Wie gestaltet man einen wertorientierten Vertrag für eine neue Gentherapie?

Gene Therapies coming into the rare disease space are coming often with an in-mature data set. Uncertainties regarding outcomes e.g. the long term response to the treatment can be contractually address. Furthermore there are financial and cash-flow risks associated with one time payments that can be managed through installment agreement. The “art” of these contracts rely on addressing both clinical and financial uncertainties in the simplest way possible, without unnecessary burden for the healthcare system.

Seek long term benefits and additional cost savings . Design your trials with the payer in mind. Understand the processes of reimbursement needs and nuances of different countries

There are rare diseases that can be targeted by gene therapy. The value that patients receive is the aim however, safety comes first. The whole regulations and guidelines should be considered at the application point. Till this starts, full imagination proposal should be drawn including the payment, the service, and even the marketing of the project.

Understand what is the most appropriate payment model by country. What has worked well and what has not. Be prepared to track data for several years

Some countries may wish to examine societal impact and also what the Soc delivers? What is the tolerability of Soc ? Who would not be able to take the gene therapy? What is the role of caregiver and parent burden

Personally, I believe in the numbers in any project. If you can transfer any achievement into number, you do well. Otherwise, just talking about the performance without measuring its not producing. Key performance indicators is a simple accurate way to know where we were and where we are? In gene therapy it's applicable to set our KPIs and follow up the quality assurance plan.

Local data and registries, patient advocacy, slrs and landscape assessments. What is the pathway currently for this condition and what are the bottle necks that the therapy addresses

Depending upon the amount of literature that exists for the condition, natural history studies should be considered early on to compensate for paucity of evidence on outcomes, disease trajectory, etc. In rare disease, international studies may be required to gather sufficient data, while accounting for differences in standard of care, reimbursement, etc.

Be clear in understanding the process for contract as some countries require legal support. Who are the stakeholders who will drive the contract and who are the stakeholders who can drive implementation? What additional data or endpoints could be captured

The biggest success factors we see in the markets are: - 3rd party provided solutions that automate the process of monitoring and evaluating contract performance - Easily accessible information - Transparency for payers and pharma on contract performance

There are two sides to consider. 1 the efficacy side, no long term proof availability and in case of degenerative diseases, damage done cannot be repaired which means stabilization is in fact proof of efficacy 2 the value side. PFP with a long term perspective is the holy grail. I believe however nearly not implementable! Time needed to have right assessments (clear, objective, measurable). A registry to populate the outcome. A sound evaluation of the information allowing a correct objective assessment. As there are many stakeholders a lot of mutual trust is needed! It would be great to have a group of clever people to work on a general format of a PFP without the pressure and urgency of access for a therapy needed for patients.

The final set of considerations perhaps is how readily it can be committed to by all stakeholders. Does the right infrastructure exist to allow it to be administered easily? How will governance operate and who will make final decisions where an impartial perspective is needed? In the event that it proves not to be an effective approach (and doesn’t meet interests satisfactorily) how can it be adjusted?

I think this article provides a decent overview of how one would design a value based contract for gene therapies. Conceptually this looks straightforward but having been involved in one of the first performance related contracts with the NHS in the late 1990's the challenge is in constructing a process that is practical to implement, that takes account of confounding factors and that can be finessed to take account of and react to unexpected developments. Setting up a steering group with members drawn from the commercial organisation and healthcare providers is an effective way of dealing with the initial arrangements and ongoing management of the contract.

It's value mentioned to tell this.. I remember a friend who ha an interview for biochemical position in a transplantation institute. This guy was a highly skilled on exceptional way. Although he experienced a very good one, he didn't get passed! Later on he knew the candidate who was choosen he had business administration degree beside the Biochemistry. The chief of the team said.. We are lab scientist but in INDUSTRY. It's a market.