Expert Perspectives: Biopharma CEOs and Industry Leaders Sign a New Commitment to Patients

On January 8, 2020, a group of 215 Biopharma CEOs and Industry Leaders published a co-signed “New Commitment” to center patient needs and to conduct ethical pharmaceutical business. Through this document, the signors pledged to ensure access to their treatments through responsible pricing, investing in health policy discussions, and encouraging commitment to ethical business practices within their own organizations, as well as the broader industry.

Not sure what to make of it? Our experts weigh in.

Rena Conti, PhD
Associate Research Director of
Biopharma & Public Policy
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We have arrived at a special moment in health care: Innovative, life-changing gene and stem-cell therapies are here that will cure or ease debilitating diseases. Particularly exciting are new medicines designed to treat the most vulnerable patients, children with rare and otherwise dreaded disease. Yet, these medicines are entering the American market – a market that entails both significant incentives for pharmaceutical companies to price excessively and too many patients without adequate financial resources to access needed care.

This announcement by some of the leading biotechnology firms engaged in the most cutting edge therapy development to treat rare disease and their investors is a very welcome step to restore the public’s trust. The signers’ commitment to insure access to patients standing to benefit from treatment above all else is laudable.

I am particularly excited to see the signers’ endorsement of the use of innovative payment models to insure equitable access to clinically valuable therapy.  What is critical here is to ensure these models are tried out among payers covering children and young adults’ access to these new therapies, including state Medicaid programs and commercial insurers. These payers face annual budget constraints and may be particularly cash strapped.

Here, the ‘subscription model’ – a guarantee of coverage and access for all eligible for treatment for a defined period of time in exchange for a lower unit price pioneered by the state of Louisiana and Gilead Pharmaceuticals to ensure access to new hepatitis C treatments in 2019 – might be particularly applicable for medicines offering cures. For gene and stem cell therapies with less certain benefits, outcomes-based payment models might be a good approach to pilot.

I hope to see these commitments made by the signatories of the announcement backed with tangible steps soon.  I also hope they are met with insurance plans and policymakers willing to think creatively about how best to ensure affordable and equitable access to clinically meaningful gene and stem cell therapy.

Anand Devaiah, MD, FACS
Biomedical & Health Technology
Development & Transfer Domain Director
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I applaud the 215 CEOs and industry leaders who have authored and signed this commitment to patients and the public. As someone whose roles include innovation, investment, and policy, I see this as a very positive step. As someone who also has an active surgical and medical practice, on the “frontline” caring for patients, this is an especially important document.

For context, it is important to recognize the contribution of small and medium sized biotechnology companies.  These companies – working alone or in conjunction with other companies – accounted for 73% of all Phase I, II, III drug trials and new drug applications in 2019. Based on 2017 data worldwide sourced from Bio Industry Analysis, small biotechnology companies were responsible for 70% of all drug trials (43% in conjunction with a partnering company).

These small and medium sized companies therefore embrace significant risk. Further compounding this risk is the fact that they typically depend on non-public investments, which adds both opportunity and complexity to their work. There is a careful balance between being responsible innovators for scientific discovery, forwarding human health, remaining a viable organization, and satisfying the expectations of those who have put their trust in the company in one form or another. This trust comes from many sources including patients, subjects, providers, researchers, investors, and other partners who support the work being done and the health outcomes being sought.

Wearing the hat of a provider, and as one who often deals with rare and unusual diseases, I find their statement especially important. It reflects how these biotechnology companies want to be proactive partners in healthcare, recognizing that the healthcare system is intricately interwoven fabric; one strand secures another, and broken ones can lead to unraveling. At the patient and provider level, it is important to know that these companies who have signed on are willing to address the issues facing biotechnology development and deployment, and ultimately touch the lives of the people who need their work the most.

Jonathan Woodson, MD
IHSIP Director

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The “New Commitment” is long overdue and should signal a new era of corporate social responsibility. Pharma is unlike other industries and markets in that its products are essential to the health, well-being and survival of people who require these products despite their ability to pay. While the industry needs to be profitable to survive and provide new therapeutics, excessive pricing compromises the health and financial well-being of the population.

How this new contract will support emerging proposals for health care payment reform is unclear, but it at least signals a willingness by the pharma industry to work with policy-makers and payers to address the affordability issues related to drug prices. A more cynical view maybe that pharma is trying to get out ahead of the growing sentiment that Congress adopt legislation to control drug prices–a legislative priority for 2020 for several health policy advocates and law-makers. There is a lot of work needed on this issue. We will follow with interest how this new recognition of ethical responsibility translates to new business practices for pharmaceutical companies.

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