Developing and Submitting 
Proposed Draft Guidance 
Relating to Patient 
Experience Data 
Guidance for Industry and Other 
Stakeholders 
 
 
 
DRAFT GUIDANCE 
 
  This guidance document is being distributed for comment purposes only. 
 
Comments and suggestions regarding this draft document should be submitted within 90 days of 
publication in the Federal Register of the notice announcing the availability of the draft 
guidance. Submit electronic comments to https://www.regulations.gov.  Submit written 
comments to the Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 
Fishers Lane, Room 1061, Rockville, MD 20852. All comments should be identified with the 
docket number listed in the notice of availability that publishes in the Federal Register. 
 
For questions regarding this draft document, contact (CDER) Meghana Chalasani at 240-402-
6525 or (CBER) Office of Communication, Outreach and Development, 800-835-4709 or 240-
402-8010.  
 
 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Drug Evaluation and Research (CDER) 
Center for Biologics Evaluation and Research (CBER) 
 
December 2018 
Procedural 
 
   
Developing and Submitting 
Proposed Draft Guidance 
Relating to Patient 
Experience Data 
Guidance for Industry and Other 
Stakeholders 
 
 
Additional copies are available from: 
Office of Communications, Division of Drug Information  
Center for Drug Evaluation and Research 
Food and Drug Administration 
10001 New Hampshire Ave., Hillandale Bldg., 4th Floor  
Silver Spring, MD 20993-0002  
Phone: 855-543-3784 or 301-796-3400; Fax: 301-431-6353 
Email: druginfo@fda.hhs.gov  
https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm 
 
and/or 
 
Office of Communication, Outreach and Development  
Center for Biologics Evaluation and Research 
 Food and Drug Administration 
10903 New Hampshire Ave., Bldg. 71, Room 3128 
Silver Spring, MD 20993-0002 
Phone: 800-835-4709 or 240-402-8010 
Email: ocod@fda.hhs.gov 
https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm  
 
 
 
U.S. Department of Health and Human Services 
Food and Drug Administration 
Center for Drug Evaluation and Research (CDER) 
Center for Biologics Evaluation and Research (CBER) 
 
December 2018 
Procedural 
  Contains Nonbinding Recommendations 
Draft  Not for Implementation 
 
 
TABLE OF CONTENTS 
I.  INTRODUCTION............................................................................................................. 1 
II.  BACKGROUND ............................................................................................................... 1 
III.  QUESTIONS AND ANSWERS ....................................................................................... 4 
A.  Development and Submission of Proposed Draft Guidance Relating to Patient Experience 
Data ........................................................................................................................................... 4 
1.  What factors should stakeholders consider when planning and determining whether to develop a 
proposed draft guidance relating to patient experience data to submit to FDA? ........................... 4 
2. How can stakeholders communicate with FDA that they plan to develop a proposed draft 
guidance relating to patient experience data? ................................................................................. 5 
3. What are some general considerations regarding the format of a proposed draft guidance? ........ 6 
4. How should stakeholders submit proposed draft guidance to FDA? .............................................. 7 
5. What will happen after stakeholders submit the proposed draft guidance to FDA? ....................... 7 
6. Can a stakeholder submit proposed revisions to an existing FDA guidance?................................. 8 
7. What are other ways for a stakeholder to participate and provide input on FDA policy and 
guidance development? .................................................................................................................... 8 
B.  Other Opportunities for Stakeholders ......................................................................................... 8 
1.  What are other opportunities for stakeholders to help advance patient-focused drug 
development? ................................................................................................................................... 8 
2. How can patient experience data inform medical product development and regulatory decision 
making? .......................................................................................................................................... 10 
3.    What work products relating to patient experience data may be developed by stakeholders? ... 12 
4.    How can stakeholders submit work products relating to patient experience data? .................... 12 
 
  Contains Nonbinding Recommendations 
Draft  Not for Implementation 
1  Developing and Submitting Proposed Draft Guidance Relating to 
2  Patient Experience Data 
3  Guidance for Industry and Other Stakeholders1 
4   
5   
6  This draft guidance, when finalized, will represent the current thinking of the Food and Drug 
7  Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not 
8  binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the 
9  applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible 
10  for this guidance as listed on the title page. 
11   
12   
13   
14  I.  INTRODUCTION  
15   
16  This guidance provides information on how a person can submit a proposed draft guidance 
17  relating to patient experience data for consideration by FDA. This guidance is intended to assist 
18  stakeholders seeking to develop and submit such proposed draft guidance to the Agency.  In 
19  addition, FDA recognizes that stakeholders may have other information on patient experience 
20  data that they would like to share with FDA outside of the guidance process and thus provides 
21  information on other ways stakeholders can advance drug development by sharing patient 
22  experience data. Section 3002(c)(5) of the 21st Century Cures Act (Cures Act) directs FDA to 
23  issue guidance on how a person seeking to develop and submit a proposed draft guidance 
24  relating to patient experience data for consideration by FDA may submit such proposed draft 
25  guidance to the Agency.2  
26   
27  In general, FDAs guidance documents do not establish legally enforceable responsibilities. 
28  Instead, guidances describe the Agencys current thinking on a topic and should be viewed only 
29  as recommendations, unless specific regulatory or statutory requirements are cited. The use of 
30  the word should in Agency guidances means that something is suggested or recommended, but 
31  not required.  
32   
33   
34  II.  BACKGROUND 
35   
36  Under Section 569C(c) of the Federal Food, Drug, and Cosmetic Act (as amended by the Cures 
37  Act), patient experience data includes data that (1) are collected by any persons (including 
38  patients, family members, and caregivers of patients, patient advocacy organizations, disease 
39  research foundations, researchers, and drug manufacturers); and (2) are intended to provide 
40  information about patients experiences with a disease or condition, including (A) the impact 
41  (including physical and psychosocial impacts) of such disease or condition, or a related therapy 
                                                 
1 This guidance has been prepared by the Office of the Center Director and the Office of Regulatory Policy in the 
Center for Drug Evaluation and Research (CDER), in cooperation with the Center for Biologics Evaluation and 
Research (CBER), at the Food and Drug Administration.  
2 See 21st Century Cures Act:  https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf. 
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42  or clinical investigation on patients lives; and (B) patient preferences with respect to treatment 
43  of such disease or condition. 
44   
45  Patient experience data can capture patients experiences, perspectives, needs, and priorities 
46  related to (but not limited to):  
47   
48    the symptoms of their condition and its natural history;  
49    the impact of the condition on their functioning and quality of life;  
50    their experience with treatments;  
51    input on which outcomes are important to them;  
52    patient preferences for outcomes and treatments; and  
53    the relative importance of any issue as defined by patients.3 
54    
55  Throughout the medical product lifecycle, patient perspectives may be valuable in addressing 
56  specific topics and questions. For instance, patient input on the therapeutic context (e.g., 
57  debilitating or most bothersome symptoms, disease impacts that matter most to patients, how 
58  well current treatment options help manage the condition) can be helpful at any stage of 
59  development.  
60   
61  Patient perspectives on clinical endpoints in clinical trials and clinical outcomes can inform 
62  stages of development from discovery through premarket review. This includes discussions on 
63  how well the most commonly studied endpoints in clinical trials align with outcomes or aspects 
64  of disease that matter most to patients, or whether patients chief complaints about their 
65  condition are captured in clinical trials. During the design and conduct of a clinical trial, patient 
66  input can be gathered on: how patients excluded from clinical trials can be included in future 
67  trials, difficulties and challenges patients may be facing in participating in clinical trials, and 
68  various measures that may be taken to increase the likelihood of patient enrollment and retention 
69  in a trial.  
70   
71  As medical product development moves into premarket review and then the postmarket setting, it 
72  may be informative to collect patient input on benefits and risks and better understand attitudes 
73  toward or tolerance of potential medical product risks and how therapy side effects vary by 
74  subpopulation. Once a product is on the market, it is then helpful to understand whether the 
75  approved labeling is clearly communicating the information that patients need to safely and 
76  effectively use the product and whether there are challenges to adherence with prescribed 
77  therapies. 
78   
79  Patient experience data should be collected and analyzed in a methodologically sound and fit-
80  for-purpose manner. There are several options for contributing patient experience data to the 
81  medical product development and regulatory decision-making process. One option is for 
82  stakeholders to submit proposed recommendations and considerations informed by patient 
83  experience data in the form of a proposed draft guidance.4 Proposed draft guidance relating to 
                                                 
3 See Patient-Focused Drug Development Glossary: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm610317.htm.  
4 See 21 CFR 10.115(f)(3). 
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84  patient experience data that is developed and submitted by external stakeholders can be helpful 
85  in bringing the patients perspective into medical product development and regulatory decision-
86  making. A stakeholder-submitted proposed draft guidance may be used to inform FDAs 
87  decision-making and future guidance development work, if applicable.  
88   
89  Under FDAs Good Guidance Practices regulations, FDA guidance documents describe the 
90  Agencys interpretation of or policy on a regulatory issue and are prepared for use by FDA staff, 
91  applicants/sponsors, and the public.5 FDA guidance documents do not establish legally 
92  enforceable rights or responsibilities and do not legally bind the public or FDA.6 When issuing a 
93  Level 1 guidance, FDA will typically issue draft guidance, request public comment, and 
94  following review of received comments, finalize the guidance document.7 FDA may also revise 
95  existing guidance if warranted, typically using the same process.  
96   
97  Submitting proposed draft guidance for FDAs consideration is not the only option for 
98  contributing patient experience data. Patients, caregivers, patient and disease advocacy groups, 
99  and other stakeholders with knowledge of or access to the patient community, may be well-
100  positioned to also make broader contributions to advance medical product development.  
101   
102  Recognizing that stakeholders may be interested in pursuing other pathways to contribute patient 
103  experience data, this guidance addresses questions relating to both guidance development and 
104  other potential pathways for contributing patient experience data. The questions addressed in this 
105  guidance are as follows: 
106   
107    Development and submission of proposed draft guidance relating to patient experience data: 
108    What factors should stakeholders consider when planning and determining whether to 
109  develop a proposed draft guidance relating to patient experience data to submit to FDA?  
110    How can stakeholders communicate with FDA that they plan to develop a proposed draft 
111  guidance relating to patient experience data? 
112    What are some general considerations regarding format of the proposed draft guidance? 
113    How should stakeholders submit proposed draft guidance to FDA? 
114    What will happen after stakeholders submit the proposed draft guidance to FDA? 
115    Can a stakeholder submit proposed revisions to an existing FDA guidance? 
116    What are other ways for a stakeholder to participate and provide input on FDA policy and 
117  guidance development? 
118   
119    Other opportunities for stakeholders: 
120    What are other opportunities for stakeholders to help advance patient-focused drug 
121  development? 
122    How can patient experience data inform medical product development and regulatory 
123  decision making? 
                                                 
5 See 21 CFR 10.115(b). 
6 Although guidance documents generally reflect FDAs current thinking, FDA staff may depart from guidance 
documents with appropriate justification and supervisory concurrence. See 21 CFR 10.115(d). 
7 Procedures for issuing and commenting on FDA draft guidance documents may vary depending on the type of 
draft guidance issued. For specific information about FDAs Good Guidance Practices, see 21 CFR 10.115.   
 
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124    What work products relating to patient experience may be developed by stakeholders? 
125    How can stakeholders submit other work products relating to patient experience data? 
126  Resources for stakeholders relating to patient-focused drug development (e.g., decision tools, 
127  flow charts, templates) will be posted on FDAs CDER Patient-Focused Drug Development web 
128  page,8 and updated as new resources are developed. 
129   
130   
131  III.  QUESTIONS AND ANSWERS 
132    
133  A.  Development and Submission of Proposed Draft Guidance Relating to Patient 
134  Experience Data 
135   
136  1.  What factors should stakeholders consider when planning and determining whether to 
137  develop a proposed draft guidance relating to patient experience data to submit to FDA? 
138   
139  Before initiating development of a proposed draft guidance, stakeholders should consider the 
140  following: 
141   
142  a.  Is there a stage in a medical product lifecycle that could be particularly informed 
143  by patient experience data for a given disease area (e.g., in discovery phase, to 
144  identify appropriate unmet needs to suggest potential drug targets; during 
145  development to suggest appropriate disease populations, enrollment criteria for 
146  trials, and important trial endpoints; late stage in development to understand 
147  acceptable benefit-risk balance and treatment burden; and post-approval to 
148  understand acceptable safety)? 
149   
150  b.  Is the development of a proposed draft guidance the best way to address an 
151  identified need in a given disease area? 
152   
153  FDA guidance documents cover a range of topics, including: (1) clinical guidance 
154  that may provide more general treatment of issues related to drug development for 
155  a given disease and with broad discussion of subpopulation issues; and (2) 
156  methodological guidance that may provide general treatment of methodological 
157  issues and cover a range of research trial settings, patient populations, cultural 
158  contexts, and sociodemographic considerations. 
159   
160  It may be appropriate for stakeholders to develop a proposed draft guidance if: 
161   
162    FDA has issued a clinical guidance in a given disease area, but the guidance 
163  does not address considerations and/or examples for a specific subpopulation. 
164  A stakeholder may want to provide subpopulation-specific considerations, 
                                                 
8 See CDER Patient-Focused Drug Development Homepage: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm579400.htm.  
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165  examples, and/or recommendations related to the disease area discussed in the 
166  FDA guidance. 
167   
168    FDA has issued a methodological guidance (e.g., adaptive clinical trial 
169  designs) that is applicable to a range of disease areas and patient populations, 
170  but the guidance does not provide specific considerations or examples of how 
171  the methods can be applied or adapted for a specific disease area and/or 
172  subpopulation. A stakeholder may want to provide disease- or subpopulation-
173  specific considerations, examples, and/or recommendations related to the 
174  methods discussed in the FDA guidance. 
175   
176    FDA has not issued a clinical guidance in a given disease area, but there 
177  appears to be a need for a disease-specific guidance to address topics related 
178  to patient experience data (e.g., clinical outcome assessments, endpoints, 
179  clinical trial considerations). Please note that CDER and CBER each publish 
180  on FDAs website a list of guidance topics that the respective Center is 
181  considering for development during the current calendar year.  
182     
183  c.  Are resources, expertise, and stakeholder capacity available to collect any relevant 
184  patient experience data, conduct required analysis, and further develop a proposed 
185  draft guidance? Would the available resources be more suitable to focus on other 
186  efforts (e.g., those discussed in Section II, Other Opportunities for Stakeholders)? 
187   
188  FDA encourages contacting FDA staff early to discuss patient experience data 
189  that may be useful to collect and submit to the FDA. Please refer to the series of 
190  FDA Patient-Focused Drug Development guidances before collecting patient 
191  experience data.9 
192   
193  d.  If patient experience data has been collected and analyzed, is it suitable to provide 
194  recommendations or considerations to include in a proposed draft guidance? If 
195  not, stakeholders should consider other opportunities to share patient experience 
196  data (see Section II, Other Opportunities for Stakeholders). 
197   
198  2.  How can stakeholders communicate with FDA that they plan to develop a proposed draft 
199  guidance relating to patient experience data? 
200   
201  Stakeholders are not required to communicate with FDA in advance of submitting a proposed 
202  draft guidance. However, when developing proposed content for disease- and indication-specific 
203  recommendations for FDAs consideration, it may be helpful (for FDA awareness) to provide 
204  FDA with a brief summary of the expected content, along with a projected timeframe for 
205  submission and a point of contact for potential follow-up. Ideally, these summaries will be brief 
206  (2-3 pages at maximum) and should be sent only to PED-
                                                 
9  See FDA draft guidance for industry and other stakeholders, Patient-Focused Drug Development: Collecting 
Comprehensive and Representative Input (June 2018). When final, this guidance will represent FDAs current 
thinking on this topic.  For the most recent version of a guidance, check the FDA guidance web page at 
https://www.fda.gov/RegulatoryInformation/Guidances/default.htm.  
  5  Contains Nonbinding Recommendations 
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207  ProposedGuidanceSummary@fda.hhs.gov.  No other documents or inquiries should be sent to 
208  that email address. The email title should reflect that the summary is a Development Summary 
209  for Anticipated Proposed Draft Guidance and indicate the subject matter (e.g., disease area) for 
210  the anticipated proposed draft guidance. After receipt by the Agency, the summaries will be 
211  directed internally to appropriate points of contact for awareness and consideration. While FDA 
212  does not intend to issue acknowledgement letters or other communications in response to these 
213  summaries as a matter of course, the Agency may reach out to points of contact identified should 
214  the Agency believe follow-up would be useful.   
215   
216  3.  What are some general considerations regarding the format of a proposed draft guidance? 
217   
218  A proposed draft guidance should be formatted so that it provides clear and concise 
219  recommendations for FDA to consider should FDA develop a guidance on the topic, and should 
220  not give the impression that it is an FDA-drafted document.  A proposed draft guidance should: 
221   
222  a.  Address one or two topics relevant to a specific disease area drug development 
223  plan. 
224   
225  b.  Be written from the stakeholders perspective, not as FDA. For example, language 
226  such as FDA suggests, FDA encourages, and FDA recommends should not 
227  be used. Instead, FDA recommends language such as The available patient 
228  experience data suggests that patients seek X or We recommend that sponsors 
229  consider. 
230    
231  c.  Include a unique title page. The proposed draft guidance should not follow FDAs 
232  visual identity program or mirror the formatting style of FDA guidance 
233  documents. 
234   
235  d.  Refrain from making any statement that indicates, implies, or suggests that FDA 
236  has endorsed the document, including by use of the FDA logo. 
237   
238  e.  Be succinct and generally no more than 5 pages. 
239   
240  f.  Include an introduction clearly stating the purpose of the proposed draft guidance; 
241  provide high-level background that includes a brief description of the disease, 
242  patient population, severity of the condition, available treatment options, and 
243  topics or issues to be addressed in the document; and propose considerations and 
244  recommendations on the topics or issues relevant to the purpose of the proposed 
245  draft guidance. Please refer to examples of FDAs short disease-specific bulleted 
246  guidance documents (e.g., (1) Inborn Errors of Metabolism That Use Dietary 
247  Management: Considerations for Optimizing and Standardizing Diet in Clinical 
248  Trials for Drug Product Development, (2) Hypertension: Conducting Studies of 
249  Drugs to Treat Patients on a Background of Multiple Antihypertensive Drugs, and 
250  (3) Pediatric HIV Infection: Drug Development for Treatment) and consider a 
251  similar format. 
252    
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253  g.  If applicable, include specific examples. Examples could be related to a specified 
254  subpopulation or special issues defined by factors such as patient age, disease 
255  severity, co-morbidities, or other concerns. They might also relate to how specific 
256  methods recommended in existing FDA guidance would or would not be applied 
257  in specific trial settings for specified patient subpopulations, including variations 
258  in economic and cultural context, language ability, literacy, numeracy, mobility, 
259  or age group. Stakeholders may also propose examples of alternative methods that 
260  may be most applicable. 
261     
262  h.  Include a study report and protocol when submitting methodologically collected 
263  patient experience data as supporting information with a proposed draft guidance. 
264  If applicable, also include additional information such as the primary data capture. 
265  For additional guidance on submission of patient experience data, please 
266  reference FDA draft guidance on Patient-Focused Drug Development: Collecting 
267  Comprehensive and Representative Input.10  
268   
269  4.  How should stakeholders submit proposed draft guidance to FDA? 
270   
271  As specified in FDAs Good Guidance Practices regulations,11 drafts of proposed guidance 
272  documents should be marked Guidance Document Submission, and submitted to:  
273   
274  Division of Dockets Management (HFA-305),  
275  5630 Fishers Lane, Rm. 1061,  
276  Rockville, MD 20852 
277   
278  Stakeholders may also submit a proposed draft guidance electronically via  www.regulations.gov 
279  under the shell docket 2013-FDA-S-0613. Instructions for filing a proposed draft guidance 
280  electronically are available in that shell docket and on FDAs guidances web page.12   
281   
282  5.  What will happen after stakeholders submit the proposed draft guidance to FDA? 
283     
284  For stakeholders interested in submitting a proposed draft guidance, FDAs Division of Dockets 
285  Management will open a new docket for each proposed draft guidance document submission and 
286  will send a letter to acknowledge receipt of the proposed draft guidance. The proposed draft 
287  guidance document will be available to the public. After receiving the proposed guidance 
288  document submission from the Division of Dockets Management, FDAs CDER or CBER will 
289  ensure it is sent to the relevant office(s) and/or division(s) within each Center. 
290   
291  Submission of a proposed draft guidance to FDA does not mean that FDA will publish its own 
292  draft guidance on the topic(s) identified. FDA has its own process for developing guidance based 
                                                 
10 See FDA draft guidance Patient-Focused Drug Development: Collecting Comprehensive and Representative 
Input. 
11 See 21 CFR 10.115. 
12 See FDAs Instructions for Submitting Drafts of Proposed Guidance Documents Electronically available on 
FDAs web page at: 
https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm601464.htm.  
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293  on several factors including the state of the science in a given area, policy priorities, and Agency 
294  resources. The stakeholder-submitted proposed draft guidance can be used to inform FDAs 
295  thinking and future guidance development work, if applicable.  
296    
297  6.  Can a stakeholder submit proposed revisions to an existing FDA guidance? 
298   
299  Under FDAs Good Guidance Practice regulations, stakeholders can provide comments on a 
300  guidance document at any time. They may also suggest that FDA revise or withdraw an existing 
301  guidance document.13 The suggestion should address why the guidance document should be 
302  revised or withdrawn and, if applicable, how it should be revised. The suggestion should be 
303  submitted as a comment to the public docket assigned to that specific guidance. To find an 
304  assigned public docket, please visit www.regulations.gov and search for the specific guidance.  
305   
306  7.  What are other ways for a stakeholder to participate and provide input on FDA policy and 
307  guidance development? 
308   
309  There are other opportunities for stakeholders to provide input on FDA policy and guidance 
310  development, such as participating in FDA-led meetings. Stakeholders are encouraged to attend 
311  FDA-led public meetings on policy development, patient-focused drug development, or technical 
312  issues in a given disease area. In addition to participating in meetings, stakeholders can submit 
313  written comments to the public docket that is assigned to each meeting. 
314      
315  B.   Other Opportunities for Stakeholders 
316    
317  Stakeholders, including patient and disease advocacy groups, may be well positioned to make 
318  contributions to advance the understanding of disease burden, progression, treatment burden, 
319  challenges to clinical trial participation, and other issues and policy considerations from the 
320  perspective of patients and caregivers. This work can be pursued by stakeholders on their own, 
321  independent of FDA involvement. 
322   
323  1.  What are other opportunities for stakeholders to help advance patient-focused drug 
324  development? 
325   
326  There are several other opportunities stakeholders may wish to pursue depending on their 
327  particular capabilities, expertise, and priorities. Other areas of opportunity may include:  
328   
329  a.  Developing a patient registry: The purposes for patient registries can range 
330  widely, including use in recruiting patients for clinical trials; providing 
331  information on a given disease area; developing therapeutics; learning about 
332  behavior patterns and their association with disease development; and developing 
333  research hypotheses. Patient registries can also be used to monitor outcomes and 
334  study best practices in care or treatment.14 
                                                 
13 See 21 CFR 10.115(f)(4). 
14 See Engaging Patients in Information Sharing and Data Collection: The Role of Patient-Powered Registries and 
Research Networks [Internet]: https://www.ncbi.nlm.nih.gov/books/NBK164514/.  
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335   
336  b.  Conducting natural history studies: Stakeholders may collaboratively develop and 
337  conduct natural history studies. Natural history studies track the course of disease 
338  over time, identifying demographic, genetic, environmental, and other variables 
339  that correlate with its development and outcomes in the absence of treatment. 
340  These studies can inform the basis for recruiting and retaining patients in clinical 
341  trials. 
342   
343  Stakeholders, including investigators from industry, academia, patient and disease 
344  advocacy groups, and government, may also request a Critical Path Innovation 
345  Meeting with FDA to discuss topics regarding natural history study designs and 
346  implementation, biomarkers in the early phase of development, clinical outcome 
347  assessments in the early phase of development, innovative conceptual approaches 
348  to clinical trial design and analysis, and plans to collect patient experience data.15 
349   
350  c.  Coordinating work among patient groups and other stakeholders: It is helpful for 
351  patient groups to align efforts to advance work in a disease area. This can help 
352  ensure that patient groups avoid duplicative efforts and help maximize the use of 
353  resources and valuable patient and staff time. 
354   
355  Stakeholders may also wish to establish public-private partnerships or consortia to 
356  bring multiple stakeholders together, including FDA, to address issues that are 
357  beyond the capacity and resources of a single organization.16  
358   
359  d.  Communicating, educating, and conducting outreach: Stakeholders, particularly 
360  patient groups, may be well positioned to communicate and conduct outreach with 
361  the patient community through sources such as emails, newsletters, and social 
362  media. This outreach will help ensure that communities are aware of continuing 
363  work in the disease area, along with opportunities to engage with other 
364  stakeholders. 
365   
366  Patient groups may be well positioned to help develop and conduct trainings to 
367  educate communities on a given disease and medical product development for 
368  that disease. Topics for trainings may include types of research and testing needed 
369  to develop and manufacture safe and effective treatments, timeframes required for 
370  development, phases of clinical development, importance of clinical trials, and 
371  where to access clinical trials. 
372   
373  e.  Convening meetings: Stakeholders can convene meetings to advance discussion 
374  on topics related to medical product development for a given disease. For 
                                                 
15 See FDA Critical Path Innovation Meeting website: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm395888.htm. 
16For information about CDERs policy and procedures governing its staff participation in public private partnership 
and consortia, see  CDER Manual of Policies and Procedures on CDER Staff Participation in Public-Private 
Partnerships and Consortia: 
https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/Manualof
PoliciesProcedures/UCM532571.pdf. 
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375  example, stakeholders can host meetings on scientific or technical issues to 
376  discuss challenges or opportunities in a given disease area related to enhancing a 
377  clinical development program. This can be an opportunity to convene experts 
378  from patient and disease advocacy groups, federal agencies, industry, academia, 
379  and healthcare institutions to discuss specific scientific or technical issues that 
380  may need further structuring, identification of areas for further research, or 
381  enhanced data collection.  
382   
383  f.  Collecting patient experience data: Patient input can help inform the therapeutic 
384  context for regulatory review. Patient input can also provide a direct source of 
385  evidence regarding the benefits and risks of a medical product, if 
386  methodologically-sound data collection tools are developed and used within 
387  clinical trials of an investigational therapy. 
388   
389  In the early stages of drug development (e.g., discovery), patient and disease 
390  advocacy groups may conduct an externally-led Patient-Focused Drug 
391  Development meeting or focus group to gather patients perspectives on disease 
392  and treatment burden. 17 The information and perspectives obtained in these 
393  meetings can be used to develop meeting reports or other work products 
394  summarizing what was heard.  
395   
396  Stakeholders, in collaboration with appropriate methodologists, can conduct 
397  methodologically-sound surveys to collect representative input on patients 
398  experiences living with their disease, using available treatments, accessing and 
399  participating in clinical trials, and weighing acceptable levels of benefits and 
400  risks. 
401   
402  Stakeholders may also want to consider collaborating with one or more sponsors 
403  to collect patient input on a given disease, including patient input on the 
404  significant symptoms of the given disease, currently available treatments for the 
405  given disease, or other types of patient experience data. Stakeholders may also 
406  want to consider sharing their independently collected patient experience data 
407  with sponsors for potential use in decision-making. 
408   
409  For methodological considerations when collecting patient experience data, please 
410  refer to the series of methodological Patient-Focused Drug Development 
411  guidances being developed by FDA.18 
412   
413  2.  How can patient experience data inform medical product development and regulatory 
414  decision making? 
415   
                                                 
17 See FDA Externally-led Patient-Focused Drug Development Meetings website: 
https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm.  
18 See FDA Patient-Focused Drug Development Methodological Guidance Series for Enhancing the Incorporation of 
the Patients Voice in Medical Product Development and Regulatory Decision Making: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm610279.htm. 
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Draft  Not for Implementation 
416  Patient experience data that is collected and corresponding work products that are generated can 
417  serve as a valuable resource to multiple stakeholders throughout medical product development 
418  and decision-making, including FDA, other federal partners, industry, patient and disease 
419  advocacy groups, healthcare providers, and academic researchers. For a high-level overview of 
420  how patient experience data could enhance medical product development and decision-making, 
421  please see Table 1.  
422   
423  Table 1: How Patient Experience Data Could Enhance Medical Product Development and 
424  Decision Making 
Type of Stakeholder  
Type of Patient 
Medical Product 
Experience Data  Patient Stakeholders*  Regulators 
Developers/Researchers 
  Inform communications,    Help identify biomarkers and    Enhance the understanding of 
education and outreach efforts  clinical outcome measures that  the course of disease over time, 
for the patient community  will show how well a patient  identifying demographic, 
Patient registry or 
  Inform future research  responds to a treatment in  genetic, environmental, and 
natural history 
  Provide basis for recruitment in  clinical trials  other factors that correlate with 
study data 
clinical trials    Inform clinical trial design  its development and outcomes 
  Support clinical trial  in the absence of treatment (or 
recruitment  while on available therapies) 
  Identify burden of disease and    Informs Target Product Profile     Inform FDA decision-making  
unmet medical needs that    Identify clinical domains (e.g.,  throughout medical product 
warrant further scientific  most bothersome symptoms) of  lifecycle 
Study report or  discussion   the condition that could be    Enhance the understanding of 
survey data on the    Identify opportunities and gaps  targeted for new treatment  the therapeutic context  for 
therapeutic context  where further development and  development  benefit-risk assessments 
(severity of  research may be needed     Identify how the condition may    Enhance understanding of 
condition and    Identify considerations for  vary by sociodemographic  meaningful endpoints and 
unmet medical  clinical endpoints and clinically  factors, subgroups, culture, and  outcomes to patients to 
need), including  meaningful outcomes   disease severity  appropriately advise sponsors 
perspectives on    Inform patients on possibilities    Inform the selection,  on a medical product 
disease  to participate in development  development and modification  development plan in early 
background,  and validation of clinical trial  of meaningful clinical  phases of development 
severity of  endpoints and patient-reported  endpoints and outcomes, and    Inform FDA guidance on 
condition, and  outcomes  tools that measure what matter  disease-specific clinical, 
available treatment  most to patients  scientific and regulatory 
options    Inform clinical trial design,  matters 
including appropriate inclusion    Inform FDA assessments of 
and exclusion criteria  medical product development 
programs 
  Help clinical trial participants    Enhance recruitment and    Enhance understanding of 
better prepare for the trial,  retention for clinical trials  patients experience with 
Clinical trial 
including the informed consent    Inform development of  clinical trial design and 
experience data, 
process  informed consent documents  inclusion/exclusion criteria to 
including 
  Inform patients on opportunities    Provide insight into clinical  better advise sponsors 
perspectives on 
to participate in clinical trials  trial participant burden, 
trial visits and 
and improve overall recruitment  including frequency and 
assessments 
  Help individual decision making  conduct of trial visits and 
on whether to enroll in a trial  assessments 
  Inform future research    Enhance the understanding of    Enhance the understanding of 
  Identify unmet medical needs  patient input on benefits and  patient input on benefits and 
Patient input on  that warrant further scientific  risks to inform benefit-risk  risks to inform benefit-risk 
benefits and risks  discussion  assessment  assessment 
  Enhance the understanding of   
benefits and risks for patients 
  11  Contains Nonbinding Recommendations 
Draft  Not for Implementation 
Type of Stakeholder  
Type of Patient 
Medical Product 
Experience Data  Patient Stakeholders*  Regulators 
Developers/Researchers 
Perspectives on    Inform communications and    Improve the overall    Inform how to convey key 
ways to  education for the patient  communication of information  information that helps 
communicate  community to enhance shared  to patients and prescribers  facilitate patients informed 
information to  decision-making between  decision-making 
patients and  patients and prescribers 
prescribers 
* Patient stakeholders include patients, caregivers, and patient advocates. To provide standardized nomenclature and terminologies related to 
patient-focused medical product development, these terms are defined in FDAs Patient-Focused Drug Development Glossary.   
425   
426  3.    What work products relating to patient experience data may be developed by stakeholders?  
427   
428  A range of work products related to patient experience can be developed to provide helpful data 
429  and information to facilitate patient-focused drug development in a given disease area. Work 
430  products may include: 
431    Meeting reports summarizing patient perspectives on disease and treatment burden 
432    Methodologically-sound patient surveys  
433    White papers or peer-reviewed journal articles describing topics such as background on 
434  disease, and considerations for clinical trials in a given disease area 
435    Case examples to address disease-specific considerations related to medical product 
436  development 
437    Natural history study report 
438    Proposed draft guidance relating to patient experience data. 
439   
440  4.    How can stakeholders submit work products relating to patient experience data? 
441    
442  Stakeholders who would like to share work products (e.g., meeting reports, surveys) with FDA 
443  may visit the FDAs External Resources and Information Related to Patient Experience web 
444  page.19 This web page provides links to publicly available reports and resources relating to 
445  patient experience data. Key stakeholders including patient communities, patient advocates, 
446  researchers, drug developers, and federal agencies may find these materials useful. Please note 
447  that although FDA reviews the materials at these links before posting them to ensure that the 
448  materials are within the scope of the web page, FDA does not assess their scientific merit or 
449  compliance with applicable regulatory requirements. FDAs decision to post links to these 
450  materials does not reflect an endorsement of their authors, sponsors, or content.  
451   
452  Please visit the web page for more information regarding what types of resources may be 
453  included on this web page, how to submit a publicly available website link for this web page, and 
454  other general questions.  
455   
456  To submit proposed draft guidance documents, follow the process outlined in this guidance (See 
457  Section III.A, Development and Submission of Proposed Draft Guidance Relating to Patient 
458  Experience). 
                                                 
19 See FDAs External Resources or Information Related to Patients Experience web page: 
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm579132.htm.  
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