Our CEO Herve Lillu recently led a discussion on this topic at the 2020 World Pharma pricing, Evidence, and Market Access Congress.
Before we get to this let’s address a few things first.
What are ATMPS, and why are they different from other pharmaceutical products?
Recent pharmaceutical advances in Advanced therapy medicinal products (ATPMs). have forged a path for innovative treatment options, particularly for rare diseases with a high unmet medical need., These products fall under the following categories:
· Genetic therapy medicinal products (GTMPs),
· Cell-based therapy medicinal products (CTMPs),
· Tissue-engineered products (TEPs),
· And products integrally combined with medical devices
These products hold a lot of a lot of promise as they could lead a groundbreaking shift in therapeutics, changing the scope from disease management to cures in a
wide range of conditions. However these therapies involve the use of living cells or viral vectors and are highly complex and tend to have higher upfront costs than conventional therapies.
As of October 2020 are 8 ATMPS have received marketing authorization in the European Union: Imlygic®, Strimvelis®, Zalmoxis®, Holoclar®, Spherox®,and Alofisel® but there are over 500 clinical trials performed at various stages with, signaling the potential for significant changes in the treatment
landscape in the near future.
So what exactly could the change to the landscape look like?
In the short-term, we expect around 20 new gene therapies to hit the market in the next couple of years by which time, it might be unrealistic to expect a fundamental change in any framework to how payers access these products. This does not mean, however, that there will not be technical adjustments made for how these drugs are appraised. For instance, there will be challenges in determining discounting rates for ATMPs given that they have the unique position of hitting the market with 6 months of clinical data but with potential benefits ranging from 20 years to a lifetime.
Thinking longterm, however, there are over 10,000 diseases for which a single gene has been identified. It would be prudent to think about larger fundamental
changes needed in the assessment process before the flood gates open. Examples of which are new payment models such as early access schemes or conditional reimbursement schemes. These types of models will have to become the new normal as opposed to exceptions. Changes to the approval frameworks used to enable the authorization of ATMPs, that consider their wide benefits will also be required.
Could there be barriers to access?
While there may be many barriers, one significant one is the high bar of evidence required by payers that can be difficult to meet especially in the case of curative gene therapies. A possible solution is currently being trialed in France where their Temporary Authorization for Use scheme allows for the use of products that do not have marketing authorisation. This scheme is in place to allow patients access to pharmaceutical products intended to treat serious or rare diseases in which there is an absence of appropriate treatment.
What impact will COVID-19 have on gene therapy pricing and reimbursement?
No doubt COVID-19 will be a game changer. The real challenge is determining which of the changes caused by the pandemic will stay and which ones are temporary. The pandemic has catalyzed changes that were already on the horizon before this pandemic. One such change that will likely stay is prioritizing assessments of assets with a ‘high impact’ France’s HAS announced at the beginning of the year that it would redirect its resources towards the assessment of ‘high impact’ therapies and UK’s NICE’s accesses products that fall under the ‘Highly Specialised Technology’ category separately. However, the Covid-19 pandemic has made this need for prioritization more eminent and obvious. This change would possibly benefit ATMPs given that they tend to have an orphan designation
meeting a large unmet clinical need.
Another trend that might remain after the pandemic, is an emphasis placed on how particular products might place a burden on the hospital care system. The pandemic has shone a spotlight on the decades of structural issues that have gone unresolved in the hospital care systems, given its inability to quickly adapt to
the increase in care demand. This could have a negative impact on ATMPs. CAR-T therapies for example, place a significant burden on hospitals as patients may have to remain in the hospital for several weeks after the infusion to monitor their response to the treatment. This should not be overlooked by the manufacturer in their value demonstration. Pharmaceutical manufacturers must reflect on how their new products can reduce the workload of public and private hospitals by
prioritizing less invasive and less resource-hungry treatment administration.
In conclusion, we anticipate substantial changes to the pricing and reimbursement framework in the near future.
More information about the conference can be found here: https://www.terrapinn.com/conference/pharma-pricing/our-story.stm
And if you’re interested in finding out more information about how Inbeeo can leverage its extensive expertise and best support your product value demonstration, please
reach out by email at email@example.com