On 30th September, thought leaders in the field of personalised medicine from Europe, the United Kingdom and the United States met in Bad Gastein, Austria to discuss how Medicine’s Adaptive Pathways to Patients (MAPPs) and breakthrough designation will impact patients. In a moderated discussion with the experts, the audience and live participation from the public via Twitter, the panel examined the potential long-term implications of these new models for personalised medicines, and what their adoption means for regulators, payers, patients and society as a whole. Full video of the live panel is available via the link below. 

Organised by the European Alliance of Personalised Medicine (EAPM), European Federation of Pharmaceutical Industries and Associations (EFPIA) and Vital Transformation with the support of AstraZeneca, the panel examined the changes being developed and introduced in Europe and the USA to speed up the regulatory approval process for pharmaceuticals, in particular, personalised medicines.

Moving ahead from concept to implementation

Gordon

Gordon McVie, Senior Consultant, European Institute of Oncology, Secretary European Alliance for Personalised Medicine (EAPM)

To start off the discussion, moderator Duane Schulthess, Managing Director, Vital Transformation, introduced the panel by emphasising the importance of differentiating MAPPs in Europe from breakthrough designation in the US. “MAPPs is an evidence approach – this is a new way to look at evidence,” he noted, while in the US, the FDA’s breakthrough designation is “a way to make things faster using the existing pathway” – with requests for breakthrough designation requiring the US FDA (Food and Drug Administration) to respond within 60 days.

The conversation kicked off with a European perspective, with Gordon McVie, Senior Consultant, European Institute of Oncology, Secretary European Alliance for Personalised Medicine (EAPM) presenting on the state of precision medicines in Europe. Speaking on what it will take for the successful implementation of precision medicines, Mr McVie outlined three points for progress in terms of foundational policy actions:

  • Liberate the data but do no harm;
  • Bring it now – ensure the adoption and translation of talk into action in the clinical setting;
  • Prepare the future – education is needed for future implementation and success, while necessary ICT infrastructure is still lacking.

In terms of preparing for the future, Mr McVie acknowledged there was much to be done – notably in the field of biomarkers, which are integral to the success of personalised medicines.  “We have got a million biomarkers and only about two that work,” he said, when asked about the current status of biomarkers, adding, “It’s the highest priority of research in most cancer research institutes that I know.”

Patient safety remains a central concern

Stanimir Hasurdjiev, General Secretary, Patient Access Partnership, posed a common question that arises in discussions around MAPPs, asking: “Can we expect the same level of safety for patients?”

Carole

Carole M Longson, Director Centre for Health Technology Evaluation, NICE

Addressing this, Carole M Longson, Director Centre for Health Technology Evaluation, NICE, said “The issue with early access is that the promise of earlier access comes with greater uncertainty – that’s in itself not necessarily a bad thing but it does present risks.” “The issue that we face if we want that earlier access, is that we have to have a way to manage that risk – in a way that healthcare systems can both understand and do. That’s what the MAPPs concept is trying to achieve.”

Chris Hoyle, Director, Health Economics & Payer Analytics (Oncology), AstraZeneca, noted that early access isn’t necessarily about lowering the evidence threshold – an oft-cited concern in terms of patient safety. “We’re using new technology to identify subsets of patients much earlier than we would otherwise,” he said, pointing out that this doesn’t necessarily mean a lower evidence threshold.

Elaborating on the safety issue from the US perspective, Amy M Miller, Executive VP, Personalized Medicine Coalition, spoke on the mechanics of breakthrough designation in the US. “When the FDA releases a product through the breakthrough pathway in phase II, that’s not the end of the FDA’s investigation of the drug. Investigation continues into phase III of the trial, and companies also continue to do research.” A product being released in phase II doesn’t end the regulatory burden on companies, she added, and it certainly doesn’t end the process of examination.

Inequalities in implementation need to be avoided

Stanimir

Stanimir Hasurdjiev, General Secretary, Patient Access Partnership

Another issue raised both among the panelists and the audience via Twitter was that of inequality. In particular, in the EU, where inequalities already exist in healthcare, this can be a touchy subject when discussing innovations such as MAPPs and their future implementation.

“There is diversity in Europe, which is nice – but this diversity can create difficulties,” noted Mr Hasurdjiev. “So we have to make sure that everything we are trying to achieve is implementable across the board, or we risk creating more inequalities.”

Infrastructure and existing systems – i.e. ICT – within countries would play a significant role in implementation between EU member states, it was agreed. There are practical consequences of not ensuring uniform access, especially when it comes to innovative treatments for unmet medical need. “We have the responsibility to find solutions,” said Hasurdjiev.

Investing in the future in the face of cost and resource burdens

A discussion of inequalities naturally raises the issue of cost burden and investment across the board. New technologies are difficult to implement as well as difficult to implement equally across the EU, where differences in government healthcare budgets, reimbursement policies, and Health Technology Assessment (HTA) still prove problematic.

Chris

Chris Hoyle, Director, Health Economics & Payer Analytics (Oncology), AstraZeneca

Ms Longson, speaking on the question of investment, highlighted this, noting that there is a pre-exiting healthcare system sustainability issue, in particular when it comes to the introduction of new drugs, which do come at a cost. “Is the cost worth the buy? This is the issue that we are talking about when we talk about earlier introduction,” she said – and this is what HTA’s seek to address.

Speaking on the hesitance of HTA bodies to accept a medicine earlier, Mr Hoyle noted that there needs to be more confidence in the evidence that is coming through. “As the evidence comes through it’s pointing to specific patient groups that would benefit with the existing evidence,” he said, emphasising the utility of iterative assessments across the product lifespan.

Keeping up with the science

Amy

Amy M Miller, Executive VP, Personalized Medicine Coalition

Despite the hurdles acknowledged by the panel throughout the conversation, the session concluded leaving no doubt as to the momentum of personalised medicines. Noting the fast pace of growth of precision medicines in the US, Ms Miller said, “last year 20% of the new drugs approved by the US FDA were personalised medicines” – and said that expectations in the US are for this trend to continue, with 1 in 5 medicines approved by the FDA likely to be precision medicines in 2015. The rapid developments of science and technology, it seems, are outpacing existing regulations. The regulatory issues touched upon during the panel, from both the US and Europe, made this clear.

Mr McVie, outlining the progress made thus far, called the audience to action: “Things are moving so fast… I hope you get some energy out of this conversation and that you’ll go back to your jobs and say yes, we’ve got to do this. We’ve got to get a move on.”

More photos of the session are available here.

 

Watch the full video.