Support for a Biotech Company entering the french market with an orphan drug.
We have successfully incorporated MCDA in all the stages of the PMA process, helping companies to optimise market access in Spain in an innovative way:
- Pre-European Commission approval:
- To create awareness of a specific disease or clinical problem among key stakeholders, identifying current unmet needs.
- To develop a disease-specific MCDA framework for complex diseases where there is a lot of available information on the disease and there are multiple treatment alternatives like Multiple Sclerosis (1).
- Pre-P&R submission in Spain: To determine the relative value contribution of an innovative medicine with respect to its comparator(s) in Spain involving a multidisciplinary group of key stakeholders (2).
- Post-P&R approval: relative value contribution determination of a new product involving a large group of stakeholders across Spain in order to support regional and local (hospital) negotiations (3).
We have worked with a wide range of stakeholders at national, regional and local level, including patient associations:
- We have developed a MCDA framework for the evaluation of Orphan Drugs at a national level, involving clinicians, hospital pharmacists, decision-makers, payers and patients (4).
- We have worked together with the Catalan Health Service (CatSalut) to implement MCDA in the Catalan health plan (6) to assess the added value of Orphan Drugs at regional level (5).
- We have also worked with MCDA at a hospital level, creating a MCDA framework to support decision-making among members of a hospital pharmaco-therapeutic committee (7).
- We have used MCDA to raise oncology patients’ capability to provide their point of view to healthcare evaluations by developing a specific oncology MCDA framework from patients’ perspective (8).
- Alerany C, Fernández R, Landete L, Rodríguez A, Sandoval S, Valdivia M, et al. PND98 – DETERMINING THE VALUE OF OCREVUS® (OCRELIZUMAB) FOR THE TREATMENT OF MULTIPLE SCLEROSIS IN SPAIN BY MULTI-CRITERIA DECISION ANALYSIS (MCDA). Value Heal [Internet]. 2018 Oct 1;21:S345. Available from: https://linkinghub.elsevier.com/retrieve/pii/S109830151835366X
- Álvarez MT, Cuervo-Arango I, Perez-Santamarina R, Poveda J, Romero JA, Santamaría A, et al. PSY155 – DETERMINING THE VALUE OF EMICIZUMAB (HEMLIBRA®) FOR THE PROPHYLAXIS TREATMENT OF HAEMOPHILIA A PATIENTS WITH INHIBITORS IN SPAIN BY MULTI-CRITERIA DECISION ANALYSIS (MCDA). Value Heal [Internet]. 2018 Oct 1;21:S462–3. Available from: https://linkinghub.elsevier.com/retrieve/pii/S1098301518360315
- Jiménez A, Ais A, Beaudet A, Gil A. Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA). Orphanet J Rare Dis [Internet]. 2018 Dec 10;13(1):220. Available from: http://www.ncbi.nlm.nih.gov/pubmed/30526673
- Badia X, Chugani D, Abad MR, Arias P, Guillén-Navarro E, Jarque I, et al. Development and validation of an MCDA framework for evaluation and decision-making of orphan drugs in Spain. Expert Opin Orphan Drugs [Internet]. 2019 Aug 14;7(7–8):363–72. Available from: https://www.tandfonline.com/doi/full/10.1080/21678707.2019.1652163
- Guarga L, Badia X, Obach M, Fontanet M, Prat A, Vallano A, et al. Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut). Orphanet J Rare Dis [Internet]. 2019 Dec 27 [cited 2019 Sep 30];14(1):157. Available from: https://ojrd.biomedcentral.com/articles/10.1186/s13023-019-1121-6
- Generalitat de Catalunya: Departament de Salut. Pla de Salut de Catalunya 2016-2020 [Internet]. 2016. Available from: http://salutweb.gencat.cat/web/.content/_departament/pla-de-salut/Pla-de-salut-2016-2020/documents/Pla_salut_Catalunya_2016_2020.pdf
- Roldán ÚB, Badia X, Marcos-Rodríguez JA, de la Cruz-Merino L, Gómez-González J, Melcón-de Dios A, et al. MULTI-CRITERIA DECISION ANALYSIS AS A DECISION-SUPPORT TOOL FOR DRUG EVALUATION: A PILOT STUDY IN A PHARMACY AND THERAPEUTICS COMMITTEE SETTING. Int J Technol Assess Health Care [Internet]. 2018 Oct 23 [cited 2019 Sep 30];34(5):519–26. Available from: http://www.ncbi.nlm.nih.gov/pubmed/30348241
- Badia X, Aguarón A, Fernández A, Gimón A, Nafria B, Gaspar B, et al. Patient involvement in reflective multicriteria decision analysis to assist decision making in oncology. Int J Technol Assess Health Care [Internet]. 2019 Feb 7;35(1):56–63. Available from: http://www.ncbi.nlm.nih.gov/pubmed/30730288
‘MAP BioPharma’s team of experts has supported over 200 pricing and reimbursement procedures in the UK and Ireland since being founded in 2012. One example, for a very small population in an ultra-rare orphan indication was being scoped by NICE for inclusion in their single technology appraisal programme.
RPN has experience with a wide range of therapeutic areas and particularly with orphan and advanced-therapy medicinal products. RPN support includes early access and P&MA strategic assessments, advisory boards, stakeholder management and full support with national and regional P&R negotiations.
SmartStep Healthcare Strategy & Market Access Consulting provides personalized and specialized consulting services to the pharmaceutical and medical device industries to provide fast and effective market access to their products in Germany.