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  • Tiered Pricing for Varied Markets: Is It of Value to Patient Access?

    Tiered Pricing for Varied Markets: Is It of Value to Patient Access?
    Tiered Pricing for Varied Markets: Is It of Value to Patient Access?

    Ensuring equitable access to life-saving medications in the pharmaceutical industry is a significant challenge. Tiered pricing, a strategy that adjusts drug prices based on the economic status of different markets, offers a promising solution. This approach aims to balance the financial sustainability of pharmaceutical companies with the urgent need for affordable medications in lower-income regions.[1]

    Tiered pricing, sometimes also referred to as differential pricing, involves setting different prices for the same product in different markets. In the pharmaceutical sector, this means offering drugs and vaccines at lower prices in developing countries compared to developed ones. The tiered pricing approach aims to improve access to medicines for economically disadvantaged populations, and is supported by industry, policymakers, civil society, and the academia. It allows manufacturers to adjust prices based on each market’s ability to pay, increasing the accessibility of pharmaceuticals in lower-income regions.[1,2]

    Tiered pricing enhances access to essential medications in low- and middle-income countries (LMICs) by lowering drug prices, aligning with global health goals to reduce disparities. This approach allows the higher drug  prices in wealthy markets to cover research and development costs while keeping prices lower in developing regions. Consequently, tiered pricing not only enhances social welfare by making medications more accessible, but also enables pharmaceutical companies to tap into new markets. Additionally, it reflects corporate social responsibility by focusing on patient welfare and contributing to global health improvements, which enhances the company’s reputation and relationships with stakeholders.[1,3-6]

    Despite its benefits, tiered pricing presents several challenges that need addressing. One major issue is pricing transparency: a lack of clarity around pricing structures can lead to mistrust among patients and healthcare providers. Clear communication about the rationale behind tiered pricing is crucial for building public trust. Market segmentation also poses difficulties, as internal market divisions may not always be equitable. Variations in the public and private sector’s role in medicine distribution and differing economic statuses within countries can complicate accurate segmentation. Additionally, current tiered pricing approaches vary widely, with some companies using World Bank income classifications while others rely on development indicators or regional health burdens. A major flaw in using per capita Gross National Income for pricing is that it does not account for high inequality within countries, which can limit affordability for poorer populations. The absence of a clear international norm for setting price tiers and the political nature of distributing R&D costs further complicates the implementation. Moreover, tiered pricing can unintentionally foster parallel trade, where lower-priced drugs intended for LMICs are resold in higher-income markets, undermining the pricing strategy and causing financial losses for pharmaceutical companies. To maintain the integrity of tiered pricing, robust measures are needed to prevent such trade practices.[6-8]

    Several pharmaceutical companies have successfully implemented tiered pricing strategies to enhance patient access. The willingness and ability to pay for drugs vary across countries and pharmaceutical companies often engage in price discrimination based on purchasing power and socio-economic segments. [5,9] As the global healthcare landscape evolves, tiered pricing is set to play a crucial role in improving patient access to medications. Advances in data analytics and market research will enhance pharmaceutical companies’ ability to accurately segment markets and set appropriate prices. To ensure access to medicines for populations in need, alternative strategies should harness the power of competition, avoid arbitrary market segmentation, and recognize government responsibilities While competition can be effective for achieving affordability, tiered pricing remains a legitimate mechanism to improve consumer welfare by reducing the price of health technologies without compromising innovation. Another method is charging higher prices in insured markets while offering lower prices for other sectors. However, many developing countries have limited insurance systems. To achieve equitable pricing, governments need to promote generic competition. Importantly, differential pricing offers limited hope of price reduction for drugs specific to the developing world and fails to address access to medicines for neglected diseases. Increased collaboration between governments, NGOs, and the pharmaceutical industry can help address these challenges and ensure the successful implementation of tiered pricing.[1,6,10]

    In conclusion, tiered pricing offers a viable solution to the challenge of equitable access to medications across varied markets. By balancing affordability with financial sustainability, this strategy can enhance patient access, improve public health outcomes, and demonstrate corporate social responsibility. However, careful consideration of the associated challenges and a commitment to transparency and market segmentation are essential for its success. As the pharmaceutical industry continues to innovate, tiered pricing will remain a key component of efforts to make life-saving medications accessible to all, regardless of economic status.

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    References:

    1. Moon S, Jambert E, Childs M, et al. A win-win solution?: A critical analysis of tiered pricing to improve access to medicines in developing countries. Global Health. 2011;7:39.
    2. Yadav P. Differential pricing of pharmaceuticals: Review of current knowledge, new findings and ideas for action. A study conducted for the UK Department for International Development (DFID). Zaragoza, Spain: MIT-Zaragoza International Logistics Program; 2010. Available from: http://www.dfid.gov.uk/Documents/publications1/prd/diff-pcing-pharma.pdf
    3. Danzon PM, Towse A. Differential pricing for pharmaceuticals: reconciling access, R&D and patents. Int J Health Care Finance Econ. 2003;3:184.
    4. Chalkidou K, Claxton K, Silverman R, et al. Value-based tiered pricing for universal health coverage: an idea worth revisiting. Gates Open Res. 2020;4:16.
    5. World Health Organization. Promoting access to medical technologies and innovation: intersections between public health, intellectual property, and trade. Geneva, Switzerland: World Health Organization; 2012. p. 159.
    6. Abbas MZ. COVID-19 and the global public health: Tiered pricing of pharmaceutical drugs as a price-reducing policy tool. J Gen Med. 2021;17(3):115-21.
    7. Cameron A, Ewen M, Ross-Degnan D, Ball D, Laing R. Medicine prices, availability, and affordability in 36 developing and middle-income countries: A secondary analysis. Lancet. 2009;373:240-9.
    8. Wong EV. Inequality and pharmaceutical drug prices: An empirical exercise. Disc Pap Econ. Center for Economic Analysis, Department of Economics, University of Colorado, Boulder, CO; Working Paper 02-19.
    9. World Health Organization. WHO and Novartis join forces to combat drug-resistant malaria [Internet]. 2001 May 23. Available from: https://www.who.int/news/item/23-05-2001-who-and-novartis-join-forces-to-combat-drug-resistant-malaria
    10. Gurry F. Innovation driving human progress: WIPO and the Sustainable Development Goals (SDGs). Geneva: World Intellectual Property Organization; 2018. p. 15.

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  • Access to Gene Therapies: The Evolving Landscape, Opportunities, and Challenges

    Access to Gene Therapies: The Evolving Landscape, Opportunities, and Challenges

    Once relegated to the realm of science fiction, gene therapy has emerged as a powerful force with the potential to revolutionize medicine. By delivering healthy genes to correct underlying genetic defects, these therapies offer the tantalizing prospect of curing previously untreatable diseases like cystic fibrosis, sickle cell anemia, and hemophilia. However, ensuring patient access to gene therapy and translating this potential into tangible benefits for patients necessitates confronting a complex landscape of challenges, embracing emerging opportunities, and continuously learning from ongoing experiences.[1]

    One of the main challenges is the high cost of gene therapy: the exorbitant cost often renders it unattainable for many patients and healthcare systems alike. This cost stems from multiple factors, including extensive research and development, intricate manufacturing processes, and limited patient populations. Years of research, clinical trials, and regulatory approvals precede market access, incurring significant financial investments. Manufacturing gene therapies often involves complex procedures utilizing specialized vectors and facilities, contributing to high production costs. Additionally, many gene therapies target diseases affecting small patient populations, leading to high per-patient costs due to economies of scale limitations.[1]

    Beyond the financial considerations, gene therapy raises a spectrum of ethical concerns that warrant careful consideration, including issues relating to equity, safety, and informed consent. Unequal access due to cost can exacerbate existing healthcare disparities and raise concerns about justice and fairness. The long-term effects of gene editing on future generations are not fully understood, necessitating careful evaluation and monitoring. Ensuring patients fully understand the potential risks and benefits, especially in the context of novel and evolving technologies, is crucial. Balancing the promise of innovation with the imperative of ensuring safety and addressing ethical concerns is paramount for fostering public trust and responsible advancement in this field.[1,2]

    Navigating the regulatory landscape for gene therapy presents another layer of complexity. Balancing the need for innovation with rigorous safety assessments is crucial to protect patients and maintain public trust. However, lengthy approval processes and evolving regulatory frameworks can hinder market access and delay patient treatment. Striking a balance between agility and thoroughness remains a delicate process, requiring streamlined approval processes and adaptive regulations. Implementing efficient review systems and data harmonization across regulatory agencies can expedite approvals without compromising safety. Keeping regulatory frameworks adaptable to accommodate technological advancements while upholding safety standards is vital.[2]

    Delivering the promise of gene therapy hinges not only on scientific breakthroughs but also on robust infrastructure and a skilled workforce. The specialized infrastructure needed for manufacturing, administration, and long-term monitoring may not be readily available in all regions, especially in under-resourced settings. This infrastructure gap makes accessibility even more challenging. Furthermore, a trained workforce capable of handling these complex therapies is critical, yet shortages exist in many areas. Addressing these critical gaps requires investment in infrastructure development and workforce training programs. Building specialized facilities and ensuring access to necessary equipment and resources globally is crucial. Upskilling healthcare professionals and creating training programs focused on gene therapy are essential to address personnel shortages.[3]

    Despite these challenges, the landscape of gene therapy is not devoid of opportunities. Innovative pricing models offer promising avenues for making these therapies more affordable. Outcomes-based agreements, which link payment to successful treatment outcomes, incentivize developers to focus on effective therapies while reducing upfront costs for patients and healthcare systems. Implementing flexible pricing structures that consider affordability across different regions and income levels can promote broader access through tiered pricing based on income levels.[3]

    Advancements in gene editing technologies like CRISPR hold the potential for more targeted therapies and broader applications. Delivering gene edits to specific cells can minimize unintended effects and increase safety while expanding the scope of treatable conditions beyond rare diseases can lead to economies of scale and potentially lower costs in the long run. Collaboration across borders emerges as a powerful tool for overcoming the challenges and seizing the opportunities presented by gene therapy. By sharing knowledge, resources, and infrastructure, a global collective can accelerate access to these life-altering treatments. Fostering public education plays a pivotal role in building trust and informing policy decisions. Enhancing awareness about the potential and limitations of gene therapy is crucial for engaging the public and garnering support for continued research and development. Openly discussing ethical considerations and potential risks alongside the therapeutic possibilities can cultivate a more informed and engaged public, laying the groundwork for responsible advancement in this transformative field.[3,4]

    A sustained commitment from researchers, clinicians, policymakers, industry players, and the public alike is necessary for enhancing patient access to responsible, safe, and efficacious gene therapy delivered ethically. By navigating the challenges wisely, embracing emerging opportunities, and prioritizing continuous learning, it is possible to unlock the full potential of gene therapy and bring hope and healing to countless lives.

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    References

    1. Carvalho M, Sepodes B, Martins AP. Patient access to gene therapy medicinal products: a comprehensive review. BMJ Innovations. 2020 Oct 27:bmjinnov-2020.
    2. Cornetta K, Bonamino M, Mahlangu J, Mingozzi F, Rangarajan S, Rao J. Gene therapy access: global challenges, opportunities and views from Brazil, South Africa, and India. Molecular Therapy. 2022 Jun 1.
    3. Kennedy C, O’Dea E, Reidy D, Towns J, Pigott M, Kevans D, O’Rourke D, Hennessy M. Challenging times: Delivering gene therapies and an opportunity for shared learning. British Journal of Clinical Pharmacology. 2022 May;88(5):2444-6.
    4. Hidalgo‐Simon A, Fibbe WE. Advanced therapies are ready to take centre stage: Academia’s involvement with regulation needs to raise its game. British Journal of Clinical Pharmacology. 2021 Jun;87(6):2412-3.



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  • Expanded Access Programme: an Important Initiative to Enhance Patient Access

    Expanded Access Programme: an Important Initiative to Enhance Patient Access

    Patients with serious, life-threatening health conditions and who have no available treatment alternatives, often want to know if and how they can receive early access to investigational products, sometimes even before they are approved by the regulatory agencies. “Expanded access programme” (EAP), also referred to as “compassionate use programme” or “managed access programme”, is a potential pathway for such patients to obtain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials.(1) This way, eligible patients can have an access to the medical products whose efficacy and safety is made known through clinical trials, and the natural delay that is involved in the process leading to the regulatory approval and ‘proper’ market access of these medical products does not hamper the patient access to such medical products.

    The EAP is an initiative exclusive to the purview of the US Food and Drug Administration (USFDA). The USFDA’s history of enabling access to investigational therapies dates back to the 1970s; however, precise regulations around the same did not establish until 1987.(2) The USFDA later published specific regulations in 2009, further clarifying the expanded access process.(3)

    The EAP was conceived to cater to the patients who are suffering from a serious, life-threatening condition with no feasible treatment options available. This means, either there are no approved treatments in their home country, or the approved available treatments have proven ineffective for them, or they are unable to enrol in an active clinical trial. Essentially, there needs to be an unmet clinical need for this programme to be considered. The access to investigational drug through EAP can only be granted by the drug manufacturer (sponsor), i.e., no regulatory body or a third-party provider can facilitate it in the absence of an approval from the sponsor.(4)

    There are three categories under EAP, which are as follows: (5)

    1. Individual patient EAP (both regular and emergency access): when the patient does not have time to gain access via a written permission from the USFDA,(6)
    2. Intermediate-size patient population EAP, and
    3. Widespread treatment use EAP under an Investigational New Drug (IND) application: the patient must have a serious or immediately fatal disease and have no suitable alternative therapy; the potential benefit of the IND must justify the potential risks; and access to the IND must not interfere with the drug development process.

    The primary difference in the above three categories is the number of participating patients. The individual patient EAP is for a single patient, and is usually the preferred way of providing access in an emergency setting. An intermediate-size patient population EAP has no requirement for number of patients, but it certainly applies for more than one patient and when the IND is not actively being developed for marketing. A widespread treatment use EAP usually applies when the access is to be provided to a large population, often bridging the gap between the end of the clinical trials and marketing approval.(5)

    Expanded access for an individual patient depends on the cooperation and expertise of more than just the patient himself. The physician applying for access on behalf of the patient, the drug manufacturer, i.e., the sponsor, the institutional review board (IRB), and the USFDA, all play crucial roles and must work together for the success of the EAP. Expanded access is possible only if and when the drug company (sponsor) agrees to provide the investigational product. The USFDA cannot instruct the sponsor to provide the drug,(7) and the sponsor’s denial to do so may have valid reasons, such as the option for the patient to join an ongoing clinical trial and/or limited drug supply. Moreover, the apprehension about ‘clinical holds’ or unfavorable outcomes in case of any adverse events reported during an EAP is a common concern. However, a review of around 11,000 expanded access requests throughout a 10-year period showed only two clinical holds during drug development programs, the reason being ‘temporary’ adverse events observed in patients receiving expanded access.(8) Therefore, the occurrence of an adverse event is extremely rare during expanded access to adversely impact drug development.(5)

    An example for drugs made available through compassionate use program was when a composite of three chimeric monoclonal antibodies against ebolavirus (ZMapp) was administered to six Ebola-infected patients following the disease outbreak in West Africa, pending regulatory approval for the same.(9) This was in line with the World Health Organization’s (WHO’s) statement ethicizing the alternative to provide needy patients with access to investigational drugs that were not yet approved by regulators or even tested on human beings, with the aim of limiting the epidemic to save the lives of patients.(10, 11) The drug had shown 100% survival in animal trials; however, in the EAP, four out of six patients improved and two patients died.(9, 12)

    The USFDA facilitates the EAP; however, its success depends on the initiative and cooperation of other involved parties, including drug manufacturers (sponsors) and physicians, along with the USFDA’s review and authorization.(5)

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    References

    1. Expanded Access. The US Food and Drug Administration. March 2021. Available at: https://www.fda.gov/news-events/public-health-focus/expanded-access
    2. 21 CFR Parts 312, 314, 511, and 514: New Drug, Antibiotic, and Biologic Drug Product Regulations (IND Rewrite). March 1987, Updated August 2010. Available at: http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/ucm120111.htm
    3. Federal Register. 2009; 74(155); Available at: https://www.gpo.gov/fdsys/pkg/FR-2009-08-13/pdf/E9-19005.pdf
    4. Expanded Access Programs. Available at: https://www.wepclinical.com/expanded-access-programs/
    5. Jarow JP, Lurie P, Ikenberry SC, Lemery S. Overview of FDA’s Expanded Access Program for Investigational Drugs. Ther Innov Regul Sci. 2017; 51(2):177-179.
    6. Gaffney A. Regulatory Explainer: FDA’s Expanded Access (Compassionate Use) Program. February 2014. Available at: https://www.raps.org/regulatory-focus%E2%84%A2/news-articles/2014/2/regulatory-explainer-fda-s-expanded-access-(compassionate-use)-program
    7. Expanded Access to Investigational Drugs for Treatment Use—questions and answers. June 2016. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM351261.pdf
    8. Jarrow J, Lemery S, Bugin K, Khosin S, Moscicki R. Expanded access of investigational drugs: the experience of the Center of Drug Evaluation and Research over a 10-year period. Therapeutic Innovation & Regulatory Science 2016; 50(6):705–709.
    9. Gallagher J. Ebola: Experimental drug ZMapp is ‘100% effective’ in animal trials: BBC News; 29 August, 2014. Available from: http://www.bbc.com/news/health-28980153
    10. Geneva: World Health Organization; 2014. World Health Organization. Ethical Considerations for Use of Unregistered Interventions for Ebola Viral Diseases: Report of an Advisory Panel to WHO. Available at: http://www.who.int/mediacentre/news/statement/2014/ebola-ethical-review-summary/en/
    11. Hantel A, Olopade CO. Drug and vaccine access in the Ebola epidemic: Advising caution in compassionate use. Ann Intern Med. 2015; 162:141–2.
    12. Patil S. Early access programs: Benefits, challenges, and key considerations for successful implementation. Perspect Clin Res. 2016; 7(1):4-8.

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  • Importance of Tapping Payer’s Data to Document the Effect of a New Therapy

    Importance of Tapping Payer’s Data to Document the Effect of a New Therapy

    Traditionally, the pharmaceutical industry has always been dependent upon the ‘push’ strategy for successful market access for products. The drug approval process, involving submission of data on efficacy, safety, and tolerability to the regulatory agencies, used to be simple; which ended with the drug being marketed to the targeted physicians and dispensed by pharmacies post approval. Thus, this whole process involved a limited set of stakeholders, viz. physicians, regulatory agencies, and pharmacies. Conversely, over the years, the market access landscape has evolved primarily due to two factors: (1)

    1. Rising healthcare costs owing to an increasing prevalence of chronic diseases, growing geriatric population, and higher prices of new therapies
    2. Competitive pricing and reimbursement environment

    This has further led to the emergence of a new and diverse set of stakeholders over the years, i.e. the ‘Payer’(s), increasing the complexity of drug access to the market in general, and to patients in particular. Payer exercises the greatest degree of control over pricing and reimbursement for any new drug, and will continue to dominate the market access scenario to ensure successful market access. (2,3)

    Pharmaceutical advancements are increasingly conflicting as countries attempt to accommodate healthcare costs via different tools. New criteria for recognizing unique drugs and differences among those within the same therapeutic area or concerning the same molecule are being introduced, even though ‘price’ remains the main driver. (4) There is a surge of criticism towards the increasing prices of drugs that adds growing pressure on pharma companies and manufactures to limit future price increases, and eventually on payers to be more cost-effective in their approach to setting budgets and managing costs. (5) Global pharma operations need to keep up with the pace of these changes to approach pharma tendering as a strategy that spans pricing and commercialization.

    In order to document the effect of a new therapy in the real world, pharma companies are trying to justify prices by tapping payer’s data. Payers encourage pharma to collect post-launch evidence of product performance in the real world, thus turning it in pharma’s favor. This can help verify a price agreement or even clarify uncertainties about the clinical and/or safety outcomes outlined at registration. (6)

    The successful market access will involve collaborative team work between sales and marketing departments. The strategy itself should be well equipped to respond to market evolution and also, to accommodate all known interactions. There is no ‘one-size-fits-all’ solution. The challenges in the market will constantly vary as per the product, therapy area and the setting in which the treatment will be used.i,vi

    Payers are increasingly focusing on “real-world” outcomes to form their decisions, encouraging new policies to be formed, in order to assimilate evidence from different sources. These policies prioritize the evidence that goes beyond information collected during clinical development in randomized controlled trials (RCTs), required by regulatory authorities for marketing approval. ‘Administrative data’- that normally use retrospective or real-time patient data – are an example of the real world data sources, as they are collected primarily for reimbursement, but contain some clinical diagnosis and procedure use with detailed information on charges. Retrospective analyses (longitudinal and cross-sectional) of clinical and economic outcomes at patient, group, or population levels can be performed with the help of claims databases. Such analyses can be performed in short time and at low costs. (7)

    In conclusion, payer data from real-world such as claims data can most certainly impact the sound coverage, payment, and reimbursement decisions. It is critical that payers recognize – a) the benefits, limitations, and methodological challenges in using these data, and b) the need to carefully consider the costs and benefits of different forms of data collection in different situations.

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    References

    1. Kumar A, et al. Pharmaceutical market access in emerging markets: concepts, components, and future. Journal of Market Access & Health Policy 2014; 2:10.3402/jmahp.v2.25302.
    2. McClearn C, et al. Big pharma’s market access mission. Deloitte University Press; 2013.
    3. Arx RV, et al. Leveraging success factors for market access in the life sciences industry. Capgemini Consulting and Cegedim dendrite; 2009.
    4. Skinner JS. The costly paradox of healthcare technology. September, 2013. 
    5. Pharmaceutical pricing and market access 2017.
    6. Wechsier J. Measuring the value of prescription drugs. Pharmaceutical Executive 2017; 37(5).
    7. Garrison LP Jr. Using real-world data for coverage and payment decisions: The ISPOR Real-World Data Task Force Report. Value Health 2007; 10(5):326-225.

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  • Is Off-Label Drug Use Impacting the Access to Patients with Rare Diseases?

    Is Off-Label Drug Use Impacting the Access to Patients with Rare Diseases?

    What could be the better time – to talk about patients with rare diseases – than today, on the occasion of 11th Rare Disease Day! (1)

    The term ‘rare disease’ is defined as, ‘a disease or disorder affecting less than 1 in 2000 people’. (2) Rare diseases make up for one of the most scientifically complex health concerns today. Currently, about 7,000 rare diseases are known, half of which seem to affect children. (3) Moreover, evidence suggests that almost 95% of rare diseases did not have a single FDA-approved treatment (4) until last year (2017); when FDA’s Center for Drug Evaluation and Research (CDER) approved many new drugs, including some for patients with rare diseases, such as Batten disease, Chagas disease, and hemophilia A with inhibitors. (5)

    A lot of patients with rare disease resort to using off-label drugs with limited data, since there is no alternative available. FDA defines ‘off-label’ drug use as ‘unapproved use of an approved drug’, indicating that the drug can be: (6)

    • Used for a disease or medical condition that it is not approved to treat
    • Given in a different way
    • Given in a different dose

    Off-label use can be the best available intervention for a patient, as well as the standard of care for a particular health problem. Off-label prescribing mostly has clear therapeutic goals, and in certain practice areas like rare diseases, it is extremely common as well as necessary. Physicians treating rare disease patients strongly rely on off-label drug use. This is because the commercial sponsors often require financial incentives to develop products for small population of patients with rare diseases, which makes it impossible to evaluate products according to ordinary clinical trial criteria. (7) During the 1980s, Congress passed legislation to create incentives for manufacturers for developing products aimed at rare diseases, but many of these diseases still lack effective approved therapies. (8)

    Many pharmaceutical and biopharmaceutical companies, academic researchers, patient groups and forums are trying to expedite the development of new treatment options with the help of their ever-increasing knowledge of rare diseases. The Orphan Drug Act of 1983 (9) has been and continues to be a crucial factor in driving treatment innovation for rare diseases. It created economic incentives to promote the development of new treatments for rare diseases, which include 7 years of market exclusivity, tax incentives for certain R&D costs, and user fee waivers. (10) Such drugs that are helpful in treating rare diseases are also entitled for “fast track” FDA review that expedites the review process. (11)

    The “off-label promotions” of these drugs lack adequate clinical data, as they are not supported by clinical studies to confirm efficacy and safety. Though FDA provides for a comprehensive review of new pharmaceuticals before they obtain marketing approval, physicians are free to prescribe them for any indication deemed medically fit. This clinical practice of off-label use/prescribing allows physicians to maintain autonomy in the practice of medicine, creating opportunities in which pharmaceuticals can be prescribed for alternative uses, ultimately improving treatment. (12)

    While off-label use has proved to be an important resource for physicians, the recently released FDA memorandum highlights the increased possibility of adverse events from off-label use (owing to lack of enough evidence) further stating that, with off-label use, there is a greater potential for wasted health care costs. (13) Unfortunately, the current system does not incentivize the evaluation of already approved drugs in rigorous clinical trials for rare indications. As a result, most rare disease patients never receive a repurposed treatment that could potentially save their lives (which may be easily available at the nearest drug store) and others receive off-label treatments backed by poor clinical data, which were destined to fail from the start. (14)

    It is believed that the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatments) would fix this serious gap in the drug development setting. (15) The legislation, initially passed in the House in 2015 as a part of the 21st Century Cures Act, was later removed before final passage. It is slated to provide six months of extended exclusivity for any FDA-approved drug repurposed for a new rare disease indication. This legislation could also increase the number of treatments available to rare disease patients at a cost that is lower than the average orphan drug price. However, critics argue that this legislation might offer too generous returns against the costs incurred. On the contrary, the OPEN ACT is expected to change this dynamic by repurposing the clinical studies with the help of experts who know the drug and can fund the expensive work. Post approval, these repurposed orphan therapies could present a lower-cost alternative priced for the larger markets, unlike specialized drugs specifically developed only for a rare disease, which often involve high costs and more resources.

    Today, patients with rare diseases have pinned their hopes on these upcoming legislations in order to gain access to the best available treatments for their prevailing conditions. We believe that employing the appropriate measures, which will facilitate rigorous and robust research in off-label drugs, could transform the lives of millions of rare disease patients.

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    References

    1. Rare Disease Day.
    2. About Rare Diseases.
    3. Pariser A, et al. “Rare Diseases and Orphan Drugs.” Pediatric Drug Development: Concepts and Applications. Edited by Mulberg, A, et al. 2013. 13:130-148.
    4. A decade of innovation in rare diseases. 2005-2015.
    5. Woodcock J. Many “Firsts” for CDER’s 2017 Drug Approvals Reflect Innovation and Enhanced Patient Care. FDA Voice. January, 2018.
    6. Understanding Unapproved Use of Approved Drugs “Off Label”. FDA- For patients.
    7. Dresser R, et al. Off-Label Prescribing: A Call for Heightened Professional and Government Oversight. J Law Med Ethics 2009; 37(3):476–396.
    8. Hampton T. Experts Weigh in on Promotion, Prescription of Off-Label Drugs. JAMA 2007; 297(7):683–684.
    9. Orphan Drug Act. U.S. Food and Drug Administration; Public Law: 97-414. January, 1983.
    10. U.S. Food and Drug Administration. “Orphan Drug Act, Relevant Excerpts.”
    11. U.S. Food and Drug Administration. “Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics.” May 2014.
    12. Mackey T, et al. Off-label promotion reform: A legislative proposal addressing vulnerable patient drug access and limiting inappropriate pharmaceutical marketing. University of Michigan Journal of Law Reform 2011; 45(1):1-54.
    13. U.S. Food and Drug Administration Memorandum: Public Health Interests and First Amendment Considerations Related to Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products. January, 2017.
    14. Bronstein M, et al. For Rare Disease Patients, A Pathway To Hundreds Of New Therapies. Health Affairs. March, 2017.
    15. H.R.971 – Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2015. 114th Congress (2015-2016).

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  • Is India Gearing up for Patient Centric Healthcare Model?

    Is India Gearing up for Patient Centric Healthcare Model?

    Following the recent announcement by PM Narendra Modi hinting at a legal framework for doctors to prescribe generic medicines cheaper than equivalent branded generic drugs, doctors appeared to welcome the move and opined that it will benefit patients as the life-saving drugs could be obtained at much cheaper rates. (1,2)

    In reality, India being the largest supplier of generic medicines globally (20% of global export volume); the quality of the generics should not be an issue. Generic drugs dominate India’s pharmaceutical space, wherein generics account for about 70% of the market. Also, India accounts for approximately 30% (by volume) and about 10% (value) in the US$ 70-80 billion US generics market. Therefore, quality of the manufacturing has got nothing to do with branding. It is purely a manufacturer’s decision and ability enforced by law which is paid for by the customer. (3)

    However, PM Modi did attract the wrath of many following the announcement. This is because a lot of doctors questioned the accessibility and quality of the generic drugs. However, many feel this to be just an excuse, as doctors fear that this move would upset their nexus with the drug companies. (4,5) Furthermore, industry associations reaching out to the health ministry, hoping to approach the PMO to prevent the law from being passed, were opposing proposal on the grounds that the onus of decision-making (what patients should consume) would shift from doctors to chemists. This group further pointed out that the chemists are unregulated and have no ethical or commercial obligation on what they sell patients. However, the Ministry of Health and Family Welfare announced to seek only qualified candidates in the workforce. (6)

    Consequently, the health ministry has made bio-equivalence (BE) studies mandatory for all drug manufacturers before launching any generics in the market. This is primarily to guarantee the same quality and efficacy of generics as their branded counterparts. The health ministry has also issued a gazette notification to this effect. (7)

    Frankly, this move is worth applauding for, since it will ensure the quality as well as appropriate distribution of the generic drugs. This is because most of the doctors have been avoiding generic prescriptions in the name of lack of quality; while actually focusing on the incentives from the pharma marketing.iv On the other hand, in reality, prescribing generics would only benefit the patient in terms of both quality and affordability. But going by certain decisions the government has made since last couple of years, we can surely say that India has started accepting the patient-centric healthcare model.

    Big pharma players and even the smaller (generic manufacturers) ones need to think of ways to rebrand themselves. Certainly, not every company can manufacture a drug for every possible disease condition; however, the future of pharma business is predicted to go by branding based on robust ’therapy area’ leadership. For instance, Mylan Pharmaceuticals, the biggest generic drug manufacturers globally, has been manufacturing more Parkinson’s medicines than any other company in the world. (8) Furthermore, small players will have to invest money in proper and ethical ways of marketing, i.e. publishing results from BA/BE studies to prove the similar efficacy of generics with reference to their branded counterparts. (9)

    It has always been a patient-centric approach. The solution to all these complications is ‘patient empowerment’. Doctors primarily make diagnosis; they cannot polarize patients to earn their own incentives from the pharma players. Also, learned chemists/pharmacists should hopefully provide patients with cheaper generics. In the end, it is patients who must choose what is right for them.

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    References

    1. Chatterji S. Modi says govt will bring law to push doctors to prescribe generic medicines. Hindustan Times. April, 2017.
    2. PTI. PM Narendra Modi Hints At Rules For Doctors To Prescribe Generic Drugs. NDTV India.  April, 2017.
    3. Pharmaceutical Exports From India. India Brand Equity Foundation (IBEF). October, 2017.
    4. Pardasani S. Health for all: Not just Prime Minister’s call. Mumbai Mirror. April, 2017. Available at:
    5. Kaul R. Doctors welcome move to prescribe generic drugs, say accessibility may be an issue. Hindustan Times. April, 2017.
    6. Rajagopal D. `Chemists, and Not Doctors, Could Decide Brand of Drug Use’. The Economic Times. April, 2017.
    7. Thacker T. Health ministry warns on rushed implementation of generics plan. livemint. August, 2017.
    8. Mylan Receives FDA Approval for Generic Version of Parkinson’s Treatment Parcopa® ODT.
    9. Phukan RS. Generic drugs in India: More awareness required. November, 2014.

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