Ensuring Access to Innovative Medicines: Big Pharma’s Role

Today’s healthcare systems, in both developed and developing countries, face serious challenges in managing health care spend. Moreover, the pressure will continue to increase over the next few years as we deal with dramatic shifts in health care issues resulting from the current epidemiological transition. Unfortunately, the poorest countries will carry the highest burden in dealing with communicable diseases, non-communicable diseases, along with the risk of potential new diseases. 

The good news is that in recent decades, novel medicines have not only improved survival rates and quality of life for many patients around the world; they have also changed the history of diseases such as HIV and certain cancers. Anti-retroviral therapies have transformed HIV from a terminal illness to a manageable chronic disease, while the once-daily single-tablet regimen has simplified the daily lives of patients. In the last 15 years, the five-year survival rate for patients with chronic myeloid leukemia has improved from less than 20%  percent to more than 90%,  percent, thanks to the advent of a class of drugs known as tyrosine kinase inhibitors (TKIs). With direct direct-acting anti-virals (DAAs), Hepatitis C, is now curable in more than 90% of treated patients with as little as 8-12eight weeks of treatment. Moreover, think about what vaccines and antibiotics have done to improve health in general.

Nevertheless, access to innovative medicines has been a challenge for one-third of the worldwide population, especially in under-developed countries, and increasingly in middle-income countries. Several new approaches based on collaborations, intellectual property, and pricing have helped stimulate innovation, promote healthcare delivery, and reduce global health disparities. It’s clear no one single approach suffices, and to have medicines more available, stakeholders need to engage further in partnerships promoting knowledge and technology transfer in assuring essential medicines be manufactured, approved, and distributed, in an effort of to making them accessible.

Notwithstanding, the pharmaceutical industry has been questioned significantly regarding the high costs of treatments. The truth is the cost of treatment is more than just the expenses of a medication.  Of course, affordability and pricing are critical but part of the discussion is also evaluating the overall burden of research and treating a disease. We should explore the entire system from the slowdown in medicine cost growth to the rising cost of chronic disease to insurance coverage of medicine to the role of intermediaries, and finding realistic solutions on what we can all do to make medications more affordable. 

Medicines account for a stable share of health care spending and we should not lose sight that they help patients avoid expensive hospital services. Prescription medicines are often the most cost-effective means of disease prevention and treatment. Medicines can actually reduce the projected impact of diseases and avoid billions in health care costs when you increase adherence to treatments, control or delay the onset of a disease, reduce symptoms, and so much more. In the midst of all the progress in treating debilitating diseases, the share of spending remains the same as it was 50 years ago. To put spending on medicines in perspective, the growth in other health care services will be five times total the medicine spending growth through the next decade.

Now, developing new treatments and cures is a complex, costly, lengthy and risky undertaking. The expected market rewards are sometimes insufficient to incentivize the development of some urgently needed products. Today, the successful development of a novel drug can take an average of 10 to 15 years with an estimated investment of USD$1.2 to US$1.5 billion +/-. As our understanding of science grows, so does the complexity of developing new medicines, particularly as treatments are increasingly tailored to the unique needs of individual patients. Setbacks are an inherent part of this complex process and the odds of success are low. For example, did you know that just a 12 percent of drugs entering clinical trials ever make it to patients? Moreover, thousands will not even go past the early discovery and pre-clinical testing stages. The productivity of pharmaceutical R&D, measured as the number of assets developed, has declined as it has in other research-intensive industries, partly because “ideas are harder to find”. 
So, what can be done to make the system work better for all and at the end, speed up access? 

There is growing consensus that existing policies need to be rethought and new approaches need to be found to strike the delicate balance between stimulating, promoting and rewarding true innovation in access. This is true particularly towards addressing unmet needs, and ensuring both financial sustainability for health systems and accessibility for patients.

1)Robust IP framework

A strong global IP framework is essential to guarantee rights to the state-of-the-art developed, while maintaining control over how creations, protected by IP rights (IPRs), are used. As such, IPRs have a facilitating role for improved healthcare and should not be perceived as a hindrance. In pharma, patents are considered as the most important IP protection tools, providing the owner exclusive rights to prevent use of the patented product or process without the consent of the owner for 20 years, in a particular territory, which will in turn ensure fair competition and innovation in a safe environment. Data protection and market exclusivity rights are IPRs granted to the market authorization (MA) holder for a period of eight to 10 years, respectively, after MA. Generic or biosimilar products are not permitted to enter the market as long as such IPRs are in force. In many cases, at the time of a new medication being introduced, at least half of the patent term may have expired. Patents should not be the focus of the debate on access to medicines. Although they provide desirable incentives and are a precondition for successful research and development of pharmaceutical products, they are not necessarily the sole (or even key) reason for lack of access to essential medicines. Factors other than patents have been identified as barriers to accessing essential medicines. Barriers to access include regulatory approval for the use of drugs (including non-price -related patent barriers), high out-of-pocket payments (especially for catastrophic illnesses), poor medicine supply & and distribution systems and insufficient health facilities. Similarly, absence of patent protection does not guarantee the availability or acceptability of generic medicines in low- and middle-income countries. Improving access to pharmaceutical products for patients requires an appropriate mix of public and private research, policy and regulatory initiatives. 

2)Partnerships 

Stakeholders bundle forces in assuring essential medicines are manufactured, authorized and distributed at affordable conditions. However, challenges remain, such as guaranteeing high distribution coverage, ensuring affordability, and adoption of essential medicines, both at the provider and end-user levels. Furthermore, developing countries lack adequate infrastructure needed to increase access to medicines. 
Public-private partnerships (PPPs), and product development partnerships (PDPs), in particular, are vehicles suitable for delivering treatment and strengthening healthcare systems. Such multi-stakeholder efforts are able to ensure product registration, increase local production and distribution capacity, and ensure governance for global health. PPPs leverage knowledge and technology transfer of new medical technologies whereas PDPs strengthen research capacity by building infrastructures at trial sites, providing equipment and setting up training in good clinical practice and dedicated disease-specific research platforms. 

3)Pricing Models 

An effective way of ensuring patients in countries at different levels of development can access innovative treatments is to apply differential (or tiered) pricing. Under this paradigm, countries that are more affluent pay higher prices than poorer countries.  At the same time, tiered pricing can increase consumer’s welfare by creating access to medicines.

4)Regulatory harmonization 

Harmonizing regulatory requirements would encourage mutual recognition. This could potentially reduce the number and costs of clinical trials and thus overall costs. Of course, the challenge lies in gaining agreement among global agencies and appropriate methods and outcomes measures. 

5)Transparent criteria for determining willingness to pay

The development and application by public payers of transparent criteria for determining willingness to pay for added health benefits could enable developers to know in advance what level of predictability they might expect and give some certainty to the risk investment they would be embarking on, hence accelerating market access for medicines with significant potential benefit. They could also increase the returns from current spending, better align spending with public priorities, improve the bargaining power of national authorities and payers, and provide greater predictability of decisions to the industry. 

At Novartis, for example, we recognize it is not enough just to introduce transformative therapies; we must get them to patients who need them as fast as possible, especially underserved populations who are in dire need. Therefore, it is imperative we share responsibility among all key stakeholders. In response, we have evolved our business model from transactional (that of only selling medicines) to a patient-centric model, in which we offer value-based solutions for the end-to-end patient experience.  Our commitment with patients, caregivers, health care professionals, payers and health care systems is to become the partner of choice that drives collaborative relationships to improve outcomes for patients and, as a consequence, be a trusted cohort in changing the practice of medicine. 

Examples of what Novartis does and will continue doing to help expand access to our drugs under this new model include:

• Patient access programs that focus on ensuring availability and affordability of medicines – depending on the ability to pay – are donated. Novartis program examples are the Glivec International Patient Assistance Program (GIPAP) / Novartis Oncology Access (NOA) / Novartis Access Program offering a portfolio of 15 meds available to governments and institutional payers at a price of USD US$1 per treatment per month. 
• Clinical Trials are a way of expanding early access and reducing costs to patients and systems.
• Value-based models such as risk-sharing (outcomes-based, testing, diagnosis & and tracking, sub-populations, etc.), patient support programs.
• Managed Access Programs, which give access to patients of an unapproved product during the development phase, through a requirement of an institution or physician. 
• Patient Organizations Support that offer disease awareness, improving adherence, diagnosis and monitoring as well as education and other support.

By collaborating with all key stakeholders at different levels and intensities (academia, medical communities, government health care systems, charities, patients and other payers), Novartis can extend affordable access to more of our medicines for a broader number of patients. This partnership-based approach allows for local adaptation of different models based on each country’s specific needs.  

Access strategies should be based on three principles: 1) addressing the needs of underserved populations through R&D, 2) further improving the affordability of drugs, and 3) strengthening health systems with quality care. Quality care means getting the right medicine, in the right dose, at an affordable price, for all the people who need it. 

Accessible, affordable, and properly used medicines save lives. What is needed is an overall vision with greater policy coherence and backed by strong political commitment and transparency.

DISCLAIMER: Thoughts, and opinions expressed in the text above belong solely to the author, and not necessarily to the author's employer, organization, committee or other group or individual.