You have to let go of the marketing
9.2.Information and engagement
9.3.A contribution to life and lifestyle
9.4.A conversation of equals
9.5.Talking and listening are both important
9.6.It comes back to trust
9.7.Addressing patient needs
9.8.Engaging early in R&D
9.9.Engaging with patients from top to bottom
10. Actively involving patients in R&D
10.1.Case study: the EUPATI project
10.2.Inolvement in R&D is selective
10.3.More expertise is needed
11.Case study: EPDA encourages national engagement
12.Conclusion: stepping up to the plate

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