Frontiers in Pharmacology, Год журнала: 2024, Номер 15
Опубликована: Ноя. 5, 2024
The rising cost of medicines burdens healthcare systems and limits access to novel therapies worldwide. Therefore, sustainable solutions enhance foster innovation are crucial. To highlight current strategies exchange ideas, we created a collection titled "Towards continued affordable accessibility innovative drugs: development efficient use medicines." Our primary goal was provide practical recommendations insights support systems. Here discuss the key topics covered in collection:1. Addressing uncertainty regarding clinical valueHealth Technology Assessment (HTA) bodies funders often evaluate value new drugs before reimbursement. Several factors can contribute value; e.g. validity surrogate endpoints, concerns about generalisability lack long-term efficacy data. In our collection, Vallano et al.1 emphasize importance evaluating clinically relevant variables (i.e., overall survival quality life) over endpoints. Broader eligibility criteria improve real-world representativeness studies.Fagereng al.2 studied impact these uncertainties on reimbursement decisions Norway. Drugs with higher certainty relative effectiveness were more likely be reimbursed, at costs, than those lower certainty. This underscores robust relative-effectiveness data for guiding policy resource allocation.In Netherlands, costs have resulted halt automatic high budget used hospitals. Since 2015, so-called 'Coverage Lock' has been implemented by government assess establish financial arrangements manufacturers. Bomhof al.3 explored ethical aspects reduced drug under this policy. Although most stakeholders interviewed favoured through free-of-charge programmes manufacturers during Coverage Lock, they expressed transparency unequal access. Creating national platform such as Dutch Drug-Access Protocol4, that provides equal access, gathers data, incorporates pragmatic, outcome-based risk-sharing model, well finding common ground pharmaceutical companies5, may offer solution.Another challenge discussed Advanced Therapy Medicinal Products (ATMPs). Rejon-Parrilla al.6 identified barriers, initial insufficient which burden With HTA regulation starting 2025, anticancer ATMPs will undergo joint assessments. could enable collaborative evidence generation potentially future.2. Increase treatment (cost)effectiveness preventing overtreatment de-escalation strategiesWith launch every therapy, optimisation studies essential refining use, improving patient outcomes, and, when possible, enhancing cost-effectiveness. Subjecting patients unnecessary long duration or doses medicine exposes them avoidable side effects, negatively their life. Furthermore, strains environment resources, including time, personnel facilities. Pharmaceutical companies themselves usually incentives address explore alternative dosing regimens, slow down jeopardise developmental business outcomes. Once is commercially available, personalised approach7 exploring 8,9,10 interest both patients, payers, professionals society. Walia al.7 an indefinite anticoagulation strategy unprovoked venous thromboembolism Buma al.8 regimens immune-checkpoint inhibitors lung cancer, alongside extensive biomarker research overtreatment.9 latter funded fund (Treatmeds Foundation)11 specifically supports cost-efficiency studies.9 However, ideal setting, dose minimisation should already part process. 3. Reducing wasteReducing waste preserves valuable resources minimises environmental impact. Dane al.10 propose same day scheduling reduces waste, prepared IV therapy no-show given another patient. For administered home, chance being left unused (for instance, due disease progression, death), hospital pharmacies deliver supply two weeks monthly prevent spillage, though frequent deliveries carefully considered holistic strategy. Also collecting re-dispensing oral reduce save money.10,12 make possible routine care, local regional regulatory guidance needs developed determine circumstances acceptable. absence guidance, oncologists aim prescribe just enough medication minimising risk wasting surplus changes necessary.10 while mindful not increase care patients. 4. Combination challenges single technology relatively straightforward, involving analysis outcomes particular intervention. assessing combination multiple technologies (several examples submitted collection)13 14,15,16 complex. complexity arises, from synergy, cumulative complex negotiations various companies. Consequently, cost-effectiveness some show unfavourable per QALY, even high-income countries.14,15In myeloma, paradigm involves upfront triplet quadruplet result toxicity costs. It unknown whether sequential agents lead similar superior al.17 advocate trial justifying multi-drug regimens.In He al.16 economic analyses combined first-line advanced hepatocellular carcinoma China, where varies significantly among provinces. Also, EU, diverse conditions varying thresholds members, resulting disparities Academic hospitals10 cooperative study groups17 well-suited champion agendas focused studying rather combinations classes, thereby reducing contributing sustainability affordability 5. In-house productionAcademic hospitals possess specialised expertise agility develop and/or produce response unmet medical needs. several today had prototypes academic hospitals.10 A recent EMA (European Medicines Agency) pilot aims help academics further ATMPs. Netherlands currently reimburses in-house adoptive cell tumour-infiltrating lymphocytes (TILs)18 melanoma non-profit radiopharmaceuticals, hospitals.10Compounding play crucial role producing scarce discontinued companies, ensuring ongoing cases, compounded also serve cost-effective commercial counterpart.10,19 Overall, academia-driven instrumental public-private partnerships towards therapies.6. Repurposing precision oncologyDrug repurposing uses approved indications, offering treatments addressing approach lowers R&D since passed safety assessments demonstrated efficacy. Drug Rediscovery Protocol (DRUP)20,21 adaptive trial, off-label targeted based molecular tumour profiles. offers who exhausted standard-of-care options generates necessary Currently, eighteen European countries collaborate PRIME-ROSE consortium (Precision Cancer Medicine System Using Pragmatic Clinical Trials)22 create DRUP-like accelerate rare indications. other EU platforms REMEDI4ALL REPO4EU boost repurposing.Nevertheless, relying solely public funding trials unrealistic. clear path registration developing transparent, cost-based-plus pricing models appeal private investors stimulate repurposing. centre Future Affordable Sustainable (FAST)23 explores area better align development.ConclusionIn 'Towards medicines', present snapshot insights, efforts aimed drugs, promoting medicines. important realise there substantial knowledge gap after drugs. systematically independently addressed optimise regimens. initiative Forum (CMF),24 established identify questions priorities, gaps. National funds supporting initiatives overtreatment.
Язык: Английский
