Market access strategies differ between broad and rare disease indications. In broad disease markets, access often depends on demonstrating cost-effectiveness to support reimbursement across large patient populations.¹ In contrast, rare disease access is driven by high unmet needs and meaningful clinical benefit to justify premium pricing for smaller, specialised populations.^2,3 

There is substantial opportunity to maximise the value of existing therapies by tailoring them to better align with patient needs, clinical practice, and payer priorities – ultimately improving healthcare efficiency.⁴ Customised therapeutic approaches can drive improved outcomes across healthcare systems.⁴ Repurposing existing medicines could have the potential to address these unmet needs in both settings by identifying new therapeutic uses for established compounds that improve efficacy, safety, or convenience.^4,5 

General market access challenges 

Geographic barriers can hinder medicine development, as specialist treatment centres and clinical trial sites are frequently difficult to access, with 28% of patients traveling over an hour to receive care.^6,7 Additionally, limited awareness of new treatments among healthcare professionals can also contribute to delayed patient access by reducing treatment recommendations.^8,9 Alongside developing novel therapies, optimising existing medicines through new formulations offers an opportunity to improve convenience, increase treatment options, and prevent therapeutic escalation.⁴ 

Market access challenges in rare disease indications 

A key challenge in developing rare disease medicines is the small patient population, which limits commercial viability and economies of scale.¹⁰ As development and post-authorisation costs must be recovered from relatively few patients, these medicines often require higher prices and have a higher cost per patient.¹¹ Although the development of these medicines is driven by high unmet needs, marketing authorisation still requires robust evidence of safety and efficacy, which can be difficult to generate because of limited patient numbers.^11,12 In these situations, value-added medicines (VAMs) can help with cost-effectiveness through strategies such as drug repositioning, reformulation, and combination.⁴ 

Market access challenges in broad disease indications 

Pharmaceutical companies face increasing challenges in navigating the complex requirements of regulators, government bodies, payers, and other stakeholders, which can make the development of new medicines challenging.¹² In the broad disease medicine market, intense competition can also affect formulary positioning and market access, requiring manufacturers to demonstrate additional value.¹³ Loss of market position may lead to product discontinuation, forcing patients to switch to alternative formulations or treatments which may cause further repercussions to their health.¹⁴  

Furthermore, the average time from clinical testing initiation to regulatory submission is 81 months, and fewer than 12% of drug candidates entering Phase I clinical trials ultimately receive regulatory approval.¹⁵ VAMs can help address these challenges by repurposing existing medicines through collaboration between healthcare professionals, academic institutions, and industry – accelerating the development of products with new formulations and for different therapeutic indications. 

How do VAMs help address these challenges? 

VAMs are derived from known molecules and address unmet needs by delivering meaningful benefits for patients and healthcare professionals.¹⁶ They can improve treatment accessibility through new formulations or delivery methods that can in turn enhance convenience, support adherence, and improve patient outcomes.¹⁷ Older patients may experience swallowing difficulties, which can lead to crushing tablets to combat this challenge; however, this can lead to dosing errors and raises concerns around bioavailability and stability.¹⁸ Liquid oral formulations can support accurate dosing during dose titration, which may be key in paediatric patients who may need low doses.^,18,19 Better adherence can also improve health outcomes by reducing urgent care visits and hospitalisations.²⁰ In addition, the streamlined research and development pathway for VAMs accelerates development and commercialisation while enhancing market competitiveness.¹⁶ By enabling more tailored and targeted treatment approaches, VAMs also address the practical challenges and diverse needs of different patient populations.²¹ 

A path to the future  

VAMs are an important way to address unmet patient needs, but are still reimbursed at similar level to generics – Article 84 proposes a data protection period of four years for repurposed medicines which will help to protect a brighter future of VAMs.²² As a member of the Value Added Medicines group within Medicines for Europe, Colonis Pharma Ltd are one of the leaders in VAMs: supporting unseen and forgotten patient groups by developing established molecules into treatment options that patients and healthcare professionals truly need. Using a patient-centred approach that goes beyond data analysis, we develop innovative VAMs and commercialise them across Europe. 

References: 

1. Kakkar AK and Dahiya N. Drug Dev Research. 2014;75:231–234. 

2. Berdud M et al. Cost Eff and Resource Allo. 2020;18:1–18. 

3. Drummond M et al. Eur J Healh Econ. 2011;12:263–271. 

4. Value Added Medicines. VALUE ADDED MEDICINES TOOLKIT. May 2016. Available from: https://www.medicinesforeurope.com/wp-content/uploads/2016/05/TOOLKIT-Value-Added-Medicines-20160531-final.pdf [Accessed September 2026]. 

5. Pushpaakom S et al. Nat Rev Drug Dis. 2019;18;41–58. 

6. Bowen AE and Nargundkar S. J Rare Dis. 2025;4:1–18. 

7. Sciencus. The real burden of treatment access for rare disease communities. Available from: https://www.sciensus.com/knowledge-base/the-real-burden-of-treatment-access-for-rare-disease-communities/ [Accessed September 2026].  

8. Iordatii M et al. BMC Med Inform Decis Mak. 2015;15:1–13.  

9. Medlinskiene K et al. Health Services Research. 2021;21:1198. 

10. Vavassori S et al. Fron in Pharm. 2024;15:1–8.  

11. Medic G et al. J Mark Access Health Policy. 2017;5:1299665. 

12. Kumar A et al. J Mark Access Health Policy. 2014;1:1–4.  

13. Mills M et al. BMC Health Serv Res. 2023;23:1–17. 

14. NHS Specialist Pharmacy Service. Switching brand and generic anti-seizure medicines for epilepsy. Available from: https://www.sps.nhs.uk/articles/considerations-when-switching-between-solid-doses-and-liquids/ [Accessed September 2026].​ 

15. Ramagopalan SV et al. J Comp Eff Res. 2025;14:1–9. 

16. Toumi M and Rémuzat C. J Mark Access Health Policy. 2016;5:1264717.  

17. Sasha E. J Pharm Pharm Res. 2023;7:019.  

18. Taylor S and Glass BD. Aust Prescr. 2018;41:191–193. 

19. Meyers RS. J Pediatr Pharmacol Ther. 2024;29:22–31. 

20. Roebuck CM et al. Health Aff (Millwood). 2011;30:91–99.  

21. Medicines for Europe. Our 5 pillars. Available from: https://www.medicinesforeurope.com/value-added-medicines/our-5-pillars [Accessed September 2026].  

22. Medicines for Europe. Pharmaceutical Directive – Article 84: Repurposed medicinal products. Available from: https://www.medicinesforeurope.com/wp-content/uploads/2024/04/Medicines-for-Europe-Position-Paper-Repurposing-v2.pdf [Accessed September 2026].  

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September 2026 |  GB-CPL-0-467 

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