Source: https://www.value-dossier.com/atmps-advanced-therapeutic-medicinal-products
Timestamp: 2019-04-24 20:25:58+00:00

Document:
Advanced therapy medicinal products cover gene therapeutics, somatic cell therapeutics and biotechnologically engineered tissue products or their combinations. As defined in Directive 2001/83/EC of the European Parliament and the Council, ATMPs are biological medicinal products if they possess properties for the curing or prevention of human disease or if they demonstrate in or on the human body a pharmacological, immunological or metabolic action intended primarily to restore, correct or modulate human physiological functions. The European Union has specifically adopted the EU Regulation No. 1394/2007 of the European Parliament and the Council to harmonise the rules governing the movement of these pharmaceuticals in order to address the specificities of these biopharmaceuticals and their complex application.
It contains an active substance which is or comprises a recombinant nucleic acid that is used or administered to human beings to regulate, repair, replace, add or remove a nucleic acid sequence.
Additionally, its prophylactic, diagnostic or therapeutic effect is directly connected to the containing recombinant nucleic acid sequence or to the expressed product of that sequence.
Vaccines against infectious diseases do not belong to the category of gene therapeutics.
ATMPs are approved as biological drugs by the EMA. In Germany, market access and reimbursement of these drugs is generally equivalent with innovative drugs or, in case of an orphan drug status, with orphan drug regulations. As a result, ATMPs used in outpatient care are also subject to the early benefit assessment procedure. For ATMPs used only for inpatient purposes, an exemption application can be submitted to the Federal Joint Committee at least 3 months before market entry. This applies only, if the expected sales volume is of minor importance, i.e., less than 1 Mio Euro.
However, it emerged that ATMPs were not always treated as drugs by the G-BA, but rather, depending on the extent and proportion of medical services involved in the use of ATMPs, were evaluated as a new examination and treatment method in accordance with § 135 SGB V (Social Code, Book V). Three examples are the gene therapy C-Cure® and the biotechnological tissue products Holoclar® and Maci®. ue to the novelty of the ATMPs, it should be noted that different legal bases have been applied reagrding the reimbursement of the products. Consequently, ATMP manufacturers are faced with different requirements, time constraints and price negotiation options for each procedure. From this point of view, the early benefit assessment according to § 35a SGB V provides benefits for the companies in terms of clear formal guidelines for the AMNOG dossier and a defined time-line for the evaluation as well as the price negotiations. The process of early benefit assessment according to § 35a SGB V provides clear formal guidelines for the AMNOG dossier, which defines the time frame. Moreover, the benefit assessment leads to price negotiations for the new product, which result in an agreement on the reimbursement amount after 12 months. The new product is immediately reimbursable upon market launch, the product price can be freely set by the pharmaceutical company and is transferred to the negotiated reimbursement amount following the early benefit assessment procedure with subsequent price negotiations.
If an ATMP is assessed according to the early benefit assessment, the new product is immediately reimbursable at market entry, the price can freely be freely chosen by the pharmaceutical entrepreneur and the final reimbursement price is negotiated within 6 months. The price negotiations are based on the outcome of the benefit assessment and the comparators.
However, if an ATMP is classified as a new examination and treatment method, its reimbursement is not initially possible, as the outpatient area is subject to the so-called reservation of authorisation. The reservation of authorisation states that examination and treatment methods are not covered by statutory health insurance (GKV) and thus shall not be applied or prescribed by physicians on behalf of the GKV until the G-BA has voted in favour for them. The G-BA evaluates whether a certain examination or treatment method is available for an adequate, appropriate and economical care of the insured, considering the current state of medical knowledge. Article § 135 SGB V differs from the early benefit assessment according to AMNOG regarding the eligible applicants, the time frame and pricing. Applications for the assessment of new examination and treatment methods in accordance with § 135 SGB V may only be submitted by impartial members of the G-BA, a Federal Association of Statutory Health Insurance Physicians, a Statuory Health Insurance Fund or the hospital association.
It is composed of substantially engineered cells or tissues resulting in altered biological characteristics, physiological functions or structural properties relevant to the intended clinical use.
Is attributed with characteristics for the treatment, prevention or diagnosis of diseases by pharmacological, immunological or metabolic effects of the contained cells or tissues and is used or administered for that purpose in humans.
it is thought to be capable of regenerating, restoring or replacing human tissue.
the cells or tissues have been substantially engineered to achieve biological characteristics, physiological functions or structural properties relevant to the intended regeneration, restoration or replacement.
Or, the cells or tissues are not intended to perform essentially the same function(s) in the recipient as in the donor (non-homologous use).
The term „biotechnologically engineered products“ does not include products which contain or solely consist of non-viable human or animal cells and/or tissues and which do not act primarily pharmacologically, immunologically or metabolically.
While ATMPs remain subject to early benefit assessment, price negotiations follow the rationale of innovative medicines. The systematic and framework of the price negotiations are defined in § 130b SGB V.
Price negotiations are determined by the results of the benefit assessment, by the price level of comparative therapies and by European reference prices.
Especially gene therapies create as one-time treatments new challenges for the price negotiations between companies and payers. The annual therapy costs for chronic diseases have to be compared in terms of benefits, risks and costs of new ATMPs. The mechanism and duration of the measures provide an opportunity to negotiate new pricing and payment models.
Please contact us for any further discussion on new payment models, price strategies and negotiation aspects which are related to ATMPs in the outpatient and hospital sector.

References: § 135
 § 35
 § 35
 § 135
 § 135
 § 130
 V.