A New Commitment to Patients

The legendary physician founder of Johns Hopkins, Dr. William Osler, once famously said:

“Just listen to your patient; he is telling you the diagnosis.” 

Many leaders of the nation’s biopharmaceutical industry are listening to America’s patients, to their families and to their caregivers, who have told us that they find our medicines too expensive, and that they have lost trust in our industry. 

We need to regain this lost trust through bold actions that put patients first. We cannot wait on policymakers in Washington who are stuck in partisan gridlock. We as industry leaders need to acknowledge that we have a moral obligation to ensure that our medicines are fairly priced and accessible to all in need.   

We need to acknowledge that what we do is truly special. We provide the medicines that doctors and nurses use to help heal the sick. As they have taken an oath, so too must we. Our products are not luxuries or “nice to haves.” They are the preventions, treatments and cures that extend and enhance human lives.

That is why earlier this month, I joined with more than 200 biotechnology CEOs and senior executives, life science investors, and leading researchers in pledging our New Biotechnology and Pharmaceutical Industry Commitment to Patients and the Public.

So many companies in our industry do act in the best interests of patients and do put patients at the core of their business. Many business practices of the biopharmaceutical industry, though, are simply unacceptable when the public health is at stake. Many practices do not put patients first. This must change - and we must be the agents of that change.

The signatories of this New Commitment are holding ourselves accountable for ensuring patient access to our products and meeting the highest ethical standards and business practices. We recognize that we have a moral imperative to lead the biopharmaceutical industry towards more responsible business practices; and again, a moral obligation to ensure that our medicines reach every person who can benefit from them.   

As the parent of two children with a rare, life-threatening neuromuscular disorder, I know that anyone, at any point in their lives, can be impacted by a life-changing diagnosis for themselves or a loved one. Nature is not cruel, just brutally random.

When disease or sickness strike, worrying about how to afford that medicine should be the least of patients’ or caregivers’ concerns. High out-of-pocket costs for even highly effective medicines have led some patients with serious chronic illnesses to ration or forgo life-saving care. Many patients, such as those with cancers or rare diseases, have enough “skin in the game” - they should not have the added burden of paying for high co-pays.

Every signatory to this New Patient Commitment pledges to pricing our medicines at launch to reflect innovation, a genuine commitment to achieve broad access for patients, and ensuring that price increases are sustainable and guided by the need for uninterrupted patient access.

We also commit ourselves to work with all public and private insurers to find ways to limit or eliminate co-pays and deductibles and to supporting robust market competition through the approval of safe and effective generic and biosimilar medicines after our legitimate patent and regulatory protections expire. Pharmaceutical company actions that pay generic companies to “delay” entry of generic competition have no place in a system where true competition must be fierce and fair.

Signatories to this New Patient Commitment also pledge to support only ethical business practices that drive innovation, competition and patient choice. We also promise to connect directly with patients and their families, supporting patient advocacy as a key business practice in how we build and operate our companies.

Finally, we will not tolerate companies and other stakeholders who abuse this commitment to patients, or who abuse policies aimed at fairly rewarding innovation in pursuit of short-term financial gain. We will call out bad actors and bad practices.

The principles of this New Patient Commitment will expand and preserve access to effective therapies and restore public trust in market-based medical innovation. It will foster a more vibrant and sustainable business model, as well.

At Amicus Therapeutics, the rare disease focused biotechnology company that I helped to found and that I have the privilege to lead, these principles already guide our work every day.

When we founded Amicus, we chose the name “Amicus” because it is the Latin word for “Friend”. We wanted Amicus to be the among the most patient focused companies in this industry. Every day I ask each of our 600 global employees to think: “If you had this disease, or you were the parent of a child with this disease, how would you make your business decisions?” It’s a patient-first mindset that leads to business actions that always put patients first. 

At Amicus, we price our medicines to be at or below competitive FDA-approved products that are already on market – encouraging payors to grant rapid coverage and to ensure broad access.

Importantly, at Amicus we committed with the launch of our first FDA-approved product in 2018 to limiting annual price increases for all of our medicines to the Consumer Price Index (CPI). This is an extraordinary commitment to patients and to the healthcare system. It has in many cases accelerated insurance approvals for patients because it gives payers the ability to budget more predictably and reduces barriers to patient access.

We’ve also committed to reinvest a share of the revenue from each of our FDA-approved products back into R&D into new treatments for those exact same diseases until the day that the disease is not just treated, but cured. 

To maintain and expand our extraordinary patient dedication, we’ve also created the position of Chief Patient Advocate, a C-Suite executive who ensures 100% patient focus, who is “the conscience” of the company and who sits on my executive team and reports directly to me as CEO. 

These are but a few examples at Amicus of how we have tried to live and to practice what we say. And we still have a long way to go.

With this “New Commitment to Patients” we as industry leaders have taken a bold step. Hold us accountable ahead.