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According to the USFDA, biologic products are the fastest growing class of therapeutic products in the United States and make up an increasing and substantial share of health care costs.³¹ In the past five years, there has been an increase in drug approvals and spending on new drugs globally. The types of drugs being approved are also changing. New drug approvals continue to trend toward biologic, orphan, and oncology products³²:

 Oncology will make up 30% of new drug approvals.

 Orphan drugs could represent 45% of new drug approvals.

Orphan disease is a term used to describe rare diseases. The definitions vary by country, with some using prevalence rates and others basing the definition on the number of affected individuals. The United States defines a disease as rare when it affects fewer than 200 000 individuals. Europe defines a rare disease as one that affects fewer than 5 individuals per 10 000, whereas Taiwan's definition is fewer than 1 in 10 000. Brazil is in line with the World Health Organization's definition where a disease is considered rare when it affects less than 65 per 100 000 individuals. There are an estimated 5000–8000 rare diseases identified globally, and while individually they are rare, collectively they may impact 6–8% of the population.³³

As the number of orphan drugs being approved continues to increase, and the use of biomarkers and precision medicine‐driven principles continue to gain traction, it is expected that the number of patients treated per new drug will go down, and the price per treatment will go up. Theoretically, a drug that costs $1 million per treatment and treats one person with a rare disease would generate the same return as a drug that costs $1 and treats one million people with a more common disease. There are complex variables associated with either treatment example above, such as costs related to R&D, manufacturing, marketing, regulatory frameworks, and clinical care and monitoring. All these variables impact how much effort and money is spent on R&D by pharmaceutical companies, which is similar to the issues noted earlier in this chapter with regards to antibiotic R&D. There is an unmet need for treatment of orphan diseases as well as for more effective cancer treatments. Regulatory agencies around the world have taken steps to address development and patient access to rare disease medicines. National plans can encompass funding for orphan disease drug access, research incentives, diagnosis programs, care coordination, and early access programs.³³

Access to life‐saving medications brings immeasurable benefit to patients; however, it comes at a significant cost for payers. Across the world, some of the recently approved biologic drugs, including gene therapies, have placed immense strains on payment systems and put into question their long‐term sustainability especially considering the pharmaceutical pipeline that lays ahead. Public and private organizations around the world are working with pharmaceutical companies, insurers, governments, and patient advocacy groups in attempts to better align financial expenditures with clinical benefit. Several organizations perform health technology assessments (HTA) that evaluate new drugs in a systematic fashion to assess clinical safety and efficacy as well as cost‐effectiveness. It is expected that independent review of drug pricing by groups like the Institute for Clinical and Economic Review (ICER) may be able to place downward pressure on drug go‐to‐market prices in the coming years. Outcomes‐based contracts that tie reimbursement for a given pharmaceutical to meeting certain clinical metrics are also expected to become more prevalent. It is predicted that by 2022, 30 out of the 50 top drugs will have some form of outcomes‐based contracts in place between pharmaceutical manufacturers and payers.³²

Biosimilars, which are similar versions of innovator biologic drugs, are expected to generate cost‐savings compared to the innovator/reference brand that they compete with. The adoption of biosimilars in the United States has been relatively slow compared to other developed markets like the European Union. It is expected that the adoption of biosimilars in Europe will continue to outpace the United States for the upcoming decade; however, major events are on the horizon in the United States including the introduction of the first biosimilars to Humira^®, currently the world's top‐selling drug.³² A concerted effort by countries and payers across the globe to educate prescribers and patients on the safety and efficacy of biosimilars will be required to see similar type of savings that small molecule generics generate today.