CRA Insights

First “Challenge of the Cure” discussion: P&MA in disruptive therapies

June 14, 2018

Here’s a peek at the advantages, obstacles, and possible solutions generated from our recent “Challenge of the Cure” discussion, which focused on pricing and market access (P&MA) in disruptive therapies, such as gene and cell therapy (GCT).
The first in our series of dinner discussions was held at Baur au Lac in Zurich, Switzerland. The hotel was named to the Gold List by Condé Naste travel editors in its 2018 ranking of favorite hotels in the world. After the panel session, all participants were invited to socialize before and during dinner, where the discussions continued.
GCT—the perfect storm for payers
Only a couple of years ago, P&MA issues surrounding Sovaldi captured the industry’s attention—and made media headlines. Now the focus has switched to gene and cell therapies, where a perfect storm is brewing for payers based on:

  • Clinical uncertainty: unproven commercial results, duration questions; higher perceived failure risk
  • Benefit asymmetry: long-term clinical benefits versus up-front costs = motivation tradeoffs for payers
  • Rare-disease focus: significant payer pressure to provide patient access; limited competitive pricing pressures make launch negotiations critical
  • Price challenges: limited reference points for policy makers; post-Sovaldi curative therapies perceived as ‘break the bank’ options

Despite these issues, collaboration and innovation between payers and manufacturers is growing. To help facilitate discussions around these ongoing challenges and collaborations, we enlisted the help of three distinguished panelists who represent the payer point of view. They included:

  • Dr Paul S.J. Miller (UK)
  • Dr Christoph J. Rupprecht (Germany)
  • Dr Joan Antoni Vallès Callol (Spain)

Our payer panel addressed the following questions from their personal and country-specific perspectives:
How has the balance between potential curative status and longer-term uncertainty affected the willingness to fund gene and cell therapies?
UK: The funding challenges reside in the evidence provided to the payers and the uncertainty surrounding lasting effectiveness. There are three possible remedies. The first would be to provide more evidence. The second would be to try to analyze the clinical data in a different way that reaches payers’ expectations. The third is to change the contract agreement, creating an innovative contracting agreement that allows for dealing with uncertainty.
Spain: The establishment of a European Medicines Agency (EMA) patient registry would increase the level of treatment evidence, which would facilitate discussions with payers. For every drug there are respondent and non-respondent results, it’s not a GCT-specific issue.
Germany: While gene and cell therapies are potentially a cure, there is plenty of uncertainty and there is, often, no comparator (single-arm trial).
Highly innovative treatments often lack a comparator. How have clinical assessments and price negotiations been approached in these instances?
Germany: There are no strict rules. One must look at the specificity of the disease. It is case-by-case.
Spain: What really matters is the benefit to the patient. Patient registry data would help clear the compounded issues rising from the lack of a comparator and an incomplete data package.
UK: This problem is not unique to GCT. For instance, single-arm trials are quite common in oncology and rare disease. One can use historical data as a comparator.
How have regional budgets been affected by recent high-cost launches? How has this been managed?
Spain: An unusually high density of patients at launch shouldn’t have a big impact because of the budget margins used to facilitate access to new efficient treatments. Risk-pooling across regions is possible.
Germany: The system cannot currently accommodate a large cohort of patients wanting same-time access to new treatments, particularly given the uncertainty regarding long-term effectiveness. Something would need to change to give a lot of people access to high-cost therapies.
Overall, what do you consider to be the biggest challenge impacting access to innovative therapies and why?
UK: Decision makers need to understand the pricing model. We need new contracting arrangements that consider the uncertainty of these treatments from a payer perspective.
Spain: We consider fairness. Decisions are not based on price.
Germany: 20% of healthcare expenditures bring no benefit to patients. We need to reallocate resources and confirm the benefit to patients.
How will healthcare systems need to adapt with many gene/cell therapy options offering cures across a broader range of diseases?
Germany: The challenge will not be price, but that populations in general will live longer.
Spain: The price will diminish as technologies evolve.
UK: Innovative contracting agreements.
Informally exploring divergent perspectives
The evening’s discussions also included robust participant-generated explorations of some of the following topics:

  • Shortening clinical development times to make treatment accessible earlier.
  • Measuring efficacy under real-world scenarios.
  • Disconnected points of view between pharmaceutical companies and payers regarding actual product innovation.
  • Empowering patients, especially regarding value assessments.
  • Weighing outcome-based versus indication-based pricing.
  • Differentiating gene- versus cell-based therapies.

Working toward solution
Several potential solutions were debated, including:

  • Give more voice to patients and use endpoints that matter to them.
  • Remove public rebates in systems like Germany to enable greater pricing flexibility within the constraints of international price referencing.
  • Increase political will to put aside a separate “regenerative medicine” fund outside of the system to allow for more innovative agreements as evidence is gathered.
  • Create a leasing model or delayed payment model to address clinical uncertainty and upfront costs. (This would require assurances from politicians that agreements would remain unchanged when authorities/people in power change.)
  • Develop better analytics to support why an innovative payment method is better than a straight discount upfront.
  • Generate more interest from both payers and policymakers/politicians.